StemCell Therapy

PALO ALTO, Calif., June 24, 2020 (GLOBE NEWSWIRE) -- BridgeBio LLC Pharma, Inc. (NASDAQ: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that it has added three new independent directors to its board: former Allergan CEO and biopharma deal-maker, Brent Saunders; genomics pioneer and entrepreneur, Randy Scott, Ph.D.; and renowned economist and BridgeBio co-founder, Andrew Lo, Ph.D.

We are privileged to welcome these world-class company builders, innovators, and thought leaders to our board where they can help us construct and advance a vast pipeline of meaningful medicines for patients with genetic disease, said BridgeBio CEO and founder Neil Kumar, Ph.D. Brent has a remarkable track record of leading companies to growth across many therapeutic areas. Randy is a giant in genomic medicine who has used his entrepreneurial skills to connect genetic information to patients in profound ways. Andrews groundbreaking financial engineering work led to the founding of BridgeBio and inspires us to find new ways to accelerate the drug development process. I look forward to working with and learning from these leaders as we seek to discover, develop, and deliver life-changing medicines at scale. Its day one at BridgeBio and we are ready to go.

Brent Saunders

Mr. Saunders will bring his considerable experience to bear as he advises BridgeBio on scaling its business across new product and therapeutic areas, expanding into new geographies, developing commercial expertise, and utilizing new corporate structures.

Mr. Saunders most recently served as chairman, president and chief executive officer of Allergan. In this capacity, he led the company to launch more than 15 products and achieve 9.4% revenue growth until its merger with AbbVie in 2020. He previously served as president and CEO at Actavis, where he led a $15 billion global pharmaceutical business until its merger with Allergan. He initially joined Actavis as part of the companys acquisition of Forest Laboratories, where he served as president and CEO. Before joining Forest Laboratories, Mr. Saunders served as president and CEO of Bausch & Lomb. Mr. Saunders received his bachelors degree in economics and East Asian studies from the University of Pittsburgh, a Juris Doctor degree from Temple University School of Law and his Master of Business Administration from Temple University School of Business.

I appreciate BridgeBios ability to accelerate the development of therapeutics for patients in need and I have been impressed with their unheard-of progress in pipeline growth in such a short time. As the company moves toward potential commercialization of its lead products, Im eager to bring my experience leading and growing global pharmaceutical companies to help guide BridgeBio in its game-changing efforts, said Mr. Saunders.

Randy Scott, Ph.D.

Dr. Scott will bring his deep expertise to BridgeBio and advise the company on how best to utilize the broad ecosystem of genomic medicine (beyond the pill) so that a wider universe of patients can be served. BridgeBios decentralized business model will also benefit from Dr. Scotts guidance on developing and maintaining a strong culture of excellence.

Dr. Scott pioneered the introduction of genetics into everyday medical care as the founder of multiple cutting-edge biotech companies. Dr. Scott recently served as chief executive officer and executive chairman at Invitae, a company he co-founded to bring genetic information into routine medical practice. Prior to Invitae, he founded Genomic Health and served as the companys CEO and later executive chairman leading the company to develop and launch genomic diagnostic tests for breast, colon, and prostate cancer. Earlier in his career, Dr. Scott served as the president and chief scientific officer for Incyte, one of the first geneticinformationcompanies. He co-founded and currently serves as chairman of Genome Medical, a telegenomics-based clinical care company. Dr. Scott earned his bachelors degree in chemistry from Emporia State University and his doctorate in biochemistry from the University of Kansas.

For the last 30 years, Ive focused on building genomics companies to better understand the fundamental basis of human disease and improve the quality of treatment decisions through genomic diagnosis.It is with great excitement that I now join the BridgeBioboard to take the next step inbringing multipletherapies for genetic disorders to market, said Dr. Scott.Genetic disorders are much more common than previously thought, and BridgeBio has the unique opportunity toefficiently bring multiple life-saving products to the clinic at an accelerating pace.With my experience in building and scaling companies, I plan to focus on helping the BridgeBio team to scale the organization and build a world-class pharmaceutical company focused on solving genetic disease."

Andrew Lo, Ph.D.

Dr. Lo brings his vast knowledge and understanding of economics and financial engineering to BridgeBio and will advise the company on how to continue accessing financing from a range of markets to broaden its work for patients and pursue more groundbreaking scientific innovation. As a famously innovative and iconoclastic thinker, he will also challenge the company to continue to innovate in areas as diverse as R&D process, clinical trial statistics, and talent management.

Dr. Lo is the Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of the MIT Laboratory for Financial Engineering, a principal investigator at the MIT Computer Science and Artificial Intelligence Laboratory, and an affiliated faculty member of the MIT Department of Electrical Engineering and Computer Science. His research spans several areas of financial economics, but his most recent focus is on developing new statistical tools for predicting clinical trial outcomes, incorporating patient preferences into the drug approval process and accelerating biomedical innovation through novel financing structures. His work formed the foundation for BridgeBios business model, and he is one of the companys co-founders. Dr. Lo earned his bachelors degree in economics from Yale University and a masters degree and doctorate in economics from Harvard University.

Its remarkable to see the significant progress that BridgeBio has made in the five years since its founding to develop new medicines for genetically driven diseases, said Dr. Lo. "I'm excited and honored to join the BridgeBio board and look forward to contributing in whatever ways I can to help them bring new therapies to patients who have no other alternatives."

About BridgeBio PharmaBridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio's pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visitbridgebio.com.

Contact:Grace RauhBridgeBio Pharma, Inc.Grace.rauh@bridgebio.com(917) 232-5478

Source: BridgeBio Pharma, Inc.

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BridgeBio Pharma, Inc. Appoints Biotech Trailblazers Brent Saunders and Randy Scott and Renowned Economist Andrew Lo to Board of Directors - BioSpace

Application of ImmTOR plus Sareptas investigational gene therapies will be evaluated for Duchenne Muscular Dystrophy and Limb-Girdle Muscular Dystrophies

CAMBRIDGE, Mass. and WATERTOWN, Mass., June 18, 2020 (GLOBE NEWSWIRE) --Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, and Selecta Biosciences, Inc. (NASDAQ: SELB) today announced that they have entered into a Research License and Option agreement granting Sarepta an option to license the rights to develop and commercialize Selectas immune tolerance platform, ImmTOR, for use in Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMDs). In advance of exercising its option, Sarepta will conduct research and evaluate the utility of ImmTOR to minimize or prevent the formation of neutralizing antibodies (NAb) to adeno-associated virus (AAV) in connection with the administration of Sareptas DMD and LGMD gene therapy candidates.

Sareptas late-stage gene therapy candidates are delivered using AAV in particular, AAVrh74. AAVrh74 was selected because of its safety profile, superior muscle tropism, empirical demonstration of high expression, and low screen-out rate for pre-existing antibodies. Currently, however, all systemic AAV-delivered constructs are one-time therapies that cannot be re-dosed due to the robust post-administration development of NAbs specific to the AAV vector. Selecta is a leader in immune tolerance and has generated strong preclinical evidence to support the potential for re-dosing patients receiving gene therapy.Selecta has reported that in preclinical studies, when used in combination with AAV gene therapy vectors,Selectas ImmTOR immune tolerance platform inhibits the development of NAbs to the vector, permitting re-dosing of the gene therapy. i

As we build our enduring gene therapy engine, we intend not only to rapidly advance treatments for rare, life-ending diseases, but at the same time, to advance the state of genetic medicine science by continually improving the utility of gene therapy. If successful, the ability to re-dose will be an enormous leap forward in the science of gene therapy and provide invaluable benefits to patients beyond those we anticipate with one-time dosing. We are encouraged by the data generated on the ImmTOR platform and excited to join with Selecta to explore the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed, said Doug Ingram, President and Chief Executive Officer, Sarepta Therapeutics.

We are pleased to build on our already strong foundation of strategic partnerships and expand the clinical application of the ImmTOR platform into neuromuscular diseases. The ability to re-dose gene therapy addresses one of the major challenges of one-time therapies today, said Carsten Brunn Ph.D., President and Chief Executive Officer of Selecta Biosciences. We are excited to collaborate with a leader in genetic medicine like Sarepta, and are confident that their expertise in rare diseases combined with our immune tolerance platform has the potential to enhance the long-term therapeutic benefit to patients with these debilitating conditions.

DMD is a rare, degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. LGMDs are a group of over 30 distinct diseases that cause weakness and wasting of the muscles around the hips and shoulders, eventually progressing to the arms and legs. LGMD can be caused by a single gene defect that affects specific proteins within the muscle cell, including those responsible for keeping the muscle membrane intact.

Under the terms of the research license and option agreement, Sarepta will make an initial payment to Selecta, and Selecta is eligible to receive certain pre-clinical milestone fees. If Sarepta exercises its options to enter any commercial license agreements, Selecta will be eligible for additional development, regulatory, and commercial milestone payments, as well as tiered royalties on net product sales. Additional financial details are not being disclosed.

About Selecta Biosciences, Inc.

Selecta Biosciences, Inc. is a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its pioneering immune tolerance platform (ImmTOR). Selecta is committed to utilizing ImmTOR to potentially improve the efficacy of biologics, enable re-dosing of life-saving gene therapy, and create novel immunotherapies for autoimmune diseases. Selectas late-stage product candidate, SEL-212, is designed to be a monthly treatment for chronic refractory gout, a debilitating rare disease with a significant unmet medical need. SEL-212 consists of a combination of our ImmTOR platform co-administered with pegadricase, an enzyme designed to treat patients with symptomatic gout, refractory to standard uric acid lowering treatment. Selectas proprietary gene therapy product candidates are in development for certain rare inborn errors of metabolism and incorporate our ImmTOR platform with the goal of addressing barriers to repeat administration. In addition to our own pipeline of core discovery and clinical candidates, Selecta has established collaborative relationships with leading biopharmaceutical companies, including Asklepios BioPharmaceutical (AskBio) for gene therapy, and Swedish Orphan Biovitrum AB (Sobi) for SEL-212. Selecta is based in Watertown, Massachusetts. For more information, please visit http://www.selectabio.com.

