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Archive for the ‘Molecular Genetics’ Category

OpGen Subsidiary Curetis GmbH Secures Access to Additional EUR 5.0 Million Non-Dilutive Debt Financing Tranche from EIB – GlobeNewswire

Thursday, July 9th, 2020

- European Investment Bank (EIB) and OpGen, Curetis GmbH and Ares Genetics GmbH enter into Amendment to the EIB Financing Agreement

-Additional EUR 5 Million Tranche With 5-Year Interest Only Period Related to COVID-19 R&D Programs Across Multiple Platforms

-EIB Financing Tranche Available for Draw-Down at Curetis Sole Discretion for a 9-Month Period and Subject to 0.7% Participating Interest in OpGen Equity Value at Maturity

GAITHERSBURG, Md. and HOLZGERLINGEN, Germany, July 09, 2020 (GLOBE NEWSWIRE) -- OpGen, Inc. (Nasdaq: OPGN, OpGen), a precision medicine company harnessing the power of molecular diagnostics and bioinformatics to help combat infectious disease, announced today that its wholly owned subsidiary Curetis GmbH will have access to another EUR 5.0 million debt financing tranche under the amended EIB financing facility.

Under the EIB debt financing facility originally put in place in December 2016, Curetis now stands to receive another EUR 5.0 million tranche of non-dilutive debt financing. This additional tranche is earmarked to co-fund R&D programs across several of the platforms and the entire product portfolio of OpGen group companies Curetis and Ares Genetics as it relates to COVID-19.

This additional tranche, which can be drawn down at the sole discretion of Curetis within nine months from the Effective Date of this amendment, will also have a five-year term to maturity from such draw-down date. All interest payments during that five-year term are compounded and become payable only upon maturity of the principal amount of this tranche. The EIB tranche disbursement will become available subject to typical conditions precedent including a pledge of certain Curetis IP rights as security to EIB. The parties have furthermore agreed on a 0.7% participation percentage interest (PPI). Upon maturity of the tranche, i.e. not before H2-2025 (and no later than early 2027 depending on draw-down date), EIB will be entitled to an additional payment that is equity-linked and equivalent to 0.7% of the then total equity valuation of OpGen. The parties have also adjusted the PPI percentage applicable to the previous EIB tranche of EUR 5.0 million which was funded in June 2019 from its original 2.1% PPI in Curetis N.V.s equity value upon maturity to a new 0.3% PPI in OpGens equity value upon maturity between mid-2024 and mid-2025. This adjustment follows the respective stockholder ownership interest in OpGen following the business combination with Curetis as of April 1, 2020. All other terms and conditions of the EIB financing contract with Curetis remain unchanged.

We are very pleased to see continued strong support and funding commitment from the EIB, said Oliver Schacht, PhD, CEO of OpGen and Managing Director of Curetis GmbH. The additional funding will allow us to continue executing on our R&D programs such as the Unyvero A50 and A30 RQ platforms with a focus on COVID-19 related applications, as well as the Ares Genetics and combined OpGen R&D programs in AI-powered bioinformatics.

About OpGen, Inc.

OpGen, Inc. (Gaithersburg, MD, USA) is a precision medicine company harnessing the power of molecular diagnostics and bioinformatics to help combat infectious disease. Along with our subsidiaries, Curetis GmbH and Ares Genetics GmbH, we are developing and commercializing molecular microbiology solutions helping to guide clinicians with more rapid and actionable information about life threatening infections to improve patient outcomes, and decrease the spread of infections caused by multidrug-resistant microorganisms, or MDROs. OpGens product portfolio includes Unyvero, Acuitas AMR Gene Panel and Acuitas Lighthouse, and the ARES Technology Platform including ARESdb, using NGS technology and AI-powered bioinformatics solutions for antibiotic response prediction.

For more information, please visit http://www.opgen.com.

Forward-Looking Statements

This press release includes statements regarding the amendment of the EIB financing agreement for purposes of R&D programs related to COVID-19 diagnostic tests by OpGen and its subsidiaries, Curetis and Ares Genetics. These statements and other statements regarding OpGens future plans and goals constitute "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and are intended to qualify for the safe harbor from liability established by the Private Securities Litigation Reform Act of 1995. Such statements are subject to risks and uncertainties that are often difficult to predict, are beyond our control, and which may cause results to differ materially from expectations. Factors that could cause our results to differ materially from those described include, but are not limited to, our ability to successfully, timely and cost-effectively develop, seek and obtain regulatory clearance for and commercialize our product and services offerings, the rate of adoption of our products and services by hospitals and other healthcare providers, the realization of expected benefits of our business combination transaction with Curetis GmbH, the success of our commercialization efforts, the impact of COVID-19 on the Companys operations, financial results, and commercialization efforts as well as on capital markets and general economic conditions, the effect on our business of existing and new regulatory requirements, and other economic and competitive factors. For a discussion of the most significant risks and uncertainties associated with OpGen's business, please review our filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which are based on our expectations as of the date of this press release and speak only as of the date of this press release. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

OpGen:Oliver SchachtPresident and CEOInvestorRelations@opgen.com

OpGen Press Contact:Matthew BretziusFischTank PR matt@fischtankpr.com

OpGen Investor Contact:Joe Green Edison Groupjgreen@edisongroup.com

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OpGen Subsidiary Curetis GmbH Secures Access to Additional EUR 5.0 Million Non-Dilutive Debt Financing Tranche from EIB - GlobeNewswire

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Racing against time: Rapid, high-throughput discovery of antibody therapeutics for SARS-CoV-2 – Science Magazine

Thursday, July 9th, 2020

Since the World Health Organization declared it a pandemic in March 2020, the novel coronavirus SARS-CoV-2 has caused millions of infections and hundreds of thousands of deaths worldwide. Currently there is no cure, and many initial treatments being tested against COVID-19 were designed for other viral infections. Multiple drugmakers are scrambling to find treatments that might help fight off the coronavirus or prevent infections altogetherin a Herculean effort to collapse the typical 1015-year drug development timeline to under a year.

Antiviral human monoclonal antibodies (mAbs) are promising drug candidates for preventing or treating severe viral diseases, but the long timelineson the order of yearsneeded for antibody discovery, functional analysis, preclinical studies, and manufacturing limit their rapid deployment and use as immunotherapeutics. Vanderbilt University Medical Center researchers Robert Carnahan and Pavlo Gilchuk are part of the scientific team attempting to compress the timeline for potent antiviral antibody discovery and characterization by integrating a series of advances in single-cell messenger RNA sequence analysis, bioinformatics, synthetic biology, and high-throughput functional analysis. Their work enabled the rapid discovery of a diverse panel of highly potent antiviral human mAbs against the SARS-CoV-2 Spike protein and the validation of their activity both in vitro and in vivo. These results provide a potential framework for expedited antibody discovery programs against viral pathogens of global concern.

During the webinar, viewers will:

This webinar will last for approximately 60 minutes.

For Research Use Only. Not for use in diagnostic procedures.

Vanderbilt Vaccine CenterNashville, TN

Dr. Pavlo Gilchuk received his M.S. in biochemistry and his doctorate in biotechnology from Taras Shevchenko National University of Kyiv, Ukraine. His work there was largely focused on single-chain antibody design and discovery. From 2002 to 2009, he was employed by Phage Biotechnology Corporation in San Diego, California, where he advanced from engineer biologist to research group manager. Dr. Gilchuk also held the position of staff scientist at the Institute of Molecular Biology and Genetics, National Academy of Sciences of Ukraine (Kyiv), and served as laboratory head at the State Institute of Genetic and Regenerative Medicine, Academy of Medical Sciences of Ukraine (Kyiv). Upon transitioning to the Vanderbilt University Medical Center in Nashville, Tennessee, his initial research was directed to large-scale vaccinia virusderived T cell epitope discovery, elucidation of immunological features of lung-resident memory CD8+ T cells, and development of platforms for T celltargeted mucosal vaccination. Dr. Gilchuk is now a senior staff scientist in the laboratory of James E. Crowe, Jr. at the Vanderbilt Vaccine Center (VVC) and leads antibody-discovery technology development and evolution within this large academic team. In addition, he is currently working on methods development for rapid identification of therapeutic antibodies against viral diseases (the Defense Advanced Research Projects Agency [DARPA] Pandemic Prevention Platform program).

Vanderbilt Vaccine CenterNashville, TN

Dr. Robert Carnahan is associate director of the Vanderbilt Vaccine Center at Vanderbilt University Medical Center and an associate professor in pediatrics and radiology. He has a high level of experience with monoclonal antibodies and antibody engineering. He directed the Vanderbilt Antibody and Protein Resource (VAPR) for over 10 years. The facility grew to the point of conducting an average of 150 antibody projects per year, comprising more than 70 unique Vanderbilt investigators as well as several academic and industry partners from across the country (e.g., the University of California, Los Angeles; the Fred Hutchinson Cancer Research Center; Purdue University; Kolltan Pharmaceuticals; Becton Dickinson). Both academic and industry projects focused increasingly on therapeutic targets (cancer, viral pathogens, autoimmunity, diabetes, etc.), with work in the lab ranging from discovery to preclinical activities. Dr. Carnahan has brought this focus on innovation applied to antibody generation and detailed characterization to the Crowe Lab and the Vanderbilt Vaccine Center. During this time, he has also held numerous regional and national leadership roles within the Association of Biomolecular Resource Facilities (ABRF), to develop advances in methodologies and technical standards, oversee collaborative studies, and implement training opportunities for lab directors and personnel. Furthermore, he is also a national leader in the design and implementation of Lean management systems for biological laboratories.

Science/AAASWashington, D.C.

Dr. Oberst did her undergraduate training at the University of Maryland, College Park, and her Ph.D. in Tumor Biology at Georgetown University, Washington D.C. She combined her interests in science and writing by pursuing an M.A. in Journalism from the Philip Merrill College of Journalism at the University of Maryland, College Park. Dr. Oberst joined Science/AAAS in 2016 as the Assistant Editor for Custom Publishing. Before then she worked at Nature magazine, the Howard Hughes Medical Institute, The Endocrine Society, and the National Institutes of Mental Health.

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Acer Therapeutics Announces Administration of ACER-001 in a Fasted State Increased Systemic Exposure of Phenylbutyrate in Healthy Volunteer Food…

Thursday, July 9th, 2020

Model suggests ACER-001, Acers taste-masked, immediate release formulation of sodium phenylbutyrate, may offer improved disease management in patients with Urea Cycle Disorders compared to current treatments

Anticipate submitting ACER-001 NDA in H1 2021 assuming successful completion of additional nonclinical work and long-term stability data, and subject to additional capital

NEWTON, Mass., July 08, 2020 (GLOBE NEWSWIRE) -- Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced data from a food effect study in healthy volunteers showing that administration of ACER-001 in a fasted state increased systemic exposure of phenylbutyrate (PBA), phenylacetate (PAA) and phenylacetylglutamine (PAGN) levels compared to fed state, and therefore based on modeling data may improve disease management in patients with urea cycle disorders (UCDs) when compared to currently approved treatments requiring administration with food.

Results from Part B of the ACER-001 bioequivalence (BE) trial in healthy volunteers (n=36) announced in February 2020 showed that ACER-001 was bioequivalent to BUPHENYL (sodium phenylbutyrate) and were within the parameters recommended by the FDAs Guidance for Industry, Statistical Approaches to Establishing Bioequivalence. The BE trial included a food effect study, which evaluated the pharmacokinetics (PK) of sodium phenylbutyrate (NaPBA) showing that administration of ACER-001 in a fasted state achieved more than two times the maximum concentration (Cmax) of PBA compared to administration of the same dose of ACER-001 in a fed state. These results are consistent with previously published data by Nakano, et al1 that evaluated PK of NaPBA in patients with progressive familial intrahepatic cholestasis, also demonstrating that administration of NaPBA in a fasted state significantly increased PBA peak plasma concentration compared to administration of NaPBA in a fed state.

Currently approved therapies for UCDs, including BUPHENYL2 and RAVICTI3 (glycerol phenylbutyrate), are required to be administered with food. BUPHENYL is required to be administered in a fed state due to its aversive odor and taste, with side effects including nausea, vomiting and headaches, which often lead to discontinuation of treatment.4 Additionally, prescribing information states that BUPHENYL food effect is unknown. RAVICTI PK and pharmacodynamic (PD) properties were determined to be indistinguishable in fed or fasted states.5 ACER-001 is uniquely formulated with its multi-particulate, taste-masked coating to allow for administration in a fasted state, while still allowing for rapid systemic release.

Based on the results from the food effect study within the ACER-001 BE trial, Acer commissioned Rosa & Co. LLC to create a PhysioPD PK model to evaluate the potential food effect on exposure, tolerability and efficacy of ACER-001 in UCDs patients. Results from this in silico model suggest that administration of ACER-001 in a fasted state required approximately 30% less PBA to achieve comparable therapeutic benefit in a fed state. In addition, the model predicted that administration of ACER-001 in a fasted state compared to administration of BUPHENYL or RAVICTI (same amounts of PBA) in their required fed states is expected to result in higher peak blood PBA, PAA and PAGN concentrations, predicting a 43% increase in urinary PAGN levels (a negative correlation between blood ammonia area under the curve and 24-hour urinary PAGN amount has been demonstrated6).

For nearly a quarter century, phenylbutyrate has been prescribed to UCD patients with food while its effect on phenylbutyrate absorption was never determined. The results of the ACER-001 food effect study, published literature and in silico modeling suggest that ACER-001 administered in a fasted state, and likely just 10 minutes prior to meals, could offer UCD patients a safe and better disease management option compared to currently approved products that are required to be taken with food, said Chris Schelling, CEO and Founder of Acer. We formulated ACER-001 to specifically improve palatability and tolerability, and we expect that this formulation should allow ACER-001 to be successfully administered without food. We look forward to discussing these findings with the FDA later in the third quarter. Schelling continued Interestingly, the increased exposure seen under fasted conditions may have benefit in other patient populations we intend to study, such as Maple Syrup Urine Disease (MSUD), where the Cmax of phenylbutyrate is the active moiety.

About UCDsUCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.7,8

The current treatment of UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.

