Archive for the ‘Gene Therapy Research’ Category

Dublin, Feb. 15, 2021 (GLOBE NEWSWIRE) -- The "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" report has been added to ResearchAndMarkets.com's offering.

Over time, several gene therapies have been developed for the treatment of both simple and complex genetic disorders. In fact, there are 10 approved gene therapies (recent examples include Zolgensma, ZyntegloT and Collategene) to date, and more than a thousand product candidates being evaluated in clinical trials, worldwide. 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 and genome editing and manipulation technologies, 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.

The 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.

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. GENE DELIVERY VECTORS

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

6. MARKET OVERVIEW

7. COMPETITIVE LANDSCAPE

8. MARKETED GENE THERAPIES

9. KEY COMMERCIALIZATION STRATEGIES

10. LATE STAGE GENE THERAPIES

11. EMERGING TECHNOLOGIES

12. KEY THERAPEUTICS AREAS

13. PATENT ANALYSIS

14. MERGERS AND ACQUISITIONS

15. FUNDING AND INVESTMENT ANALYSIS

16. CLINICAL TRIAL ANALYSIS

17. COST PRICE ANALYSIS

18. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES

19. DEMAND ANALYSIS

20. MARKET FORECAST AND OPPORTUNITY ANALYSIS

21. VECTOR MANUFACTURING

22. CASE STUDY: GENE THERAPY SUPPLY CHAIN

23. CONCLUSION

A Selection of Companies Mentioned Include:

For more information about this report visit https://www.researchandmarkets.com/r/c6r4ih

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Global Gene Therapy Market Outlook to 2030 - by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of...

The new gene therapy target,GJB2 coding for the Connexin 26 protein, has been added to Sensorions development portfolio: with the target the third candidate to emerge from the R&D collaboration with Institut Pasteur. It represents the largest gene therapy opportunity for the French biotech to date.

The GJB2 program will focus on major new markets with an estimated patient population of 300,000 children and adults in Europe and the US alone.

Sensorion, a French clinical-stage biotech based in Montpellier, was founded in 2009 to develop novel therapies to restore, treat and prevent hearing loss disorders.

The GJB2 program draws on new research from Institut Pasteur which shows that the same genes that underly congenital deafness are also involved in severe forms of presbycusis (age-related hearing loss). These forms of presbycusis appearing to be monogenic types of hearing loss that can be potentially treated by gene therapy.

Although the types ofGJB2mutations in children and adults may differ, Sensorion says gene therapy could potentially provide a solution to both.

Mutations inGJB2are believed to alter a gap junction protein widely expressed in the inner ear, disturbing intercellular exchanges of molecules and leading to hearing loss that is severe-to-profound in a majority of cases.

Institut Pasteur research now shows three pathologies related to GJB2 mutations: congenital deafness;age-related hearing loss in adults; and progressive forms of hearing loss in children. Sensorion will prioritize the latter two forms, saying it is the first company to address these needs and offering the potential of large market opportunities.

The emergence of a new gene therapy target candidate validates our conviction that long-term solutions for restoring hereditary hearing loss will arise from an in-depth analysis of the "genetic landscape" of hearing loss," saidNawal Ouzren, CEO of Sensorion.

"It was clear that mutations in the GJB2 gene are important in severe to profound childhood hearing loss. However, the new discovery made by our collaborators at Institut Pasteur shows that alteration of this gene in adults offers new opportunities for Sensorion. It marks significant potential expansion of our pipeline and supports our goal of becoming a global leader in the field of gene therapies for hearing loss disorders.

Sensorions collaboration with Institut Pasteur initiated in 2019 has already led to gene therapy candidate programs in two other indications. Its USHER-CT gene therapy development program aims to restore inner ear function for patients suffering from Usher Syndrome Type 1 by providing a healthy copy of the USH1G gene coding for the SANS protein.

Meanwhile, the OTOF-GT gene therapy development program seeks to restore hearing in people with Otoferlin deficiency, one of the most common forms of congenital deafness.

Both of these have been proved in concept in preclinical studies.

Originally posted here:
Sensorion and Institut Pasteur announce new gene therapy collaboration - BioPharma-Reporter.com

WILMINGTON, Mass.--(BUSINESS WIRE)--Charles River Laboratories International, Inc. (NYSE: CRL) announced today that it has signed a definitive agreement to acquire Cognate BioServices, Inc., a premier, cell and gene therapy contract development and manufacturing organization (CDMO), for approximately $875 million in cash, subject to customary closing adjustments. The transaction is expected to close by the end of the first quarter of 2021, subject to regulatory requirements and customary closing conditions.

James C. Foster, Chairman, President and Chief Executive Officer of Charles River Laboratories, commented, Cognate BioServices presents a unique opportunity to expand into a high-growth, value-added sector of the CDMO market and enhance our ability to meet our clients needs in emerging areas of scientific innovation. This acquisition will be an exceptional strategic fit, adding to our comprehensive suite of early-stage research and manufacturing support solutions and enabling us to achieve our goal of establishing a single scientific partner to provide biopharmaceutical clients with an integrated solution to help accelerate their cell and gene therapy programs from discovery and non-clinical development through commercialization.