Selecta Forward-Looking Statements:

SelectaBiosciences, Inc. (the company), including without limitation, the companys actions regarding the monitoring and assessment of COVID-19 on the companys operations, clinical trials and manufacturing, Sareptas plans to evaluate its gene therapies in combination with the companys ImmTOR technology, the possibility of Sarepta exercising an option to enter into a commercial license agreement, the unique proprietary technology platform of the company and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the ability of the companys ImmTOR platform to unlock the full potential of biologic therapies, the potential treatment applications for product candidates utilizing the ImmTOR platform in areas such as enzyme therapy and gene therapy, the novelty of treatment paradigms that Sarepta is able to develop in combination with the companys ImmTOR technology, the potential of any therapies developed by Sarepta in combination with the companys ImmTOR technology to fulfill unmet medical needs, the companys plan to apply its ImmTOR technology platform to a range of biologics for rare and serious diseases, the ability of Sareptas existing therapies to target the heart and skeletal muscle, expected payments to be made to the company under the Research License and Option Agreement, the potential of the ImmTOR technology platform generally and the companys ability to grow its strategic partnerships, the sufficiency of the companys cash, cash equivalents and short-term investments, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including their uncertain outcomes, the effect of the COVID-19 outbreak on any of the companys planned or ongoing clinical trials, manufacturing activities, supply chain and operations, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the unproven approach of the companys ImmTOR technology, Sareptas ability to research and develop therapeutic candidates using the companys ImmTOR technology, undesirable side effects of the companys product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials as well as the impact of the COVID-19 outbreak on those third parties and their ability to continue their operations, the companys inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, managements ability to perform as expected, potential delays in regulatory approvals, Sareptas ability to make up-front and milestone payments, the companys business development strategy, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the companys recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock including stock market fluctuations that occur as a result of the COVID-19 outbreak, and other important factors discussed in the Risk Factors section of the companys most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

AboutSarepta Therapeutics

At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Sarepta Forward-Looking Statement

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the parties undertakings under the agreement and potential payments and fees; the potential benefits of Sareptas gene therapy product candidates; the potential of ImmTOR to enable re-dosing of AAV gene therapy; Sareptas intention to rapidly advance treatments for rare, life-ending diseases, and to advance the state of the genetic medicine science by continually improving the utility of gene therapy; the potential of re-dosing to provide invaluable benefits to patients beyond those Sarepta anticipates with one-time dosing;the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed; and the potential of the collaboration between Sarepta and Selecta to enhance the long-term therapeutic benefit to patients with these debilitating conditions.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Selecta may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; success in preclinical trials does not ensure that later clinical trials will be successful; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Selecta Contacts:For Media:Joshua R. MansbachSolebury Trout+1-646-378-2964jmansbach@soleburytrout.com

For Investors:Lee M. SternSolebury Trout+1-646-378-2922lstern@soleburytrout.com

Sarepta Contacts: Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

_____________________________iNature Communications,October 2018.

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Sarepta Therapeutics and Selecta Biosciences Enter into Research License and Option Agreement for Selecta's ImmTOR Immune Tolerance Platform in...

Image: Prof. Dr. David Ellinghaus and Frauke Degenhardt, both first authors of the study (Photo courtesy of UKSH Kiel)

The world's first large-scale genome-wide study conducted by scientists at the University Medical Center Schleswig-Holstein (UKSH Kiel, Germany) and the Kiel University (CAU Kiel, Germany), in cooperation with a research group from Norway, has found gene variants that significantly influence the course of the disease-one of them concerns the gene for the blood group trait. This suggests that different blood groups may be responsible for why some people become severely ill with COVID-19 while others show hardly any symptoms.

Doctors from several hospitals of the corona epicenters in Northern Italy and Spain, sent blood samples of a total of 1,980 intensive care COVID-19 patients who had to be treated with oxygen or connected to a ventilator. For the control group, 2,205 randomly selected women and men from the population of these countries were obtained. Within only three weeks, DNA was isolated from the blood samples and 8.5 million positions of the genetic material from each individual were measured with so-called biochips (SNP arrays). The study showed that people with blood group A had an approximately 50% higher risk of severe COVID-19 progression than people with other blood groups. In contrast, people with type 0 blood groups were almost 50% better protected against serious COVID-19 disease. Thus, the study confirmed for the first time by means of a comprehensive genome-wide analysis two earlier studies by international researchers who had already described a possible correlation between blood group characteristics and the disease using the blood serum of COVID-19 patients.

In addition to the significant abnormality in the AB0 blood group locus, the gene locus by which the individual blood group is determined, the researchers found an even higher effect strength for a genetic variation on chromosome 3. Which of the several candidate genes located in this locus is responsible for this cannot be determined precisely at present, but the analysis was able to show that carriers of the gene are at a twofold higher risk of contracting severe COVID-19 than people who do not carry this variation. Among the Italian and Spanish patients who were so ill that they not only had to be supplied with oxygen but also connected to a ventilator, a particularly high number carried this genetic disposition. A result that was also evident in the distribution of blood groups: Among the particularly seriously ill, there were also a particularly large number of people with blood group A.

The results were very exciting and surprising for us. The region on chromosome 3 in particular had not previously been associated with COVID-19 by scientists. In other regions of the genome for which an effect on the disease had been suspected, no statistically significant differences were found between the healthy volunteers and the patients; neither in the chromosome section 6p21, which is associated with the immune system and many infectious diseases, nor in the gene IFITM3, which is associated with influenza, said Prof. Dr. Andre Franke, Director of the Institute of Clinical Molecular Biology (IKMB) and member of the steering committee of the cluster of excellence "Precision Medicine in Chronic Inflammation" (PMI). With chromosome 3 and the AB0 blood group locus we describe real causes for a severe course of COVID-19. Our results, therefore, create an excellent basis for the development of active substances that can target the candidate genes found. It has been proven that a clinical study in which a drug is tested has twice as much success if genetic evidence for the target is already available. The results could also contribute to an improved risk assessment for a severe course of COVID-19 in patients.

Related Links:University Medical Center Schleswig-Holstein (UKSH)UKSH Kiel

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Genes and Blood Type Determine Risk of COVID-19 Severity - HospiMedica

The Global Precision Medicine market report presents market dynamics focusing on all the important factors market movements depend on. It includes current market trends with a record from historic year and prediction of the forecast period. This report is a comprehensive market analysis of the Precision Medicine market done on a basis of regional and global level. Important market analysis aspects covered in this report are market trends, revenue growth patterns market shares and demand and supply along with business distribution.

Get Research Insights @Precision Medicine Market 2019-2025 (USD Bn)

Precision medicines are about bringing the right treatment to the right patient at the right time. It is one of the key vehicles by which the healthcare system of tomorrow can achieve better outcomes for patients and financial efficiency.

Global Precision Medicine market size is estimated based on the population genetic screening data, disease prevalence rate and adoption of the precision medicines across the globe. Global Precision Medicine manufacturers have varying products, depending on consumer genetic screening data. The report also provides various key strategies adopted by the leading players as they mainly target prevalent diseases in the developed and developing economies. Precision Medicine market share in various diseases has also been derived on the basis of thorough understanding of the forecast and dynamics of various products under pipeline in various geographies. Also, the global Precision Medicine market size has been laid down based on an extensive value chain analysis and patent analysis.

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The global precision medicine market size is projected to be valued USD 119.90 billion by 2025. Advancement in genomic and proteomic sequencing with reduced costs, is bringing in the so-called genomic revolution. By identifying the optimal point of intervention for treatment, healthcare providers massively improve the quality of care. Additionally, these technologies offer optimized cost by effectively timing treatment intervention and eliminating the need for insufficient or excessive treatment regimens.

Next generation sequencing has shifted the paradigm of genomics by accomplishing entire genome sequencing in a matter of hours. The enhanced speed and ease of DNA sequencing has drastically reduced the cost. The cost of deciphering the entire human genome has dropped by an order of magnitude from USD 10,000 in 2011 to about USD 1,000 today. As genome sequencing costs are declining rapidly healthcare providers and patients are likely to be more inclined to seek treatments that are targeted to a particular illness.

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Cost is a major issue for precision medicines. Precision medicines may overburden many healthcare systems which are currently under significant financial constraints. It is a major factor hindering the patients access to treatment. The new targeted drugs that are launched in the market, are so highly priced that the patients can hardly afford them unless the cost is fully covered by the payers.

Geographically, Asia Pacific led the precision medicine market share in the global market in 2017, growing with a CAGR of 14.38% from 2018 to 2025. Globally, 40 countries have their individual versions of precision medicine ingenuities. United States and China being one of them. China, is spending $43 for every $1 the United States is spending on its precision medicine initiatives. The Beijing Genome Institute has the worlds largest sequencer and repository of genetic material. Additionally, developments in computational power and artificial intelligence are also in progress to discover new drugs, treatment and delivery methods. Wuxi Nextcode and Huawei are collaboratively developing cloud computing infrastructure to store and compute enormous amounts of data for precision medicines.

Furthermore, the Korean government is strategizing framework for implementation of precision medicine into clinical practice. The Korean Ministry of Food and Drug Safety, conducted a survey in 2012-2013, in order to explore the public awareness and attitude towards precision medicine. Results of the survey demonstrated low levels of public awareness regarding precision medicine, however, family income showed a positive correlation with precision medicine knowledge, thus reducing potential health disparities will increase the access to precision medicine.

Some of the leading players operating in the market include Novartis International AG, F. Hoffmann-La Roche AG, AstraZeneca plc, Eli Lilly and Company, Pfizer Inc., Teva Pharmaceutical Industries Ltd., Abbott Laboratories, Merck & Co. and others.

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Key segments of the global precision medicine market

Application Overview, 2015-2025 (USD Million)

Regional Overview, 2015-2025 (USD Million)

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Precision Medicine Market 2020 | Increasing Demand, Current Trend, Scope, Business Strategies, Challenges, New Innovations, Future Projections and...

London: Genes may soon reveal who would develop severe COVID-19 symptoms, remain asymptomatic or only have a mild coronavirus attack, say researchers.

Data from popular home genetic-testing kits could help scientists shed light on why some people who catch coronavirus have no symptoms while others become very ill, according to the team from the University of Edinburgh in the UK.

Researchers are now asking people who have used DNA testing services to gain ancestry or health insights to join a study that aims to identify key genes involved in the body's response to the infection.

"Understanding the effect genes have on susceptibility to COVID-19 could aid efforts to tackle the pandemic, and help combat future disease outbreaks," said the researchers.

More than 30 million people worldwide have used genetic testing services. Researchers are now urging them to share their DNA data to help speed up discoveries that could help fight the virus.

"Some people suffer no ill effects from coronavirus infection, yet others require intensive care. We need to identify the genes causing this susceptibility, so we can understand the biology of the virus and hence develop better drugs to fight it," said Jim Wilson, Professor of Human Genetics at the University of Edinburgh.

By providing gene data, volunteers will help the team avoid the costly, time-consuming task of collecting the hundreds of thousands of DNA samples that would otherwise be needed to map the genes involved.

The team aims to identify genes that influence the risk of developing COVID-19 and those that affect disease severity, by comparing volunteers' symptoms -- or lack of them -- with their DNA.

Researchers also aim to analyse the long-term health consequences of infection and self-isolation.