Current medical treatments for UCDs include nitrogen scavengers RAVICTI and BUPHENYL in which the active pharmaceutical ingredients are glycerol phenylbutyrate (GPBA) and sodium phenylbutyrate (NaPBA), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications can be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons given for non-compliance include the unpleasant taste associated with available medications, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.9

About ACER-001ACER-001 is a taste-masked, immediate-release proprietary formulation of sodium phenylbutyrate developed by Acer using a microencapsulation process. ACER-001 is being developed for the treatment of various inborn errors of metabolism, including UCDs and Maple Syrup Urine Disease (MSUD). ACER-001 microparticles consist of a core center, a layer of active drug, and a taste-masking coating that quickly dissolves in the stomach, allowing taste to be neutralized while still allowing for rapid systemic release. This taste-masked formulation may result in better patient tolerability allowing for administration in a fasted state, and likely prior to a meal. Acer has been granted orphan drug designation by the FDA for the MSUD indication. ACER-001 is under clinical investigation and its safety and efficacy have not been established. There is no guarantee that this product will receive FDA approval or become commercially available for the uses being investigated.

About Acer Therapeutics Inc.Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acers pipeline includes four clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; EDSIVO (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. Each of Acers product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit http://www.acertx.com.

References

Forward-Looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund the ACER-001 program; the adequacy of our capital to support our future operations and our ability to successfully initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on managements current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, the substantial costs and diversion of managements attention and resources which could result from pending securities litigation, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

Investor Contact:Hans VitzthumLifeSci AdvisorsPh: 617-430-7578hans@lifesciadvisors.com

Jim DeNikeAcer Therapeutics Inc.Ph: 844-902-6100jdenike@acertx.com

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Acer Therapeutics Announces Administration of ACER-001 in a Fasted State Increased Systemic Exposure of Phenylbutyrate in Healthy Volunteer Food...

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Current research: 2020 Emerging Gene Therapies Market Report – WhaTech Technology and Markets News

Thursday, July 9th, 2020

This report provides statistics on the market situation, size, regions and growth factors. Emerging Gene Therapies Market report contains emerging players analyze data including competitive situations, sales, revenue and market share of top manufacturers.

The research reports onEmerging Gene Therapies Marketreport gives detailed overview of factors that affect global business scope. Emerging Gene Therapies Market report shows the latest market insights with upcoming trends and breakdowns of products and services.

Get FREE PDF Sample of the report @www.reportsnreports.com/contactme=1854862

Emerging Gene Therapies Market Top Company Analysis-Sangamo TherapeuticsCRISPR TherapeuticsCasebia TherapeuticsEditas MedicineIntellia TherapeuticsHomology Medicines

and more

Emerging Gene Therapies Market provides a comprehensive overview of the emerging gene therapy market. The report discusses gene therapy and the technology behind gene editing, outlining the advantages, limitations and current evidence for the platforms under development.

The report discusses relevant clinical studies targeting specific therapeutic indications and highlights examples of current challenges within the field, with a focus on therapies that target the eye, liver, and blood.

Additionally, the report provides a background to the CRISPR patent litigation, a key factor within the gene editing company landscape.

It provides profiles of six companies developing gene editing platforms, considers the gene therapy interests of the main pharmaceutical companies, and discusses current regulatory trends in the development of gene therapies.

The report explores how emerging gene editing products will compete with established products, their relative competitive strengths, and upcoming value inflection points within the field.

Scope of Emerging Gene Therapies Market Report- What are the key emerging products within the gene therapy landscape? Which companies have the strongest pipeline of innovative products? How will gene editing disrupt existing gene therapy products? What are the regulatory trends for emerging gene therapies? What are the interests of pharmaceutical companies within the field?

Get this Report @www.reportsnreports.com/purchasme=1854862

Reasons to accessthis Report- Achieve an up-to-date understanding of the area, with a comprehensive reference of key products within the gene therapy landscape, compared across technology-specific relevant characteristics such as editing mechanism and delivery vector. Conduct competitive analysis using indication-specific, side-by-side comparisons of the latest data for key gene therapy products in the strategically relevant areas of eye, blood, and liver. Conduct strategic analysis using an overview of gene therapy specific considerations for evaluating and developing gene therapy products the CRISPR patent space, emerging regulatory trends, innovation leaders and the interests of pharma in gene therapy.

Table of Contents in this Report-1 Table of Contents 51.1 List of Tables 71.2 List of Figures 82 Introduction 102.1 Gene Therapy Definitions 102.2 Report Coverage the Emerging Gene Therapy Pipeline 112.3 History of Gene Therapy 122.4 Limitations of Gene Transfer 132.5 The Development of Targeted Gene Editing 132.6 Overview of Gene Editing Platforms 132.6.1 Zinc Fingers (1996) 132.6.2 Transcription Activator-Like Effectors (2011) 142.6.3 The CRISPR/Cas System (2013) 152.6.4 Effectors for Targeting Domains 192.6.5 Comparison of Gene Editing Systems 192.6.6 Summary of Gene Editing Systems 192.7 Overview of In Vivo Gene Therapy 212.7.1 Editing is Dependent on Cell Type, Stage, and Repair Pathway 212.7.2 Delivery 212.7.3 Emerging Safety Concerns with Editing Platforms 242.7.4 Editing Products are Reliant on the Target Cells Cycle Stage and DNA Repair Machinery 272.7.5 Advantages of Gene Editing over Gene Transfer 282.7.6 Integration into Safe Harbor Sites 282.7.7 The Increasing Complexity of Gene Therapy 302.7.8 Summary of In Vivo Gene Therapy 313 Gene Therapy Near Term Product Pipeline 333.1 Leber Congenital Amaurosis 333.1.1 Unmet Need 333.1.2 Molecular Genetics 333.1.3 Luxturna (Voretigene neparvovec) 333.1.4 Editas Medicine: EDIT-101 353.1.5 Trial Design 363.1.6 EDIT-101 and Off-Target Effects 373.1.7 The Potential Advantage of EDIT-101 is the Longevity of its Therapeutic Effect 373.1.8 Summary LCA 383.2 Choroideremia 383.3 Hurler Syndrome (MPS I) 393.3.1 Key Clinical Studies 403.3.2 Regenex: RGX-111 403.3.3 Sangamo Therapeutics: SB-318 403.4 Hunter Syndrome (MPS II) 413.4.1 Unmet Need 413.4.2 Sangamo Therapeutics: SB-913 413.4.3 Immusoft Corporation: Cell Therapy 433.5 Sanfilippo Syndrome (MPS III) 433.5.1 Lysogene: LYS-SAF302 433.6 Summary MPS Disorders 443.7 Hemophilia 443.7.1 Hemophilia A 463.7.2 Summary Hemophilia A 503.7.3 Hemophilia B 513.7.4 Summary Hemophilia B 533.8 Hemoglobinopathies 543.8.1 Beta Thalassemia: Unmet Need 543.8.2 Beta Thalassemia: Molecular Genetics 553.8.3 Sickle Cell Disease: Unmet Need 563.8.4 Sickle Cell Disease: Molecular Genetics 563.9 Cellular Therapies for Hemoglobinopathies 573.9.1 Blue Bird Bio: BB-305 (LentiGlobin) 573.9.2 Sangamo: ST-400 603.9.3 CRISPR Therapeutics: CTX-001 613.9.4 Summary: Cellular Therapies for Hemoglobinopathies 623.10 Duchenne Muscular Dystrophy 633.10.1 Unmet Need 633.10.2 Molecular Genetics 633.10.3 ExonDys 51 Sarepta Therapeutics 643.10.4 Solid BioSciences: SGT-001 663.10.5 Exonics Therapeutics: CRISPR Approach 673.10.6 Summary Duchenne Muscular Dystrophy 684 Competitive Landscape 694.1 Regulatory Considerations for Developing Gene Therapy Products 694.1.1 Product Characteristics 694.1.2 Clinical Study Design for Gene Therapy Products 694.1.3 Disease specific guidance 704.1.4 Reimbursement and Payment 714.1.5 Summary Regulatory Considerations 724.2 Intellectual Property CRISPR/Cas 724.2.1 Licensing, Exploitation, and MPEG Pool 744.3 Company Analysis: Gene Editing Companies 754.3.1 Sangamo Therapeutics 754.3.2 CRISPR Therapeutics 794.3.3 Casebia Therapeutics 814.3.4 Editas Medicine 824.3.5 Intellia Therapeutics 844.3.6 Homology Medicines 864.4 Company Analysis: Pharma 874.4.1 Amgen 874.4.2 Gilead Sciences 874.4.3 Novartis 874.4.4 Sanofi 884.4.5 GlaxoSmithKline 884.4.6 Pfizer 885 Appendix 895.1 References 895.2 Report Methodology 98

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Current research: 2020 Emerging Gene Therapies Market Report - WhaTech Technology and Markets News

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Myriad Announces New Studies Validating the Ability of Myriad’s riskScore Test to Modify Breast Cancer Risk Prediction | 2020-07-07 | Press Releases -…

Thursday, July 9th, 2020

SALT LAKE CITY, July 07, 2020 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in molecular diagnostics and precision medicine, announced today two recent publications validating the polygenic risk score (PRS) component of Myriad’s breast cancer risk stratification tool riskScore®. The publications clinically validate both the ability of the PRS component of riskScore to predict breast cancer risk in asymptomatic women and modify risk estimations for patients identified with pathogenic mutations.

Historically we’ve considered breast cancer risk most significant for women diagnosed with pathogenic mutations in hereditary cancer genes. These studies demonstrate clearly that other genetic factors evaluated through Myriad’s riskScore test can dramatically alter breast cancer risk both independent of, and in combination with, gene mutations,” said Nicole Lambert, president of Myriad International, Oncology and Women’s Health. This information can dramatically change patient clinical management and Myriad is currently working diligently to provide access to this important information for all women.”

The first study published in JCO Precision Oncology described the PRS component of riskScore in over 150,000 women. It showed that independent of other hereditary breast cancer gene mutations (e.g., BRCA1), Myriad’s polygenic risk score can add great value and precision to breast cancer risk estimates. The PRS was highly associated with breast cancer risk with an odds ratio of 1.47 (95% confidence interval 1.45 to 1.49) per unit standard deviation in the PRS. This translated to women in the top PRS percentile having a three-fold higher risk of breast cancer than an average risk patient.

The second study published in the Journal of the American Medical AssociationNetwork Open demonstrates the ability of Myriad’s polygenic risk score to improve breast cancer risk stratification in women diagnosed with pathogenic mutations in common breast cancer genes. The study evaluated over 150,000 patients and approximately 10,000 patients who were carriers of pathogenic mutations in the BRCA1, BRCA2, CHEK2, ATM and PALB2 genes who were tested at Myriad. The study demonstrated that patients with high penetrant genes such as BRCA1 and BRCA2 did not warrant changes in clinical management; however, breast cancer risks in patients with moderate penetrant genes such as CHEK2, ATM, and PALB2 could vary significantly, warranting different clinical management considerations. For example, patients with a PALB2 mutation historically have been assessed to have an approximately 50 percent lifetime risk for breast cancer. However, after incorporating the data from Myriad’s 86 single nucleotide polymporphism (SNP) riskScore test, patient risks varied between 26 percent to 79 percent (see Graph 1 below).

To view Graph 1: PRS Significantly Modifies Lifetime Breast Cancer Risk in Mutation Carriers please visit the following link: https://www.globenewswire.com/NewsRoom/AttachmentNg/11ac3a62-dd7e-417a-9f08-a3a3110e01db

These are some of the largest polygenic risk score studies ever published. Patient medical management can vary dramatically depending on where patients with and without pathogenic mutations fall within the risk spectrum,” said Thomas P. Slavin M.D., senior vice president for Medical Affairs in Oncology at Myriad Genetic Laboratories. This information will help empower patients and clinicians to make more informed decisions based upon the most precise breast cancer risk estimates availiable.”

About riskScore® riskScore is a new clinically validated personalized medicine tool that enhances Myriad’s myRisk® Hereditary Cancer test. riskScore helps to further predict a women’s lifetime risk of developing breast cancer using clinical risk factors and genetic-markers throughout the genome. The test incorporates data from more than 80 single nucleotide polymorphisms identified through 20 years of genome wide association studies in breast cancer and was prospectively validated in our laboratory to predict breast cancer risk in women of European descent. This data is then combined with a best-in-class family and personal history algorithm, the Tyrer-Cuzick model, to provide every patient with individualized breast cancer risk.

About Myriad Genetics Myriad Genetics Inc., is a leading personalized medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on three strategic imperatives: transitioning and expanding its hereditary cancer testing markets, diversifying its product portfolio through the introduction of new products and increasing the revenue contribution from international markets. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice CDx, Vectra, Prequel, Foresight, GeneSight, riskScore and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor Statement This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to providing access to this important information for all women; and the Company’s strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties associated with COVID-19, including its possible effects on our operations and the demand for our products and services; our ability to efficiently and flexibly manage our business amid uncertainties related to COVID-19; the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers’ reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decisions in Mayo Collab. Servs. v. Prometheus Labs., Inc., 566 U.S. 66 (2012), Ass’n for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013), and Alice Corp. v. CLS Bank Int’l, 573 U.S. 208 (2014); risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2019, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

Graph 1

PRS Significantly Modifies Lifetime Breast Cancer Risk in Mutation Carriers

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Applied DNA Forms Clinical Testing Subsidiary to Maximize Utility of FDA EUA-Approved Linea COVID-19 Assay Kit and Expands Utility with Successful EUA…

Thursday, July 9th, 2020

STONY BROOK, N.Y.--(BUSINESS WIRE)-- Applied DNA Sciences Inc. (NASDAQ: APDN) (Applied DNA or the Company) a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing that enables in vitro diagnostics, and pre-clinical nucleic acid-based therapeutic drug candidates, today announced the formation of a wholly-owned subsidiary, Applied DNA Clinical Labs, LLC (ADCL), dedicated to the commercial development of its tests in Virology, for the detection of COVID-19, and in Oncology, for the detection and enumeration of invasive Circulating Tumor Cells (iCTCs). With the formation of ADCL, the Company is executing on a strategy to expand its market reach through value-added services complementary to a growing portfolio of diagnostic assays attractive to a broader number of qualified labs. ADCL allows Applied DNA to be a direct beneficiary of its own diagnostics development beyond the sales of kits to qualified laboratories, and provides the clinical sampling framework necessary to obtain the data required by State and Federal regulatory authorities to develop and improve diagnostics and vaccines in the face of a pandemic with rapidly shifting genetics and infectious behavior.

In mid-May, Applied DNA received Emergency Use Authorization (EUA) for a high sensitivity, high-throughput complex molecular test for SARS-CoV-2, the coronavirus that causes COVID-19. The Linea COVID-19 SARS-CoV-2 Assay Kit is a real-time polymerase chain reaction (RT-PCR) test for the qualitative detection of SARS-CoV-2 RNA in respiratory specimens. It provides a high-throughput solution to help laboratories address the urgent need for patient testing during the Coronavirus pandemic.

