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Archive for the ‘Gene therapy’ Category

Rentschler Biopharma establishes Center of Excellence for cell and gene therapy in the UK – GlobeNewswire

Thursday, February 11th, 2021

LAUPHEIM, Germany and STEVENAGE, United Kingdom, Feb. 11, 2021 (GLOBE NEWSWIRE) -- Rentschler Biopharma SE, a leading global contract development and manufacturing organization (CDMO) for biopharmaceuticals, today announced that the Company is establishing a Center of Excellence for cell and gene therapy, which will be located in Stevenage, UK. With the newly formed UK subsidiary, Rentschler ATMP Ltd., the CDMO will establish development and manufacturing capability in Advanced Therapy Medicinal Products (ATMPs). The Companys UK site is being set up at an already existing facility run by the Cell and Gene Therapy Catapult (CGT Catapult) through a unique collaborative model. Over the next five years, Rentschler Biopharma plans to make a significant investment at the site to set up state-of-the-art manufacturing of viral vectors for clinical supply and expects to be ready for cGMP manufacturing by the first half of 2022.

Dr. Frank Mathias, CEO of Rentschler Biopharma, said: With more than 1000 cell and gene therapies currently in clinical development, the demand for manufacturing capabilities and support is ever increasing. Leveraging our specific expertise in process development and manufacturing for challenging molecules with highest quality and within demanding timelines, we are ideally suited to implement this knowledge in the field of viral vector production for new modalities. Our well-planned move into this growing area of biopharmaceuticals fits well with our strategic plans to expand internationally ensuring proximity to our clients and important biopharma hubs.

Rentschler Biopharma will continue its well-established practice of forming true partnerships with its clients. At this new Center of Excellence, the company plans to partner with entrepreneurial players to enable them to transform their ideas into real products with the potential to treat and even cure patients with serious and life-threatening diseases. Rentschler Biopharma utilizes a highly flexible business model that addresses the specific needs of each client.

About Rentschler Biopharma SE

Rentschler Biopharma is a leading contract development and manufacturing organization (CDMO), focused exclusively on client projects. The company offers process development and manufacturing of biopharmaceuticals as well as related consulting activities, including project management and regulatory support. Rentschler Biopharma's high quality is proven by its long-standing experience and excellence as a solution partner for its clients. A high-level quality management system, a well-established operational excellence philosophy and advanced technologies ensure product quality and productivity at each development and manufacturing step. In order to offer best-in-class formulation development along the biopharmaceutical value chain, the company has entered into a strategic alliance with Leukocare AG. Rentschler Biopharma is a family-owned company with about 1,000 employees, headquartered in Laupheim, Germany with a second site in Milford, MA, USA. In Stevenage, UK, Rentschler Biopharma launched a company dedicated to cell and gene therapies, Rentschler ATMP Ltd.

For further information, please visit http://www.rentschler-biopharma.com. Follow Rentschler Biopharma on LinkedIn and Facebook.

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Launch of Novel Gene Therapy Company, VectorY, to Develop Innovative Vectorized Antibodies – BioSpace

Thursday, February 11th, 2021

Feb. 10, 2021 08:00 UTC

AMSTERDAM--(BUSINESS WIRE)-- VectorY, a fully integrated gene therapy company focused on the development of innovative vectorized antibodies for muscular and neurodegenerative disorders, today announces its official launch. The Company has been operational since October 2020 and has established its laboratories and offices at the Amsterdam Science Park in The Netherlands.

VectorY develops proprietary and partnered programs based on a novel AAV platform and innovative antibody-based targeted degradation technologies. The Company is creating a pipeline of innovative vectorized antibodies targeting muscular and CNS diseases, aimed at overcoming limitations of current therapies, specifically by improving delivery, durability and accessibility of targeted tissues and cells. These novel viral vectors will have enhanced properties of non-immunogenicity, cell type specificity and expression in selected sub-cellular compartments, thus unlocking new treatment opportunities in these disease areas.

This year, the Company is setting up its own state-of-the-art GMP manufacturing facilities in Amsterdam. Its technology platform will be developed for scalability and yield, with the aim of significantly lowering the costs of advanced gene therapies.

VectorY has already negotiated its first corporate collaboration agreement, gaining access to a highly relevant neuro-inflammation target to develop a vectorized antibody therapy for treatment of neurological disorders. VectorY will continue to consider further collaborations in parallel to progressing its proprietary programs.

The highly experienced management team will be led by Marco Boorsma, General Partner at Forbion, who is joining the management team as Interim CEO. Co-founders Carlo Incerti, Pavlina Konstantinova, Anthony Newcombe, and Barbara Sanders all bring significant longstanding experience in gene therapy, vector development, and biopharmaceutical manufacturing expertise. Carlo Incerti, Operating Partner at Forbion and previously Chief Medical Officer of Sanofi Genzyme, will join VectorYs board as Chairman, together with Sander Slootweg, Managing Partner at Forbion, as board member.

VectorY was seeded by Forbion, following previous investments in successful gene vector companies including bluebird bio (then named Genetix), Biovex, Hookipa, EnGene, Amsterdam Molecular Therapeutics, uniQure and Replimune.

Carlo Incerti, Co-Founder and Chairman of the Board of VectorY, said: I have been working in the gene therapy field for over 30 years, and, from experience, know that accurately targeting the right cells with the transgene has always been of paramount importance. Using viral vectors developed at VectorY to deliver the genetic sequence for therapeutic antibody expression in target cells has the potential to address some of the key shortcomings of intravenously injected molecules. Our aim is to create novel treatments for patients.

Marco Boorsma, Interim CEO of VectorY, said: VectorY is developing innovative technologies to create novel, potentiated vectorized antibodies that will target muscle and CNS disorders, for which substantial unmet medical needs exist. Our aim is to bring new, powerful treatments to patients suffering from serious diseases. The Company is hitting the ground running, capitalizing on the extensive experience of the co-founders, and the support and expertise from Forbion as a longstanding gene therapy investor.

-Ends-

Notes to Editors

About VectorY VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for muscular and neurodegenerative diseases with high unmet medical need.

Founded in August 2020, and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics based on a novel AAV gene therapy platform, antibody-based targeted degradation technologies, and proprietary manufacturing technology.

VectorY develops proprietary & partnered programs and product candidates are based on new technologies that will enable the next generation of highly scalable manufacturing processes within VectorYs own manufacturing facilities. VectorYs manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands, with the capability to deliver suspension based AAV viral vector manufacturing of up to 2000L for both clinical and commercial supply.

About Forbion Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands, Germany and Singapore. Forbion invests in life sciences companies that are active in the (bio-) pharmaceutical space.

Forbion manages well over EUR 1.7 billion across multiple fund strategies that cover all stages of (bio)pharmaceutical drug development. Forbions current team consists of 20 life sciences investment professionals that have built an impressive performance track record since the late nineties with successful investments in over 69 companies.

The firm is a signatory to the United Nations Principles for Responsible Investment. Besides financial objectives, Forbion selects investments that will positively affect the health and well-being of patients.

Its investors include the EIF, through its European Recovery Programme (ERP), LfA, Dutch Venture Initiative (DVI), AMUF and EFSI facilities and KfW Capital through the Programme, ERP Venture Capital Fonds investments. Forbion operates a joint venture with BGV, the manager of seed and early-stage funds, especially focused on Benelux and Germany.