The study is supported by the Medical Research Council, Biotechnology and Biological Science Research Council, Health Data Research UK and Wellcome Trust.

"To identify the genes that explain why some people get very sick from coronavirus and others don't, we need the solidarity of a large proportion of people from different countries who can share their DNA testing results with us. In this case, size really matters," said Albert Tenesa, Professor of Quantitative Genetics at the University of Edinburgh.

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Researchers to identify genes that put some at severe corona risk - ETHealthworld.com

Precision medicine (PM) is an approach to patient care that allows doctors to select treatments that are most likely to help patients based on a genetic understanding of their disease. Personalized nanomedicine involving individualized drug selection and dosage profiling in combination with clinical and molecular biomarkers can ensure the maximal efficacy and safety of the treatment. The major hindrance toward the development of such therapies is the handling of the Big Data, to keep the databases updated. Robust automated data mining tools are being developed to extract information regarding genes, variations, and their association with diseases. Phenotyping, an integral part of PM, is aimed at translating the data generated at cellular and molecular levels into clinically relevant information.Precision Medicine Moves Care from Population-Based Protocols to Truly Individualized Medicine as President of the US announced the Precision Medicine Initiative in his 2015 State of the Union address. Under the initiative, medical care would transition from a one-size-fits-all approach to an individualized approach, in which data on each patients genomic makeup, environment, and lifestyle (the exposome) helps medical professionals tailor treatment and prevention strategies. To achieve the Precision Medicine Initiative mission statement, to enable a new era of medicine through research, technology, and policies that empower patients, researchers, and providers to work together toward development of individualized care, researchers and clinicians need vast and varied amounts of data and the technology to ensure that data is widely accessible and usable.

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Insights Presented in the Report

Based on technology type, the market is fragmented into big data analytics, bioinformatics, gene sequencing, drug discovery, companion diagnostics, and others.Recent technological and analytical advances in genomics, have now made it possible to rapidly identify and interpret the genetic variation underlying a single patients disease, thereby providing a window into patient-specific mechanisms that cause or contribute to disease, which could ultimately enable the precise targeting of these mechanisms

Based on the market segment by application type, the market is segmented into oncology, respiratory diseases, central nervous system disorders, immunology, genetic diseases and others. With the advent of precision medicine, cancer treatment is moving from a paradigm in which treatment decision isprimarily based on tumor location and histology followed by molecular information to a new paradigm whereby treatment decisions will be primarily based on molecular information followed by histology and tumor location

Based on the market segment by end-user, the market is fragmented into hospitals & clinics, pharmaceuticals, diagnostic companies, Healthcare-IT firms and others. The precision medicine suppliers that understand technology and the goals of value-based healthcare can create value in the precision medicine value-chain by offering value-based solutions and platforms to interpret and connect data points. There are a number of technology companies who work in the field of precision medicine and more will be founded in the years to come

For better understanding on the market dynamics of Precision Medicine market, detailed analysis was conducted for different countries in the region including North America (United States, Canada, Mexico and Rest of North America), Europe (Germany, UK, France, Italy, Spain and Rest of Europe), Asia-Pacific (China, Japan, Australia, India and Rest of APAC), and Rest of World

Some of the major players operating in the market includeHoffmann-La Roche, Medtronic, Qiagen, Illumina, Abbott Laboratories, GE Healthcare, NanoString Technologies, bioMrieux SA, Danaher Corporation, and AstraZeneca

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Precision Medicine Market Size, Share & Trends Analysis Report by Product Types, And Applications Forecast To 2026 - Cole of Duty

A new University of California, Irvine-led study reveals a protein responsible for genetic changes resulting in a variety of cancers, may also be the key to more effective, targeted cancer therapy.

The study, published today inNature Communications, titled, Quantification of ongoing APOBEC3A activity in tumor cells by monitoring RNA editing at hotspots, reveals how the genomic instability induced by the protein APOBEC3A offers a previously unknown vulnerability in cancer cells.

Each day, in human cells, tens of thousands of DNA damage events occur. In cancer cells, the expression of the protein APOBEC3A is one of the most common sources of DNA damage and mutations. While the mutations caused by these particular proteins in cancer cells contribute to tumor evolution, they also cause breaks in the DNA, which offer a vulnerability.

Targeting cancer cells with high levels of APOBEC3A protein activities and disrupting, at the same time, the DNA damage response necessary to repair damages caused by APOBEC3A, could be key to more effective cancer therapies, saidRemi Buisson, PhD, senior investigator and an assistant professor in the Department of Biological Chemistry at the UCI School of Medicine. However, to exploit the vulnerability of the cancer cells, it is critical to first quantitatively measure the proteins activity in tumors.

To understand the role of APOBEC3A in tumor evolution and to target the APOBEC3A -induced vulnerabilities, the researchers developed an assay to measure the RNA-editing activity of APOBEC3A in cancer cells. Because APOBEC3A is difficult to quantify in tumors, developing a highly sensitive assay for measuring activity was critical. Using hotspot RNA mutations, identified from APOBEC3A-positive tumors, the team developed an assay using droplet digital PCR and demonstrated its applicability to clinical samples from cancer patients.

Our study presents a new strategy to follow the dysregulation of APOBEC3A in tumors, providing opportunities to investigate the role of APOBEC3A in tumor evolution and to target the APOBEC3A-induced vulnerability in therapy, said Buisson. We anticipate that the RNA mutation-based APOBEC3A assay will significantly advance our understanding of the function of the protein in tumorigenesis and allow us to more effectively exploit the vulnerabilities it creates in cancer therapy.

This study was funded in part by the National Institutes of Health, a California Breast Cancer Research Program grant and an MPN Research Foundation Challenge grant.

Reference: Jalili, P., Bowen, D., Langenbucher, A. et al. (2020) Quantification of ongoing APOBEC3A activity in tumor cells by monitoring RNA editing at hotspots. Nat Commun. DOI: https://doi.org/10.1038/s41467-020-16802-8

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Exploiting Cancer Cell Vulnerabilities To Create More Effective Cancer Therapies - Technology Networks

Parasite infections are a constant presence for many people who live in tropical regions, particularly in less industrialized areas. These often chronic conditions are at best unpleasant; more seriously, children with parasite diseases that cause diarrhea can die of malnutrition or dehydration.

In Genome Biology, a study led by University of Pennsylvania scientists investigated the links between parasite infection and the gut microbiome. Using genetic methods to characterize the gastrointestinal microbiome of 575 ethnically diverse Cameroonian people representing populations from nine villages with meaningful differences in lifestyle, the researchers discovered that the presence of parasites was strongly associated with the overall composition of the microbiome.

"We found that we could look at someone's microbiome and use it to predict whether someone had a gastrointestinal parasite infection," says Meagan Rubel, who completed her doctorate degree at Penn and is now a postdoc at the University of California, San Diego. "Whether or not it was parasites changing the microbiome or something in the resident microbiota of a person that made them more susceptible to infection, we can't say, but the association was strong."

Rubel led the study in collaboration with Penn's Sarah Tishkoff, a Penn Integrates Knowledge Professor in the Perelman School of Medicine and School of Arts and Sciences, and Frederic Bushman, a microbiologist in the medical school. In addition to the microbiome and parasites, the research also examined markers of immune function, dairy digestion, and pathogen infection, a rich dataset.

The investigation entailed six months of field work, collecting fecal and blood samples from Mbororo Fulani pastoralists, cattle herders with a diet high in meat and dairy; Baka and Bagyeli rainforest hunter-gatherers, who practice a limited amount of farming but also forage for meat and plant-based foods; and Bantu-speaking agropastoralists, who both grow crops and raise livestock. As a comparison group, the study included data from two groups of people living in urban areas of the United States, with a diet heavier in animal fats, proteins, and processed foods.

In the field, the researchers tested for malaria and a number of other pathogens that infect both the blood and gastrointestinal system.

Of the 575 people tested in Cameroon, the researchers found nearly 40% were infected with more than one parasite before receiving an antiparasitic treatment, with hunter-gatherers, on average, most likely to be co-infected with multiple parasites. In particular, the team found that four soil-transmitted gut parasites tended to co-occur at a rate much higher than chance: Ascaris lumbricoides, Necator americanus, Trichuris trichiura, and Strongyloides stercoralis, or ANTS.

"Gut parasites are a global public health concern," says Rubel. "And you tend to see several of these parasites together in resource-poor settings where people may not have access to clinical care, piped water, and soap, so there's more opportunity for them to be transmitted."

Back in the lab at Penn, the researchers used genomic sequencing tools to take a snapshot of the participants' gut microbiomes. The composition of the microbiome, they found, could accurately predict a person's country (U.S. or Cameroon) and lifestyle (urban, pastoralist, agropastoralist, or hunter-gatherer). But after these two variables, the presence of ANTS parasites could be predicted with greater accuracy by the microbiome structure than any other variable the research team studied. Taken together, the microbiome could predict the presence of these four gut parasites with roughly 80% accuracy.

Infection with these parasites also led to upticks in immune system activation, specifically turning on pathways that promote inflammatory responses. Parasite infection was also associated with a greater likelihood of having bacteria from the order Bacteroidales, which are known to play a role in influencing digestion and immune system function.

In a second part of the study, the Penn-led team assessed the relationship between the gut microbiome and milk consumption in the Fulani pastoralist population. Earlier work by Tishkoff and colleagues illuminated how genetic mutations enabling lactose digestion arose in pastoralist communities in Africa, selected through evolution because of the important nutritional benefits of consuming dairy. In looking at the Fulani's microbiomes, they also tended to have an abundance of bacterial genes capable of breaking down galactose, a component of lactose, and fats, compared to other groups. "This enrichment of genes could help you extract more nutrition from the food you eat," Rubel says.

The researchers believe their findings, the largest-ever study on the link between gut microbiome composition and parasite infection from sub-Saharan Africa, can open new possibilities for future work. "The kinds of microbiome markers we found could be useful to predict the type of pathogens you have, or to shed light on the interplay between the microbiome and the immune system," says Rubel.

Eventually, she adds, more research could even illuminate strategies for purposefully modulating the microbiome to reduce the risk of a parasite infection or minimize the harm it causes to the body.

ReferenceRubel, M.A., Abbas, A., Taylor, L.J. et al. Lifestyle and the presence of helminths is associated with gut microbiome composition in Cameroonians. Genome Biol 21, 122 (2020). https://doi.org/10.1186/s13059-020-02020-4.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Investigating the Link between Parasites and the Gut Microbiome - Technology Networks

DALLAS June 14, 2020 Recently developed MRI techniques used to more precisely target a small area in the brain linked to Parkinsons disease and essential tremor may lead to better outcomes without surgery and with less risk of negative effects, a new study led by UT Southwestern researchers suggests.