The Company also announced that the amendment of its EUA was approved by FDA to include extraction and concentration of the viral RNA by a magnetic method of nucleic acid extraction that lends itself easily to high throughput scaling with robotics and diversifies the two extraction methods already covered by the existing EUA. A series of EUA amendments are planned to introduce automation for increased throughput and accuracy, gain approval for additional RNA extraction methods and PCR devices to expand the footprint where our EUA assay can be used, develop point-of-care testing and simplify sample acquisition from patients.

Commenting on the amendment, Dr. James Hayward, president and CEO of Applied DNA, said, We are extremely grateful to our collaborators at Stony Brook University Hospital and to the FDA for their guidance during the development and validation of our Linea COVID-19 Assay Kit. Our goal is to service the COVID-19 patient community and their families, and to facilitate the reopening of universities and businesses with reliable, well-tested science. The demand for COVID testing is diverse, from asymptomatic patients and those in the early stages of disease, to the commercial imperative to operate.

The Company believes that as the demand for testing grows and diversifies, the existing approval of the Companys Linea COVID-19 Assay Kit for use with anterior nasal swabs will facilitate uptake by certified testing labs. As opposed to nasopharyngeal swabs, anterior nasal swabs only enter less than 1 inch into the nose, allowing for sampling with minimal irritation or discomfort. Additionally, anterior nasal swabs may be self-collected at a healthcare location or collected by a healthcare worker.

Dr. Hayward continued, With anterior nasal swabs, we believe patient compliance is not an issue. This is a simple approach to early testing, repetitive testing if begun early in infection, or high-frequency testing, such as in schools, universities, government office, and companies struggling with reopening. When combined with our high-sensitivity and high-throughput Linea COVID-19 Assay Kit, we believe the combined platform to be well suited to combat both the healthcare and economic challenges of the pandemic.

ADCL, the Companys new subsidiary was formed to enable clinical testing of patient specimens using methods developed by the Companys research teams. Access to clinical specimens will also facilitate more rapid development of improvements and new assays. All of these continuous improvements are designed to help drive sales to other approved testing labs.

ADCL was also formed to facilitate the use and development of the Companys Liquid Biopsy platform for detecting iCTCs in the blood of cancer patients, far simpler and safer than surgical or needle biopsies, and is already in use under contract in a Phase III clinical trial of a promising drug for the treatment of Stage IV pancreatic cancer. The trial involves 3,000 samples and the quantification of metastatic cancer cells in the blood. Dr. Hayward continued, Eventually, with the utility of ADCL, we plan to seek regulatory approval for our iCTC assay and add it to the tools used in modern oncology to ensure better patient outcomes.

The Company has obtained its Permanent Facility Identifier for ADCL, has hired a New York State Licensed Clinical Laboratory Director for Oncology and Virology and Certified Laboratory Scientists, and has applied for a license from the New York State Department of Health.

There can be no assurance that this license will be obtained, or that FDA will maintain the Emergency Declaration that enabled the EUA program for COVID-19 diagnostics. There can be no assurance that the Company will obtain approved 510K status (or other necessary regulatory approval) for its COVID-19 In Vitro Diagnostics in the event that the Emergency Declaration is lifted. In addition, the Companys iCTC assay is currently limited to RUO (Research Use Only), and there can be no assurance that the Company will obtain the necessary regularly approval for its iCTC assay to expand its use outside of RUO limitations.

About Applied DNA Sciences

Applied DNA is a provider of molecular technologies that enable supply chain security, anti-counterfeiting and anti-theft technology, product genotyping, and pre-clinical nucleic acid-based therapeutic drug candidates.

Visit adnas.com for more information. Follow us on Twitter and LinkedIn. Join our mailing list.

The Companys common stock is listed on NASDAQ under ticker symbol APDN, and its publicly traded warrants are listed on OTC under ticker symbol APPDW. Applied DNA is a member of the Russell Microcap Index.

Forward-Looking Statements

The statements made by Applied DNA in this press release may be forward-looking in nature within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. Forward-looking statements describe Applied DNAs future plans, projections, strategies and expectations, and are based on assumptions and involve a number of risks and uncertainties, many of which are beyond the control of Applied DNA. Actual results could differ materially from those projected due to the possibility of a failure to make timely payment on its outstanding secured convertible notes and resulting enforcement by noteholders of remedies on collateral which includes substantially all of Applied DNAs assets, its history of net losses, limited financial resources, limited market acceptance, the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Applied DNAs diagnostic candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. FDA or equivalent foreign regulatory agencies, the unknown outcome of any applications or requests to U.S. FDA, equivalent foreign regulatory agencies or New York State Department of Health, the unknown limited duration of any Emergency Use Authorization (EUA) approval from U.S. FDA, disruptions in the supply of raw materials and supplies, and various other factors detailed from time to time in Applied DNAs SEC reports and filings, including our Annual Report on Form 10-K filed on December 12, 2019 and our subsequent quarterly reports on Form 10-Q filed on February 6, 2020 and May 14, 2020, and other reports we file with the SEC, which are available at http://www.sec.gov. Applied DNA undertakes no obligation to update publicly any forward-looking statements to reflect new information, events or circumstances after the date hereof or to reflect the occurrence of unanticipated events, unless otherwise required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200709005245/en/

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July 2020: New NIEHS Director Rick Woychik will lead with innovation – Environmental Factor Newsletter

Tuesday, July 7th, 2020

National Institutes of Health (NIH) Director Francis Collins, M.D., Ph.D., announced selection of Richard (Rick) Woychik, Ph.D., to serve as director of NIEHS. Woychik was acting director of NIEHS from October 2019 until June 7, the official start of his new role.

Innovation has been a hallmark of Ricks scientific career and its at the center of his vision for leading NIEHS, said Collins. He will be working to support new technologies and scientific approaches throughout the field of environmental health sciences applying his proven skills in scientific excellence, creativity, and rigor to improving public health.

As NIEHS director, Woychik takes on additional responsibility as director of the National Toxicology Program (NTP), which reports directly to the U.S. Department of Health and Human Services. NTP coordinates toxicology research among NIEHS, the National Center for Toxicological Research at the U.S. Food and Drug Administration, and the National Institute for Occupational Safety and Health at the Centers for Disease Control and Prevention.

Woychik is highly respected for a long list of accomplishments in environmental epigenetics and mammalian genetics. His laboratory discovered that mutations in a gene that codes for a protein in a class called protocadherins interfered with mammalian hearing. This gene was ultimately linked to hearing loss in Cushings disease patients.

Other firsts included identifying a gene associated with polycystic kidney disease, and the first cloning of an obesity-related gene called agouti. Woychiks passion for epigenetics and environmental health sciences is rooted in his research groups discovery that the obesity trait associated with an agouti mutant mouse line was influenced during embryonic development by the epigenome.

Woychik said he will explore opportunities to embrace new technologies and implement state-of-the-art and potentially transformative scientific approaches for the NIEHS research enterprise. I am particularly interested in better integrating environmental health sciences into the All of Us Research Program and Precision Medicine programs at NIH, he wrote in an email to employees.

He added that achieving insights into the molecular mechanisms of toxicity and other physical responses to environmental exposures will be fundamental to improving risk assessments of human health impacts.

Individuals, with their unique biological make-up, respond to the environment in different ways. Our current research strategies mostly employ a one-size-fits-all approach that does not factor in this critically important variable, Woychik explained. One important element of my vision for the institute is to deal with individual genetic, epigenetic, and biological variability when establishing research strategies for studying public health and environmental toxicology.

Woychik earned his Ph.D. in molecular biology in 1984 from Case Western Reserve University in Cleveland. He moved into the field of molecular genetics during postdoctoral training in the lab of the late Philip Leder, M.D., in Harvard Medical School Department of Genetics.

In 2010, he moved to NIEHS in the role of deputy director, bringing basic science expertise in using genetics and epigenetics to study the influence of the environment. Woychik successfully led strategic planning exercises and guided implementation across a broad range of scientific disciplines. In nearly a decade of leadership at NIEHS, he built an in-depth awareness of and appreciation for the broad range of scientific activities such as toxicology, epidemiology, and more that are necessary to effectively support the environmental health sciences community.

That experience contributes to the wealth of knowledge he brings to his new role. Prior to NIEHS, Woychik served nearly 10 years as president and CEO of The Jackson Laboratory in Bar Harbor, Maine, where he established a robust record of achievements.

Citations:Alagramam KN, Murcia CL, Kwon HY, Pawlowski KS, Wright CG, Woychik RP. 2001. The mouse Ames waltzer hearing-loss mutant is caused by mutation ofPcdh15, a novel protocadherin gene. Nat Genet. 27:99102.

Bultman SJ, Michaud EJ, Woychik RP. 1992. Molecular characterization of the mouse agouti locus. Cell 71(7):11951204.

Moyer JM, Lee-Tischler MJ, Kwon HY, Schrick JJ, Avner ED, Sweeney WE, Godfrey VL, Cacheiro NL, Wilkinson JE, Woychik RP. 1994. Candidate gene associated with a mutation causing recessive polycystic kidney disease in mice. Science 264:13291333.

(Christine Bruske Flowers is director of the NIEHS Office of Communications and Public Liaison.)

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Myriad Announces New Studies Validating the Ability of Myriad’s riskScore Test to Modify Breast Cancer Risk Prediction – BioSpace

Tuesday, July 7th, 2020

Publications Demonstrate the Ability of the PRS Component of riskScore to Accurately Stratify Breast Cancer Risk in Women Both With and Without Pathogenic Mutations

SALT LAKE CITY, July 07, 2020 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in molecular diagnostics and precision medicine, announced today two recent publications validating the polygenic risk score (PRS) component of Myriads breast cancer risk stratification tool riskScore. The publications clinically validate both the ability of the PRS component of riskScore to predict breast cancer risk in asymptomatic women and modify risk estimations for patients identified with pathogenic mutations.

Historically weve considered breast cancer risk most significant for women diagnosed with pathogenic mutations in hereditary cancer genes. These studies demonstrate clearly that other genetic factors evaluated through Myriads riskScore test can dramatically alter breast cancer risk both independent of, and in combination with, gene mutations, said Nicole Lambert, president of Myriad International, Oncology and Womens Health. This information can dramatically change patient clinical management and Myriad is currently working diligently to provide access to this important information for all women.

The first study published in JCO Precision Oncology described the PRS component of riskScore in over 150,000 women. It showed that independent of other hereditary breast cancer gene mutations (e.g., BRCA1), Myriads polygenic risk score can add great value and precision to breast cancer risk estimates. The PRS was highly associated with breast cancer risk with an odds ratio of 1.47 (95% confidence interval 1.45 to 1.49) per unit standard deviation in the PRS. This translated to women in the top PRS percentile having a three-fold higher risk of breast cancer than an average risk patient.

The second study published in the Journal of the American Medical AssociationNetwork Open demonstrates the ability of Myriads polygenic risk score to improve breast cancer risk stratification in women diagnosed with pathogenic mutations in common breast cancer genes. The study evaluated over 150,000 patients and approximately 10,000 patients who were carriers of pathogenic mutations in the BRCA1, BRCA2, CHEK2, ATM and PALB2 genes who were tested at Myriad. The study demonstrated that patients with high penetrant genes such as BRCA1 and BRCA2 did not warrant changes in clinical management; however, breast cancer risks in patients with moderate penetrant genes such as CHEK2, ATM, and PALB2 could vary significantly, warranting different clinical management considerations. For example, patients with a PALB2 mutation historically have been assessed to have an approximately 50 percent lifetime risk for breast cancer. However, after incorporating the data from Myriads 86 single nucleotide polymporphism (SNP) riskScore test, patient risks varied between 26 percent to 79 percent (see Graph 1 below).

To view Graph 1: PRS Significantly Modifies Lifetime Breast Cancer Risk in Mutation Carriers please visit the following link:https://www.globenewswire.com/NewsRoom/AttachmentNg/11ac3a62-dd7e-417a-9f08-a3a3110e01db

These are some of the largest polygenic risk score studies ever published. Patient medical management can vary dramatically depending on where patients with and without pathogenic mutations fall within the risk spectrum, said Thomas P. Slavin M.D., senior vice president for Medical Affairs in Oncology at Myriad Genetic Laboratories. This information will help empower patients and clinicians to make more informed decisions based upon the most precise breast cancer risk estimates availiable.

About riskScoreriskScore is a new clinically validated personalized medicine tool that enhances Myriads myRisk Hereditary Cancer test. riskScore helps to further predict a womens lifetime risk of developing breast cancer using clinical risk factors and genetic-markers throughout the genome. The test incorporates data from more than 80 single nucleotide polymorphisms identified through 20 years of genome wide association studies in breast cancer and was prospectively validated in our laboratory to predict breast cancer risk in women of European descent. This data is then combined with a best-in-class family and personal history algorithm, the Tyrer-Cuzick model, to provide every patient with individualized breast cancer risk.

About Myriad GeneticsMyriad Genetics Inc., is a leading personalized medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on three strategic imperatives: transitioning and expanding its hereditary cancer testing markets, diversifying its product portfolio through the introduction of new products and increasing the revenue contribution from international markets. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice CDx, Vectra, Prequel, Foresight, GeneSight, riskScore and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to providing access to this important information for all women; and the Companys strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties associated with COVID-19, including its possible effects on our operations and the demand for our products and services; our ability to efficiently and flexibly manage our business amid uncertainties related to COVID-19; the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decisions in Mayo Collab. Servs. v. Prometheus Labs., Inc., 566 U.S. 66 (2012), Assn for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013), and Alice Corp. v. CLS Bank Intl, 573 U.S. 208 (2014); risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2019, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

Graph 1

PRS Significantly Modifies Lifetime Breast Cancer Risk in Mutation Carriers

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Myriad Announces New Studies Validating the Ability of Myriad's riskScore Test to Modify Breast Cancer Risk Prediction - BioSpace

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Myriad Announces Partnership with OptraHEALTH to Deliver Gene a New AI Based Information Tool for Hereditary Cancer Patients – GlobeNewswire

Tuesday, July 7th, 2020

SALT LAKE CITY, July 06, 2020 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in molecular diagnostics and precision medicine, today announced a new collaboration with OptraHEALTH to implement a cognitive ChatBOT named Gene to provide genetic and financial assistance information to prospective patients. Gene is an AI-powered, HIPAA-compliant knowledge platform for genetic health with BOT interfaces and can answer over 500,000 health related questions pertaining to hereditary cancer. Gene interfaces with Myriads market leading online hereditary cancer quiz, which is now taken by approximately one million people per year.