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

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Launch of Novel Gene Therapy Company, VectorY, to Develop Innovative Vectorized Antibodies - BioSpace

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The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 – GlobeNewswire

Thursday, February 11th, 2021

New York, Feb. 10, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" - https://www.reportlinker.com/p06021776/?utm_source=GNW

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually. Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes. There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

The following factors are likely to contribute to the growth of the Europe cell and gene therapy market during the forecast period: CMOs Offering Vector Manufacturing Services for Cell and Gene Therapy Companies Robust Cell & Gene Therapies in the Pipeline Increase in Strategic Acquisitions Regulatory Support for Cell and Gene Therapy Products

The study considers the present scenario of the Europe cell and gene therapy market and its market dynamics for the period 2020?2026. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Europe Cell and Gene Therapy Market Segmentation The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies. While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe. Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patients quality of life. Cell and gene therapies for certain types of cancers have been promising results. The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer. Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

Product Cell Therapies Gene Therapies End-user Hospitals Cancer Care Centers Wound Care Centers Others Application Oncology Dermatology Musculoskeletal Others

INSIGHTS BY GEOGRAPHY Germany, France, the UK, Italy, and Spain play a significant role in the Europe cell and gene therapy market. Clinical trials and the number of manufacturing facilities are increasing slowly in the European region. The region has become a major R&D destination for several vendors as the funding for cell & gene therapies is increasing. Europe has supported collaborative efforts in gene transfer and gene therapy research. In addition, the target patient population is increasing across Europe; there were an estimated 3.9 million new cases of cancer and 1.9 million cancer deaths in Europe in 2018. In addition, the prevalence surveys in the UK and Denmark indicate that there are 34 people with one or more wounds per 1,000 people. Favorable government support in terms of product approvals, reimbursement and coverage, and high R&D funding to academic institutes that are involved in the development of cell and gene therapies are expected to boosting the market in Europe.

Geography Europe o UK o Germany o France o Italy o Spain o Switzerland o Netherlands

INSIGHTS BY VENDORS Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors. While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors Novartis Spark Therapeutics Amgen Gilead Sciences Organogenesis

Other Prominent Vendors APAC Biotech AVITA Medical bluebird bio CHIESI Farmaceutici CollPlant CO.DON Human Stem Cells Institute PJSC (HSCI) Medipost NuVasive Nipro Orchard Therapeutics RMS Regenerative Medical System Orthocell Osiris Therapeutics Sibino GeneTech Shanghai Sunway Biotech Takeda Pharmaceutical Company Terumo Vericel

Emerging Investigational Vendors In Europe Adaptimmune Therapeutics AgenTus Therapeutics Autolus Cellecits Celyad CombiGene EUKARS Freeline Therapeutics Innoskel PsiOxus Therapeutics Ltd SparingVision uniQure

KEY QUESTIONS ANSWERED 1. What is the Europe cell and gene therapy market size and growth rate during the forecast period? 2. What are the factors driving the demand for CAR-T therapy in the European region? 3. How are strategic acquisitions aiding in market growth of cell and gene therapy products? 4. Which segments are expected to generate the highest revenues during the forecast period? 5. Who are the leading vendors in the European cell and gene therapy market?Read the full report: https://www.reportlinker.com/p06021776/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Global Genes Announces New Multimedia Series Focused on Advances in Gene Therapy and Editing, in Collaboration with the National Institutes of Health…

Thursday, February 11th, 2021

ALISO VIEJO, Calif.--(BUSINESS WIRE)--As gene therapies and editing technologies rapidly advance, it is more urgent than ever to provide updates and information to the rare disease community on how these technologies can be applied across multiple diseases. Global Genes, a leading rare disease patient advocacy organization, is pleased to announce they will be publishing a multimedia series, titled Platforms of Hope: Advances in Gene Therapy and Gene Editing, throughout 2021 regarding upcoming data announcements and information on gene therapy and editing technology advances with thought leaders from the National Center for Advancing Translational Sciences (NCATS), the NIH Common Funds Somatic Cell Genome Editing (SCGE) program and other leading voices in these fields.

In addition to ongoing coverage through Global Genes videos, online publication RARE Daily and RARECast podcast, the organization will be publishing a special report at the end of 2021 on gene therapy and gene editing innovation. This will address a wide range of topics, with a focus on efforts to accelerate the translation of discoveries into genetic medicines that benefit patients with rare diseases.

With more than 7,000 rare diseases, there is an urgent need to keep the rare disease community abreast of developments in the rapidly changing fields of gene therapy and genome editing, said P.J. Brooks, program director at the Office of Rare Diseases Research at the NCATS. In this collaboration, NIH will help Global Genes identify the ideas, technologies and advances that have broad implications for many patients and families affected by rare diseases and provide information that could positively impact their lives and care in the future.

This collaboration will bring visibility into cutting-edge science at the frontier of genetic medicines and provide the rare disease community with insights into emerging technologies and therapies in development for rare diseases, said Christian Rubio, vice president, strategic advancement at Global Genes. Its critically important to educate the rare disease community on these rapidly evolving events.

For more information, visit http://www.globalgenes.org/media-hub.

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf -- helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease, or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.

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Avrobio Gene Therapy Shows Early Promise in Fabry, Other Rare Lysosomal Diseases – BioSpace

Thursday, February 11th, 2021

Shares of AVROBIO wereup nearly 20% in premarket trading after the company posted positive clinical data from its gene therapy trials in three different rare lysosomal diseases, Fabry, Gaucher type 1 and cystinosis.

The data from the Phase II study assessing AVR-RD-01, an investigational ex vivo lentiviral gene therapy for Fabry disease, was particularly promising. This morning, Cambridge, Mass.-based Avrobio said a second kidney biopsy conducted on the first patient dosed with AVR-RD-01 showed 100% clearance of the toxic substrate Gb3.

Kidney substrate reduction is the primary endpoint of the Phase II study and has been a cornerstone for evaluating and approving treatments in Fabry disease, Avrobio noted. Avrobio said the patient came in with significant toxic buildup in his kidneys, which is quite common with Fabry disease. One year after the gene therapy treatment was administered in the trial, two independent pathologists foundzero markersof toxic substrate across all the 99 biopsy slides each evaluated.

The first biopsy conducted on the patient showed an 87% clearance of the substrate. In addition to the substrate clearance in the Fabry disease study, AvroBio reported continued strong and durable results in other key metrics across all nine Phase I and Phase II Fabry patients. All patients are now producing the functional enzyme they need to clear toxic substrate from their cells and seeing a concurrent drop in plasma substrate. The farthest patient is out 3.5 years, the company said.

Avrobio Chief Executive Officer Geoff MacKay hailed the data and said it was a thrilling way to begin 2021. The data announced this morning builds on the breadth of strong clinical data weve reported across our leading lysosomal disorder pipeline of single-dose gene therapies, he added.

The Fabry disease study wasnt the only positive news from Avrobio. The company also announced six-month data from the first patient dosed in the Phase I/II study of AVR-RD-02, an investigational ex vivo lentiviral gene therapy for Gaucher disease type 1. That data showed plasma chitotriosidase and the toxic metabolite lyso-Gb1, which are key biomarkers of Gaucher disease, had both dropped 49% below the patients baseline levels that had been achieved on enzyme replacement therapy (ERT) before gene therapy was administered. Also, Lyso-Gb1, the toxic metabolite that builds up in cells throughout the body in Gaucher, is down 44% below the patients ERT baseline. Avrobio said this is an early sign of efficacy.

Based on the data observed to date, we believe lentiviral gene therapy drives down toxic metabolites below levels of ERT, supporting our view that gene therapy has the potential to prevent, halt or even reverse progression of these devastating diseases with a single infusion, MacKay said.

For the cystinosis study, Avrobio said three patients who are taking part in the study are now off of standard-of-care treatment. The first study patient has had sharp reductions in crystal density in the eyes and skin and a marked improvement in photophobia, which is an extreme sensitivity to light that is associated with the disease.

With 13 patients dosed across three clinical programs, we have observed sustained and potentially transformative improvements in key biomarkers and functional metrics, with data from our Fabry disease program out 3 years after dosing. Additionally, enrollment activities for our Fabry disease trial are accelerating, giving us added confidence in our efforts to meet our goal of having dosed a cumulative 30 patients across all our clinical programs by the end of the year. With this strong momentum, we look forward to clarifying the regulatory pathway with regulatory agencies, MacKay said in a statement.

Full data from the studies will be presented later this week and WORLDSymposium, an annual meeting dedicated to lysosomal disorders.

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Amicus Therapeutics Presents Positive Preclinical Fabry Disease Gene Therapy Data at the 17th Annual WORLDSymposium 2021 – GlobeNewswire

Thursday, February 11th, 2021

Amicus Optimized Transgene Show Greater Substrate Reduction than Wild Type Construct Across All Tissues and Doses

Further Validates Combining Amicus-Engineered Transgenes with Penns AAV Gene Therapy Technologies to Develop Next Generation Gene Therapies

PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Fabry disease in mice. The results are featured in a virtual poster presentation at the 17th Annual WORLDSymposium 2021, being held February 8-12, 2021. The poster is also available in the Events and Presentations section of the Amicus Therapeutics corporate website.