The study, published today in Brain, describes recently refined MRI methods designed to allow neuroradiologists to zero in on a pea-sized region in the brains thalamus involved in movement. Using the images, doctors then can use high-intensity focused ultrasound (HIFU) to ablate, or burn away, problem tissue, says Bhavya R. Shah, M.D., first author of the study and an assistant professor of radiology and neurological surgery at UT Southwesterns Peter ODonnell Jr. Brain Institute.

The benefit for patients is that we will be better able to target the brain structures that we want, Shah says. And because were not hitting the wrong target, well have fewer adverse effects. The procedures are already Food and Drug Administration-approved for use in patients, and UTSW plans to begin employing them to treat patients when its Neuro High Intensity Focused Ultrasound Program opens this fall.

Adverse effects from imprecise targeting include problems walking or slurring words. While such effects are usually temporary, they can be permanent in 15 to 20 percent of cases, says Dr. Shah.

According to the National Institutes of Health, essential tremor affects up to 10 million Americans and Parkinsons disease impacts more than 1 million. Both are neurologic diseases thought to have genetic links. The first line of treatment for the involuntary trembling or shaking seen with these diseases is medication. However, approximately 30 percent of patients do not respond well to drugs, according to the study.

In the late 1990s, neurosurgeons began using a procedure called deep brain stimulation, opening the skull to permanently implant metal electrodes that could then be stimulated via a battery pack.

About a decade ago, a new MRI-guided procedure emerged that uses high-intensity ultrasound waves to heat and eliminate a small section of the thalamus linked to the disorders. MRI-guided HIFU is currently approved for treatment of essential tremor and tremors seen in Parkinsons disease patients. The outpatient procedure does not require opening the skull, and the patient is awake while it is performed, says Dr. Shah. No cuts. No anesthesia. No implanted devices.

A challenge in both procedures has been locating the precise area inside the brains thalamus to treat the pea-sized ventral intermediate nucleus, says Dr. Shah.

Traditionally, doctors have relied on either landmarks or maps of the brain drawn from cadavers to help them pinpoint the correct location. However, every brain is different, Dr. Shah says, and tiny errors can lead to damage in surrounding tissue, or to missing portions of the correct target.

Three newly refined MRI techniques are better at delineating the target tissue, according to the study.

The most widely studied and perhaps most promising imaging method is called diffusion tractography, says Dr. Shah. It creates precise brain images by taking into account the natural water movement within tissues.

The other methods described are quantitative susceptibility mapping which creates contrast in the image by detecting distortions in the magnetic field caused by substances such as iron or blood and fast gray matter acquisition TI inversion recovery which operates much like a photo negative, turning the brains white matter dark and its gray matter white in order to provide greater detail in the gray matter.

Dr. Shah and his team plan to participate in a multicenter clinical trial with collaborators at the Mayo Clinic in Rochester, Minnesota, testing the diffusion tractography method in patients.

Senior author of the study was Rajiv Chopra, Ph.D., director of image-guided therapy development and associate professor of radiology in the Advanced Imaging Research Center at UTSW. Researchers at the Mayo Clinic also participated in the study.

About UTSouthwestern Medical Center

UTSouthwestern, one of the premier academic medical centers in the nation, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty has received six Nobel Prizes, and includes 25 members of the National Academy of Sciences, 16 members of the National Academy of Medicine, and 14 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,500 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in about 80 specialties to more than 105,000 hospitalized patients, nearly 370,000 emergency room cases, and oversee approximately 3 million outpatient visits a year.

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Advanced MRI Scans May Improve Treatment of Tremor, Parkinsons Disease - Newswise

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Direct-to-Consumer Genetic Testing market.

Trusted Business Insights presents an updated and Latest Study on Direct-to-Consumer Genetic Testing Market 2019-2026. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Direct-to-Consumer Genetic Testing market during the forecast period (2019-2029).It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

Get Sample Copy of this Report @ Direct-to-Consumer Genetic Testing Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19)

Abstract, Snapshot, Market Analysis & Market Definition: Direct-to-Consumer Genetic Testing MarketIndustry / Sector Trends

Direct-to-Consumer Genetic Testing Market size was valued at USD 831.5 million in 2018 and is expected to witness 15.2% CAGR from 2019 to 2025.

U.S. DTC Genetic Testing Market Size, By Test Type, 2018 & 2025 (USD Million)

Rising prevalence of genetic diseases such as cystic fibrosis and Alzheimers globally is one of the major factors fostering direct-to-consumer genetic testing market growth. According to Global Genes, over 300 million people globally suffer from rare diseases. Recently developed DTC genetic tests allows consumer to identify probability of acquiring a specific genetic disease. Therefore, increasing adoption of DTC genetic testing for early disease detection and identification of genetic diseases will boost the industry growth over forecast timeline.

Increasing demand for personalized medications to treat genetic diseases will positively impact industry growth in forthcoming years. Individuals genome must be tested to develop personalized medicines. This increases the demand for DTC genetic kits since, it provides detailed information about individuals genetic predisposition. As detailed information regarding genetic makeup of individuals is easily available with the use of DTC genetic kits, researchers can easily design and develop personalized medicine that would help in faster patient recovery. Aforementioned factor is expected to drive the industry growth. However, high cost of DTC genetic testing kits may hamper industry growth to some extent during the forecast period.

Market Segmentation, Outlook & Regional Insights: Direct-to-Consumer Genetic Testing Market

Direct-to-Consumer Genetic Testing Market, By Test Type

Predictive testing segment will experience around 17% growth throughout the analysis period. Considerable segmental growth can be associated with rising prevalence of genetic diseases. Recently developed DTC genetic tests help to identify mutations that increase the chances of acquiring specific disease accurately. Surging awareness regarding benefits of such presymptomatic testing has reduced the mortality rates by enabling effective management of disease. Above mentioned factors have stimulated the segmental growth that is predicted to continue over the forecast timeframe.

Ancestry and relationship testing segment accounted for over 43% revenue share in 2018. Increasing awareness regarding ethnicity tests amongst the American and European population has increased the demand for DTC genetic tests. Accuracy and efficiency possessed by these tests has fostered segmental growth. Moreover, ancestry tests developed by companies such as Ancestry.com are user friendly. Availability of robust DTC ancestry tests providing meaningful clinical, genealogical and even forensic information will positively impact the segment growth.

Direct-to-Consumer Genetic Testing Market, By Technology

Targeted analysis segment was valued over USD 310 million in 2018. Targeted analysis is utilized for determining the defects in genes that are responsible for a particular disorder. Targeted genotyping can accurately measure an individuals gene pool that encodes important information regarding various diseases. Targeted analysis can be conducted at significant low cost compared to other available techniques that should augment its adoption rate over forecast timeframe.

Single nucleotide polymorphism segment will experience around 15% growth throughout the forecast period. Single nucleotide polymorphism chips specifically detect changes in single nucleotide that increases the efficiency of tests. For instance, SNP chips utilized for diagnosing hereditary cancers have detected 1300 mutations in BRCA2 genes. Various companies such as Ancestry.com and Color Genomics utilize SNP arrays that analyse gene sequences at a specific resolution and reveal detailed analysis about the defective genes that may in future cause certain disease. Increasing adoption of such advanced SNP chips in DTC testing kits will trigger the segmental growth.

Germany DTC Genetic Testing Market Size, By Technology, 2018 (USD Million)

Direct-to-Consumer Genetic Testing Market, By Region

North America direct-to-consumer genetic testing market accounted for around 39% regional share in 2018. Regional market growth can be attributed to increasing prevalence of genetic diseases. Rare genetic diseases such as thalassemia, hemophilia and anaemia require continuous and critical monitoring. According to CDC, every year more than 1,000 people are affected by thalassemia. Furthermore, American population has higher literacy rate and also, awareness regarding DTC tests is high amongst the American population that augments demand for DTC genetic tests.

Europe is estimated to experience around 15% growth over the coming years. European direct-to-consumer genetic testing market is highly regulated and for carrying out some of the genetic tests through DTC kits, customers are required to have physicians prescription. However, currently, European regulatory bodies are working on improving regulations set on DTC tests due to improved accuracy and efficiency possessed by them. Thus, improvement in regulatory scenario will positively impact regional market growth.

Latin America DTC Genetic Testing Market Size, By Country, 2025 (USD Million)

Key Players, Recent Developments & Sector Viewpoints: Direct-to-Consumer Genetic Testing Market

Few of the eminent industry players operating in direct-to-consumer genetic testing market are Ancestry, 23andMe, Color, Family Tree DNA, EasyDNA, Helix, Identigene, Full Genomes, Genesis HealthCare, Karmagenes, MyHeritage, MapMyGenome, Living DNA and Pathway Genomics. Chief industry players implement numerous initiatives such as mergers, acquisitions and new product launch to maintain their market position. Receiving approvals from regulatory bodies for new products will also foster companys revenue share. For instance, in October 2018, 23andme received first U.S. FDA approval for de novotechnology utilized in pharmacogenomic tests. This approval will enable company to launch innovative products, thereby fostering companys growth.

Direct-to-Consumer (DTC) Genetic Testing Industry Viewpoint

Direct-to-consumer genetic testing industry can be traced back to early 2000s. Earlier DTC tests were thought to be convenient as they would allow the patients to access their genetic information without involvement of physician. Although, DTC genetic testing kits had several benefits, in the initial days, they were stringently regulated by regulatory bodies. Regulatory scenario has always been stringent since the introduction of DTC genetic kits in European countries. Currently, there has been change in the regulatory scenario and European countries have started receiving approval for DTC genetic kits. Defects in the DTC kits have been reduced and people have started relying on these kits. DTC genetic testing market is sort of matured in North America due to numerous technological advancements and is still in developing phase in Asian countries. With further advancements in technology, DTC genetic tests industry will experience numerous growth opportunitie

Key Insights Covered: Exhaustive Direct-to-Consumer Genetic Testing Market1. Market size (sales, revenue and growth rate) of Direct-to-Consumer Genetic Testing industry.2. Global major manufacturers operating situation (sales, revenue, growth rate and gross margin) of Direct-to-Consumer Genetic Testing industry.3. SWOT analysis, New Project Investment Feasibility Analysis, Upstream raw materials and manufacturing equipment & Industry chain analysis of Direct-to-Consumer Genetic Testing industry.4. Market size (sales, revenue) forecast by regions and countries from 2019 to 2025 of Direct-to-Consumer Genetic Testing industry.