Because of the synergistic fit with Charles River, the market growth potential, and the emerging role of advanced drug modalities as treatments for oncology and rare disease, we believe Cognate will meaningfully enhance our long-term revenue and earnings growth potential. We look forward to welcoming Cognates dedicated employees to the Charles River family, Mr. Foster concluded.

Cognate is a premier, cell and gene therapy CDMO offering comprehensive manufacturing solutions for cell therapies, as well as for production of plasmid DNA and other inputs in the CDMO value chain. The planned acquisition will establish Charles River as a premier scientific partner for cell and gene therapy development, testing, and manufacturing, providing clients with an integrated solution from basic research and discovery through CGMP production. Cognate has extensive experience producing various cell types and technologies used in cellular immunotherapy and immuno-oncology, regenerative medicine, and advanced cell therapy. Headquartered in Memphis, Tennessee, Cognate has operations in North America and Europe with over 500 employees.

Strategic Rationale

The acquisition of Cognate will expand Charles Rivers scientific capabilities into the emerging, high-growth cell and gene therapy CDMO sector, establishing a comprehensive solution from discovery and non-clinical development through CGMP manufacturing in advanced drug modalities.

Cognate will be particularly synergistic with the Companys Biologics Testing Solutions business. It will enable clients to seamlessly conduct analytical testing, process development, and manufacturing for advanced modalities with the same scientific partner, enabling them to achieve their goal of driving greater efficiency. Clients will also have access to the Companys cellular products as the starting point for their cell therapy programs and will be able to work with Charles River through every step of the research and early-stage development process before moving into CGMP production with Cognate, accelerating clients speed to market for advanced drug modalities.

Additional Financial and Transaction Details

Based on the anticipated completion of the acquisition by the end of the first quarter, Cognate is expected to add approximately $110 million to Charles Rivers 2021 consolidated revenue for the partial year. The transaction is expected to be neutral to non-GAAP earnings per share in 2021, and accretive thereafter. Items excluded from non-GAAP earnings per share are expected to include all acquisition-related costs, which primarily include amortization of intangible assets, advisory fees, certain costs associated with efficiency initiatives, and certain third-party integration costs.

The acquisition and associated fees are expected to be financed through Charles Rivers existing credit facility and cash. The Company is evaluating further optimizing its debt structure which could be used to finance the acquisition and for general corporate purposes.

Cognate is expected to be reported as part of Charles Rivers Manufacturing Support segment.

Advisors and Cognate OwnershipGordon Dyal & Co., LLC is acting as the exclusive financial advisor to Charles River. Davis Polk & Wardwell LLP is acting as Charles Rivers transactional legal counsel, and Weil, Gotshal & Manges LLP is acting as antitrust counsel. Dark Horse Consulting Group, Inc. is acting as Charles Rivers strategic advisor.

Cognate is supported by its majority shareholder, EW Healthcare Partners, as well as minority shareholders, Medivate Partners, BlackRock, and a sovereign wealth fund. Morgan Stanley & Co. LLC is acting as the exclusive financial advisor and Kirkland & Ellis LLP is acting as legal counsel to Cognate and its shareholders.

Use of Non-GAAP Financial Measures

This news release contains non-GAAP financial measures, such as non-GAAP earnings per diluted share, which exclude the amortization of intangible assets, integration costs, advisory fees, and other charges related to our acquisitions and expenses associated with evaluating acquisitions. We exclude these items from the non-GAAP financial measures because they are outside our normal operations. There are limitations in using non-GAAP financial measures, as they are not prepared in accordance with generally accepted accounting principles, and may be different than non-GAAP financial measures used by other companies. In particular, we believe that the inclusion of supplementary non-GAAP financial measures in this news release helps investors to gain a meaningful understanding of our core operating results and future prospects without the effect of these often-one-time charges, and is consistent with how management measures and forecasts the Company's performance, especially when comparing such results to prior periods or forecasts. We believe that the financial impact of our acquisitions (and in certain cases, the evaluation of such acquisitions, whether or not ultimately consummated) is often large relative to our overall financial performance, which can adversely affect the comparability of our results on a period-to-period basis. In addition, certain activities, such as business acquisitions, happen irregularly and the underlying costs associated with such activities do not recur on a consistent basis. Non-GAAP results also allow investors to compare the Companys operations against the financial results of other companies in the industry who similarly provide non-GAAP results. The non-GAAP financial measures included in this news release are not meant to be considered superior to or a substitute for results of operations prepared in accordance with GAAP. The Company intends to continue to periodically assess the potential value of reporting non-GAAP results consistent with applicable rules and regulations. A reconciliation of the effect of this transaction on non-GAAP earnings per share for 2021 to the most directly comparable GAAP financial measure has not been included because it is impracticable to determine the allocation of the purchase price for the proposed acquisition and other necessary adjustments at this time.