Fabry disease is an inherited lysosomal disorder caused by deficiency of the enzyme alpha-galactosidase A (GLA). Reduced or absent levels of GLA lead to accumulation of disease substrate leading to cellular disfunction and organ damage, which results in the clinical manifestations of Fabry disease. Amicus, in collaboration with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania (Penn), is developing a novel gene therapy for Fabry disease that combines the Amicus protein-engineering expertise and deep knowledge and experience in Fabry disease with Penns adeno associated virus (AAV) gene transfer technologies.

This initial preclinical study assessed a range of single doses of AAV in Gla knockout (KO) mice with either natural unmodified hGLA (wildtype hGLA) or Amicus/Penn engineered hGLA transgenes (engineered hGLA). The Amicus/Penn engineered hGLAs are designed for improved stability which is believed to provide a larger window for the enzyme to stay active while in circulation prior to being taken up into the target tissues and for additional stabilization after cell uptake. The lead Amicus/Penn engineered hGLA declared as an IND candidate is designated as AT-GTX-701.

Preclinical Poster Highlights for Amicus/Penn AAV Gene Therapy for Fabry Disease:

Hung Do, Ph.D., Chief Science Officer of Amicus Therapeutics, stated, These very important preclinical results validate our capabilities to develop engineered proteins via a gene therapy that can result in superior substrate reduction compared with a wildtype transgene. This is the second program in our collaboration with Penn that has demonstrated the potential advantages of optimizing the target protein in these disorders, and may be applicable to other lysosomal disorders as we continue to combine our understanding of the molecular basis of these diseases and expertise in protein engineering, together with Penns vector engineering expertise, to develop novel gene therapies.

Amicus is currently developing AAV gene therapies in collaboration with Penn for Pompe disease, Fabry disease, CDD, CLN1, MPS IIIB, a next generation program in MPS IIIA, as well as Angelman Syndrome. The agreement between Amicus and Penn is a Research, Collaboration and License Agreement, providing funding to Penn to advance the preclinical research programs in the Wilson Lab and to license certain technologies invented under the funded Research Collaboration.

About Fabry DiseaseFabry disease is an inherited lysosomal disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to irreversible organ damage regardless of the time of symptom onset.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Fabry disease in mice and the potential implications of these data for the future advancement and development of a gene therapy for Fabry disease and other lysosomal disorders and development of potential platform technologies. Words such as, but not limited to, look forward to, believe, expect, anticipate, estimate, intend, "confidence," "encouraged," potential, plan, targets, likely, may, will, would, should and could, and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the preliminary data reported before completion of the study will not be predictive of future results, that results of additional preliminary data or data from the completed study or any future study will not yield results that are consistent with the preliminary data presented, that later study results will not support further development, or even if such later results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully commercialize. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2019 and the Quarterly Report filed on Form 10-Q for the quarter ended September 30, 2020. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof.

CONTACTS:

Investors:Andrew FaughnanSr. Director, Investor Relationsafaughnan@amicusrx.com(609) 662-3809

Media:Diana MooreHead of Global Corporate Communicationsdmoore@amicusrx.com(609) 662-5079

FOLDG

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Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 – Insight, Epidemiology and Forecasts – ResearchAndMarkets.com – Yahoo…

Thursday, February 11th, 2021

The "Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Market Insight, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

This report delivers an in-depth understanding of the AAV Vector Based Gene Therapy, historical and forecasted epidemiology as well as the AAV Vector Based Gene Therapy market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The AAV Vector Based Gene Therapy market report provides emerging drugs, AAV Vector Based Gene Therapy market share of the individual diseases, current and forecasted AAV Vector Based Gene Therapy market size from 2017 to 2030 segmented by seven major markets. The Report also covers current AAV Vector Based Gene Therapy market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

The AAV Vector Based Gene Therapy epidemiology division provides the insights about historical and current AAV Vector Based Gene Therapy patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted AAV Gene Therapies epidemiology [segmented as Total cases of AAV Gene Therapy Targeted Indications, Total diagnosed indication-specific cases, Number of Patients Eligible for AAV Gene Therapy and Total treated cases] scenario of AAV Gene Therapy in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Country Wise- AAV Vector Based Gene Therapy Epidemiology

Estimates show that the highest cases of AAV vector based gene therapy in the 7MM were in the United States, followed by Germany, Japan, France, the United kingdom, Italy, and Spain in 2017.

Story continues

AAV vector based gene therapy Drug Chapters

Drug chapter segment of the AAV vector based gene therapy report encloses the detailed analysis of AAV vector based gene therapy marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the AAV vector based gene therapy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Scope of the Report

The report covers a detailed overview of the development of AAV vectors and gene therapies based on them across the various therapeutic areas and several selected indications, explaining its various serotypes, benefits, and challenges for future

Comprehensive insight has been provided into the AAV Vector Based Gene Therapy epidemiology and treatment in the 7MM.

Additionally, an all-inclusive account of both the current and emerging therapies for AAV Vector Based Gene Therapy is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.

A detailed review of AAV Vector Based Gene Therapy market; historical and forecasted is included in the report, covering drug outreach in the 7MM.

The report provides an edge while developing business strategies, by understanding trends shaping and driving the global AAV Vector Based Gene Therapy market.

AAV Vector Based Gene Therapy Report Insights

Patient Population

Therapeutic Approaches

AAV Vector Based Gene Therapy Pipeline Analysis

AAV Vector Based Gene Therapy Market Size and Trends

Market Opportunities

Impact of upcoming Therapies

AAV Vector Based Gene Therapy Report Key Strengths

AAV Vector Based Gene Therapy Report Assessment

SWOT Analysis

Current Treatment Practices

Unmet Needs

Pipeline Product Profiles

Conjoint Analysis

Market Attractiveness

Market Drivers and Barriers

Reasons to Buy

The report will help in developing business strategies by understanding trends shaping and driving the AAV Vector Based Gene Therapy market.

To understand the future market competition in the AAV Vector Based Gene Therapy market and Insightful review of the key market drivers and barriers.

Organize sales and marketing efforts by identifying the best opportunities for AAV Vector Based Gene Therapy in the US, Europe (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Identification of strong upcoming players in market will help in devising strategies that will help in getting ahead of competitors.

Organize sales and marketing efforts by identifying the best opportunities for AAV Vector Based Gene Therapy market.

To understand the future market competition in the AAV Vector Based Gene Therapy market.

Companies Mentioned

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/p3sdwo

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Contacts

ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.com

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Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 - Insight, Epidemiology and Forecasts - ResearchAndMarkets.com - Yahoo...

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Taysha Gene Therapies Announces Participation in Upcoming Investor Healthcare Conferences – Yahoo Finance