Research Methodology: Direct-to-Consumer Genetic Testing Market

Quick Read Table of Contents of this Report @ Direct-to-Consumer Genetic Testing Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19)

Trusted Business InsightsShelly ArnoldMedia & Marketing ExecutiveEmail Me For Any ClarificationsConnect on LinkedInClick to follow Trusted Business Insights LinkedIn for Market Data and Updates.US: +1 646 568 9797UK: +44 330 808 0580

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Direct-to-Consumer Genetic Testing Market Analysis Of Global Trends, Demand And Competition 2020-2028 - 3rd Watch News

Three people with the inherited blood diseases sickle cell and beta thalassemia remain free of burdensome blood transfusions and their worst symptoms, months after receiving an infusion of genetically modified stem cells.

One of the three, a young woman with a severe form of beta thalassemia, has now been followed for over a year since she was treated, while the second, a woman in her 30s with sickle cell disease, is more than nine months removed from her infusion. They are the first two patients in pioneering studies of a therapy, developed by CRISPR Therapeutics and Vertex, that's based on the gene editing technology known as CRISPR.

Both patients continue to respond to treatment, bolstering evidence of genetic medicine's potential to permanently alter the course of devastating hereditary conditions like sickle cell and transfusion-dependent beta thalassemia. A gene therapy developed by Bluebird Bio has shown similar potential.

First results from the two studies, disclosed last November, were "taking the promise of CRISPR and turning that into a reality," said Samarth Kulkarni, CRISPR Therapeutics' CEO, in an interview. The additional data and follow-up now available "show these effects can be long-lasting and durable."

And in beta thalassemia, the first patient's experience is now supported by results from another patient who was treated about five months ago. This individual has also been able to stop receiving blood transfusions.

Taken together, the two patients responses are "proof of concept," CRISPR Therapeutics and Vertex claim, that their approach to treating beta thalassemia has the potential to be curative.

In sickle cell, the companies are also hopeful. The one patient for whom they have data has not had a vaso-occlusive crisis, a painful episode caused by the disease's characteristic sickling of red blood cells, since her treatment.

"The clinical manifestation of the disease is different, but we see consistent outcomes across both diseases," said Bastiano Sanna, Vertex's head of cell and genetic therapies, in an interview.

Three other beta thalassemia patients and one other sickle cell disease patient have been treated in the two studies of CRISPR Therapeutics and Vertex's therapy, dubbed CTX001. If results continue to look positive, CTX001 could be another powerful way to help people for whom treatment options have long been limited.

CRISPR, an easy-to-use method of genetic surgery that's derived from a bacterial defense system, has become a mainstay in labs across the world for all types of experiments. Its potential use as a human therapeutic has drawn closer as companies harnessing the technology CRISPR Therapeutics, Editas Medicine and Intellia Therapeutics have advanced their research. CRISPR Therapeutics is the first of the three to deliver results from a clinical trial.

CRISPR and Vertex unveiled their updated results at the European Hematology Association's virtual meeting on Friday. Also being presented were the latest data from Bluebird's gene therapy, known as LentiGlobin.

Bluebird is much further along, having treated 60 patients with beta thalassemia and 37 with sickle cell disease across six different studies.

Updated results from three of those studies showed 23 of 27 evaluable patients with beta thalassemia were transfusion independent for at least a year following treatment. And in sickle cell, no serious vaso-occlusive crises were observed in the 18 patients who had at least six months of follow-up. An episode was previously reported in one patient several months after LentiGlobin treatment, but was judged to be non-serious.

One sickle cell patient died suddenly 20 months following infusion with LentiGlobin, Bluebird reported Friday. Both the treating physician and an independent study committee concluded the death, ruled to be cardiovascular in nature, was unlikely to be related to the gene therapy.

Both beta thalassemia and sickle cell are diseases caused by mutations in the beta globin gene, faulty DNA that results in either absent or warped hemoglobin. Without enough hemoglobin, patients' red blood cells can't carry needed oxygen throughout the body. And those with sickle cell have abnormal hemoglobin that makes red blood cells fragile and stiff, causing them to stick in blood vessels.

Both diseases require chronic blood transfusions, and can lead to organ damage and reduced lifespans. Treatment options are limited, although that's now changing. The Food and Drug Administration, over the past few years, has approved Reblozyl, for beta thalassemia, and Oxbryta and Adakveo, for sickle cell.

Adakveo reduces the frequency of vaso-occlusive crises, while Reblozyl and Oxbryta are chronic medicines meant to boost patients' hemoglobin levels.

CRISPR Therapeutics and Vertex, along with Bluebird, are trying to accomplish the same goal but in more dramatic fashion: raising hemoglobin levels high enough so patients can stop blood transfusions and, in sickle cell, avoid pain crises altogether.

CRISPR and Vertex use CRISPR/cas9 gene editing to modify the DNA of stem cells extracted from a patient's bone marrow. The cells are engineered to produce a type of hemoglobin that's present at birth but normally replaced soon after. Once returned to the body and engrafted in the bone marrow, these CRISPR'd cells substitute this so-called fetal hemoglobin for the missing adult hemoglobin.

In the three patients treated so far, that appears to be what's happened. Both beta thalassemia patients are producing hemoglobin at levels considered normal. The sickle cell patient now has enough fetal hemoglobin to dilute the effects of sickled hemoglobin, potentially helping to preserve red blood cells.

Crucially, CRISPR and Vertex shared data for the first time indicating a high percentage of edited cells are present in each patient's bone marrow, supporting their confidence that the effects of treatment might last.

Bluebird, by contrast, doesn't edit the DNA of extracted stem cells, but rather inserts a modified gene into those cells. Once infused and engrafted in a patient, the cells can produce gene therapy-derived hemoglobin.

In most beta thalassemia and sickle cell patients treated with Bluebird's LentiGlobin, hemoglobin levels rose to normal or near-normal levels.

LentiGlobin is already approved for certain beta thalassemia patients in Europe as Zynteglo. In the U.S., Bluebird has hit delays and pushed back when it expects to submit an application to the middle of next year. A filing for an accelerated approval in sickle cell would likely follow sometime in the second half of 2021.

CRISPR and Vertex, meanwhile, plan to enroll more patients into their two studies, which they hope could serve as sufficient for an approval application if positive, Kulkarni said.

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New CRISPR, gene therapy results strengthen potential for treatment of blood diseases - BioPharma Dive

Finding Provides New Potential Approaches to Treating Fatal Disease

Researchers at the University of Maryland School of Medicine (UMSOM) have identified how certain gene mutations cause amyotrophic lateral sclerosis (ALS), also known as Lou Gehrigs disease. The pathway identified by the researchers may also be responsible for a certain form of dementia related to ALS. The finding could offer potential new approaches for treating this devastating condition, which causes progressive, fatal paralysis and sometimes mental deterioration similar to Alzheimers disease. Their discovery was published this week in theProceedings of the National Academy of Sciences (PNAS)and included collaborators from Harvard University, University of Auckland, Kings College London, and Northwestern University.

More than 5,000 Americans are diagnosed with ALS every year, a condition that is usually fatal and has no cure. Patients with ALS slowly lose the ability to move their muscles, leading to problems with basic functions such as breathing and swallowing. About half of ALS patients also develop dementia. Genetic studies of families with a predisposition to develop ALS have shown that the condition can be associated with certain gene mutations. Some of these mutations involve the gene UBQLN2 which regulates the disposal of misfolded garbage from the bodys cells. Until now, researchers did not fully understand how UBQLN2 mutations interfere with this pathway and cause ALS.

Mervyn Monteiro, PhDWe mapped out the process by which ubiquilin-2 (UBQLN2) gene mutations disrupt an important recycling pathway that cells use to get rid of their trash, saidMervyn Monteiro, PhD, Professor of Anatomy and Neurobiology, who is affiliated with the UMSOMs Center for Biomedical Engineering and Technology (BioMET) at UMSOM. Without this recycling, misfolded proteins build up in the nerve cell and become toxic, eventually destroying the cell. This destruction could lead to neurodegenerative disorders like ALS.

To investigate how UBQLN2 mutations cause ALS, Dr. Monteiros group used both human cells and UBQLN2-mutant mouse models for their investigations. The mouse models, which they described in a 2016PNAS publication, mimic the progression of the disease in people who inherit these gene mutations.

Dr. Monteiros group first removed the UBQLN2 gene from human cells and found it completely stalled the recycling pathway. They then reintroduced either the normal gene or one of five gene mutations into the cells. They found that reintroduction of normal UBQLN2 restored the recycling pathway while all five of the gene mutations failed to restart the pathway.

Using the mouse model, Dr. Monteiro and his colleagues outlined the reason for the pathway disruption in the presence of gene mutations. They found that the mice with the gene mutations had reduced levels of a certain protein called ATP6v1g1, which is an essential part of a pump that acidifies the cells trash container in order to initiate the breakdown and recycling process.

Our new findings are exciting because similar acidification defects have been found in Alzheimers, Parkinsons and Down syndrome, Dr. Monteiro said. This suggests that restoration of the defect could have broad implications for not only treating ALS, but possibly other neurodegenerative diseases as well.

The research study was supported by grants from the Packard Center for ALS Research at Johns Hopkins, the ALS Association, and the National Institutes of Health (grant number: R01-NS100008).

The BioMET research team led by Dr. Monteiro continues to make important advances in understanding the mechanisms that give rise to ALS, saidDean E. Albert Reece, MD, PhD, MBA, who is also Executive Vice President for Medical Affairs, UM Baltimore, and the John Z. and Akiko K. Bowers Distinguished Professor, University of Maryland School of Medicine. Future treatments and preventive measures for this devastating disease would not be possible without this foundational work.

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UM School of Medicine Researchers Identify New Genetic Defect Linked to ALS - BioSpace

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Biorepository project initiated early in pandemic to streamline coronavirus research efforts

Philip Mudd, MD, PhD, picks up research samples at the BJC Institute of Health at Washington University School of Medicine in St. Louis. Mudd, an emergency medicine physician, and his colleague, Jane O'Halloran, MD, PhD, an infectious diseases specialist, have led efforts to create a repository for storing and managing specimens collected from patients with COVID-19. The samples, including blood, urine and saliva specimens, are being distributed to investigators conducting COVID-19 research across the university.

In the weeks before the St. Louis region saw its first patients with COVID-19, physician-scientists at Washington University School of Medicine began planning and preparing how best to collect blood and other biological samples from such patients so specimens could be quickly disseminated to researchers seeking strategies to treat, prevent and contain the novel coronavirus.

With financial support from The Foundation for Barnes-Jewish Hospital, Siteman Cancer Center and Washington Universitys Institute of Clinical and Translational Sciences (ICTS), as well as input from the Community Advisory Board of Washington Universitys Institute for Public Health and ICTS, the School of Medicine created a repository to store and manage specimens collected from adult and pediatric patients who have tested positive for SARS-CoV-2 the virus that causes COVID-19. The sample-collection efforts are led by Philip Mudd, MD, PhD, an assistant professor of emergency medicine, and Jane OHalloran, MD, PhD, an assistant professor of medicine.