Caution Concerning Forward-Looking Statements

This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words such as anticipate, believe, expect, will, may, estimate, plan, outlook, and project and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements include statements in this news release regarding the acquisition of Cognate BioServices, expectations regarding the timing of the closing of the acquisition, and Charles Rivers expectations with respect to the impact of Cognate on the Company, its product and service offerings, client perception, revenue, revenue growth rates, and earnings per share; Charles Rivers projected future performance including revenue and earnings per share; as well as Charles Rivers future growth in the area of cell and gene therapy CDMO services. Forward-looking statements are based on Charles Rivers current expectations and beliefs, and involve a number of risks and uncertainties that are difficult to predict and that could cause actual results to differ materially from those stated or implied by the forward-looking statements. Those risks and uncertainties include, but are not limited to, the ability to successfully complete the acquisition of Cognate, our ability to successfully integrate Cognate, and risks and uncertainties associated with Cognates products and services which are in areas in which Charles River does not currently operate. A further description of these risks, uncertainties, and other matters can be found in the Risk Factors detailed in Charles River's Annual Report on Form 10-K as filed on February 11, 2020 and the Quarterly Report on Form 10-Q as filed on October 29, 2020, as well as other filings we make with the Securities and Exchange Commission. Because forward-looking statements involve risks and uncertainties, actual results and events may differ materially from results and events currently expected by Charles River, and Charles River assumes no obligation and expressly disclaims any duty to update information contained in this news release except as required by law.

About Charles River

Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit http://www.criver.com.

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Charles River Laboratories to Acquire Cognate BioServices to Create a Premier Scientific Partner for Cell and Gene Therapy Development - Business Wire

-- Gene editing expert Charles Albright, Ph.D., joins as chief scientific officer --

-- Gene therapy development expert Petra Kaufmann, M.D.,joins as chief medical officer

WALTHAM, Mass., Feb. 17, 2021 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and non-rare diseases, today announced the completion of its leadership team. Collectively, the executives represent a diverse team of proven leaders in gene therapy who have successfully developed novel platforms and translated them to medicines that have made a transformative difference in the lives of those affected by devastating diseases.

Our calling is to broaden the reach of gene therapies for patients in need, and this starts with our proven leadership team. Members of our team have been at the forefront of the field and come from a variety of eminent organizations to innovate in the gene therapy science and programs at Affinia Therapeutics, said Rick Modi, chief executive officer at Affinia Therapeutics. We believe in a vastly different future medical era of one-time potentially curative medicines for patients affected by diseases, rare and non-rare. But realizing this vision requires moving beyond conventional AAV serotypes. At Affinia Therapeutics, we are uniquely positioned to engineer novel vectors and gene therapies that direct tissue tropism and have the potential to improve clinical efficacy, as well as immunogenicity and safety. Together, Dr. Albright, Dr. Kaufmann and the rest of our leadership team will leverage their extensive experience to advance our platform and programs.

Dr. Albright joins Affinia Therapeutics from Editas Medicine, where he served as executive vice president and chief scientific officer and led the development of the CRISPR gene editing technology platform. In this role, he industrialized and matured the platform and translated it to pioneering programs through Investigational New Drug (IND) submissions that led to clinical trials. Prior to joining Editas, Dr. Albright spent over 12 years at Bristol Myers Squibb, where he most recently held the position of vice president, genetically defined diseases and genomics. As a research leader at both biotech and large pharmaceutical companies, he has honed his expertise across a range of therapeutic areas including neurology, neuromuscular, cardiovascular, ophthalmology and oncology. Dr. Albright earned a Ph.D. in biology at MIT and was a postdoctoral fellow at the Whitehead Institute.

Dr. Kaufmann is an expert in translational medicine and clinical development focused on neuroscience, rare disease and gene therapy. She recently served as senior vice president and head of clinical development, analytics and translational medicine at Novartis Gene Therapies. She directed clinical development activities that included the global development of Zolgensma and translational strategy for several pipeline programs. This followed many years of clinical research and development positions in academia and at the National Institutes of Health (NIH), where she held leadership roles of increasing responsibility, most recently as director of the Office of Rare Diseases Research. Dr. Kaufmann earned an M.D. at the University of Bonn and an M.S. in biostatistics at Columbia University, where she also trained in neurology and served as a tenured faculty member, advancing research and caring for patients.

It is exciting to see the platform generate advances beyond the conventional AAV serotypes discovered many years ago, said Dr. Albright. Affinia Therapeutics platform for engineering next-generation vectors with specific pharmacodynamic properties enables us to explore the potentially curative benefits of gene therapy in new indications. I look forward to leading the scientific team as we advance these insights into translational opportunities in the clinic and, ultimately, into transformative therapies for patients.

I am thrilled to join this team of gene therapy experts who are helping to broaden the reach of gene therapies, said Dr. Kaufmann. I look forward to putting my clinical research and development experience into practice as we translate our differentiated AAV vectors into transformative medicines for people suffering from diseases that have been inadequately addressed by conventional AAVs and traditional therapies.

Dr. Albright and Dr. Kaufmann join the following individuals on Affinia Therapeutics leadership and scientific advisory team:

This team has collectively authored more than 450 publications, holds more than 15 patents, held meaningful roles on more than 15 BLAs, supplemental BLAs and product launches, and has executed on more than $16 billion in value for IPOs and M&As.