Thursday, February 11th, 2021

TipRanks

Investors have been fixated on growth companies over the past year, and one segment which has been on the rise is the fledgling cannabis industry. The sector offers a unique proposition and the prospect of further growth, as there is still a major catalyst on the horizon which will completely alter the industry. As expected, a Democrat led senate has been good news for those banking on marijuana reform at the federal level; And it looks like the anticipated changes could happen faster than initially expected. Backed by Senate majority leader Chuck Schumer, Democratic Senators have stated that they will push for federal-level legalization of marijuana, promising a unified discussion draft on comprehensive [cannabis] reform in the first half of this year. The statement feeds expectations that the Democratic Congressional majority will pass and that President Biden will sign a bill to legalize marijuana. Investors are also looking at further state-level legalization moves; one key state in this regard is New York. So, the cannabis industry is looking up. There is an expanding network of state legalization regimes, and expectations of a change in federal policy; both are putting upward pressure on cannabis shares. Against this backdrop, we used TipRanks database to find two cannabis stocks that have been earmarked as 'Strong Buys' by the analyst consensus. Both have posted impressive year-to-date performances, and stand to rise even more in the year ahead. Village Farms International (VFF) We will start with Village Farms International, a company that has long been involved in the niche agricultural business. The company started out as a farmer, producing high-quality greenhouse vegetables year-round for sale in the North American market. That background fit the company well for a transition to the cannabis industry Village Farms has experience in greenhouse production and industrial-scale growing. Village Farms shares are showing a tremendous growth profile, up 327% in the past 12 months with a strong spike in recent days. Two important pieces of news precipitated the surge since the end of January. First, the company has fully repaid ahead of schedule the $15 million debt it incurred during its November acquisition of the cannabis growing company Pure Sunfarms. And second, Village Farms increased its investment in the Asian cannabinoid company Altum by 50%, to hold a 10% stake in the company. The move increases the international reach of Village Farms, and its ability to increase Altum holdings in the future. The company was able to fund these moves because it had a successful equity sale in January, putting an additional 10.8 million shares on the market, and raising US$135 million in new capital. In addition to its strong capital and expansion positions, Village Farms has been reporting solid financial results. The company saw US$43 million in revenue for 3Q20, a gain of 12.5% year-over-year. EPS came in at 1 cent per share, a turnaround from the US$0.10 loss in the year-ago quarter. Covering Village Farms for Craig-Hallum, 5-star analyst Eric Des Lauriers writes: Village Farms has clearly established itself as the leading cannabis producer in Canada with #1 brand share and industry-leading profitability. Canadian cannabis sales in 2020 through October (latest available) were up 128% y/y, and dispensary counts are set to accelerate through 2021, providing a tailwind to VFF revenues. Turning to the US markets, and VFFs position in Canadas larger neighbor, the analyst goes on to add, With 5.7M SF of greenhouses in TX, the company also has real US optionality, which is finally being appreciated by investors after the GA election. VFF has historically been undervalued compared to less profitable peers, but we expect shares to continue working higher as the prospect for US reform increases throughout the year. To this end, Des Lauriers rates VFF a Buy, and his $25 price target suggests the stock has room for ~26% upside in the coming year. (To watch Des Lauriers track record, click here) Overall, there are 3 recent reviews on VFF shares, and all are Buys, giving the stock a Strong Buy analyst consensus rating and showing a general agreement on Wall Street about the companys strengths. Shares are priced at $19.90, and the $24.33 average price target implies an upside of ~23% for the year ahead. (See VFF stock analysis on TipRanks) TerrAscend Corporation (TRSSF) The next cannabis stock were looking at, TerrAscend, is another major cannabis producer in both the US, Canada, and Europe. The company is involved in both the medical and recreational sides of the market, and both grows and produces cannabis and markets a range of products through numerous brand names. TerrAscends US operations are located in California, Pennsylvania, New Jersey, and Utah, and the company looks to expand as more states legalize cannabis. In a strong sign of the cannabis industrys strength, TRSSF shares are up a sky-high 624% over the past 12 months. Growth has been fueled by expansion of the cultivation operations in California and Pennsylvania, and by the move into the adult-use recreational market in New Jersey. Last month, TerrAscend closed a non-brokered private placement stock sale, putting more than 18 million common shares on the market. The sale price was C$12.35 (US$9.72), and the offering grossed C$224 million (US$176.3 million). The bulk of the proceeds some 80% of the total was put up by four large US-based institutional investors. The funds raised will be used to continue expansion of the companys cultivation operations (TRSSF has plans to expand growing and manufacturing ops in New Jersey), as well as to pursue merger & acquisition activities. TerrAscends rapid growth and strong future prospects have attracted attention from top-rated analysts, including 5-star analyst Eric Des Lauriers of Craig-Hallum (stated above). "TerrAscend is a leading multi-state operator (MSO) in the US cannabis market with top-tier management, assets, and access to deal flow. We have been bullish on the company since initiating coverage last year and are happy to say the TRSSF team has exceeded our expectations, generating rapid increases in margins and operating leverage that have earned them a place solidly in the Top Tier of MSOs," Des Lauriers noted. The analyst summed up, "[With] US$280M+ raised since the elections and federal reform moving quicker than expected, we think TRSSF does deserve a premium to peers." In line with his bullish comments, Des Lauriers rates TRSSF shares a Buy, and has a $20 price target that implies a ~31% upside potential for the next 12 months. Once again, were looking at a stock with broad agreement from Wall Streets analysts the Strong Buy consensus rating is unanimous, based on 7 recent reviews. Shares are selling for $15.30, and their recent appreciation has pushed that price almost up to the $15.43 average price target. (See TRSSF stock analysis on TipRanks) To find good ideas for cannabis stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Taysha Gene Therapies Announces Participation in Upcoming Investor Healthcare Conferences - Yahoo Finance

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Paragon Biosciences Expands Cell And Gene Therapy Platform – Contract Pharma

Thursday, February 11th, 2021

Paragon Biosciences, a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence, has launched CiRC Biosciences, a cell therapy company developing treatments for serious diseases with high, unmet needs with an initial focus on the eye."The addition of CiRC Biosciences to our portfolio builds upon our cell and gene therapy platform, an area that has tremendous potential to address serious genetic diseases," said Jeff Aronin, founder, chairman and chief executive officer, Paragon Biosciences. "CiRC Biosciences gives us the science to target retinal diseases that could lead to vision restoration with numerous other applications in the years ahead."CiRC Biosciences is currently advancing pre-clinical development of chemically induced retinal cells for vision restoration in Geographic Atrophy Age-Related Macular Degeneration (Dry AMD), which is the most common cause of irreversible vision loss over the age of 65, and advanced Retinitis Pigmentosa (RP), a genetic disorder that causes tunnel vision and eventual blindness. There are no U.S. Food & Drug Administration (FDA) approved treatments to restore vision loss in Dry AMD or RP.The company's novel mechanism of action is designed for direct chemical conversion of fibroblasts into other cell types using a cocktail of small molecules in an 11-day chemical conversion process. Pre-clinical studies have shown efficacy in blind mice that demonstrated vision restoration. CiRC Biosciences has provisional patent applications to protect its platform."Our technology transforms ordinary skin cells into specialized retinal cells using a cocktail of small molecules," said Sai Chavala, M.D., co-founder and chief scientific officer, CiRC Biosciences. "This process is potentially safer, quicker, more cost effective and easier to manufacturer than using traditional stem cells. Working with Paragon Biosciences to build and advance CiRC Biosciences provides us the opportunity to efficiently progress this technology through research and development stages.CiRC Biosciences first reported its discovery in the highly respected scientific journal Nature (April 15, 2020). A recently published New England Journal of Medicine article (Nov. 5, 2020) discussed CiRC's technology of using chemically induced cells to restore retinal function. The article concluded, "The new and emerging strategies for the rescue, regeneration, and replacement of photoreceptors suggest a bright future in the fight to preserve and restore vision in blinding eye diseases."The abstract in Nature is available here.Access to the NEJM article is available here.

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Paragon Biosciences Expands Cell And Gene Therapy Platform - Contract Pharma

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FDA Clears IND Application for Passage Bio’s Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare…

Thursday, February 11th, 2021

DetailsCategory: DNA RNA and CellsPublished on Monday, 08 February 2021 16:09Hits: 412

- Phase 1/2 trial expected to commence in first half of 2021

- Company has three INDs cleared for rare monogenic CNS disorders

PHILADELPHIA, PA, USA I February 08, 2021 I Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy being studied for the treatment of early infantile Krabbe disease (Globoid Cell Leukodystrophy). Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare lysosomal storage disease that most often presents early in a childs life, resulting in rapid progressive damage to both the brain and peripheral nervous system and mortality by two years of age. Underscoring the urgent medical need in the patient population, the FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 for treatment in Krabbe disease.

As part of our commitment to deliver a transformative, one-time gene therapy to the children and their families who suffer from the devastating effects of Krabbe disease, we are excited to advance toward clinically evaluating the potential life-changing benefits of PBKR03, said Bruce Goldsmith, Ph.D., chief executive officer of Passage Bio. The FDA clearance of our IND for PBKR03 is an important milestone for Passage Bio, paving the way for the start of our third clinical program in rare monogenic CNS disorders in the first half of 2021. Having solidified our clinical trial preparedness and manufacturing readiness during the past year, we are well-positioned to move with urgency to advance PBKR03 into the clinic.

PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C). Low GAL-C activity results in accumulation of psychosine which is toxic to the myelin-producing oligodendrocytes of the CNS and Schwann cells in the periphery, resulting in damage to both the central and peripheral nervous systems. PBKR03 has the potential to treat both the central nervous system and peripheral nerve manifestations observed in Krabbe disease patients.