To date, more than 350 patients have donated samples for the research effort, and over 7,000 samples have been distributed to more than 20 labs on the School of Medicine and Danforth campuses to help understand the basic biology of the infection and seek ways to prevent or treat it.

The biorepository is an important resource for scientists working in many different areas of COVID-19 research, Mudd said. We are hopeful that the analysis of these samples will speed the development of treatments and new diagnostics that will help patients in St. Louis and around the world. Were grateful for the support of the foundations donors and for the generosity of our patients and are optimistic that this work will lead to new and useful breakthroughs in the treatment of COVID-19.

According to the investigators, collecting such samples including blood, urine, stool and nasal swabs via one centralized process speeds research, prevents scientists from duplicating work already underway and relieves patients of the burden of being asked to participate in multiple studies.

The project includes research teams that span the entire university, including investigators who are internationally recognized for their research into other viruses, such as Ebola, SARS, West Nile, Zika and emerging strains of influenza.

Studies underway include research to:

To receive samples for research, investigators must submit an application to a committee that is part of Washington Universitys ICTS, led by William G. Powderly, MD, the J. William Campbell Professor of Medicine and co-director of the universitys Division of Infectious Diseases. The ICTS committee managing patient specimens led by Christina A. Gurnett, MD, PhD, the A. Ernest and Jane G. Stein Professor of Developmental Neurology and director of the Division of Pediatric and Developmental Neurology is fielding many requests and encouraging a team science approach to enhance collaboration and reduce duplication of efforts. The biorepository has already led to new collaborations for researchers with complementary skills.

Its important to streamline this work from a research perspective, but its equally important to ease the process of participation for our patients, OHalloran said. Ordinarily, individual researchers design studies and approach patients separately. Knowing we would have many scientists studying COVID-19, it was critical for us to find a way to facilitate the process of obtaining patient samples. Were grateful to our patients for providing such a valuable resource to help us understand and combat this new virus.

Because some of the research may involve sequencing the DNA of patients with COVID-19 to understand each persons susceptibility to the illness, the researchers consulted the Community Advisory Board for guidance on how to address treatable genetic conditions independent of COVID-19 that might be revealed during the course of the research. The board, made up of 12 people with diverse leadership experience in local health-care and community organizations, provides advice to investigators on the conduct of clinical research involving the local community.

Anytime an individual undergoes whole genome sequencing, even people who may appear perfectly healthy, there is the potential to reveal genetic mutations that indicate a very high risk of developing a disease. Because some genetic conditions have treatments or prevention strategies available, doctors conducting COVID-19 research would like to have the ability to return this important information to patients, with patient permission, in a way that will allow patients and family members to make informed decisions about their health.

As part of studying the genetics of COVID-19, the researchers may learn information about a research participants risk of developing any one of 59 hereditary conditions that lead to very high risk of developing life-threatening but preventable or treatable diseases such as breast cancer, heart arrhythmias or blood-clotting disorders, said advisory board member Doug Lindsay, a personal medical consultant who advises patients with rare diseases or complex conditions. How do you best provide this kind of information if at all to patients or family members who are totally preoccupied with the immediate crisis of COVID-19 illness?

The overall thrust of our advice is, Give patients the choice to know or not know, Lindsay said. We also feel it is best to deliver this information at a later time, to give the patients, or family members if the patient has passed away, a chance to make an appointment to go over follow-up questions about the COVID-19 research study that they or their family members were enrolled in. The genetic studies of COVID-19 can reveal hereditary conditions, so the information is relevant for family members, too. We feel it is important to give people the mental space to deal with major medical decisions independent of the immediate pandemic.

The committee also has recommended that genetic counselors be made available to patients who are found to have any of these 59 conditions and choose to learn their genetic status. The committee also wants to ensure that patients and families know the kinds of medical specialists they should follow up with based on their genetic information.

These questions are about what a healthy relationship between participants and investigators looks like, Lindsay said. Participants who donate their data to these studies are real people. The information that the doctors are identifying may shape lives over more than one generation. We hope this kind of advice can help researchers be good stewards of the information put in their care.

Added Gurnett, The guidance provided by the Community Advisory Board has been critical to helping our investigators and clinicians provide the best possible care and conduct clinical research in the most ethical and respectful ways possible. We thank the patients and their families for their central contributions to the work of understanding this virus, as their participation is key to reducing its impact on our communities.

The Institute of Clinical and Translational Sciences is supported by the National Center for Advancing Translational Sciences of the National Institutes of Health (NIH), grant number UL1 TR002345.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Patients with COVID-19 donate specimens to advance research efforts - Washington University School of Medicine in St. Louis

June 10, 2020 In a new study, Johns Hopkins researchers found that testing people for SARS-CoV-2 (COVID-19) too early in the course of infection is likely to result in a false negative test, even though they may eventually test positive for the virus.[1] This is important to understand since many hospitals are using these COVID tests to screen patients before imaging exams, diagnostic testing or procedures.

The report found even a week after infection, one in five people who had the virus had a negative test result. The findings was published in the May 13 issue of Annals of Internal Medicine.

A negative test, whether or not a person has symptoms, doesnt guarantee that they arent infected by the virus, said Lauren Kucirka, M.D., Ph.D., M.Sc., obstetrics and gynecology resident at Johns Hopkins Medicine. How we respond to, and interpret, a negative test is very important because we place others at risk when we assume the test is perfect. However, those infected with the virus are still able to potentially spread the virus.

Kucirka said patients who have a high-risk exposure should be treated as if they are infected, particularly if they have symptoms consistent with COVID-19. This means communicating with patients about the tests shortcomings. One of several ways to assess for the presence of SARS-CoV-2 infection is a method called reverse transcriptase polymerase chain reaction (RT-PCR). These tests rapidly make copies of and detect the viruss genetic material. However, as shown in tests for other viruses such as influenza, if a swab misses collecting cells infected with the virus, or if virus levels are very low early during the infection, some RT-PCR tests can produce negative results. Since the tests return relatively rapid results, they have been widely used among high-risk populations such as nursing home residents, hospitalized patients and healthcare workers. Previous studies have shown or suggested false negatives in these populations.

For the new analysis, Johns Hopkins Medicine researchers reviewed RT-PCR test data from seven prior studies, including two preprints and five peer-reviewed articles. The studies covered a combined total of 1,330 respiratory swab samples from a variety of subjects including hospitalized patients and those identified via contact tracing in an outpatient setting.

Using RT-PCR test results, along with reported time of exposure to the virus or time of onset of measurable symptoms such as fever, cough and breathing problems, the researchers calculated the probability that someone infected with SARS-CoV-2 would have a negative test result when they had the virus infection. In the published studies, healthcare providers collected nasal and throat samples from patients and noted the time of virus exposure or symptom onset and sample collection.

From this data, the Johns Hopkins researchers calculated daily false-negative rates, and have made their statistical code and data publicly available so results can be updated as more data are published.

The researchers estimated that those tested with SARS-CoV-2 in the four days after infection were 67 percent more likely to test negative, even if they had the virus. When the average patient began displaying symptoms of the virus, the false-negative rate was 38 percent. The test performed best eight days after infection (on average, three days after symptom onset), but even then had a false negative rate of 20 percent, meaning one in five people who had the virus had a negative test result.

We are using these tests to rule out COVID-19, and basing decisions about what steps we take to prevent onward transmission, such as selection of personal protective equipment for healthcare workers, Kucirka explained. As we develop strategies to reopen services, businesses and other venues that rely on testing and contact tracing, it is important to understand the limitations of these tests.

Ongoing efforts to improve tests and better understand their performance in a variety of contexts will be critical as more people are infected with the virus and more testing is required. The sooner people can be accurately tested and isolated from others, the better we can control the spread of the virus, the researchers said.

Another John Hopkins study in March found the average incubation period for COVID-19 was approximately five days.[2] This was originally used as a guide by some in developing quarantine guidelines. However, this new study shows that test COVID PCR test results are not reliable for a firm diagnosis until well after a week of infection.

Additional authors include Denali Boon, Stephen Lauer, Oliver Layendecker and Justin Lessler and of Johns Hopkins.

Funding for the study was provided by the National Institute of Allergy and Infectious Diseases (R01AI135115 and T32DA007292), the Johns Hopkins Health System and the U.S. Centers for Disease Control and Prevention (NU2GGH002000).

Reference:

1. Lauren M. Kucirka, Stephen A. Lauer, Oliver Laeyendecker, et al. Variation in False-Negative Rate of Reverse Transcriptase Polymerase Chain ReactionBased SARS-CoV-2 Tests by Time Since Exposure. Annuals of Internal Medicine. May 13, 2020. doi.org/10.7326/M20-1495.

2. Stephen A. Lauer, Kyra H. Grantz, Qifang Bi, et al. The Incubation Period of Coronavirus Disease 2019 (COVID-19) From Publicly Reported Confirmed Cases: Estimation and Application. Ann Intern Med. 2020 Mar 10 : M20-0504. Published online 2020 Mar 10. doi: 10.7326/M20-0504.

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COVID-19 Genetic PCR Tests Give False Negative Results if Used Too Early - Imaging Technology News

Newswise Irvine, CA June 12, 2020 A new University of California, Irvine-led study reveals a protein responsible for genetic changes resulting in a variety of cancers, may also be the key to more effective, targeted cancer therapy.

The study, published today in Nature Communications, titled, Quantification of ongoing APOBEC3A activity in tumor cells by monitoring RNA editing at hotspots, reveals how the genomic instability induced by the protein APOBEC3A offers a previously unknown vulnerability in cancer cells.

Each day, in human cells, tens of thousands of DNA damage events occur. In cancer cells, the expression of the protein APOBEC3A is one of the most common sources of DNA damage and mutations. While the mutations caused by these particular proteins in cancer cells contribute to tumor evolution, they also cause breaks in the DNA, which offer a vulnerability.

Targeting cancer cells with high levels of APOBEC3A protein activities and disrupting, at the same time, the DNA damage response necessary to repair damages caused by APOBEC3A, could be key to more effective cancer therapies, said Remi Buisson, PhD, senior investigator and an assistant professor in the Department of Biological Chemistry at the UCI School of Medicine. However, to exploit the vulnerability of the cancer cells, it is critical to first quantitatively measure the proteins activity in tumors.

To understand the role of APOBEC3A in tumor evolution and to target the APOBEC3A -induced vulnerabilities, the researchers developed an assay to measure the RNA-editing activity of APOBEC3A in cancer cells. Because APOBEC3A is difficult to quantify in tumors, developing a highly sensitive assay for measuring activity was critical. Using hotspot RNA mutations, identified from APOBEC3A-positive tumors, the team developed an assay using droplet digital PCR and demonstrated its applicability to clinical samples from cancer patients.