About Affinia TherapeuticsAt Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating rare and non-rare diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies with potentially improved tissue tropism, cell specificity, immunogenicity and safety. With our innovative science, we are working to broaden the reach of life-changing gene therapies to meaningful numbers of patients with an initial focus on central nervous system (CNS) and muscle diseases with significant unmet need. http://www.affiniatx.com.

Affinia Therapeutics Contacts

Investors: investors@affiniatx.com

Media: media@affiniatx.com

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Affinia Therapeutics Announces Addition of Gene Therapy Scientific and Medical Experts to Leadership Team to Advance Novel Gene Therapy Platform and...

Igor Golovniov/SOPA Images/LightRocket via Getty Images

Charles River Laboratories will acquire Cognate BioServices, a cell and gene therapy contract development and manufacturing organization, in an $875 million cash deal that will significantly expand the companys capabilities in the high-growth cell and gene therapy sector.

In addition to expanding Charles Rivers capabilities in the cell and gene therapy space, the company said the acquisition of Memphis, Tenn.-based Cognate will also establish a comprehensive solution from discovery and non-clinical development through CGMP manufacturing in advanced drug modalities. Cognate has a global presence with more than 500 employees. In January, Cognate announced plans to significantly expand cell and gene therapy manufacturing capacity, laboratory space, warehousing capabilities, and increase office support at its facilities inthe United StatesandEurope.

The company has extensive experience producing various cell types and technologies used in cellular immunotherapy and immuno-oncology, regenerative medicine and advanced cell therapy. Its primary area of expertise is in CGMP cell therapy manufacturing, which processes a variety of cellular products and other starting materials to develop and produce allogeneic (donor-derived) and autologous (patient-derived) cell therapies. Cognate also produces plasmid DNA, which is a foundational tool used in the development of gene-modified cell therapies and gene therapies, as well as other CDMO inputs, Charles River said.

James C. Foster, president and chief executive officer of Charles River Labs, said Cognate provides a synergistic fit for Charles River. He said bringing Cognate under the companys umbrella presents a unique opportunity to expand the companys capabilities and also enhance its offerings to clients in emerging areas of scientific innovation.

Additionally, Charles River said the addition of Cognate will complement its existing Biologics Testing Solutions business. Cognates capabilities will enable clients to seamlessly conduct analytical testing, process development, and manufacturing for advanced modalities with the same scientific partner, enabling them to achieve their goal of driving greater efficiency, the company said.

The addition of Cognate is also expected to provide a significant financial boost to Charles River. Cognate is expected to generate annual revenue of approximately $140 million in 2021, and is expected to grow at least 25% annually over the next five years, the company said.

This acquisition will be an exceptional strategic fit, adding to our comprehensive suite of early-stage research and manufacturing support solutions and enabling us to achieve our goal of establishing a single scientific partner to provide biopharmaceutical clients with an integrated solution to help accelerate their cell and gene therapy programs from discovery and non-clinical development through commercialization, Foster said in a statement. Because of the synergistic fit with Charles River, the market growth potential, and the emerging role of advanced drug modalities as treatments for oncology and rare disease, we believe Cognate will meaningfully enhance our long-term revenue and earnings growth potential.

The acquisition is expected to move quickly. Charles River anticipates the closing of the deal by the end of the first quarter of 2021. Based on the anticipated completion of the acquisition by the end of the first quarter, Cognate is expected to add approximately $110 million to Charles Rivers 2021 consolidated revenue for the partial year.

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Charles River Dives Deep Into Cell and Gene Therapy With Cognate BioServices Acquisition - BioSpace

A new Market Research Report by Facts and Factors Market Research (fnfresearch.com), on Cancer Gene Therapy Market Overview By Trends, Industry Top Manufactures, Size, Industry Growth Analysis & Forecast Till 2026 added to the flourishing data archive is in place to provide readers with innate detailing on market developments, comprising a detailed market overview, vendor landscape, market dimensions, vendor landscape as well as in-depth SWOT and PESTEL assessment, besides other internationally approved market assessment guidelines that play crucial roles in growth dissemination.

The report Cancer Gene Therapy Market 2020-2026 focuses on outlining and narrating the key influencing elements for the growth of the market. It also proposes a thorough study of the market stature (revenue), market share, key market segments, distinct geographic regions, dominant market players, and prime industry trends. The aim of this report is to portray the forthcoming market trends and revenue prognosis for the global Cancer Gene Therapy market for the next five years.

According to the research report, Global Cancer gene therapy market is expected to grow at a CAGR of 23% and is anticipated to reach around USD 3,400 million by 2026. Gene therapy is a method for curing or avoiding the substitution of mutated genes of health diseases. This replacement enables cells to produce valuable proteins.

We Have Recent Updates of Cancer Gene Therapy Market in Sample Copy @ https://www.fnfresearch.com/sample/cancer-gene-therapy-market-by-type-ex-vivo-898

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Cancer Gene Therapy Market by Top Manufacturers (2020-2026)

Vigene Biosciences

Sirion Biotech

Bluebird bio

Cellectis

Ziopharm

Cobra

Uniqure

Finvector

Sarepta Therapeutics

To Get an Additional List of Key Market Players Included in this Report, Request a Free PDF Brochure: https://www.fnfresearch.com/sample/cancer-gene-therapy-market-by-type-ex-vivo-898

The Market Player Analysis based on some of the below Factors:

Pre & Post Impact of COVID-19 Pandemic on Cancer Gene Therapy Market

The COVID-19 outbreak has affected economies and industries in various countries due to lockdowns, travel bans, and business shutdowns. The decrease in the overall market growth due to COVID-19 is also affecting the Post growth of the Cancer Gene Therapy market owing to the shutting down of factories, obstacles in the supply chain, and a downturn in the world economy.