We are excited to offer this innovative new tool for physicians and patients to provide best-in-class pre-test education solutions that we can supplement with live sessions when necessary, said Nicole Lambert, president of Myriad International, Oncology and Womens Health. Myriad is highly focused on making the screening and testing process as streamlined as possible for healthcare providers and the implementation of this new technology will give their patients access to unparalleled online genetic education and support tools. This is especially important in the current environment with COVID-19 where patients may not be returning to the clinic setting and pre-test education can be particularly helpful as they work remotely with the healthcare provider to determine if testing is right for them.

Gene will interactively engage individuals online, providing them with education about hereditary cancer prior to taking an online assessment to determine if they may be a candidate for genetic testing. For those who complete the preliminary assessment and meet criteria for further evaluation, Gene will automate a pre-test process that sends an educational link that displays interactive multimedia content and gives the option to start a live conversation with a patient educator, who is a certified genetic counselor. Gene can also assist in finding a healthcare provider who can help a patient make an informed, definitive decision whether testing is appropriate and then order testing if so. Myriad plans on launching the Gene chatbot for its Foresight and Prequel prenatal tests and for companion diagnostic testing in oncology later this calendar year.

About OptraHEALTH: OptraHEALTHis focused on improving outcomes for consumers and leading Life Sciences and Healthcare organizations by utilizing a next-generation Artificial Intelligence Platform. OptraHEALTHs flagship product GeneFAX is an AI-powered knowledge platform for genetic health and is available as a web plugin or mobile application.

About Myriad GeneticsMyriad Genetics Inc., is a leading personalized medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on three strategic imperatives: transitioning and expanding its hereditary cancer testing markets, diversifying its product portfolio through the introduction of new products and increasing the revenue contribution from international markets. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice CDx, Vectra, Prequel, Foresight, GeneSight, Prolaris and riskScore are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to implementation of this new technology giving patients access to unparalleled online genetic education and support tools; plans to launch the Gene chatbot for its ForeSight and Prequel prenatal tests and for hereditary cancer testing in oncology later this calendar year; details of the functionality of the Genechatbot; and the Company's strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties associated with COVID-19, including its possible effects on our operations and the demand for our products and services; our ability to efficiently and flexibly manage our business amid uncertainties related to COVID-19; the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decisions in Mayo Collab. Servs. v. Prometheus Labs., Inc., 566 U.S. 66 (2012), Assn for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013), and Alice Corp. v. CLS Bank Intl, 573 U.S. 208 (2014); risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2019, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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Global Molecular Biomarkers by Manufacturers, Regions, Type and Application, Forecast to 2026 – Jewish Life News

Tuesday, July 7th, 2020

The global Molecular Biomarkers market focuses on encompassing major statistical evidence for the Molecular Biomarkers industry as it offers our readers a value addition on guiding them in encountering the obstacles surrounding the market. A comprehensive addition of several factors such as global distribution, manufacturers, market size, and market factors that affect the global contributions are reported in the study. In addition the Molecular Biomarkers study also shifts its attention with an in-depth competitive landscape, defined growth opportunities, market share coupled with product type and applications, key companies responsible for the production, and utilized strategies are also marked.

This intelligence and 2026 forecasts Molecular Biomarkers industry report further exhibits a pattern of analyzing previous data sources gathered from reliable sources and sets a precedented growth trajectory for the Molecular Biomarkers market. The report also focuses on a comprehensive market revenue streams along with growth patterns, analytics focused on market trends, and the overall volume of the market.

Download PDF Sample of Molecular Biomarkers Market report @ https://hongchunresearch.com/request-a-sample/18434

The study covers the following key players:Epic SciencesAbbottGenomic HealthAtossa GeneticsBiophysicalBioTheranosticsDako (Agilent)20/20 GeneSystemsGenomeDxDiagnoCureGen-ProbeCynvenioFoundation MedicineLife TechnologiesAlereBioCept

Moreover, the Molecular Biomarkers report describes the market division based on various parameters and attributes that are based on geographical distribution, product types, applications, etc. The market segmentation clarifies further regional distribution for the Molecular Biomarkers market, business trends, potential revenue sources, and upcoming market opportunities.

Market segment by type, the Molecular Biomarkers market can be split into,GenomicsProteomicsOthers

Market segment by applications, the Molecular Biomarkers market can be split into,MedicineBiologyOther

The Molecular Biomarkers market study further highlights the segmentation of the Molecular Biomarkers industry on a global distribution. The report focuses on regions of North America, Europe, Asia, and the Rest of the World in terms of developing business trends, preferred market channels, investment feasibility, long term investments, and environmental analysis. The Molecular Biomarkers report also calls attention to investigate product capacity, product price, profit streams, supply to demand ratio, production and market growth rate, and a projected growth forecast.

In addition, the Molecular Biomarkers market study also covers several factors such as market status, key market trends, growth forecast, and growth opportunities. Furthermore, we analyze the challenges faced by the Molecular Biomarkers market in terms of global and regional basis. The study also encompasses a number of opportunities and emerging trends which are considered by considering their impact on the global scale in acquiring a majority of the market share.

The study encompasses a variety of analytical resources such as SWOT analysis and Porters Five Forces analysis coupled with primary and secondary research methodologies. It covers all the bases surrounding the Molecular Biomarkers industry as it explores the competitive nature of the market complete with a regional analysis.

Brief about Molecular Biomarkers Market Report with [emailprotected] https://hongchunresearch.com/report/molecular-biomarkers-market-18434

Some Point of Table of Content:

Chapter One: Molecular Biomarkers Market Overview

Chapter Two: Global Molecular Biomarkers Market Landscape by Player

Chapter Three: Players Profiles

Chapter Four: Global Molecular Biomarkers Production, Revenue (Value), Price Trend by Type

Chapter Five: Global Molecular Biomarkers Market Analysis by Application

Chapter Six: Global Molecular Biomarkers Production, Consumption, Export, Import by Region (2014-2019)

Chapter Seven: Global Molecular Biomarkers Production, Revenue (Value) by Region (2014-2019)

Chapter Eight: Molecular Biomarkers Manufacturing Analysis

Chapter Nine: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter Ten: Market Dynamics

Chapter Eleven: Global Molecular Biomarkers Market Forecast (2019-2026)

Chapter Twelve: Research Findings and Conclusion

Chapter Thirteen: Appendix continued

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List of tablesList of Tables and FiguresFigure Molecular Biomarkers Product PictureTable Global Molecular Biomarkers Production and CAGR (%) Comparison by TypeTable Profile of GenomicsTable Profile of ProteomicsTable Profile of OthersTable Molecular Biomarkers Consumption (Sales) Comparison by Application (2014-2026)Table Profile of MedicineTable Profile of BiologyTable Profile of OtherFigure Global Molecular Biomarkers Market Size (Value) and CAGR (%) (2014-2026)Figure United States Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Europe Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Germany Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure UK Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure France Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Italy Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Spain Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Russia Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Poland Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure China Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Japan Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure India Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Southeast Asia Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Malaysia Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Singapore Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Philippines Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Indonesia Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Thailand Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Vietnam Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Central and South America Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Brazil Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Mexico Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Colombia Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Middle East and Africa Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Saudi Arabia Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure United Arab Emirates Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Turkey Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Egypt Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure South Africa Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Nigeria Molecular Biomarkers Revenue and Growth Rate (2014-2026)Figure Global Molecular Biomarkers Production Status and Outlook (2014-2026)Table Global Molecular Biomarkers Production by Player (2014-2019)Table Global Molecular Biomarkers Production Share by Player (2014-2019)Figure Global Molecular Biomarkers Production Share by Player in 2018Table Molecular Biomarkers Revenue by Player (2014-2019)Table Molecular Biomarkers Revenue Market Share by Player (2014-2019)Table Molecular Biomarkers Price by Player (2014-2019)Table Molecular Biomarkers Manufacturing Base Distribution and Sales Area by PlayerTable Molecular Biomarkers Product Type by PlayerTable Mergers & Acquisitions, Expansion PlansTable Epic Sciences ProfileTable Epic Sciences Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Abbott ProfileTable Abbott Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Genomic Health ProfileTable Genomic Health Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Atossa Genetics ProfileTable Atossa Genetics Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Biophysical ProfileTable Biophysical Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table BioTheranostics ProfileTable BioTheranostics Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Dako (Agilent) ProfileTable Dako (Agilent) Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table 20/20 GeneSystems ProfileTable 20/20 GeneSystems Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table GenomeDx ProfileTable GenomeDx Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table DiagnoCure ProfileTable DiagnoCure Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Gen-Probe ProfileTable Gen-Probe Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Cynvenio ProfileTable Cynvenio Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Foundation Medicine ProfileTable Foundation Medicine Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Life Technologies ProfileTable Life Technologies Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Alere ProfileTable Alere Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table BioCept ProfileTable BioCept Molecular Biomarkers Production, Revenue, Price and Gross Margin (2014-2019)Table Global Molecular Biomarkers Production by Type (2014-2019)Table Global Molecular Biomarkers Production Market Share by Type (2014-2019)Figure Global Molecular Biomarkers Production Market Share by Type in 2018Table Global Molecular Biomarkers Revenue by Type (2014-2019)Table Global Molecular Biomarkers Revenue Market Share by Type (2014-2019)Figure Global Molecular Biomarkers Revenue Market Share by Type in 2018Table Molecular Biomarkers Price by Type (2014-2019)Figure Global Molecular Biomarkers Production Growth Rate of Genomics (2014-2019)Figure Global Molecular Biomarkers Production Growth Rate of Proteomics (2014-2019)Figure Global Molecular Biomarkers Production Growth Rate of Others (2014-2019)Table Global Molecular Biomarkers Consumption by Application (2014-2019)Table Global Molecular Biomarkers Consumption Market Share by Application (2014-2019)Table Global Molecular Biomarkers Consumption of Medicine (2014-2019)Table Global Molecular Biomarkers Consumption of Biology (2014-2019)Table Global Molecular Biomarkers Consumption of Other (2014-2019)Table Global Molecular Biomarkers Consumption by Region (2014-2019)Table Global Molecular Biomarkers Consumption Market Share by Region (2014-2019)Table United States Molecular Biomarkers Production, Consumption, Export, Import (2014-2019)Table Europe Molecular Biomarkers Production, Consumption, Export, Import (2014-2019)Table China Molecular Biomarkers Production, Consumption, Export, Import (2014-2019)Table Japan Molecular Biomarkers Production, Consumption, Export, Import (2014-2019)Table India Molecular Biomarkers Production, Consumption, Export, Import (2014-2019)Table Southeast Asia Molecular Biomarkers Production, Consumption, Export, Import (2014-2019)Table Central and South America Molecular Biomarkers Production, Consumption, Export, Import (2014-2019)continued

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It Will Consume Your Life: 4 Families Take On Rare Diseases – The New York Times

Tuesday, July 7th, 2020

I knew we had to get the kids treatment, she said.

Dr. Harald Jueppner, a pediatric nephrologist at Massachusetts General, was the researcher who first identified the mutation that caused the condition. Dr. Nizar learned that he had been studying the mutated gene, called a PTH/PTHrP receptor, for 20 years out of scientific interest. But he had never seen a patient. She told him he could now see three herself and her two sons.

She also learned that Dr. Jueppner and a colleague, Thomas Gardella, had found in lab experiments that certain peptides, or short chains of amino acids, looked promising as possible treatments for Jansens. At that point Dr. Nizar latched onto the researchers, urging them to study the peptides for Jansens. They tried one of them in animal experiments. It partially reversed some of the bone abnormalities, Dr. Jeuppner said, but, he added, remember, this mouse model of Jansens is far from being ideal.

Dr. Nizar stayed in constant contact with Dr. Jueppner and Dr. Gardella prodding them to not lose sight of the work.

Working with Neena has been an incredible experience. Dr. Jueppner said. She is a force of nature.

In 2017, Dr. Nizar set up a foundation to support research. But she was not in a good position to fund raise. We only had eight patients, and I couldnt go to GoFundMe. My family and friends are tired of giving me money, she said.

So Dr. Nizar spoke to experts at conferences hosted by the National Institutes of Health, hoping to find a way to receive research funds. With her enthusiastic prodding, Dr. Jueppner and Dr. Gardella received a grant to study and improve the peptides theyd found as a possible Jansens treatment

In 2018, Dr. Nizar asked the F.D.A. for guidance. They were surprised we didnt have a drug company, she said. I told the lady at the F.D.A. that, at this point, I am the drug company.

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Study by U of T researchers reveals how bacterial toxins evolve to cause new illnesses – News@UofT

Tuesday, July 7th, 2020

The coronavirus pandemic is a daily reminder of the far-reachingconsequences of apathogens successful invasion of human cells. And, as a new University of Toronto study on bacterial toxins shows, it does not take much for these encounters to turn deadly.

The research found that two almost identical bacterial toxins cause distinct illnesses diarrhea and fatal toxic shock syndrome by binding unrelated human receptors. It also highlights a mechanism by which pathogens have evolved distinct receptor preferences to infect different organs.

I always think of bacterial toxins as fascinating machines of death in how they find new ways to enter host tissue, saysMikko Taipale, an assistant professor of molecular genetics at the Donnelly Centre for Cellular and Biomolecular Research.

Taipaleco-led the study,published recently in the journalCell, withRoman MelnykandJean-Philippe Julien, both senior scientists at the Hospital for Sick Children and faculty members in U of Ts department of biochemistry.

Many are familiar withClostridium difficile, a gut-dwelling bacterium that can cause diarrhea. Lesser known is its close relative,Paeniclostridium sordellii, which also lives in the gut and in the female reproductive tract. Infections are rare but fatal and can occur when the bacterial toxin escapes into the bloodstream, during birth for example, and spreads into the lungs and other organs.

Both species are thought to be part of the microbiome, the bodys resident bacteria, but its not clear why they harm some people and not others.

The toxin released byC. difficileacts through Frizzled receptor proteins, which play a role in tissue regeneration. Yet, whileP. sordelliiproduces a similar toxin, it does not bind in the same way so the researchers set out to investigate.

They took an unbiased approach by systematically switching off every gene in human cells and exposing them to theP. sordelliitoxin. Cells that survived turned out to lack genes encoding cell surface proteins called semaphorins, and other experiments confirmed that two members of this class, Semaphorin6A and Semaphorin6B, are indeed the receptors for the toxin. Both receptors are present in the lungs, as expected, though their role there remains unclear.

Knowing the receptor opens the door to finding treatment. The researchers were able to halt infection in mice by co-injecting the toxin with purified semaphorin fragments, which bound and neutralized the toxin before it could reach the real receptors.

But the finding led to more surprises.

Like Frizzled, semaphorins play important roles in the body most notably in developing the nervous system, where they help guide projecting nerve fibers. Even more surprising was that it binds a receptor with no structural resemblance to Frizzled.