Our study presents a new strategy to follow the dysregulation of APOBEC3A in tumors, providing opportunities to investigate the role of APOBEC3A in tumor evolution and to target the APOBEC3A-induced vulnerability in therapy, said Buisson. We anticipate that the RNA mutation-based APOBEC3A assay will significantly advance our understanding of the function of the protein in tumorigenesis and allow us to more effectively exploit the vulnerabilities it creates in cancer therapy.

This study was funded in part by the National Institutes of Health, a California Breast Cancer Research Program grant and an MPN Research Foundation Challenge grant.

About the UCI School of Medicine

Each year, the UCI School of Medicine educates more than 400 medical students, and nearly 150 doctoral and masters students. More than 700 residents and fellows are trained at UCI Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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UCI Researchers Uncover Cancer Cell Vulnerabilities; May Lead to Better Cancer Therapies - Newswise

Parasite infections are a constant presence for many people who live in tropical regions, particularly in less industrialized areas. These often chronic conditions are at best unpleasant; more seriously, children with parasite diseases that cause diarrhea can die of malnutrition or dehydration.

In Genome Biology, a study led by University of Pennsylvania scientists investigated the links between parasite infection and the gut microbiome. Using genetic methods to characterize the gastrointestinal microbiome of 575 ethnically diverse Cameroonian people representing populations from nine villages with meaningful differences in lifestyle, the researchers discovered that the presence of parasites was strongly associated with the overall composition of the microbiome.

We found that we could look at someones microbiome and use it to predict whether someone had a gastrointestinal parasite infection, says Meagan Rubel, who completed her doctorate degree at Penn and is now a postdoc at the University of California, San Diego. Whether or not it was parasites changing the microbiome or something in the resident microbiota of a person that made them more susceptible to infection, we cant say, but the association was strong.

Rubel led the study in collaboration with Penns Sarah Tishkoff, a Penn Integrates Knowledge Professor in the Perelman School of Medicine and School of Arts and Sciences, and Frederic Bushman, a microbiologist in the medical school. In addition to the microbiome and parasites, the research also examined markers of immune function, dairy digestion, and pathogen infection, a rich dataset.

The investigation entailed six months of field work, collecting fecal and blood samples from Mbororo Fulani pastoralists, cattle herders with a diet high in meat and dairy; Baka and Bagyeli rainforest hunter-gatherers, who practice a limited amount of farming but also forage for meat and plant-based foods; and Bantu-speaking agropastoralists, who both grow crops and raise livestock. As a comparison group, the study included data from two groups of people living in urban areas of the United States, with a diet heavier in animal fats, proteins, and processed foods.

In the field, the researchers tested for malaria and a number of other pathogens that infect both the blood and gastrointestinal system.

Of the 575 people tested in Cameroon, the researchers found nearly 40% were infected with more than one parasite before receiving an antiparasitic treatment, with hunter-gatherers, on average, most likely to be co-infected with multiple parasites. In particular, the team found that four soil-transmitted gut parasites tended to co-occur at a rate much higher than chance: Ascaris lumbricoides, Necator americanus, Trichuris trichiura, and Strongyloides stercoralis, or ANTS.

Gut parasites are a global public health concern, says Rubel. And you tend to see several of these parasites together in resource-poor settings where people may not have access to clinical care, piped water, and soap, so theres more opportunity for them to be transmitted.

Back in the lab at Penn, the researchers used genomic sequencing tools to take a snapshot of the participants gut microbiomes. The composition of the microbiome, they found, could accurately predict a persons country (U.S. or Cameroon) and lifestyle (urban, pastoralist, agropastoralist, or hunter-gatherer). But after these two variables, the presence of ANTS parasites could be predicted with greater accuracy by the microbiome structure than any other variable the research team studied. Taken together, the microbiome could predict the presence of these four gut parasites with roughly 80% accuracy.

Infection with these parasites also led to upticks in immune system activation, specifically turning on pathways that promote inflammatory responses. Parasite infection was also associated with a greater likelihood of having bacteria from the order Bacteroidales, which are known to play a role in influencing digestion and immune system function.

In a second part of the study, the Penn-led team assessed the relationship between the gut microbiome and milk consumption in the Fulani pastoralist population. Earlier work by Tishkoff and colleagues illuminated how genetic mutations enabling lactose digestion arose in pastoralist communities in Africa, selected through evolution because of the important nutritional benefits of consuming dairy.

In looking at the Fulanis microbiomes, they also tended to have an abundance of bacterial genes capable of breaking down galactose, a component of lactose, and fats, compared to other groups. This enrichment of genes could help you extract more nutrition from the food you eat, Rubel says.

The researchers believe their findings, the largest-ever study on the link between gut microbiome composition and parasite infection from sub-Saharan Africa, can open new possibilities for future work. The kinds of microbiome markers we found could be useful to predict the type of pathogens you have, or to shed light on the interplay between the microbiome and the immune system, says Rubel.

Eventually, she adds, more research could even illuminate strategies for purposefully modulating the microbiome to reduce the risk of a parasite infection or minimize the harm it causes to the body.

This research was supported in part by the Lewis and Clark Fund, University of Pennsylvania, Leakey Foundation, Wenner-Gren Foundation, National Institutes of Health (grants AI007532-18, DK104339-01, GM113657-01, GM134957-01, HL113252, HL137063, HL098957, HL087115, and HL115354), National Science Foundation (grants 1540432 and 1317217), American Diabetes Association, Penn Center for AIDS Research, and PennCHOP Microbiome Program.

Rubel, Bushman, and Tishkoffs coauthors on the paper were Penn Medicines Arwa Abbas, Louis J. Taylor, and Andrew Connell; the Childrens Hospital of Philadelphias Ceylan Tanes and Kyle Bittinger; the Johns Hopkins Cameroon Programs Valantine N. Ndze; the Yaound Central Hospitals Julius Y. Fonsah and Alfred K. Njamnshi; the University of Yaounds Eric Ngwang and Charles Fokunang; and the Mbalmayo District Hospitals Andr Essiane.

Frederic Bushman is the William Maul Measey Professor in Microbiology in the Perelman School of Medicine at Penn.

Meagan Rubel earned her Ph.D. from Penn and is now a postdoctoral researcher at the University of California, San Diego.

Sarah Tishkoff is the David and Lyn Silfen University Professor and aPenn Integrates Knowledge Professorat Penn, with appointments in the Department of Genetics in thePerelman School of Medicineand the Department of Biology in theSchool of Arts & Sciences.

Link:
Parasites and the microbiome - Penn: Office of University Communications

Dublin, June 12, 2020 (GLOBE NEWSWIRE) -- The "Personalized Medicine, Targeted Therapeutics and Companion Diagnostic Market to 2025 - Strategic Analysis of Industry Trends, Technologies, Participants, and Environment" report has been added to ResearchAndMarkets.com's offering.

This comprehensive report examines the precision medicine industry and its impact on the health system. This report tackles the growing market interest in pharmacogenomics, targeted therapeutics, companion diagnostics, and the associated market environment.

This report describes the current technologies that are propelling the personalized medicine and companion diagnostic market. It examines the current genetic diagnostic tests and companion diagnostic assays that are in use by the medical and pharmaceutical industry today. Current developments in personalized medicine and the pharmacogenomics revolution are discussed. The emerging trends that appear in key markets such as the US, UK, Germany, and France are elucidated and analysed. This study reveals market figures of the overall personalized medicine market and also sub-market figures.

The study also provides a comprehensive financial and product review of key players in the personalized medicine industry. Strategic drivers and restraints of this market are revealed and market opportunities and challenges are identified.

In summary, the personalized therapeutic and associated companion diagnostic market have huge opportunities for growth. This industry is revolutionizing the healthcare system and will improve therapeutic effectiveness and reduce the severity of adverse effects. It has enormous potential for investment and the emergence of genetic-based in vitro diagnostics. This is a comprehensive account of the market size, segmentation, key players, SWOT analysis, influential technologies, and business and economic environments.

The report is supported by over 360 tables & figures over 470 pages. The personalized medicine market is presented as follows:

A wealth of financial data & business strategy information is provided including:

SWOT, Economic & Regulatory Environment specifics include:

This report highlights a number of significant players and influential company's and gives details of their operations, products, financials and business strategy:

For more information about this report visit https://www.researchandmarkets.com/r/qmotdw

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global Strategic Analysis of the Personalized Medicine, Targeted Therapeutics and Companion Diagnostic Market 2020-2025 - GlobeNewswire

The HIV virus can take refuge in the brain even when treated with antiretroviral therapies, only to later infect other organs in the body if that treatment is stopped, a new study in mice and human tissue suggests.

Untreated HIV, the virus that causes AIDS, cripples the immune system and leaves the body vulnerable to life-threatening illness. Combination antiretroviral therapy, or cART, can significantly lower concentrations of the virus in the body, to the point that the pathogen can become undetectable, symptoms largely disappear and the treated person is no longer infectious to others. But cART must be taken daily, and if treatment pauses, the virus may reemerge from hidden sanctuaries in the body.

The new study, published June 11 in the journal PLOS Pathogens, suggests that one of these hideouts is in brain cells called astrocytes. Astrocytes constitute roughly 60% of the total cells in the human brain, according to the report, and in an infected person, the study authors estimate that between 1% and 3% of these cells could harbor HIV.

Related: Going viral: 6 new findings about viruses

"Even 1% could be significant as a reservoir, as a sanctuary site, for the virus," said study author Lena Al-Harthi, a professor and chair in the Department of Microbial Pathogens and Immunity at Rush University Medical Center in Chicago. "If we're going to try to find an HIV cure, you can't neglect the role of the brain as a reservoir."

Al-Harthi and her colleagues drew their conclusions from a mouse model of HIV injected with human cells, as well as examinations of postmortem human brain tissue. While both experiments provide insight into the role of astrocytes in HIV infection, more work must be done to nail down exactly how the virus takes hold in human patients, an expert told Live Science.

"Animal models can tell us quite a bit. They're not humans, but they can inform us quite a bit," said Dr. Lishomwa Ndhlovu, a professor of immunology in medicine at Weill Cornell Medicine, who was not involved in the study. If astrocytes can act as a reservoir for HIV in human infection, and that virus can exit the brain and trigger infection elsewhere, as the mouse study indicates, "we do need to figure out how to eliminate the virus from these compartments" to devise a successful cure, he said.