This report presents a comprehensive overview, market shares, and growth opportunities of Cancer Gene Therapy market by product type, application, key manufacturers, and key regions and countries. In addition, this report discusses the key drivers influencing market growth, opportunities, the challenges, and the risks faced by key manufacturers and the market as a whole. It also analyzes key emerging trends and their impact on present and future development.

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Points Covered in the Report

The points that are discussed within the report are the major market players that are involved in the market such as market players, raw material suppliers, equipment suppliers, end users, traders, distributors and etc.

The complete profile of the companies is mentioned. And the capacity, production, price, revenue, cost, gross, gross margin, sales volume, sales revenue, consumption, growth rate, import, export, supply, future strategies, and the technological developments that they are making are also included within the report. This report analyzed 12 years of data history and forecast.

The growth factors of the market are discussed in detail wherein the different end users of the market are explained in detail.

Data and information by market player, by region, by type, by application, etc., and custom research can be added according to specific requirements.

The report contains the SWOT analysis of the market. Finally, the report contains the conclusion part where the opinions of the industrial experts are included.

The market research report also offers information on potential investment opportunities, strategic growth market analysis, and probable threats that will adhere to the client to systematically and creatively plan out the business models and strategies. The critical data analysis in the Cancer Gene Therapy market report is laid out in an upright way. This means that the information is represented in form of infographics, statistics, and uncomplicated graphs to make it an effortless and time-saving task for the client.

(**The consulting and implementation services segment to account for the highest market share during the forecast period.**)

The scope of the Cancer Gene Therapy Market Size report has a wide spectrum extending from market scenarios to comparative pricing between major players, cost, and profit of the specified market regions. The numerical data is supported by statistical tools such as SWOT analysis, BCG matrix, SCOT analysis, and PESTLE analysis. The statistics are depicted in a graphical format for a clear picture of facts and figures.

The report provides a complete view of the Cancer Gene Therapy market and encompasses a detailed type of portfolio and strategic developments of key vendors. To know the competitive landscape of the Cancer Gene Therapy market, an analysis of Porters five forces model is done. The study cover market attractiveness analysis, in which type, source type, and application segments are specialized based on the market size, growth rate, and attractiveness.

The report study further includes an in-depth analysis of industry players market shares and provides an overview of leading players market position in the Cancer Gene Therapy sector. Key strategic developments in the Cancer Gene Therapy market competitive landscape such as acquisitions & mergers, inaugurations of different products and services, partnerships & joint ventures, MoU agreements, VC & funding activities, R&D activities, and geographic expansion among other noteworthy activities by key players of the Cancer Gene Therapy market are appropriately highlighted in the report.

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Table of Contents

Research Coverage:

The Cancer Gene Therapy market has been segmented based on offering, technology, end-use application, and end-user. It also provides a detailed view of the market across four main regions: North America, Europe, APAC, and RoW.

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Most Elevated Read 2020 on Cancer Gene Therapy Market To Witness Robust Expansion Throughout The Forecast Period 2026 KSU | The Sentinel Newspaper -...

Basel, February 17, 2021 Novartis today announced that it has entered into a grant agreement with the Bill & Melinda Gates Foundation. As part of the agreement, the foundation will provide funding support for the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease (SCD). The project brings together Novartis drug discovery and gene therapy expertise with the Gates Foundations charitable objectives to expand access to healthcare in low-resource settings in an effort to address this potentially life-threatening genetic disease.

Existing gene therapy approaches to sickle cell disease are difficult to deliver at scale and there are obstacles to reaching the vast majority of those affected by this debilitating disease, said Jay Bradner, a hematologist and President of the Novartis Institutes for BioMedical Research (NIBR). This is a challenge that calls for collective action, and we are thrilled to have the support of the Bill & Melinda Gates Foundation in addressing this global unmet medical need.

SCD is a hereditary blood diseaseone of the oldest known and most common genetic disorders. The disease affects millions around the world, with over 300,000 born with the condition annually1,2. It disproportionately affects those of African descent, and sub-Saharan Africa bears roughly 80 percent of the disease burden3. It also is common among people with ancestry from South America, Central America, and India, as well as several Mediterranean countries, such as Italy and Turkey.

SCD is a complex genetic disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently and, early on, progresses to a chronic vascular disease1,4,5,6. The disease can lead to acute episodes of pain known as sickle cell pain crises, or vaso-occlusive crises, as well as life-threatening complications7,8,9. The resulting, frequent hospitalizations associated with sickle cell disease combined with an overall lack of specialized care places a significant burden on patients and their families, healthcare systems, and the overall economy. Even with todays best available care, SCD continues to drive premature deaths and disability.