Compelling preclinical data support advancement into clinical trials

PBKR03 is supported by extensive preclinical studies, conducted by our collaborator, the University of Pennsylvanias Gene Therapy Program, showing meaningful transduction of both the central and peripheral nervous system in animal models, with restoration of myelination in the brain and peripheral nerves. In a naturally occurring Krabbe animal model, a single ICM injection of an AAVhu68 capsid containing the normal canine GALC gene showed normalization of GALC activity, reduction of cerebral spinal fluid psychosine levels, normalization of peripheral nerve conduction velocity, improvement in brain myelination, reduction in brain inflammation and increased survival.

Phase 1/2 study anticipated for 1H21

Passage Bio expects to initiate a Phase1/2 clinical trial for PBKR03 in the first half of 2021. The trial is designed as a dose escalation study of a single ICM dose of PBKR03 in pediatric subjects with early infantile Krabbe disease. The primary endpoint of the Phase 1/2 study is safety and tolerability; secondary endpoints include CSF and serum GALC levels, disease biomarkers, and clinical outcome measures. Initial data from the trial is anticipated to potentially readout in late 2021 or early 2022, depending on the timing of when the first patient is treated in the study.

PENN Financial Disclosure

The University of Pennsylvania (Penn) and its Gene Therapy Program receives sponsored research funding from Passage Bio, and Penn has licensed intellectual property to Passage Bio that may result in future financial returns to Penn.

About Krabbe Disease

Krabbe disease is a rare and often life-threatening lysosomal storage disease caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine. Without adequate levels of galactosylceramidase, psychosine accumulates, causing widespread death of myelin-producing cells and progressive damage to nerves in both the brain and peripheral tissues. The early infantile form of the disease is the most severe and common, typically manifesting before six months of age and accounting for 60 percent to 70 percent of diagnoses. In these patients, the disease course is highly predictable and rapidly progresses to include loss of acquired milestones, staring episodes, apnea, peripheral neuropathy, severe weakness, unresponsiveness to stimuli, seizures, blindness, deafness and eventual death by two years of age. Late infantile patients, defined by onset between seven to twelve months of age, present similar symptoms and have a median survival of approximately five years from onset of symptoms. The estimated worldwide incidence of Krabbe disease is 2.6 in 100,000 births, which is higher than reported due to lack of adequate screening at birth.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at http://www.passagebio.com.

SOURCE: Passage Bio

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FDA Clears IND Application for Passage Bio's Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare...

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Neurons from patient blood cells enable researchers to test treatments for genetic brain disease – Brown University

Thursday, February 11th, 2021

PROVIDENCE, R.I.[Brown University] New research provides insights into the treatment of Christianson syndrome (CS), an X-linked genetic disease characterized by reduced brain growth after birth, intellectual disability, epilepsy and difficulties with balance and speech.

One of the major challenges in developing treatments for human brain disorders, like CS, is developing an experimental system for testing potential therapeutics on human neurons, said study senior author Dr. Eric Morrow, an associate professor of molecular biology, neuroscience and psychiatry at Brown University. In recent years, advanced stem cell therapies that use tissues from patients have provided powerful new approaches for engineering human neurons from the patients themselves, who may undergo the treatment in the future.

For the study, published in Science Translational Medicine on Feb. 10, 2021, Morrow and his colleagues obtained blood samples from five CS patients and the patients unaffected brothers. They then reprogrammed these blood cells into stem cells, and these stem cells were converted into neurons in a petri dish. As a result, they obtained neurons that were representative of those from CS patients, and they used these neurons to test treatments.

Morrow who directs the Center for Translational Neuroscience at the Carney Institute for Brain Science and the Brown Institute for Translational Science said the team also used a new gene-editing approach that employs CRISPR-Cas9 technologies to correct patient mutations back to a healthy gene sequence.

CS is caused by a mutation in a gene encoding for NHE6, a protein that helps regulate acid levels within cell structures called endosomes. Past research suggests that the loss of NHE6 causes endosomes to become overly acidic, which disrupts the abilities of developing neurons to branch out and form connections in the growing brain.

Loss of this important protein can arise from a variety of gene mutations in patients. The majority of CS mutations are called nonsense mutations, which prevent NHE6 from being produced at all; four of the five CS patients involved in this study exhibited this class of mutation. However, some CS patients exhibit missense mutations. Individuals with missense mutations still have some NHE6, but it is produced in smaller amounts, and the protein fails to function as it should.

The research team tested two main forms of treatment on the stem-cell-derived neurons: first, gene transfer, which involves adding a healthy NHE6 gene into the cell; and second, administration of trophic factors, which are substances that promote neuron growth and encourage neurons to develop connections with other neurons. The researchers found that the neurons response to treatment depended on the class of mutation present.

The gene transfer studies, which may represent the first steps toward developing gene therapy, were successful in neurons with nonsense mutations. After the researchers inserted a functional NHE6 gene into nonsense-mutation CS neurons, the neurons branched out properly. In neurons with missense mutations, however, gene transfer failed completely. Further tests suggested that the abnormal NHE6 produced as a result of missense mutations may interfere with normal NHE6, thereby rendering gene transfer therapy ineffective in patient cells with these mutations.

In contrast, administration of trophic factors, such as brain-derived neurotrophic factor (BDNF) and insulin-like growth factor-1 (IGF-1), successfully promoted proper branching in all the CS neurons studied, regardless of mutation type.

While these initial results are encouraging, Morrow hopes that future studies will examine these treatments in animal models.

Our results provide an initial proof-of-concept for these treatment strategies, indicating that they should be studied further, he said. However, we may ultimately need to pay close attention to the class of mutation that a patient has when we choose a specific treatment.

In addition to Morrow, the research team included scientists from Brown University, the University of South Carolina and the Icahn School of Medicine at Mount Sinai. The study was supported by multiple grants from the National Institutes of Health as well as a number of awards from foundations and academic institutions.

This news story was authored by contributing science writerKerry Benson.

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Neurons from patient blood cells enable researchers to test treatments for genetic brain disease - Brown University

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Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key…

Thursday, February 11th, 2021

New York, Feb. 05, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" - https://www.reportlinker.com/p06020737/?utm_source=GNW Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods (such as genome sequencing), and genome editing and manipulation technologies (such as molecular switches), have enabled the development of novel therapy development tools / platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

SCOPE OF THE REPORT The Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Amongst other elements, the report features: - A detailed review of the overall market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any). - A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. - An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors. - A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies. - Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company. - An elaborate discussion on the various commercialization strategies that can be adopted by drug developers across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing. - A review of the various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates. - An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2016. The analysis assesses several relevant parameters associated with the patents, including type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation. - A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area. - An analysis of the investments made at various stages of development in companies that are focused in this area, between 2015-2020, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings. - A detailed geographical clinical trial analysis of completed, ongoing and planned studies of numerous gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population. - An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies. - An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio. - An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. - A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors. - A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vectors used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals: - Adam Rogers (CEO, Hemera Biosciences) - Al Hawkins (CEO, Milo Biotechnology) - Buel Dan Rodgers (Founder & CEO, AAVogen) - Christopher Reinhard (CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)) - Michael Triplett (CEO, Myonexus Therapeutics) - Robert Jan Lamers (CEO, Arthrogen) - Ryo Kubota (CEO, Chairman & President, Acucela) - Tom Wilton (CBO, LogicBio Therapeutics) - Jeffrey Hung (CCO, Vigene Biosciences) - Cedric Szpirer (Executive & Scientific Director, Delphi Genetics) - Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) - Molly Cameron (Corporate Communications Manager, Orchard Therapeutics)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include - Annual reports - Investor presentations - SEC filings - Industry databases - News releases from company websites - Government policy documents - Industry analysts views

While the focus has been on forecasting the market over the coming decade, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWERED - Who are the leading industry players engaged in the development of gene therapies? - How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products? - Which types of vectors are most commonly used for effective delivery of gene therapies? - What are the key regulatory requirements for gene therapy approval, across various geographies? - Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development? - What are the different pricing models and reimbursement strategies currently being adopted for gene therapies? - What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies? - Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development? - What are the key value drivers of the merger and acquisition activity in the gene therapy industry? - Who are the key stakeholders that have actively made investments in the gene therapy domain? - Which are the most active trial sites (in terms of number of clinical studies being conducted) related to this domain? - How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.

Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic disorders, metabolic disorders, nervous system disorders, oncological disorders and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted in relation to gene therapy and gene editing technologies. It also features a detailed analysis, highlighting the prevalent trends related to type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation. For the purpose of this analysis, we have taken into consideration patents that have been filed / granted since 2016.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2015 and 2020, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents a detailed, geographical clinical trial analysis of completed, ongoing and planned studies focused on gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 highlights top big biopharma players engaged in the field of gene therapy, featuring a heat map analysis based on several parameters, including therapeutic area, type of vector used, therapeutic approach and type of gene therapy.

Chapter 19 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 20 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2030. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vector used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world).

Chapter 21 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 22 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 23 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 24 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Christopher Reinhard (CEO & Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (CEO, Chairman & President, Acucela), Tom Wilton (CBO, LogicBio Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Molly Cameron (Corporate Communications Manager, Orchard Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 25 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 26 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06020737/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key...

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FDA approves third gene therapy for large B-cell lymphoma – European Pharmaceutical Review

Thursday, February 11th, 2021

Breyanzi (lisocabtagene maraleucel) was approved on the 54 percent complete remission rate achieved in diffuse large B-cell lymphoma trials.

Breyanzi (lisocabtagene maraleucel), a chimeric antigen receptor (CAR) T cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma who have not responded to, or relapsed, after at least two other types of systemic treatment has been approved by the US Food and Drug Administration (FDA).

According to the agency, Breyanzi is the third gene therapy approved in the US for certain types of non-Hodgkin lymphoma, including diffuse large B-cell lymphoma (DLBCL). Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma.

Todays approval represents another milestone in the rapidly progressing field of gene therapy by providing an additional treatment option for adults with certain types of cancer affecting the blood, bone marrow, and lymph nodes, commented Dr Peter Marks, director of the FDAs Center for Biologics Evaluation and Research. Gene and cell therapies have evolved from promising concepts to practical cancer treatment regimens.

DLBCL is the most common type of non-Hodgkin lymphoma in adults. Approximately 77,000 new cases of non-Hodgkin lymphoma are diagnosed in the US each year, with DLBCL accounting for around a third of newly diagnosed cases.

Breyanzi is customised for each patient; their T cells, a type of white blood cell, are collected and genetically modified to include a new gene that facilitates targeting and killing of the lymphoma cells. Once the cells are modified, they are infused back into the patient.

The safety and efficacy of the treatment were established in a multi-centre clinical trial of more than 250 adults with refractory or relapsed large B-cell lymphoma. The complete remission rate after treatment with Breyanzi was 54 percent.

The treatment can cause severe side effects, including cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T cells, causing high fever and flu-like symptoms and neurologic toxicities. Both CRS and neurological events can be life-threatening, so the therapy is being approved with a risk evaluation and mitigation strategy (REMS) which includes elements to assure safe use (ETASU).

The requirements include, among other things, that healthcare facilities that dispense Breyanzi be specially certified, with staff involved in the prescribing, dispensing or administering of the treatment being trained to recognise and manage the risks of CRS and neurologic toxicities.

Other side effects include hypersensitivity reactions, serious infections, low blood cell counts and a weakened immune system. According to the FDA, side effects generally appear within the first one to two weeks following treatment, but some side effects may occur later.

To further evaluate the long-term safety, the FDA is also requiring the manufacturer to conduct a post-marketing observational study involving patients treated with Breyanzi.

The approval was granted to Juno Therapeutics Inc., a Bristol-Myers Squibb Company.

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Adverum to Present Data from the OPTIC Phase 1 Trial with ADVM-022 Intravitreal Gene Therapy in Wet AMD at the Angiogenesis, Exudation, and…

Thursday, February 11th, 2021

REDWOOD CITY, Calif., Feb. 10, 2021 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the presentation of data from Cohorts 1-4 of the OPTIC Phase 1 clinical trial of ADVM-022 intravitreal (IVT) injection gene therapy in patients requiring frequent anti-VEGF injections for their wet age-related macular degeneration (AMD). These data were previously presented as part of a corporate update on Saturday, November 14, 2020.

Oral Presentation Title: Intravitreal Gene Therapy for Exudative AMD and Diabetic RetinopathyDate and Time: Saturday, February 13, 2021 at 9:30 am ETSession V: Gene Therapy for Exudative AMD and Diabetic RetinopathyPresenter: Arshad M. Khanani, M.D., M.A., Director of Clinical Research, Sierra Eye Associates

A copy of this presentation will be available as the presentation begins in the publications and presentation section of Adverums website.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is advancing the clinical development of its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration and diabetic macular edema. For more information, please visitwww.adverum.com.

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Adverum to Present Data from the OPTIC Phase 1 Trial with ADVM-022 Intravitreal Gene Therapy in Wet AMD at the Angiogenesis, Exudation, and...

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World Symposium Orchard leads the crop of Hurler syndrome hopefuls – Vantage

Thursday, February 11th, 2021

Gene therapy companies have been under pressure lately, but Orchard Therapeutics got a lift yesterday from promising early data with its mucopolysaccharidosis type I candidateOTL-203.

The company is seeking to supersede the current standard of care, enzyme-replacement therapy or bone marrow transplant. But other gene therapy contenders are not too far behind, notablyRegenxbio, which in December started a proof-of-concept study of its rival project, RGX-111.

Good IDUA

Both projects seek to deliver the -l-iduronidase (IDUA) gene, which is mutated in MPS-I, leading to a deficiency of the IDUA enzyme. This enzyme usually breaks down glycosaminoglycans (GAGs), so in MPS-I patients these build up, causing tissue and organ damage. Symptoms of MSP-I, also known as Hurler syndrome, include cognitive impairment and skeletal deformity; if left untreated, patients rarely survive beyond the age of 10.

And both OTL-203 and RGX-111 are designed as one-time therapies, whereas the current enzyme replacement, Biomarin/Sanofis Aldurazyme, is given intravenously once a week.

However, the gene therapy candidates go about restoring IDUA enzyme activity in different ways. OTL-203 uses hematopoietic stem cells taken from the patient, then genetically modified using a lentiviral vector to express the IDUA gene, before being reinfused.

RGX-111, meanwhile, uses an adeno-associated viral vector to deliver the gene directly to the brain, getting around a central problem with Aldurazyme, which cannot cross the blood-brain barrier.

Getting into the brain should not be a problem for OTL-203 either, Orchards head of medical affairs, Leslie Meltzer, told Evaluate Vantage. She explained that hematopoietic stem cells naturally cross the blood-brain barrier and, once in the CNS, differentiate into a microglial-like cell.

This claim appears to be supported by the latest data, which admittedly come in just a handful of subjects. The eight-patient phase I/II trial, presented at the World Symposium yesterday, found increases in the IDUA enzyme in patients blood and cerebrospinal fluid. There was also a decrease in GAGs in the CSF and urine.

Encouragingly, this activity appears to have translated into a clinical benefit: all eight patients showed stable cognitive scores and stable motor function versus baseline, as well as growth in the normal range for patients age.

Its a progressive disease, so youd expect these things to worsen over time, but the fact they continued to be stable is very promising, Ms Meltzer said.She admitted that the data were early, with only around a year of follow-up on most of the clinical endpoints.

Orchard plans to start a registrational study by the end of this year.Ms Meltzer would not give any details ondesign, saying this would be finalised after feedback from regulators.

Regenxbios proof-of-concept study of RGX-111 is due to complete in November, putting the project about a year behind OTL-203.

One candidate that will go no further is Sangamos SB-318. The company reported disappointing data with the in vivo zinc finger nuclease genome-editing project two years ago, and has since said it would focus on second-generation zinc finger projects.

Still, even two gene therapies might be too many for an ultra-rare disease like MPS-I, which affects just one in 100,000 people. Asked whether this market could support more than one gene therapy, Ms Meltzer said newborn screening recently implemented in countries including the UScould lead to a revision of that estimate.

But, as in other rare disorders that have attracted several gene therapy players, a battle over a limited patient pool could be shaping up.