Astrocytes, named for their star-like shape, come in a variety of subtypes and play critical roles in the central nervous system, according to BrainFacts.org, a public information initiative run in part by the Society for Neuroscience. The cells help deliver nutrients to neurons, or the brain cells that transmit electrical signals, and they can spur or subdue inflammatory reactions in the brain. Astrocytes also shape and maintain the wiring of the central nervous system and fortify the blood-brain barrier, a border of tissue separating circulating blood from brain cells.

Scientists knew that the HIV virus infiltrates the brain during infection, as infected people can develop dementia and other cognitive deficits.

"The role of astrocytes in HIV infection has always been controversial," Al-Harthi told Live Science. Previous studies suggested that the star-shaped cells can become infected with HIV, but much of the research used cells in petri dishes, which may not replicate infection processes in a living animal, Al-Harthi wrote in a 2018 report published in The Journal of NeuroVirology. A few studies have utilized live animals but used "traditional" methods, such as tagging viral proteins or genetic material with fluorescent compounds, to scan for the virus that may not be sensitive enough to accurately detect the low levels of HIV present in astrocytes. No study attempted to address whether, once infected, astrocytes could somehow release HIV to organs beyond the brain.

Al-Harthi and her team developed two new mouse models to address this crucial question.

Related: Top 10 mysterious diseases

First, the authors placed human fetal astrocytes, derived from extracted brain tissue, in petri dishes and infected those cells with HIV. They then injected the infected cells into the brains of lab mice, one set of newborn mice and one set of adult mice. They found that, in both sets of mice, the infected astrocytes passed on the virus to CD4 cells a type of immune cell that helps orchestrate the body's immune response and are specifically targeted by the HIV virus.

After picking up an infection from astrocytes, infected CD4 cells migrate out of the brain and into other tissues. When "the brain is already seeded, the virus can come out and reseed peripheral organs," Al-Harthi said.

In particular, the authors noted that the spleen and lymph nodes become infected as a result of this process. By blocking the movement of CD4 cells, the authors could cut this chain of viral transmission.

To ensure that the virus could infect astrocytes on its own, without their assistance, the authors also ran an experiment in which they injected healthy human astrocytes into mice and infected the animals with HIV afterward. In this scenario, some human astrocytes still became infected and released HIV into the rest of the body. Notably, the virus could still escape from the brains of mice given cART treatment, "albeit at low levels" compared to untreated mice. If the treatment was stopped, the virus from the brain triggered a full-blown infection.

To confirm aspects of their mouse experiments, the authors examined the donated brains of four HIV-infected individuals, all of whom received effective cART treatment. (The report did not specify how each donor died, but noted that the virus was effectively suppressed by cART at the time of death.) The team found that a small percentage of astrocytes contained HIV genetic material in their nuclei, indicating that the cells had been infected.

Many questions about astrocytes and HIV remain to be answered. For instance, certain subtypes of astrocytes may serve as reservoirs of HIV, while others don't, Al-Harthi said. And while the mouse experiments demonstrated that HIV can exit the brain, the postmortem tissue analysis could not confirm that the same occurs in humans.

"Animal models, none of them are perfect," so there may be differences in how infection unfolds in people, Al-Harthi said.

For example, during natural HIV infection, the virus can accumulate genetic mutations each time it replicates, and the genetic material required for infection can be lost in the process, Ndhlovu said. To fully understand the role of astrocytes in HIV, researchers will need to determine how much of the virus present in human astrocytes can actually trigger infection, he said.

Al-Harthi and her team began to address this question by examining postmortem brain tissue and analyzing what segments of HIV genetic material could be found within but further studies will need to confirm that the found virus is both able to infect cells and migrate to other organs in the body, Ndhlovu said. In addition, scientists will need to determine the exact route HIV takes out of the brain in order to infect other organs, as that information would also be crucial for developing treatments that target the brain and finding a successful cure, he added.

Originally published on Live Science.

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HIV may hide out in brain cells, ready to infect other organs - Live Science

The increasing incidence of genetic disorders and chronic diseases is one of the biggest factors responsible for the burgeoning sales of regenerative medicine throughout the globe. The rising adoption of sedentary lifestyles and unhealthy dietary habits of the people all around the world are the main reasons causing the high prevalence of chronic diseases across the world. According to the World Health Organization (WHO), almost 17.9 million deaths are recorded every year because of cardiovascular diseases. Moreover, the cardiovascular diseases account for nearly 31% of the total deaths occurring across the world every year.

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The other major factors propelling the demand for regenerative medicine are the soaring investments being made by the governments of many countries in research and development activities in the domain of regenerative medicine, surging number of regenerative medicine companies throughout the world, and rapid technological developments in tissue engineering areas and stem cell research. Due to these factors, the global regenerative medicine market is expected to exhibit huge expansion over the coming years.

Across the globe, the regenerative medicine market is predicted to record the fastest growth in the Asia-Pacific (APAC) region in the upcoming years. This is mainly credited to the improving healthcare facilities and infrastructure in the region and the subsequent rise in stem cell research in the developing nations of APAC. For instance, the Chinese government has recently approved R&D activities pertaining to the human embryonic stem cells, which has in turn, encouraged more research on the clinical potential of the stem cells in the country.

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Global Regenerative Medicine Market By Type, Therapy, Application and Forecast Report by 2030 - Cole of Duty

- Treatment with TTP399 resulted in significant improvements in HbA1c with reduction in insulin, without increasing risk of hypoglycemia or diabetic ketoacidosis (DKA) -

HIGH POINT, N.C., June 13, 2020 (GLOBE NEWSWIRE) -- vTv Therapeutics Inc.(Nasdaq: VTVT) today made two presentations at the American Diabetes Associations 80th Scientific Sessions. The clinical data presented from the positive Phase 2 Simplici-T1 Study confirms the potential for TTP399 to provide a benefit beyond standalone insulin treatment for patients living with type 1 diabetes.

These results from the Simplici-T1 Trial are a major step towards the future of care in type 1 diabetes. They demonstrate that TTP399, a once-a-day pill, reduces HbA1c and improves time in range, without increasing hypoglycemia or any signal for adverse events including diabetic ketoacidosis, said John Buse, MD, Director of the North Carolina Translational and Clinical Sciences Institute and of the Diabetes Center at the University of North Carolina School of Medicine. Despite advances in insulin and type 1 diabetes technologies, affected patients continue to have difficulty achieving optimal glucose control. A safe and effective oral adjunctive therapy would be an important tool for patients and treating endocrinologists to improve both the daily burden of T1D and its long-term outcomes.

A copy of the poster presentations is available on the company website at http://vtvtherapeutics.com/publications/.

Details of the presentations follow:

Late Breaking Poster Presentation: Poster #122-LB, The Simplici-T1 Trial: Glucokinase Activator TTP399 Improves Glycemic Control in Patients with Type 1 Diabetes.

Presenter: John Buse, M.D., Ph.D., Director of the North Carolina Translational and Clinical Sciences Institute and of the Diabetes Center at the University of North Carolina School of Medicine

Key results presented included:

Late Breaking Poster Presentation: Poster #123-LB, The Simplici-T1 Trial: Relationship between Glycemic Control and Insulin Dose

Presenter: Carmen Valcarce, Ph.D., Chief Scientific Officer, vTv Therapeutics

Key results presented included:

The treat-to-target (FPG: ~80-130mg/dL; post meal glucose: <180-200 mg/dL) design of the study allowed changes in insulin dose after the insulin-optimization period. To evaluate the effect of these insulin adjustments on HbA1c, several pre-planned analyses were performed grouping the participants according to their change in total insulin doses (decreased, stable or increased).

About the Simplici-T1 StudySimplici-T1 was a multi-center, randomized, double-blind, adaptive study assessing the safety and efficacy of TTP399 as an adjunct to insulin therapy in adults with T1D. The primary endpoint was the change in HbA1c at week 12. The study was conducted with support from JDRF, the leading global organization funding research in type 1 diabetes.

This Phase 2 learn-and-confirm study was conducted in two parts under a treat-to-target protocol to evaluate the safety and efficacy of TTP399 in T1D patients over 12 weeks of daily dosing following a multi-week insulin optimization and placebo run-in period. Part 1 enrolled 19 patients on both insulin pumps and CGMs. The positive topline results from the learning phase - Part 1 were reported inJune 2019. The confirming phase - Part 2, enrolled 85 patients that used either insulin pumps or multiple daily injections of insulin; CGMs were allowed for those patients using the devices for at least three months prior to the start of the study. The positive topline results from Part 2 of the SimpliciT-1 Study were reported in February 2020.

About Type 1 DiabetesType 1 diabetes (T1D) is an autoimmune disease in which a persons pancreas stops producing insulin, a hormone that enables people to get energy from food. It occurs when the bodys immune system attacks and destroys the insulin-producing cells in the pancreas, called beta cells. While its causes are not yet entirely understood, scientists believe that both genetic factors and environmental triggers are involved. Its onset has nothing to do with diet or lifestyle. There is nothing you can do to prevent T1D, andat presentnothing you can do to cure it.

AboutvTv TherapeuticsvTv Therapeutics Inc.is a clinical-stage biopharmaceutical company focused on developing oral small molecule drug candidates. vTv has a pipeline of clinical drug candidates led by programs for the treatment of type 1 diabetes, Alzheimers disease, and inflammatory disorders. vTvs development partners are pursuing additional indications in type 2 diabetes, chronic obstructive pulmonary disease (COPD), and genetic mitochondrial diseases.

Forward-Looking StatementsThis release contains forward-looking statements, which involve risks and uncertainties. These forward-looking statements can be identified by the use of forward-looking terminology, including the terms anticipate, believe, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would and, in each case, their negative or other various or comparable terminology. All statements other than statements of historical facts contained in this release, including statements regarding the timing of our clinical trials, our strategy, future operations, future financial position, future revenue, projected costs, prospects, plans, objectives of management and expected market growth are forward-looking statements. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Important factors that could cause our results to vary from expectations include those described under the heading Risk Factors in our Annual Report on Form 10-K and our other filings with theSEC. These forward-looking statements reflect our views with respect to future events as of the date of this release and are based on assumptions and subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. These forward-looking statements represent our estimates and assumptions only as of the date of this release and, except as required by law, we undertake no obligation to update or review publicly any forward-looking statements, whether as a result of new information, future events or otherwise after the date of this release. We anticipate that subsequent events and developments will cause our views to change. Our forward-looking statements do not reflect the potential impact of any future acquisitions, merger, dispositions, joint ventures or investments we may undertake. We qualify all of our forward-looking statements by these cautionary statements.

Nura StrongVP of Business Developmentnstrong@vtvtherapeutics.com

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vTv Therapeutics Presents Two Late-Breaking Poster Sessions on Simplici-T1 Study at the American Diabetes Association's Virtual Sessions Supporting...