Here we have two toxins that are so similar to each other, but they use completely different receptors, says Taipale. We did not expect to find that.

The reason is a tiny differencebetween C. difficileandP. sordellii in particular, the surface though which both toxins contact their receptors, as revealed by cryo electron microscopy. Each toxin protein is composed of about 2,500 amino-acids and the researchers were able to pinpoint those that directly engage with the receptor. Swapping a mere 15 of these amino acids between the two toxins was sufficient to switch receptor preference. In other words, they created a P. sordellii toxin that targeted Frizzled and vice versa.

We were floored when we saw that the toxins shared a surface that each evolved to uniquely interact with distinct cells, says Julien.

It appears that, while the rest of the toxin is under strong evolutionary pressure to remain unchanged, the receptor-binding surface is free from such constraints. This allows toxins to evolve into variants that bind new receptors to invade other tissues and hosts.

Receptor switching is not unique to bacteria, however. SARS-CoV-2 and coronavirus strains that cause common cold use the same part of the now famous spike protein to bind to diverse receptors, which might explain differences in disease severity.

This is a nice example of how viruses and bacteria from completely different domains of life have found similar molecular tactics to change their receptor targets in human cells, says Taipale.

It also reminds us how much cool biology one can find in the microbial world.

The research was supported by the CIFAR Azrieli Global Scholar program, the Ontario Early Researcher Awards programand the Canada Research Chairs program.

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U of T and Hebrew University of Jerusalem launch research and innovation partnership – News@UofT

Tuesday, July 7th, 2020

How did environmental conditions and climate change influence early human evolution? Can protein engineering be harnessed to block the virus that causes COVID-19? How do quantum mechanics affect biological functions, and how do our memory and learning work on a cellular level in the brain?

These are some of the big questions that will be explored by researchers at the University of Toronto and the Hebrew University of Jerusalem (HUJI) as part of a new strategic partnership that will allow faculty and students from the two institutions to combine resources to carry out high-impact research.

Each year, the University of Toronto Hebrew University of Jerusalem Research and Innovation Alliance will select projects to receive funding of $150,000 a year for up to four years, with each research group comprising faculty drawn from both universities and covering a range of disciplines. The alliance will also occasionally provide one-time seed funding to help get promising projects off the ground.

Initially launched with endowed funding of $5.9 million from the Canadian Friends of Hebrew University and the family of Roz and Ralph Halbert, the allianceaims to raise another $14 million and eventually construct an innovation pipeline between U of T and HUJI to connect the entrepreneurship ecosystems in Toronto and Jerusalem and provide student entrepreneurs with exposure to each others universities and markets.

[HUJIs] mandate with respect to research is very closely aligned to U of Ts in terms of leading the world in a variety of areas, and thats always the kind of partner were looking for, said Alex Mihailidis, U of Ts associate vice-president of international partnerships and a professor in the Faculty of Medicine's department of occupational science and occupational therapy, as well as the Institute of Biomaterials and Biomedical Engineering.

We both recognize that international collaborations strengthen the research within each university, and thats why were excited to partner with them.

He added that the timing of the partnership speaks to U of Ts commitment to forge ahead with research partnerships despite the challenges of working and collaborating amid the pandemic.

From an international partnerships perspective, its business as usual, said Mihailidis, who is also cross-appointed to the department of computer science in the Faculty of Arts & Science. Weve not shut anything down and weve not stopped collaborations. Were going full-speed ahead its looking a bit different, but we are still moving ahead both with existing and new partners.

Both researchersdeveloped an interest in the Kalahari Chazan as an archeologist analyzing early evidence of human activity and Matmon as a geologist carrying out dating techniques to study the evolution of the landscape and theyre now looking to combine their perspectives.

The next phase of work with this funding is to expand Aris geological work, particularly looking for evidence of wet environments, so we can try and understand when there was a shift to modern arid conditions, said Chazan. At the same time, Ill be working in the town of Kathu in South Africa, which is a major mining area today, and were looking at some very large sites and trying to understand what the conditions were when this place supported large groups of people.

So its a really new area of study that combines geological perspectives on how the landscape and hydrology evolved with an archeological perspective which is asking in more narrowly focused locations what the human behaviour was and what was drawing people to these sites.

Oron Shagrir, vice-president for international affairs at HUJI, said the partnership brings together the two leading universities in Israel and Canada, and that the call for research proposals resulted in several exciting submissions.

In these challenging and unprecedented times for societies and universities alike, international partnerships are an invaluable source of support and inspiration, said Shagrir, a professor of philosophy and cognitive science. They are not only an important asset and tool in advancing universities on all levels, but also serve as a valuable platform to promote and support collaborative research projects.

Chazan points to his project as an example of how the two universities can combine their respective strengths.

At U of T, were strong in terms of field archeology and geophysics, he said. Hebrew University is particularly strong in looking at the evolution of landforms over the period of the last two to five million years ... [and] that requires some very specialized labs.

Among the labs that Chazan and his students will have access to is a high-tech facility that blocks out any modern magnetic signals to precisely study fluctuations in the earths magnetic field. Having access to that is a major asset for the project and for our students, who get to learn how to operate in that kind of system, said Chazan.

Meanwhile, Sachdev Sidhu, a professor appointed to U of Ts Donnelly Centre for Cellular and Biomolecular Research, the department of molecular genetics and the Institute of Biomaterials and Biomedical Engineering, will be working with Professor Julia Shifman of HUJIs Alexander Silberman Institute of Life Science to study how the fast-growing fields of protein engineering and design can be leveraged to develop treatments for diseases, including COVID-19.

Their project will use insights gained from past outbreaks of coronaviruses to understand the functions of the proteins that power SARS-CoV-2 the virus that causes COVID-19 and to develop molecules with the potential to disarm the virus and pave the way to a potential cure.

Additionally, the U of T HUJI Research and Innovation Alliance is providing $5,000 in seed funding to two projects.

The first will see Professor Dvira Segal of U of Ts departments of chemistry and physics and Professor Roi Baer of HUJIs Fritz Haber Research Center for Molecular Dynamics and Institute of Chemistry explore the role of quantum processes in natural and engineered quantum systems.

The second aims to better understand how the brain acquires and stores information in order to help prevent and treat debilitating memory and learning disorders. The principal investigators are Associate Professors Sheena Josselyn and Paul Frankland of the department of physiology in U of Ts Faculty of Medicine, Professor Melanie Woodin of the department of cell and systems biology and HUJI scholars Adi Mizrahi, Ami Citri and Inbal Goshen.

Ronald Appleby, a U of T alumnus and campaign chair for the partnership, said the research efforts made possible by the partnership speak to the two universities shared commitment to advancing interdisciplinary teams of researchers and students working on translational research, bolstered by mutual respect and friendship.

The attention paid to research in engineering and medicine, the sciences, the social sciences, humanities, and law reflects our mutual interest in creating novel solutions for some of the most pressing current issues, Appleby said.

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Invaio Sciences Announces New License Agreement with the University of California for Novel Technology – PRNewswire

Tuesday, July 7th, 2020

CAMBRIDGE, Mass., July 7, 2020 /PRNewswire/ --Invaio Sciences, Inc., a Flagship portfolio company focused on unlocking the potential of the planet's interdependent natural systems to solve pressing agriculture, nutrition and environmental challenges, today announced a new, exclusive world-wide license agreement with the University of California (UC-Riverside) for a novel technology developed by Professor Hailing Jin at the University of California Riverside.

The technology is proven to control the pathogen that causes HLB/Citrus Greening - a severe plant disease carried by an insect called the Asian citrus psyllid that has dramatically and rapidly threatened the citrus industry by devastating millions of acres of citrus crops throughout the United States and abroad.

"Invaio is enthusiastic to partner with UC Riverside and advance this innovative technology to develop solutions for combating the disease HLB/Citrus Greening," said Dr. Gerardo Ramos, Chief Science Officer at Invaio Science from his Basel, Switzerland offices. "Our novel approach with a focus on natural-based solutions in connection with plant and soil health will revolutionize agricultural practices in far more beneficial ways for the health of people and the planet. This relationship with UC Riverside will allow us to continue to learn from nature and enable biologicals to perform in a sustainable and reliable way. The prospects of addressing this type of incurable devastating crop disease to help agricultural communities and improve the environmental impact of production is exciting and rewarding."

The innovation that Invaio will now license from UC Riverside harnesses naturally-occurring compounds called anti-microbial peptides produced naturally by citrus trees, that have been shown in extensive studies conducted by Dr. Jin, to kill the bacteria responsible for HLB in infected trees, and essentially "curing" infected trees. Dr. Jin, a professor of genetics, microbiology and plant pathology at UC Riverside, studies the molecular mechanisms of plant immunity and pathogen virulence, with the goal of developing effective and environmentally friendly strategies to control plant diseases and to ensure sufficient food production.

This agreement extends Invaio's proprietary capabilities in direct delivery of biologics in trees, to combat this devastating disease. Under the terms of this agreement, UC has granted Invaio an exclusive, world-wide license with the rights to sublicense to this novel technology to develop and deploy products needed by the industry.

Invaio Sciences has pioneered a novel approach to managing insect populations in a more sustainable and targeted way by controlling the nutritional function organ called the obligate microbial symbiont (OMS) to alter insect health. This new approach, coupled with the deep understanding of the inner workings of insects by Invaio's diverse team of scientists, holds the potential to dramatically reduce the need for pesticide use globallybenefiting our agriculture, health and environment.

Invaio's focus on precision delivery allows the company to take a more mindful approach to insect management. For biological delivery, Invaio leverages naturally derived systems that can be used to produce, protect, and deliver a variety of active moleculesall of which are biodegradable. The approach may also include a physical delivery system built upon proprietary 3D-printed injection tips that deliver biological active molecules into a crop vascular system, where they rapidly move throughout the plant for maximum protection.

About Invaio Sciences Invaio Sciences is a multi-platform technology company that unlocks the potential of the planet's interdependent systems to address pressing agricultural, nutritional, and environmental challenges. Founded by Flagship Pioneering in 2018, Invaio leverages discoveries from diverse fields including human therapeutics, agriculture, environmental science, and advanced manufacturing. The company's deep understanding of the physiology of insects, together with its novel approach to managing insect populations in a more sustainable and precise way, promises to refine agricultural practices and reduce the need for pesticides globally. Invaio Sciences is dedicated to developing technology that's mindful of beneficial insects, bad for pests, and safer for us all. For more information, please visit http://www.invaio.com.

Media ContactOgilvy for Flagship [emailprotected] (202) 230-1275

SOURCE Invaio Sciences

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Invaio Sciences Announces New License Agreement with the University of California for Novel Technology - PRNewswire

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Cancer Research UK and British Heart Foundation warn Prime Minister they may have to cut 200m from research funding – Cambridge Independent

Tuesday, July 7th, 2020

Cancer Research UK could be forced to slash 150million per year from its research funding, while the British Heart Foundation anticipates cuts its spend by half from 100million to 50million due to the loss of income caused by Covid-19.

The desperate scenario has prompted more than 60 of the UKs leading cardiovascular disease and cancer research scientists - many of them based in Cambridge - to write an open letter to the Prime Minister calling for urgent financial support for medical research charities.

Cancelled events, such as CRUKs Race for Life, and closed charity shops have decimated income for the charities, which expect a reduction in research funding for the next three to five years.

In the letter, sent today (Tuesday), the scientists warn: This will have a catastrophic and long-lasting impact on both cardiovascular and cancer research in the UK, but also on the broader R&D sector.

They call for swift action to invest in a Life Sciences-Charity Partnership Fund to protect the vital and unique contribution charity-funded biomedical research makes to the UKs R&D ecosystem and the wider economy.

Medical research charities invested 1.9 billion into UK research last year, with CRUK and the BHF alone funding more than half of all non-commercial research in the country into cancer and heart and circulatory diseases.

Professor Sir Nilesh Samani, BHFs medical director, said: Without immediate action, the UKs research base faces a devastating fall in funding that will delay progress in discovering new ways of preventing, diagnosing and treating diseases including heart attack, stroke and vascular dementia. We also risk losing a generation of promising young researchers and diminishing the UKs standing as a world leader in science. We cannot afford to let this happen during a pandemic which has underlined the critical role science and research play in the UKs healthcare and economy.

The call for a Life Sciences - Charity Partnership Fund, now backed by many of the countrys most eminent scientists, is about far more than supporting charities. It would represent a government investment in UK research, returned many times over in terms of the world leading scientific discoveries it enables, the fuel it provides to the UK economy, and the lives that will be saved through the treatments and cures that will follow.

With the Chancellor setting out a plan for the UKs economic recovery tomorrow, stabilising UK science should be at the heart of it.

The letter is signed by scientists including Professor Richard Gilbertson, Li Ka Shing chair of oncology, head of the Department of Oncology and director of the Cambridge Cancer Centre, and Prof Greg Hannon, Royal Society Wolfson research professor of cancer molecular biology and director of the CRUK Cambridge Institute at the University of Cambridge.

It adds: For every 1 the BHF invests, its researchers attract 2.14 of additional funding. CRUK is the second largest licensor in oncology in the world licences that underpin tomorrows medicines and todays investment by companies such as AstraZeneca. Charities have led to the formation of over 40 spin-out companies, which in turn have raised over a 1bn in third party investment and the creation of thousands of jobs.

The idea of a co-investment scheme that provides a level of match funding for future charity research over the next three to five years is supported by the Association of Medical Research Charities (AMRC) and 151 of its charity members.

The AMRC estimates a reduction in UK medical research investment of 310million this financial year.

BHF supports a portfolio of 446million of research at 47 UK institutions, including funding more than 1,700 researchers, hundreds of whom are in the early stages of their scientific career.

CRUK spent 422million on cancer research in 2018/19 and has 90 institutions in more than 40 UK towns and cities, including its major institute in Cambridge. It funds about 4,000 researchers in labs and hospitals across the UK, including more than 500 PhD students.

The letter warns Boris Johnson: Without your support, the UK risks a slide backwards, undermining decades of pioneering and life saving research, as well as losing a generation of new researchers and a major decline in our nations international competitiveness in life sciences that we have worked so hard to achieve.

Also among the signatories are Prof Martin Bennett, BHF professor of cardiovascular sciences at the University of Cambridge, Prof Jason Carroll, professor of molecular oncology and senior group leader at the CRUK Cambridge Institute, Prof John Danesh, of the University of Cambridge; Wellcome Sanger Institute and Health Data Research UK-Cambridge, Prof Gerard Evan, of the Department of Biochemistry at the University of Cambridge, Prof Kay-Tee Khaw of the University of Cambridge and Prof Ziad Mallat, BHF chair, professor of cardiovascular medicine at the University of Cambridge.