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AGTC Executives Awarded First Place in the BioProcess International Reader’s Choice Awards, Cell & Gene Therapies Category – GlobeNewswire

Thursday, February 11th, 2021

Article reflects Companys leadership and innovation in scalable, reproducible manufacture of adeno-associated virus (AAV)-based gene therapies

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, announced that Sue Washer, President & Chief Executive Officer and Dave Knop, Vice President of Process Development, have been awarded first place in the BioProcess International (BPI) magazine inaugural Readers Choice Awards program, cell and gene therapies category, for their article, Viral-Vectored Gene Therapies: Harnessing Their Potential Through Scalable, Reproducible Manufacturing Processes.

High-productivity approaches to AAV manufacturing processes, like AGTCs HSV-helper based platform, will be crucial if we are to address the unmet clinical need growing across a variety of indications, said AGTC President and CEO, Sue Washer. There is no question that investing in the manufacturing process is imperative and our early commitment in this area has put AGTC in a strong position with respect to the purity and quality needed for late stage development and commercialization.

Concentrating on articles published from September 2019 through June 2020, and using rankings based on views, engagement, and download rates, BioProcess International identified the four most popular articles within each of its six pillars of bioprocessing coverage. The AGTC authors article received the highest number of votes from BPI readers, who ranked the nominees in terms of their innovativeness, presentability and applicability.

The eBook featuring the first-place article by Washer and Knop, as well as summarized versions of the second- and third-place articles, are available by visiting: https://bioprocessintl.com/wp-content/uploads/2020/11/18-11-eBook-RCA-CellGeneTherapies.pdf.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Companys most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Companys industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS:David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.com or glenn.silver@finnpartners.com

Corporate Contact:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

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Actinium Showcases Targeted Conditioning Program with 2 Oral Presentations Highlighting Iomab-B and Pivotal Phase 3 SIERRA Trial at 2021…

Thursday, February 11th, 2021

NEW YORK, Feb. 11, 2021 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") today highlighted its presence at the 2021 Transplantation & Cellular Therapy (TCT) Annual Meeting, which is being held virtually from February 8th 12th. The TCT meeting organizes thousands of transplant professionals from over five hundred transplant centers worldwide and is a seminal event for Actinium given its focus on targeted conditioning for bone marrow transplant (BMT), CAR-T and other adoptive cell therapies and gene therapy. At TCT, Actinium's pivotal Phase 3 trial SIERRA trial for Iomab-B was featured in 2 oral presentations, as well as CME event focused on AML and BMT and in investigator interactions led by Actinium's clinical development and medical affairs teams.

Dr. Mark Berger, Actinium's Chief Medical Officer, said, "TCT is the ideal venue to showcase Actinium's Iomab-B and Iomab-ACT targeted conditioning programs given the concentrated audience of thought leaders in these fields that TCT brings together. The timing of TCT is also ideal as it follows shortly after ASH resulting in a data rich period for Actinium that drives investigator interest. This is particularly the case this year as we have built strong momentum in SIERRA following positive data from 75% enrollment featured in 2 oral presentations at ASH and now in 2 oral presentations at this year's TCT, which has driven high levels of investigator and referring physician interactions. We have coupled this with bolstered outreach efforts, which will continue beyond TCT, that have resulted in new site activation despite the advanced stage of SIERRA and robust enrollment rates that give us great confidence in completing SIERRA enrollment rapidly."

Summary data presented in TCT oral presentations include:

-100% BMT and engraftment rate for patients receiving a therapeutic dose of Iomab-B compared to 18% of patients receiving physician's choice of salvage therapy on the control arm- 79% of all patients enrolled on SIERRA were able to proceed to BMT despite being a patient population not considered eligible for BMT with standard approaches-Iomab-B delivers high amounts of targeted radiation to the bone marrow with minimal impact on other organs resulting in lower rates and severity of adverse events

TCT Oral Presentation: Targeted Radioimmunotherapy with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] in Older Patients with Active, Relapsed or Refractory (R/R) Acute Myeloid Leukemia Results in Successful and Timely Engraftment Not Related to the Radiation Dose Delivered

Phase 3 SIERRA 75% Enrollment Results

Baseline Characteristics

Iomab-B Arm(N=56)

Conventional Care (CC) Arm(N=57)

Age (yrs, median, range)

63 (55-77)

65 (55-77)

Cytogenetic and Molecular Risk1, 2

Favorable: 4%Intermediate: 35%

Adverse: 61%

Favorable: 5%

Intermediate: 32%

Adverse: 63%

% TransplantedIntent-to-Treat Group

88% (49/56)

18% (10/57)

64% (30/47)

Results

Underwent Iomab-B based Conditioning and HCT (N=49)3

Achieved CR and received standard of care HCT (N=10)

Randomized to Conventional Care and Crossed Over to Iomab-B with HCT (N=30)4

Cross-over Rate

n/a

n/a

Received Therapeutic Dose of Iomab-B (N=30)

Transplanted (N=30)

64% (30/47)

% Transplanted

100% (49/49)

18% (10/57)

100% (30/30)

% Marrow Blast @ randomization (median, range)

29% (4-95)5

20% (5-97)

28% (6-87)

Days to ANC Engraftment

14 (9-22)6

17 (13-83)7

14 (10-37)8

Days to Platelet Engraftment

18 (4-39)6

22 (8-35)7

19 (1-38)8

Days to HCT (Post Randomization)

30 (23-60)

67 (52-104)

62 (36-100)9

Myeloablative Dose Delivered to Bone Marrow

14.7 (4.6-32) Gv

n/a

15.5 (6.3-42) Gv

592 (313-1013) mCi

646 (354-1027) mCi

100-day non-Relapse Transplant-Related Mortality

4%

(2/45 Evaluable)

20%

(2/10 Evaluable)

10.7%

(3/28 Evaluable)

1) Iomab-B arm: data unavailable (4) and patient was excluded (1)

2) Per NCCN guidelines version 3. 2020

3) No therapy dose (7) due to: declining KPS (4), Infusion reaction (1), unfavorable biodistribution (1), post- randomization eligibility (1). Two (2) did not receive DI and five (5) received DI without proceeding to TI.

4) Thirteen (13) patients ineligible for crossover due to: hospice care/progression (4), declined/ineligible for HCT (5), died pre-crossover (4). Additionally, four (4) patients were eligible for crossover and did not receive Iomab-B due to declining KPS.

5) One (1) patient with 4% blasts in the marrow had circulating AML blasts

6) ANC engraftment data not available (4), platelet engraftment data not available (7)

7) ANC and platelet engraftment data not available (1)

8) ANC engraftment data not available (1), platelet engraftment data not available (2)

9) One (1) patient at 161 days had delayed transplant due to infection & respiratory failure, received Iomab & transplant when stable, not included in range

https://tct.confex.com/tct/2021/meetingapp.cgi/Paper/16878

TCT Oral Presentation: Myeloablative Targeted Conditioning with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Spares the GI Tract and Has Low Incidence of Severe Mucositis, Febrile Neutropenia and Sepsis in the Prospective, Randomized Phase 3 Sierra Trial for Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)

Adverse Event

Iomab-B Arm (N=56)

Conventional Care Arm (N=57)

Received Iomab-B/HCT

(N=49)1

Achieved CR and received Std HCT (N=10)

No CR Crossed over to Iomab-B/HCT (N=30)

Sepsis

% (N)

4.2 (2)*

30.0 (3)

23.3 (7)

Febrile Neutropenia Gr 3-4

% (N)

41.7 (20)

50.0 (5)

40.0 (12)

Mucositis Gr 3-4

% (N)

10.4 (5)

30.0 (3)

16.7 (5)

Day +100 Non-Relapse Mortality3

2/45(4.4%)

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Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 – Arizton – PRNewswire

Wednesday, February 3rd, 2021

CHICAGO, Feb. 2, 2021 /PRNewswire/ -- In-depth analysis and data-driven insights on the impact of COVID-19 included in this Europe cell and gene therapy market report.

The Europe cell and gene therapy market is expected to grow at a CAGR of over 23% during the period 20202026.