While the genetic cause of SCD has been known for decades, its only recently that the medical world has gained the tools to potentially fix the errant gene that causes the disease. Current clinical-stage gene-based therapies require extracting cells from a patient, altering those cells in a laboratory, and then reintroducing them to the patient through a complex procedure akin to a bone marrow transplant. The lab and manufacturing facilities and hospital infrastructure for such procedures often do not exist in the areas where SCD is most prevalent, excluding the vast majority of those with the disease from these life-changing gene therapies.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, said Trevor Mundel, President of Global Health at the Gates Foundation. Whats exciting about this project is that it brings ambitious science to bear on that challenge. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought. It also holds the promise of applying lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.

Novartis envisions developing an accessible in vivo gene therapy for SCD that could potentially be administered once, directly to the patient, without the need to modify the cells in a lab. This would have the advantage of mitigating the need for long or repeated hospital stays or specialized lab infrastructure. To facilitate the research, the Gates Foundation has agreed to provide funding support for a research team within NIBR wholly dedicated to developing an approach to delivering this potential treatment. Novartis will provide in-kind support and access to its suite of technologies and resources.

In addition to research funding, the Gates Foundation lends its long history and experience in global health to this collaboration. As part of the early drug design strategy, Novartis will prioritize addressing access and distribution hurdles posed by limited healthcare infrastructure in low- and middle-income countries and the funding agreement includes specific provisions to support global access to any resulting innovations.

Novartis is proud to lead this effort to find an accessible genetic therapy for sickle cell disease, with support from the Bill & Melinda Gates Foundation, said Lutz Hegemann, Group Head of Corporate Affairs & Global Health for Novartis. In keeping with our purpose, we firmly believe we can use science and innovation to reimagine the way SCD is treated for patients around the world.

The agreement builds on the Novartis commitment to improve the lives of patients with sickle cell disease through the development of new treatments, including crizanlizumab, strategic partnerships with government and non-government organizations, and support for such interventions as newborn screenings and the distribution of existing drugs.

DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as potential, potentially, can, will, could, would, believe, commitment, pipeline, to discover, aims, to address, promise, envisions, to facilitate, to provide, lead, or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, including crizanlizumab, or regarding the collaboration to discover and develop an accessible in vivo gene therapy for sickle cell disease. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release, including crizanlizumab, will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that the activities and efforts described in this release will be achieved or succeed, in the expected time frame, or at all. In particular, our expectations regarding such products and efforts could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AGs current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About NovartisNovartis is reimagining medicine to improve and extend peoples lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the worlds top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more athttps://www.novartis.com/.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisnewsFor Novartis multimedia content, please visit https://www.novartis.com/news/media-libraryFor questions about the site or required registration, please contact media.relations@novartis.com

References

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Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

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Novartis and the Bill & Melinda Gates Foundation collaborate to discover and develop an accessible in vivo gene therapy for sickle cell disease -...

Newark, NJ, Feb. 17, 2021 (GLOBE NEWSWIRE) -- As per the report published by Fior Markets, theglobal genome editing market is expected to grow from USD 5.37 billion in 2020 to USD 17.36 billion by 2028, at a CAGR of 15.8% during the forecast period 2021-2028.

Some of the factors that drive the growth of the global genome editing market are expanding genetic research funding in North America, rising synthetic gene demand in North America & Asia Pacific regions, growing R&D expenditure for biotechnology, technological developments in gene editing, presence of strong pipeline products, increasing application fields of genomics, and rising production of GM crops. New products are being developed to assist in improving DNA targeting, with better flexibility, higher specificity, and enhanced AAV packaging.

With the outbreak of a global pandemic, many have faced salary cuts and job losses. Covid-19 has become a threat to the entire world's economy and has negatively impacted all kinds of markets, including the genome editing market. With less disposable income and broken supply chains, the market's growth is expected to slow down. A shortage of skilled professionals due to medical workers being shifted to covid-19 frontlines also negatively impacts the market. Other market growth hindering factors are low public awareness in underdeveloped nations and strict government regulations.

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Key players operating in the global genome editing market include Thermo Fisher Scientific, Horizon Discovery Limited, GenScript, Eurofins Scientific, Editas Medicine, CRISPR Therapeutics, Oxford Genetics, Synthego, Vigene Biosciences, Integrated DNA Technologies, OriGene Technologies, Transposagen Biopharmaceuticals, Agilent Technologies, Genecopoeia, and Precision Biosciences, among others. To gain a significant market share in the global genome editing market, the key players are now focusing on adopting strategies such as product innovations, mergers & acquisitions, recent developments, joint ventures, collaborations, and partnerships. Thermo Fisher Scientific and Horizon Discovery Limited are some of the biggest players in the global genome editing market.

CRISPR/Cas9 segment dominated the market and held the largest market share of 32.8% in the year 2020

The technology segment consists of ZFN, TALENs/MegaTALs, CRISPR/Cas9, and others. The CRISPR/Cas9 segment dominated the market and held the largest market share of 32.8% in the year 2020. This is primarily due to CRISPR's better ease of use as compared to TALEN & ZFN, and patient-specific treatment characteristics for illnesses like cystic fibrosis.