The letter in full

Dear Prime Minister,

We are writing to you as experts and leaders in the fields of cardiovascular and cancer research, and as British Heart Foundation (BHF) and Cancer Research UK (CRUK) funded academics carrying out research at leading UK universities, research centres and institutes. We wish to highlight the significant impact COVID-19 has had on the medical research sector and to urge you to take swift action to invest in a Life Sciences-Charity Partnership Fund to protect the vital and unique contribution charity- funded biomedical research makes to the UKs R&D ecosystem and the wider economy.

The biomedical research sector is key to the health and the wealth of the nation. We therefore welcomed the Governments commitment to make the UK the leading global hub for life sciences. We believe that the sector can be one of the key engines that will help boost the UK economy through COVID-19 recovery, leaving the EU, and beyond. Recent funding announcements, such as the university research support scheme, are positive first steps to help achieve this ambition, but we remain concerned they will not fully address the significant shortfall in charity investment in the UK science base.

The funding provided by charities plays a unique role within the wider funding mix, supporting high- risk discovery science that drives the breakthroughs in our fields (and others), as well as de-risking projects to attract commercial investment and supporting clinical trials that bring the latest innovations and life-saving treatments to patients. Charity funding invests in skills and has supported many of us earlier in our careers, allowing us to continue to build our expertise within the UK system, adding to the strength of its research base and building the UKs global reputation for research excellence.

However, both the BHF and CRUK are now seeing their incomes decimated by the COVID-19 pandemic. This will have a catastrophic and long-lasting impact on both cardiovascular and cancer research in the UK, but also on the broader R&D sector. CRUK could be forced to cut 150 million per year from its research funding, and the BHF anticipates having to cut its research spend by half this year from 100 million to around 50 million. This means a significantly lower investment in life saving high quality research, in skills and in infrastructure.

To put this challenge into context, last year, medical research charities invested 1.9 billion in research in the UK. CRUK and BHF alone funded 50% and 55% of all UK-based independent research into cancer and heart and circulatory diseases respectively something that will not be possible to maintain for the foreseeable future. Given that level of contribution, such a drastic reduction in their capacity to fund research will have a dramatic impact on the national investment in research in two key areas of unmet medical need.

Moreover, this reduction will have a knock-on impact on our partners in industry and on the inward investment into the UK life science sector. For example, for every 1 the BHF invests, its researchers attract 2.14 of additional funding. CRUK is the second largest licensor in oncology in the world licences that underpin tomorrows medicines and todays investment by companies such as AstraZeneca. Charities have led to the formation of over 40 spin out companies, which in turn have raised over a 1bn in third party investment and the creation of thousands of jobs. The Association of Medical Research Charities (AMRC) estimates a reduction in UK medical research investment of 310 million this financial year alone which will severely impact the UKs internationally recognised strength in the life sciences.

While charities are doing all they can to support existing research, we are gravely concerned for the future of biomedical research in the UK, and we fear that the latest research support package will not go far enough to plug the funding gap charities are facing. We urge you to support the proposal from CRUK and the BHF, as well as 151 other medical research charities and AMRC, to work in partnership with the sector to establish a Life Sciences-Charity Partnership Fund. This co-investment scheme is urgently needed to help protect and invest in world-class research across the UKs four nations, whose quality is assured through a stringent peer-review process, and could provide matched funding from Government for future charity research over the next 3-5 years (the period in which funding is expected to be most acutely affected).

Without your support, the UK risks a slide backwards, undermining decades of pioneering and life saving research, as well as losing a generation of new researchers and a major decline in our nations international competitiveness in life sciences that we have worked so hard to achieve. This will diminish our reputation as a world-leader in developing medical breakthroughs that save lives lost to cancer, heart and circulatory diseases and countless other conditions. We ask that you reaffirm your commitment to science and research by investing to support medical research charities in this time of need.

Signed,

Cc:

Rt Hon Rishi Sunak MP, Chancellor of the Exchequer Rt Hon Alok Sharma MP, Secretary of State for Business, Energy and Industrial Strategy Rt Hon Matt Hancock MP, Secretary of State for Health and Social Care

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Revealed: 220m vision for Cambridge Cancer Research Hospital

Leading the fight against children's brain tumours: Prof Richard Gilbertson on CRUK's new centre

Cancer Research UK Cambridge Institute developing liquid biopsies for detecting brain tumours

Prof Greg Hannon on taking over at the Cancer Research UK Cambridge Institute and creating the worlds first virtual reality tumour

Prof Ziad Mallat leads Cambridge effort to win 30m to tackle leading cause of heart attacks and strokes

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Cancer Research UK and British Heart Foundation warn Prime Minister they may have to cut 200m from research funding - Cambridge Independent

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Fine-tuning brain activity reverses memory problems in mice with autism mutation – Spectrum

Tuesday, July 7th, 2020

Social circuitry: Mice with an autism-linked mutation have better social memory after treatment that calms a related neural circuit.

Georgejason / iStock

Dampening overactive brain circuits alleviates social and spatial memory problems in a mouse model of 22q11.2 deletion syndrome, according to a new study1. The findings hint at the possibility of novel treatments for some difficulties associated with the syndrome.

Deletions of DNA in a chromosomal region known as 22q11.2 often cause intellectual disability or other cognitive difficulties, as well as psychiatric conditions such as schizophrenia. About 16 percent of people with the deletion also have autism2.

The type and severity of traits vary from person to person, in part because the deletion can span roughly 20 to 50 genes. That range makes it difficult to design targeted therapies. And many people with deletions in 22q11.2 are prone to drug-related side effects, such as seizures.

Side effects with drug treatment is one of the hardest parts of dealing with mental illness, says Julia Kahn, a postdoctoral researcher at the Childrens Hospital of Philadelphia in Pennsylvania, who worked on the study. Being able to circumvent that in a very directed manner would be really life-changing for a lot of people.

The study identifies the neural circuits responsible for select behaviors in model mice and shows that manipulating those circuits could offer a new treatment strategy.

It suggests that therapies can be symptom specific, says lead investigator Douglas Coulter, professor of pediatrics and neuroscience at the University of Pennsylvania in Philadelphia.

Coulter and his colleagues manipulated circuits in two regions of the hippocampus in 22q11.2 model mice: the ventral region, which governs social memory, and the dorsal region, involved in spatial memory. They focused on the hippocampus because it is important to social cognition in both mice and people, and previous studies have shown it is unusually small in people with 22q11.2 deletions3.

Before the manipulation, mice missing 22q11.2 perform worse than controls on tests of their social and spatial memory, the study shows. The mice do not distinguish between a new mouse and one they have already met, and they have trouble recognizing when an object in their cage has been moved. Brain imaging also showed that the model mice have overactive neurons in the hippocampus.

The team used a method known as chemogenetics to dampen this overactivity. They gave the mice an injection that prompts some neurons in the hippocampus to produce designer receptors. They then injected the animals with an experimental drug that binds only to those receptors, making the neurons less excitable.

The animals behaviors changed, depending on where they received the injection. Social memory improved when the drug targeted the ventral hippocampus, and spatial memory improved when the drug affected neurons in the dorsal area. Too much inhibition in either area caused the animals memory problems to return.

Using the same technique, the researchers also gave control mice drug-sensitive receptors that either activate or quell the same circuits in the hippocampus. After both treatments, the controls showed the same social memory problems as the mice with 22q11.2 deletions. The results indicate that disrupting the circuits in either direction is enough to change behavior, even without any underlying genetic mutations. The findings were published in May in Biological Psychiatry.

Chemogenetics is a long way off from use in people, but drugs currently on the market may be able to achieve similar outcomes by nudging circuits into a more balanced state, says Peter Scambler, professor of molecular medicine at University College London in England, who was not involved in the work.

Its a proof of principle, he says.

Manipulating circuits that govern specific behaviors should be a goal of all current work at this point, says Anthony LaMantia, professor of developmental disorders and genetics at Virginia Polytechnic Institute and State University in Blacksburg, who was not involved in the work. This is much more targeted and precise. It should make everybody in the field think through how to design their experiments.

Targeting circuits in the hippocampus could help people, because findings in the hippocampus in mice typically translate well to humans, says Rebecca Piskorowski, head of the synaptic plasticity and neuronal circuits team at the Institute of Psychiatry and Neuroscience of Paris in France, who was not involved in the work.

This kind of targeting might also help at any age. The method improved memory in adult mice, suggesting similar treatments could help older people with 22q11.2 deletions and not just children.

This paper shows if you just adjust the activity in a tiny little place, you can somehow compensate for all those developmental problems, Piskorowski says. That is particularly exciting.

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Fine-tuning brain activity reverses memory problems in mice with autism mutation - Spectrum

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University of Wisconsin forms new animal, dairy science department – Feedstuffs

Tuesday, July 7th, 2020

The University of Wisconsin-Madison College of Agriculture & Life Sciences (CALS) announced July 6 the formation of the department of animal and dairy sciences, effective July 1, 2020.

The former department of animal sciences and department of dairy science, which have been operating as a single unit since early 2019, will dissolve after positions are transferred to the new department, CALS said.

After exploring the possibility of a merged unit in fall 2018, the two departments voted to begin sharing administrative roles that December. In January, they voted to have a single leadership structure as well.

Kent Weigel, a dairy cattle geneticist who has been the chair of dairy science since 2010, became the chair of both departments, and Hasan Khatib, a molecular geneticist who was serving as associate chair of animal sciences, became the associate chair of both departments. They will continue in these roles in the new department.

While the two departments have been separate units since 1962, they have similar missions and commonalities that include animal health and welfare, nutrition, reproduction, lactation, cattle genetics and genomics, animal biologics and basic science, CALS said, noting that becoming one department will lessen administrative burdens and foster unique, collaborative research. The undergraduate and graduate academic programs will remain the same, with the single department offering majors in animal science and dairy science.

The departments prepared a formal proposal that they voted on in November 2019. The proposal was approved by the CALS Academic Planning Council and University Academic Planning Council this spring.

The formation of the department of animal and dairy sciences aligns with recommendations made by the CALS organizational Redesign Committee in 2017. With these changes, the college now has 16 academic departments.

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University of Wisconsin forms new animal, dairy science department - Feedstuffs

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A Brighter Tomorrow > News > USC Dornsife – USC Dornsife College of Letters, Arts and Sciences

Tuesday, July 7th, 2020

From environment to family, transportation to health care, from work and leisure to what well eat and how well age, USC Dornsife faculty share how they think our future world will look. [11 min read]

As the 19th century drew to a close and a new era dawned, an American civil engineer named John Elfreth Watkins consulted experts at the nations greatest institutions of science and learning for their opinions on 29 wide-ranging topics. Watkins, who was also a contributor to the Saturday Evening Post, then wrote an extraordinary magazine article based on what these university professors told him.

Published on Page 8 of the December 1900 issue of Ladies Home Journal a sister publication of the Post it was titled What May Happen in the Next Hundred Years. Watkins opened the article with the words, These prophecies will seem strange, almost impossible. In fact, many of his far-sighted predictions for the year 2000 which included the invention of digital color photography, television and mobile phones proved remarkably accurate.

For this issue of USC Dornsife Magazine, we have repeated the experiment by inviting 10 scholars drawn from USC Dornsife faculty and representing diverse disciplines to predict what the world will look like in the year 2050 and the year 2100.

A Bluer Planet

Astronauts circling the globe in 80 years may find our blue planet looking quite a bit bluer, says Naomi Levine, assistant professor of biological sciences and Earth sciences.

The middle of the Pacific or Atlantic oceans are what we call the deserts of the ocean. Theyre really low in nutrients, and things that live there are usually small. As a result, these areas look very blue because there isnt much ther except water, Levine explains. As the climate warms, we predict that these desert areas are going to expand. So, ocean waters will look bluer from space.

A Brighter Shade of Green

Our planet may also look a bit greener. Travis Williams, professor of chemistry, says that without an active plan for removing the carbon clogging our atmosphere, nature could step in.

If we dont choose a biomass thats going to utilize higher temperatures and that atmospheric carbon, nature is going to choose on our behalf, and I dont think were going to like it, he says. To avoid harmful organism explosions like algae blooms, Williams foresees a human-led reforestation of the planet, at a scale several times the size of the Amazon rainforest.

What's On the Menu?

A greening planet could also be due to changes in our agricultural systems. A move away from monoculture farming and a return to an ancient polyculture approach might be on the horizon, says Sarah Portnoy, associate professor (teaching) of Spanish. Portnoy researches indigenous food cultures of Mesoamerica and suggests that in the future we could adopt the milpa food system. Animals would be grazing on the same land where there are cover crops and squash, corn, beans and all kinds of herbs growing together, she says.

This isnt just a utopian pipe dream. Governments will have to seriously rethink agriculture if they want to reduce rising rates of chronic disease such as obesity, especially among the poor. The agriculture that is supported by the government now is skewed toward crops like soybeans and wheat. Our food system is geared to the cheapest calories, Portnoy says.

The high-calorie, processed foods produced from these monoculture, subsidized crops are less expensive than fruits and vegetables, but do little for our health. Unless we reprioritize which crops get government cash, we can expect disparities in health between economic classes to continue. By 2050, only the privileged might be able to afford strawberries or carrots.

Food supplies will alter in other ways as well, thanks to climate change. The bluer oceans will be less friendly to bigger marine organisms, which means fewer large fish to harvest.

When you change ocean temperatures, it changes what types of organisms can grow, and that cascades up the food web, says Levine. Sushi chefs in 2050 might dish up more avocados and scallops than tuna rolls. This could work for future diners, Portnoy thinks. Theres a move toward being a lot more intrepid as an eater, and toward plant-based diets, she says.

One Big, Happy Family

Starting off your day in 2050 could mean wheeling your toddler to the state-funded neighborhood day care center. Birth rates are currently plummeting across the industrialized world and governments may soon need to tackle the problem as a public health priority, says Darby Saxbe, associate professor of psychology and director of the USC Center for the Changing Family.

Well realize that, when the birth rate goes down, that affects our future workforce, she says. When were not able to replace our population, it ultimately becomes a national security issue. Child care benefits, family leave and subsidized, part-time work schedules for parents could be the governments strategy to encourage a new baby boom.

We may be well into the digital age, but you might not find too many iPads in the nurseries of the future. Increased awareness of the pitfalls of screen time could change our approach to parenting via device. The original scions of social media themselves now admit to limiting their own childrens time online, observes Saxbe. In fact, in some of the more expensive private schools in Los Angeles, you have to sign a no screen time pledge.