Key Highlights Offered in the Report:

Key Offerings:

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Europe Cell and Gene Therapy Market Segmentation

Europe Cell and Gene Therapy Market by Product

Europe Cell and Gene Therapy Market by End-user

Europe Cell and Gene Therapy Market by Application

Europe Cell and Gene Therapy Market Dynamics

Cell and gene therapy is revolutionizing the global healthcare segment. Although various new cell and gene therapies are approved, there are various hurdles that limit the penetration of new therapies, such as high cost, multiple regulatory hurdles, and other manufacturing challenges. These cell and gene therapy developers need reliable, efficient, and cost-effective manufacturing services with the flexibility to scale up production as the demand increases. Cell and gene therapy products are very complex, and their manufacturing requires skilled labor, developed infrastructure for limited patients. Such huge investments will affect vendors and contract manufacturing organizations (CMOs) work with companies to overcome these challenges.

Key Drivers and Trends fueling Market Growth:

Europe Cell and Gene Therapy Market Geography

European countries such as Germany, France, the UK, Italy, and Spain play a significant role in the cell and gene therapy market. However, clinical trials and the number of manufacturing facilities are increasing slowly in Europe. Europe has become a major R&D destination for many vendors as the funding for cell and gene therapies is increasing across many European countries. Europe stands next to North America in the global cell and gene therapy market. Initially, Europe led the cell and gene therapy market due to first product approvals. France, Germany, and Italy had a greater contribution globally and in Europe. However, from the past decade, the US has competed and increased its market share globally. Europe stands second in the market, with the increasing prevalence of cancer and rare genetic disorders that are not effectively solved by the conventional therapies are increasing in the region. This increased target population is driving the demand for cell and gene therapy in the region.

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Europe Cell and Gene Therapy Market by Geography

Major Vendors

Other Prominent Vendors

Emerging Investigational Vendors In Europe

Explore our healthcare & lifesciencesprofile to know more about the industry.

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About Arizton:

AriztonAdvisory and Intelligence is an innovation and quality-driven firm, which offers cutting-edge research solutions to clients across the world. We excel in providing comprehensive market intelligence reports and advisory and consulting services.

We offer comprehensive market research reports on industries such as consumer goods & retail technology, automotive and mobility, smart tech, healthcare, and life sciences, industrial machinery, chemicals and materials, IT and media, logistics and packaging. These reports contain detailed industry analysis, market size, share, growth drivers, and trend forecasts.

Arizton comprises a team of exuberant and well-experienced analysts who have mastered in generating incisive reports. Our specialist analysts possess exemplary skills in market research. We train our team in advanced research practices, techniques, and ethics to outperform in fabricating impregnable research reports.

Mail: [emailprotected] Call: +1-312-235-2040 +1-302-469-0707

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Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market – BioSpace

Wednesday, February 3rd, 2021

Global Retinal Gene Therapy Market: Overview

The retinal gene therapy market is estimated to expand at an exponential growth rate. For the use of gene therapy, retina is considered a highly desirable target as it an irreplaceable part of a body. The global retinal gene therapy market is likely to be influenced by the promise its holds for the treatment of various forms of inherited and non-inherited blindness. Furthermore, this therapy can also be used in the treatment of rare genetic retinal diseases, such as Leber's congenital amaurosis, which is likely to augur well for the development of the global retinal gene therapy market during the forecast period, from 2020 to 2030. It is expected that the global retinal gene therapy market is anticipated to witness the entry of new players, with the presence of promising candidates in the phases of drug approval process.

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Type, application, and region are the three important parameters based on which the global retinal gene therapy market has been classified. Such detailed analysis of the market comes with the sole purpose to provide stakeholders with a detailed and clear analysis of the global retinal gene therapy market.

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Global Retinal Gene Therapy Market: Notable Developments

One of the important market developments that give a quick view of the dynamics pertaining to the global retinal gene therapy market is mentioned as below:

There is only one player in this global retinal gene therapy market, which is mentioned as below:

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Global Retinal Gene Therapy Market: Key Trends

The global retinal gene therapy market is characterized by the presence of the following restraints, drivers, and opportunities.

Advent of High-end Technologies to Support Development of the Market

Mostly in the cases of inherited retinal diseases, retinal gene therapy is performed. Gene therapy is capable of bettering vision impairment through mutation in RPE65 gene. Luxturna, a recently introduced gene therapy is utilized for the treatment of patients suffering from type 2 Leber's congenital amaurosis. This disease is a form of inherited disease that causes impairment in vision at the time of birth, which leads to a highly progressive degeneration. At present, there are many retinal gene therapy at the clinical trial phase and those are utilizing recombinant viruses. This factor is likely to increase the scope of growth for the global retinal gene therapy market over the period of assessment, from 2020 to 2030.

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In addition to that, the emergence of new market players together with the advent of high-end technological developments is likely to encourage growth of the global retinal gene therapy market during the forecast period. It is estimated that retinal gene therapy is likely to come up as a standard form of treatment for such retina-related diseases.

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Global Retinal Gene Therapy Market: Geographical Analysis

North America is clearly at the forefront of the growth of the global retinal gene therapy market at the very moment. It is estimated that the region will continue to retain its dominance over the period of forecast, from 2020 to 2030. So far, the product that has been approved for use is from a manufacturer from this region. Europe is likely to emerge as another lucrative region in the global retinal gene therapy market over the period of forecast.

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Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market - BioSpace

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Paragon Biosciences Launches CiRC Biosciences to Expand Cell and Gene Therapy Platform – PRNewswire

Wednesday, February 3rd, 2021

CHICAGO, Feb. 2, 2021 /PRNewswire/ -- Paragon Biosciences, a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence, today announced the launch of CiRC Biosciences, a cell therapy company developing treatments for serious diseases with high, unmet needs with an initial focus on the eye.

"The addition of CiRC Biosciences to our portfolio builds upon our cell and gene therapy platform, an area that has tremendous potential to address serious genetic diseases," said Jeff Aronin, founder, chairman and chief executive officer of Paragon Biosciences. "CiRC Biosciences gives us the science to target retinal diseases that could lead to vision restoration with numerous other applications in the years ahead."

CiRC Biosciences is currently advancing pre-clinical development of chemically induced retinal cells for vision restoration in Geographic Atrophy Age-Related Macular Degeneration (Dry AMD), which is the most common cause of irreversible vision loss over the age of 65, and advanced Retinitis Pigmentosa (RP), a genetic disorder that causes tunnel vision and eventual blindness. There are no U.S. Food & Drug Administration (FDA) approved treatments to restore vision loss in Dry AMD or RP.

The company's novel mechanism of action is designed for direct chemical conversion of fibroblasts into other cell types using a cocktail of small molecules in an 11-day chemical conversion process. Pre-clinical studies have shown efficacy in blind mice that demonstrated vision restoration. CiRC Biosciences has provisional patent applications to protect its platform.

"Our technology transforms ordinary skin cells into specialized retinal cells using a cocktail of small molecules," said Sai Chavala, M.D., co-founder and chief scientific officer of CiRC Biosciences. "This process is potentially safer, quicker, more cost effective and easier to manufacturer than using traditional stem cells. Working with Paragon Biosciences to build and advance CiRC Biosciences provides us the opportunity to efficiently progress this technology through research and development stages.

CiRC Biosciences first reported its discovery in the highly respected scientific journal Nature (April 15, 2020). A recently published New England Journal of Medicine article (Nov. 5, 2020)discussed CiRC's technology of using chemically induced cells to restore retinal function. The article concluded, "The new and emerging strategies for the rescue, regeneration, and replacement of photoreceptors suggest a bright future in the fight to preserve and restore vision in blinding eye diseases."

The abstract in Nature is available here: https://www.nature.com/articles/s41586-020-2201-4

Access to the NEJM article is available here: https://www.nejm.org/doi/full/10.1056/NEJMcibr2027602

About CiRC Biosciences CiRC Biosciences is a privately held cell therapy company dedicated to developing treatments for serious diseases with high, unmet needs with an initial focus on the eye. Currently it is pre-clinical phase for Geographic Atrophy Age-Related Macular Degeneration (Dry AMD) and advanced Retinitis Pigmentosa (RP). CiRC Biosciences is a portfolio company of Paragon Biosciences. Visit our website: https://circbiosciences.com/.

About Paragon Biosciences Paragon is a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence. The company's current portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the replicable Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.

Media Contact:

Evelyn M. O'Connor Paragon Biosciences 312-847-1335 [emailprotected]

SOURCE Paragon Biosciences

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Paragon Biosciences Launches CiRC Biosciences to Expand Cell and Gene Therapy Platform - PRNewswire

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