Ex-vivo segment dominated the market and held the largest market share of 55.3% in the year 2020

Based on delivery method, the global market has been divided into in-vivo and ex-vivo. Ex-vivo segment dominated the market and held the largest market share of 55.3% in the year 2020. The large share of this segment is attributed to key factors such as control ease in DNA modification, and precise regulation of nuclease expression for highly accurate gene editing.

Cell line engineering segment dominated the market and held the largest market share of 34.1% in the year 2020

The application segment includes cell line engineering, plant genetic engineering, animal genetic engineering, and other applications. Cell line engineering segment dominated the market and held the largest market share of 34.1% in the year 2020. Some of the vital factors that drive market growth in this segment are rising government as well as private funding, increasing global awareness, and expanding industry attention on stem cell research.

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Biotechnology & pharmaceutical companies segment dominated the market and held the largest market share of 45.3% in the year 2020

Based on end-use, the genome editing market has been segmented into contract research organizations, academic & government research institutes, and biotechnology & pharmaceutical companies. Biotechnology & pharmaceutical companies segment dominated the market and held the largest market share of 45.3% in the year 2020. The large share of this segment can be attributed to factors like rising prevalence of cancer & other diseases, and the increasing number of gene engineering research projects being carried out for advancement in novel therapeutics.

In-house segment dominated the market and held the largest market share of 60.9% in the year 2020

Based on service, the market has been divided into in-house and contract. The in-house segment dominated the market and held the largest market share of 60.9% in the year 2020. Large companies like Sigma Aldrich Corporation and Thermo Fisher engineer cell lines in-house due to the availability of financial resources, technology, and facilities.

Regional Segment Analysis of the Genome Editing Market

On the basis of geography, the global genome editing market has been classified into North America, Europe, South America, Asia Pacific, and Middle East and Africa. The North America region accounted for the major market share of 48.2% in the year 2020 and is anticipated to grow throughout the projected period. The large share of this segment is attributed to key factors such as R&D activities in gene therapy, increasing cases of cancer & other diseases, increasing funding & research grants, and rising usage of GM (genetically modified) crops in the region. On the other hand, the Asia-Pacific region is expected to grow at the highest CAGR during the forecast period 2021-2028. This growth is owing to factors like advancing medical science and rising disposable income.

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The global genome editing market is analyzed on the basis of value (USD Billion). All the segments have been analyzed on a global, regional, and country basis. The study includes the analysis of more than 30 countries for each segment. The report offers an in-depth analysis of driving factors, opportunities, restraints, and challenges for gaining the key insight of the market. The study includes porter's five forces model, attractiveness analysis, raw material analysis, and competitor position grid analysis.

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Global Genome Editing Market Is Expected to Reach USD 17.36 Billion by 2028 : Fior Markets - GlobeNewswire

ROCKVILLE, Md., Feb. 16, 2021 /PRNewswire/ --

REGENXBIO Inc. (Nasdaq: RGNX) reported at the Angiogenesis, Exudation, and Degeneration 2021 conference additional positive interim data from Cohorts 4 and 5 of its RGX-314 Phase I/IIa trial for the treatment of wet age-related macular degeneration (wet AMD), and Cohort 3 of its Long-Term Follow-Up (LTFU) study. RGX-314 is a potential best-in-class, one-time gene therapy for the treatment of wet AMD.

"The continued durability of treatment effect up to three years after RGX-314 administration highlights the potential of RGX-314 as a one-time treatment option for patients with wet AMD. The results from the Phase I/IIa trial of RGX-314 using subretinal delivery have informed the key design elements of our pivotal program, in which we plan to conduct two randomized, well-controlled clinical trials, enrolling approximately 700 patients total," said Steve Pakola, M.D., Chief Medical Officer ofREGENXBIO.

"I am excited about this data out to three years, which demonstrates that one-time treatment with RGX-314 has the potential to result in long-term stability to improvement of visual acuity outcomes and retinal anatomy, while alleviating treatment burden," said Allen C. Ho, M.D., Director of Retina Research at Wills Eye Hospital and Mid Atlantic Retina and investigator surgeon in the RGX-314 clinical trials. "In our practice, and as reported by multiple real-world studies, we see many patients losing vision due to lack of compliance with standard of care, which requires frequent anti-VEGF injections. I look forward to further evaluating the effects of RGX-314 in ATMOSPHERETM, the first pivotal trial of a gene therapy for the treatment of wet AMD."

Study Design and Safety Update from Phase I/IIa Trial of RGX-314 for the Treatment of Wet AMD Using Subretinal Delivery

In the Phase I/IIa trial of RGX-314, 42 patients with severe wet AMD requiring frequent anti-vascular endothelial growth factor (anti-VEGF) injections were treated across five dose cohorts, with doses ranging from 3x109 GC/eye to 2.5x1011 GC/eye.