The keywords there might be expensive and private. A movement away from childhood spent online could leave behind children from poorer families as technology becomes cheaper and the cost of human labor rises. It will likely soon be less expensive to instruct classrooms of kids via lessons on tablets than by engaging a human teacher.

You might end up with a two-class system, Saxbe warns. You have more kids having a digital childhood thats a little less regulated, especially in neighborhoods where its not safe to play outside. Wealthier families are going to be able to afford more hands-on child care and more hands-on educational activities, instead of leaving kids alone with their technology.

However, technology can still benefit the family in the coming decades. In fact, Saxbe believes this is a largely untapped opportunity with great potential. Silicon Valley technologists primarily childless young men still havent tackled devices like the breast pump or baby monitor, which could both use a redesign.

Has there been a real focus on innovation and investment when it comes to things that serve parents and families yet? asks Saxbe. I think theres a big market there.

Working 10-4

After dropping your child off at day care, you head to work. You likely wont be putting the keys in the ignition of your own car, though. Kyla Thomas, sociologist at the USC Dornsife Center for Economic and Social Research and director of LABarometer, a quarterly internet-based survey of approximately 1,800 L.A. county residents, says that by 2030 commuters will probably rely more on public transit and shared, autonomous vehicles to get around.

Public transportation will be faster and more convenient, and increased density in neighborhoods will mitigate sprawl. Parking will be more expensive and harder to find. By 2100, Thomas says, private car ownership will be a thing of the past.

Hopping out of your driverless commuter van, you clock in at the office for your six-hour work day. Patricia Grabarek, lecturer with USC Dornsifes Online Master of Science in Applied Psychology program, believes that the traditional 40-hour work week could get phased out by 2050.

We are in the midst of a job revolution thats on the scale of the Industrial Revolution, Grabarek says. The entire nature of work will change.

Automation promises to replace many jobs, and streamline others. Combine this with the growing emphasis on work-life balance, embodied by current millennials pushing for workplace flexibility, and we could see our work week lighten in load.

Our leaders are recognizing the problem that employees are burning out. People are working too much and they are not as productive as they could be. Bosses will start modeling better behaviors for their employees, Grabarek says. After-hours emails could soon be banned, as is already the case in France and Germany.

This doesnt mean well all be aimlessly underemployed, however. There is a fear that automation will eliminate jobs but, in the past, weve always replaced the jobs that weve lost. Innovators will come out and replace them with new jobs we cant even come up with now, she says.

No matter how advanced computers become, human curiosity remains superior. Automation will be good at analyzing data, Grabarek says, but the questions will still originate with human researchers.

It's Quitting Time

Finished with work for the week, youre off to start the weekend. One item not likely to be on the agenda? Attending a traditional religious service.

In the United States, theres a trend away from institutionalized religion and toward highly individualized spirituality, says Richard Flory, associate professor (research) of sociology and senior director of research and evaluation at the USC Dornsife Center for Religion and Civic Culture. People just arent interested in institutions anymore, and nothing seems to be stepping forward to replace that interface between the individual and society.

Churches and temples could find new life as condos, bars or community centers, with religion relegated to a decorative background.

Rather than kneeling in prayer, people might find themselves downing a psychedelic drug to reach personal spiritual enlightenment. Movements that center around hallucinogens such as ayahuasca, a psychoactive tea from the Amazon, have gained traction in recent years, Flory notes.

Of course, there might just be an app for it all. Consciousness hacking aims to use science to bypass years of devotion to a spiritual practice and give everyone the hard-won benefits of such a practice instantly. In the future, I could see having some sort of implanted device to get to this level of consciousness, Flory says.

Reading the Tea Leaves

You may also use your leisure time to crack open a good book one with a slightly different texture. As climate change threatens our traditional resources, more sustainable alternatives such as seaweed could step in as a paper substitute, predicts Mark Marino, professor (teaching) of writing and a scholar of digital literature.

By 2100, literature could be written across the heavens instead.

Roboticist poets will create autonomous micro-texts that will be able to swarm into collectives, self-organize, aggregate and adapt, says Marino. Bevies of these nano-rhy-bots will create superstructures that can write epics on the Great Wall of China, on the surface of Mars or in the bloodstream of their readers.

Better Living Through Quantum Computing

Aging in the New Age may mean more nontraditional family units. Older adults prefer to age and die at home, but what happens when you dont have a big family network to support that? It may mean people might be more invested in friend networks, or the idea of chosen family, says Saxbe. Cue The Golden Girls theme song.

Sean Curran, associate professor of gerontology and biological sciences, believes that a focus on increasing our health span, the period of life during which one is free from serious disease, rather than simply elongating our life spans, will improve the quality of our longer lives as we age.

The goal is to have a personalized approach to aging that takes into account an individuals genetics, environment and life history, explains Curran. The assisted living facility of the future will be patient-centered, with each resident having a personalized prescription to maintain optimal health.

Eli Levenson-Falk, assistant professor of physics and astronomy, predicts that quantum computing could unlock the development of those drugs.

Quantum computers solve problems much more swiftly and with higher information density than todays computers. Although the technology is still in its infancy, Levenson-Falk predicts that by 2050, practical quantum technologies will be used commercially by major drug companies for research and development.

Enormously complicated computational tasks like simulating a chemicals molecular structure are much more achievable through this technology.

The idea is that with a quantum computer you can sort of emulate nature, he explains. We might have the canonical example for this by 2050: the physical shape of a protein molecule.

Predicting this shape is nearly impossible with a classical computer, Levenson-Falk says.

Measuring it is difficult and requires you to predict the shape first. With a good quantum simulator, we can emulate the protein and just let quantum mechanics do the processing for us, then measure the result at the end.

The Quantum Age

Indeed, quantum computing might solve questions that relate to the very fabric of the universe. Or at least get us closer to the answers.

Dark energy, dark matter, quantum gravity and thequantum classical transition are the principle problems existing in physics today. Quantum technologies are the best bet to solve the last one, says Levenson-Falk. Quantum sensors will probably also be used to help detect dark matter, or at least falsify some theories. And there are some proposals for using quantum technologies to poke at quantum gravity.

We cannot, of course, predict our shared future with 100 percent accuracy, but one thing we can be sure of is that it will be filled with new challenges and opportunities to create a better tomorrow. Although advances in technology will certainly help determine our future, how equitably those advances are shared in our interconnected world will also play a dominant role in shaping it.

This is a tale of two societies: You could either see things get better and more supportive for families, or you might see two-class stratification, Saxbe warns.

As the future unspools, we are given both the invaluable gift and the tremendous responsibility of deciding how we want it to look. Whether our world in 2100 takes on the dystopian qualities of Blade Runner or embodies the utopian, egalitarian ideals of Star Trek remains in the terrestrial hands of those already building that future.

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A Brighter Tomorrow > News > USC Dornsife - USC Dornsife College of Letters, Arts and Sciences

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ViraxClear Receives ISP Approval for ViraxClear Rapid Test Kit Distribution to Chile With Signed LOI for up to 3 million Test Kits – GlobeNewswire

Tuesday, July 7th, 2020

VANCOUVER, British Columbia, July 07, 2020 (GLOBE NEWSWIRE) -- Global Care Capital Inc. (CSE: HLTH, FRANKFURT: L6V1) (the Company or Global Care) a global investment company which engages in early stage investment opportunities in private and public companies, is pleased to announce that its portfolio company, ViraxClear, through its joint venture, Shanghai Biotechnology Devices Ltd. (SBD) has received approval on July 3rd, 2020 from El Instituto de Salud Pblica de Chile (ISP) for the distribution of COVID-19 Rapid Antibody Test Kits (Test Kits) supplied by its previously disclosed manufacturing partners, Innovita Biological Technology Co., Ltd and Vazyme Biotech Co., Ltd (Innovita, Vazyme), for the Chilean market. ViraxClear has signed a non-binding LOI with Biosonda Biotecnologia (Biosonda) for the distribution of up to 3 million Test Kits for the Chilean market, a contract valued at up to $17.25 million in gross revenue.

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/f21c6f66-8b2f-4b6c-a13d-fd8e9822d37b

Distribution Contract for Test Kits in Chile

ViraxClear has received initial orders for trials and market testing of their Test Kits for the Chilean market and manufacturing is expected to commence over the next 60 days. The LOI provides for a 60-day exclusivity period during which ViraxClear and Biosonda intend to sign a definitive agreement with a total contract value of up to $17.25 million.

Biosonda is a biotechnology company founded in Chile in 1992 by leading scientists. It is currently one of the main distribution companies of products for scientific research and clinical diagnosis in Chile, with a recognizable corporate image in the national market. Biosonda works with some of the top global brands in the clinical and diagnostic industries and handle everything from marketing, import, logistics, research and development and sales.

The Biosonda commercial department is made up of a sales force, with a high degree of specialization and extensive experience in the market made up of experts in molecular biology and immunology. They offer a technical service laboratory for equipment maintenance, repair and training, a personalized after-sales service, and a commercial-logistics team that manages the tasks of quotation, import, logistics, inventory, orders and dispatch to provide an optimum service.

Alex Somjen, CEO of Global Care Capital Inc., stated, This distribution deal represents the culmination of months of negotiating by ViraxClear with top distributors in key global regions. Biosonda is in a perfect position to roll out mass testing for the region with Chile already seeing 288,000 confirmed cases of COVID-19 and the population requiring Antibody Testing once the curve has flattened.

ISP Approval

The Chilean government require medical testing devices to receive ISP Approval before they can be sold in Chile. Both Innovita and Vazyme, ViraxClear suppliers, are now on the list of ISP Approved factories, allowing ViraxClear tests to be imported and sold nationally.

Established in 2006, Innovita is a Chinese high-tech enterprise specialized in R&D, manufacturing, marketing and after-sales service of In-vitro diagnostic tests. The CE and NMPA (formerly known as CFDA) approved Innovita serology tests have performed particularly well during preliminary clinical tests carried out by the COVID-19 Testing Project; a multidisciplinary team of researchers and physicians at UCSF, UC Berkeley, Chan Zuckerberg Biohub and Innovative Genetics Institute. According to their studies, Innovita tests scored a 96.3 % specificity (NPV); 83.3% Sensitivity (PPV) with an IN-HOUSE ELISA test (gold reference standard) giving 99.1 % specificity (NPV); 81.8 % Sensitivity (PPV): https://covidtestingproject.org/

Vazyme, which isa highly reputable producer of enzymes and antibodies with products covering clinical diagnosis, molecular diagnostics, high-throughput sequencing and life science research, as well as RNA sequencing, enabling customers and reagent manufacturers to get enzymes with higher resistance. Vazyme owns a dedicated 8000 m2 R&D site and have established the Biotechnology Industry Research Institute, which is comprised of a team of nearly 100 scientists.

The Test Kits are intended for the qualitative detection of IgG and IgM antibodies against 2019 Novel Coronavirus, produced by the immune system after virus infection. IgM is the earliest antibody that appears upon the first immune response, with detection indicating an early stage infection. IgG is produced later and lasts a long time in the body, indicating a prior infection. The combination of the two markers offers an insight into what stage the virus has reached.

About ViraxClear

ViraxClear focuses on commercializing novel products that address significant healthcare needs with a specifictarget on the novel coronavirus (COVID-19). The companys main focus is marketing its ViraxClear Rapid IgM-IgG Combined Antibody Test. The ViraxClear Rapid IgM-IgG Combined Antibody Test for COVID-19 is a lateral flow immunoassay used to qualitatively detect both earlyand late marker IgG/IgM antibodies.

http://www.viraxclear.com

info@viraxclear.com

About Global Care

Global Care Capital is a global investment company which specializes in providing early stage financing to private and public companies. The Company engages in new, early stage investment opportunities in previously underdeveloped assets and obtaining positions in early stage investment opportunities that adequately reflect the risk profile.

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GLOBAL CARE CAPITAL INC.:

Company Contact:

Alex Somjen, President & CEO

asomjen@globalcarecapital.com

Neither the CSE nor its regulation services provider accepts responsibility for the adequacy or accuracy of this release.

Forward-Looking Information: This news release includes certain statements that may be deemed forward-looking statements. The use of any of the words anticipate, continue, estimate, expect, may, will, would, project, should, believe and similar expressions are intended to identify forward-looking statements. Although the Company believes that the expectations and assumptions on which the forward-looking statements are based are reasonable, undue reliance should not be placed on the forward-looking statements because the Company can give no assurance that they will prove to be correct. Since forward-looking statements address future events and conditions, by their very nature they involve inherent risks and uncertainties. These statements speak only as of the date of this News Release. Actual results could differ materially from those currently anticipated due to a number of factors and risks including various risk factors discussed in the Companys disclosure documents which can be found under the Companys profile on http://www.sedar.com

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ViraxClear Receives ISP Approval for ViraxClear Rapid Test Kit Distribution to Chile With Signed LOI for up to 3 million Test Kits - GlobeNewswire

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Idorsia announces positive results in the second Phase 3 study of daridorexant – GlobeNewswire

Monday, July 6th, 2020

Allschwil, Switzerland July 6, 2020Idorsia Ltd (SIX: IDIA) today announced positive top-line results of the second pivotal Phase 3 study investigating 10 and 25 mg doses of its dual orexin receptor antagonist, daridorexant, in 924 adult and elderly patients (39.3% 65 years) with insomnia. The study confirms the findings of the first pivotal study, demonstrating efficacy of treatment with daridorexant on objective and subjective sleep parameters and showed positive effects on daytime functioning, with patients reporting no morning sleepiness and no evidence of rebound or withdrawal symptoms upon treatment discontinuation.

On April 20, 2020, the company reported (media release) the results of the first pivotal study with daridorexant where both 25 and 50 mg daridorexant significantly improved both sleep onset and sleep maintenance. Daridorexant 50 mg also significantly improved daytime functioning. All results were sustained over the 3 months of the trial.

In the second study, daridorexant 25 mg significantly improved sleep maintenance as measured objectively in a sleep lab by polysomnography. Daridorexant 25 mg also significantly improved subjective total sleep time as measured daily with a patient diary at home. The results were statistically significant at month 1 and at month 3 for these sleep measures, showing sustained benefit.

Furthermore, the effect of daridorexant 25mg on sleep onset and daytime functioning were numerically consistent with the effects seen in the first study. However, due to the control of the Type 1 error rate for 16 comparisons, these endpoints despite the low p values did not reach statistical significance.

The 10 mg dose of daridorexant showed numerical improvements, across all efficacy measures, of a smaller magnitude than observed on 25 mg, none of which reached statistical significance.

The results of the two large pivotal studies, testing daridorexant at three doses from 10 to 50 mg, now provide a deep understanding of its efficacy and tolerability profile. Furthermore, the similar design of the two Phase 3 studies allows for the twogroups of25 mgandplaceboto be pooledanda pre-planned analysis to be made. This pooled analysis willfurthercharacterize the effect of daridorexant.

Guy Braunstein, MD and Head of Global Clinical Development of Idorsia, commented:

I want to start with a thank you to the study participants, investigators and their support staff, and the Idorsia team for delivering another comprehensive set of robust data. I am delighted to see the replicated effect of 25 mg of daridorexant in this large confirmatory study. The consistency of the treatment effect across both studies is remarkable. I believe the fact that daridorexant improves daytime functioning is a real breakthrough for patients. I am looking forward to the integration of all aspects of the program, including the pooled data, the long-term extension data, the clinical pharmacology program, and all that we can learn from the patient reported outcome instruments. There is a lot of work for us to do as we interact with the health authorities and share the data with the scientific community.

About safety in the studyThe safety profile was consistent with the results of the first study. Treatment-emergent adverse events (TEAEs) during the double-blind study period were reported in 38.2% and 39.3% of the patients treated with 10 and 25 mg daridorexant, respectively (32.7% for placebo). The most frequent TEAEs reported over 3% incidenceand higher on 25 mg of daridorexant than placebo were nasopharyngitis, headache, somnolence and fatigue. The number of patients experiencing serious adverse events was low and balanced across treatment groups (10 mg, 3 patients; 25 mg, 3 patients; placebo, 4 patients). Based on independent blinded adjudication committee assessment, the number of patients reporting excessive daytime sleepiness as AE was low (10 mg, 1 patient; 25 mg, 4 patients; and placebo, 1 patient); 3 patients had AEs of special interest related to sleep paralysis and hallucination. No events denoting cataplexy-like events were reported or adjudicated. There was no next-morning residual effectas assessed every morning by the patients using a visual analog scale; 2 patients reported suicidal ideation (10 mg, 1 patient; 25 mg, 1 patient) with clear alternative causes; no suicide or self-injury were observed. There was no evidence of rebound insomnia, and no withdrawal symptoms upon discontinuation.

EmmanuelMignot,MD and Professor of Psychiatry and Behavioral Sciences at Stanford University, commented:The daridorexant program demonstrates the full potential of orexin receptor antagonism excellent effect and a good safety profile. It is exciting to see this, 20 years after the discovery of the role of orexin in sleep regulation. For me, the improved daytime functioning seen with daridorexant is most impressive. What is important to patients is not only to improve their night sleep but also how they feel during the day. By measuring the benefits of the drug through the day as well as through the night, the program has put patients back at the center of the equation and raised the standard for what we need to see with sleep medications. This ensures the patient need is at the center of prescription decisions when treating insomnia.

Jean-Paul Clozel, MD and Chief Executive Officer of Idorsia, commented:I was stunned by the excellent results of the first study with daridorexant, this time Im struck by the consistency of the efficacy results, including daytime functioning and the safety profile. I am very proud of the great science behind daridorexant and that Idorsia has designed and executed such a comprehensive program, focused on patients, in such a short time. I am convinced that with daridorexant, Idorsia has a unique drug which is going to have a disruptive impact on the insomnia market. The whole company is united in the effort to file the NDA with the US FDA around the end of this year and to prepare for a successful launch. There is certainly a lot of work to be done, but we are already making great progress on all fronts.

Detailed results of the Phase 3 studies will be made available through scientific disclosure at upcoming congresses and in peer-reviewed publications.

About the Phase 3 registration program

The Phase 3 registration program comprises two confirmatory studies of 3-month duration, together with a long-term extension study. Both pivotal studies are complete, having enrolled around 1,850 patients with insomnia at over 160 sites across 18 countries. As insomnia often presents later in life, around 40% of the recruited population was aged 65 years or older. The confirmatory multi-center, double-blind, randomized, placebo-controlled, parallel-group, polysomnography studies investigated three doses of daridorexant (10 mg, 25 mg, and 50 mg) on sleep and daytime functioning parameters, objectively in a sleep lab by polysomnography and subjectively with a daily patient diary at home. The impact of insomnia on patients daytime functioning was measured daily using the sleepiness domain score from the Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ) a Patient Reported Outcome (PRO) instrument, validated according to the US Food and Drug Administration (FDA) Guidance for Industry. 806 patients decided to continue treatment in the ongoing 40-week extension study which will measure the effect of all three doses vs. placebo, generating data for long-term treatment of insomnia.

Investor webcastOn April 20, 2020, the company held an investor webcast to discuss the results of the first Phase 3 study with daridorexant. On that occasion, Martine Clozel, MD, Chief Scientific Officer presented a brief overview of the tailored drug discovery efforts that led to the synthesis of daridorexant. This was followed by Guy Braunstein providing an overview of insomnia, the objectives of the Phase 3 program, the methodologies used to measure the effect of daridorexant on patients with insomnia, and the results of the first study. This webcast is available for replay on the corporate website.

The company will hold an investor conference call and webcast to discuss the results of the second Phase 3 study with daridorexant. On the call, Guy Braunstein will present the study results, followed by a Q&A session with Jean-Paul Clozel, Guy Braunstein, and Martine Clozel.

Date: Monday July 6, 2020Time: 14:00 CEST | 13:00 BST | 08:00 EDT

Webcast participants should visit Idorsia's website http://www.idorsia.com 10-15 minutes before the webcast is due to start.

Conference call participants should start calling the number below 10-15 minutes before the conference is due to start.

Dial-in CH: +41 (0)44 580 65 22 / UK: +44 20 3009 2470 / US: +1 (877) 423-0830PIN: 24890393#

Notes to the editor

About insomniaInsomnia is a condition of overactive wake signaling that can have a profound effect on the lives of patients. Insomnia can be defined as difficulty falling asleep and / or staying asleep, occuring at least three times a week for a minimum of three months.

It is estimated that as many as one in ten people suffer from insomnia and its impact is often underestimated. In reality, it can be a distressing condition that can impair quality of life. Sleepless nights can leave people feeling irritable and out of sorts this may affect many aspects of daily life, from studying and employment to social activities and relationships. People who suffer from insomnia may lack the energy or motivation to exercise or to take part in social activities. It can also have a significant economic impact as it increases the risk of accident and injury on the road or in the workplace, and is a leading cause of absenteeism and reduced productivity at work. People with insomnia are more likely to experience feeling down or depressed, lack concentration, and suffer from poor energy levels during the day compared with people who sleep well. In addition, worrying about sleep can cause stress and may lead to negative thought patterns which may in turn make it more difficult to sleep, setting up a vicious circle. Chronic insomnia is associated with cardiovascular and cerebrovascular diseases, and increased mortality.

The goal of treatments for insomnia is to improve sleep quality and quantity, as well as reducing insomnia-related impaired daytime functioning, while avoiding adverse events and next morning residual effect. Current treatment of insomnia includes cognitive behavioral therapy, sleep hygiene recommendations, and pharmacotherapy. The most widely prescribed products on the market that are indicated for insomnia enhance the effects of gamma-aminobutyric acid (GABA), the major inhibitory neurotransmitter in the central nervous system. Such medications are only approved for short-term use and are associated with side effects such as next-morning residual effects, anterograde amnesia, and risk of tolerance and dependence.

About the orexin systemWake and sleep signaling is regulated by intricate neural circuitry in the brain. One key component of this process is the orexin system, which helps promote and consolidate wakefulness. There are two forms of orexin neuropeptides Orexin A and OrexinB. Orexin promotes wakefulness through its receptors OX1R and OX2R. In combination, these neuropeptides and receptors comprise the orexin system. The orexin system stimulates target neurons in the wake system leading to the release of several chemicals (Dopamine, Serotonin, Histamine, Acetylcholine, Norepinephrine) which promote wakefulness. Under normal circumstances, orexin levels rise throughout the day as wakefulness is promoted and then consolidated and fall at night. Overactivity of the orexin system is thought to be an important driver of insomnia.

Idorsias research team has been working on the science of orexin and orexin receptors since they were first described in 1998. The teams initial work led to the conclusion that antagonism of the orexin system was the key to preserving a natural sleep architecture for patients with insomnia. With this as the target the team started to design a dual antagonist with a rapid effect, and a duration of action sufficient for the night but short enough to avoid any negative residual activity the following morning at optimally effective doses.

About dual orexin receptor antagonismDual orexin receptor antagonists or DORAs are an entirely different approach to treating insomnia than previous drug classes, turning down overactive wakefulness by blocking the activity of orexin. DORAs specifically target the orexin system by competitively binding with both receptors and thereby reversibly blocking the activity of orexin. It is hypothesized that blocking orexin receptors reduces the downstream activity of the other wake promoting neurotransmitters that are overactive in insomnia, leading to the clinical efficacy demonstrated by orexin receptor antagonists.

Data supporting daridorexant in insomnia Results of the first Phase 3 study, investigating daridorexant doses 25 and 50mg, were reported in April 2020. The study demonstrated efficacy of treatment with daridorexant on objective and subjective sleep parameters and daytime functoning with no residual effect in the morning, and no evidence of rebound or withdrawal symptoms upon treatment discontinuation.

Daridorexant at both 25 and 50 mg significantly improved sleep onset and sleep maintenance as measured objectively in a sleep lab by polysomnography. Daridorexant also significantly improved subjective total sleep time as measured daily with a patient diary at home. The results were consistently statistically significant at month 1 and at month 3, indicating sustained benefit. Furthermore, treatment with daridorexant improved patients daytime functioning from baseline at month 1 and month 3.

The rate of adverse events was comparable between placebo and daridorexant at both treatment doses. Treatment-emergent adverse events (TEAEs) during the double-blind study period were reported in 37.7% and 37.7% of the patients treated with 25 and 50 mg daridorexant, respectively (34.0% for placebo). The most frequent TEAE reported over 3% incidence and higher than placebo was nasopharyngitis, headache.

Prior to the Phase 3 program, the safety and efficacy of daridorexant in adult and elderly patients with insomnia was evaluated in a comprehensive Phase 2 program, comprising two studies, one of which included zolpidem 10 mg as an active reference. Both studies showed the desired effect on sleep maintenance and onset, with a significant dose-response relationship; treatment was generally well tolerated.

A comprehensive clinical pharmacology program is being conducted totaling approximately 20 studies and including, amongst others, studies assessing abuse liability, drug-drug interactions, next-morning driving, the effect of daridorexant on respiratory function in patients with chronic obstructive pulmonary disease (COPD) or obstructive sleep apnea (OSA), and the pharmacokinetics of daridorexant in patients with liver and renal impairment.

EmmanuelMignot,MD and Professor of Psychiatry and Behavioral Sciences at Stanford University He is a former student of the Ecole Normale Superieure (Ulm, Paris, France) and received his M.D. and Ph.D. from Paris V and VI University in France. He practiced medicine in France for several years before joining Stanford as a faculty member in 1991 and was named Director of the Stanford Center for Narcolepsy in 1993. Dr. Mignot was named the Craig Reynolds Professor of Psychiatry and Behavioral Sciences in 2001. He served as the Director of the Stanford Center of Sleep Sciences and Medicine from 2009 to 2019.

Dr. Mignot is internationally recognized for discovering the cause of narcolepsy. His findings led to the development of new hypnotics that block the hypocretin (orexin) receptor and is likely to have other therapeutic applications as well. His research also demonstrated that narcolepsy is a selective autoimmune disease of the hypocretin system showing the involvement of molecular mimicry in humans with influenza A.

He has received numerous research grants and honors including National Sleep Foundation and National Institute of Health Research Awards, Howard Hughes Medical Institute Investigator and McKnight Neuroscience awards, the Narcolepsy Network professional service award, the Drs. C. and F. Demuth 11th Award for Young Investigators in the Neurosciences, the WC Dement Academic Achievement Award in sleep disorders medicine, the CINP and ACNP awards in neuropharmacology and the Jacobaeus prize.

Dr. Mignot is an elected member of the Association of American Physicians, the Institute of Medicine, and of the National Academy of Sciences (USA). He is the co-author of more than 200 original scientific publications, and he serves on the editorial board of scientific journals in the field of sleep and biology research. Dr. Mignot is an active member of several professional and governmental organizations. He has served as President of the Sleep Research Society, Chair of the National Center on Sleep Disorders Research Advisory board of the National institutes of Health, and Chair of the Board of Scientific Counselors of the National Institute of Mental Health.

Most of Dr. Mignot's current research focuses on the neurobiology, genetics and immunology of narcolepsy, a disorder caused by hypocretin (orexin) cell loss, with indirect interest in the neuroimmunology of other brain disorders. His laboratory uses state of the art human genetics techniques, such as genome wide association, exome or whole genome sequencing in the study of human sleep and sleep disorders, with parallel studies in animal models. His laboratory is also interested in web-based assessments of sleep disorders, computer-based processing of polysomnography (PSG), and outcomes research. Dr. Mignot serves as a consultant to Idorsia.

References

About IdorsiaIdorsia Ltd is reaching out for more - We have more ideas, we see more opportunities and we want to help more patients. In order to achieve this, we will develop Idorsia into one of Europes leading biopharmaceutical companies, with a strong scientific core.

Headquartered in Switzerland - a biotech-hub of Europe - Idorsia is specialized in the discovery and development of small molecules, to transform the horizon of therapeutic options. Idorsia has a broad portfolio of innovative drugs in the pipeline, an experienced team, a fully-functional research center, and a strong balance sheet the ideal constellation to bringing R&D efforts to business success.

Idorsia was listed on the SIX Swiss Exchange (ticker symbol: IDIA) in June 2017 and has over 800 highly qualified specialists dedicated to realizing our ambitious targets.

For further information, please contactAndrew C. WeissSenior Vice President, Head of Investor Relations & Corporate CommunicationsIdorsia Pharmaceuticals Ltd, Hegenheimermattweg 91, CH-4123 Allschwil+41 58 844 10 10www.idorsia.com

The above information contains certain "forward-looking statements", relating to the company's business, which can be identified by the use of forward-looking terminology such as "estimates", "believes", "expects", "may", "are expected to", "will", "will continue", "should", "would be", "seeks", "pending" or "anticipates" or similar expressions, or by discussions of strategy, plans or intentions. Such statements include descriptions of the company's investment and research and development programs and anticipated expenditures in connection therewith, descriptions of new products expected to be introduced by the company and anticipated customer demand for such products and products in the company's existing portfolio. Such statements reflect the current views of the company with respect to future events and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements of the company to be materially different from any future results, performances or achievements that may be expressed or implied by such forward-looking statements. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected.

Read more:
Idorsia announces positive results in the second Phase 3 study of daridorexant - GlobeNewswire

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