As of January 22, 2021, RGX-314 continued to be generally well-tolerated across all cohorts, with 20 serious adverse events (SAEs) reported in 13 patients, including one possibly drug-related SAE of significant decrease in vision in Cohort 5. The most common nonserious adverse events in the eye were generally assessed as mild (87%). These included post-operative conjunctival hemorrhage (69% of patients), post-operative inflammation (36% of patients), eye irritation (17% of patients), eye pain (17% of patients), and post-operative visual acuity reduction (17% of patients). In 67% of patients across all cohorts, and in 83% of patients in Cohorts 3 through 5, retinal pigmentary changes were observed on imaging, the majority of which were in the peripheral inferior retina. Retinal hemorrhage was observed in 26% of patients and is an anticipated event in patients with severe wet AMD. There have been no reports of clinically-determined immune responses, drug-related ocular inflammation, or post-surgical inflammation beyond what is expected following routine vitrectomy.

Summary of Data for Cohorts 4 and 5

Today's update includes data from Cohorts 4 and 5 as of January 22, 2021. Each cohort enrolled 12 patients each at doses of 1.6x1011 GC/eye and 2.5x1011 GC/eye, respectively.

Patients in Cohorts 4 and 5 at 1.5 years after administration of RGX-314 demonstrated stable visual acuity with a mean Best Corrected Visual Acuity (BCVA) change of +1 letters and -1 letters from baseline, respectively, as well as decreased central retinal thickness (CRT), with a mean change of -46 m and -93 m, respectively.

There was a meaningful reduction in anti-VEGF treatment burden in both Cohorts 4 and 5 compared to the mean annualized injection rate during the 12 months prior to RGX-314 administration. Patients in Cohort 4 received a mean of 4.4 injections over 1.5 years following administration of RGX-314, a 58.3% reduction in anti-VEGF treatment burden. Patients in Cohort 5 received a mean of 1.7 injections over 1.5 years following administration of RGX-314, a reduction in anti-VEGF treatment burden of 81.2%.

In Cohort 4, four out of 12 (33%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of +2 letters at 1.5 years. Eight out of 11 (73%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of -2 letters at 1.5 years.

Summary of Long-Term Follow-Up (LTFU) Study Data

Following the Phase I/IIa trial, patients are encouraged to enroll in a LTFU study to assess safety and efficacy up to five years after RGX-314 administration. Patients in the LTFU study have scheduled visits every six months for the first year and then annual visits until the end of the study. Patient management is per physician discretion. Data collected during the scheduled study visits include safety, BCVA, and CRT. In addition, chart reviews are conducted at each scheduled study visit to collect the number of retina specialist visits and anti-VEGF injections each patient has received since the prior scheduled study visit.

As of January 22, 2021, RGX-314 continued to be generally well-tolerated in patients enrolled in the LTFU study, with no new drug-related ocular adverse events reported.

All six patients from Cohort 3 of the Phase I/IIa trial enrolled in the LTFU study, and long-term treatment effect was demonstrated over three years. These patients demonstrated a mean BCVA improvement of +12 letters from baseline at three years. Retinal anatomy as measured by machine-read CRT remained stable at three years compared to the two-year timepoint.

Patients also demonstrated long-term reductions in anti-VEGF treatment burden over three years with a mean annualized rate of 2.4 anti-VEGF injections after administration of RGX-314, which is a reduction of 66.7% from the mean annualized injection rate during the 12 months prior to administration of RGX-314. Three out of six (50%) patients received no anti-VEGF injections over three years following one-time administration of RGX-314. Four out of six (67%) patients have received no anti-VEGF injections from nine months to three years after RGX-314 administration. The four patients who did not receive anti-VEGF injections after nine months demonstrated a mean BCVA improvement from baseline of +11 letters at three years.

About Wet AMD

Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

About RGX-314

RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

REGENXBIO is advancing two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

About the Phase I/IIa Clinical Trial of RGX-314 and Long-Term Follow-Up Study

RGX-314 is being evaluated in a Phase I/IIa, multi-center, open-label, multiple-cohort, dose-escalation study in adult patients with wet AMD in the United States. The study includes patients previously treated for wet AMD who are responsive to anti-VEGF therapy. The study is designed to evaluate five escalating doses of RGX-314, with six patients in the first three dose cohorts and 12 patients in the fourth and fifth dose cohorts. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic immune suppressive oral corticosteroid therapy before or after administration of RGX-314. The primary endpoint of the study is safety at 6 months following administration of RGX-314. Secondary endpoints include visual acuity, retinal thickness on SDOCT, ocular RGX-314 protein expression, and the need for additional anti-VEGF therapy. Following completion of the primary study period, patients enter a follow-up period and will continue to be assessed until week 106 for long-term safety and durability of effect. After completion of the Phase I/IIa clinical trial, patients are encouraged to enter a Long-Term Follow-Up study to continue to follow safety and efficacy for a total of 5 years following administration of RGX-314.

About ATMOSPHERE

ATMOSPHERE is a multi-center, randomized, active-controlled trial to evaluate the efficacy and safety of a single-administration of RGX-314 versus standard of care in patients with wet AMD. The trial is designed to enroll 300 patients at a 1:1:1 ratio across two RGX-314 dose arms (6.4x1010 genome copies (GC)/eye and 1.3x1011 GC/eye delivered subretinally) and an active control arm of monthly intravitreal injections of ranibizumab (0.5 mg/eye). The primary endpoint of the trial is non-inferiority to ranibizumab based on change from baseline in Best Corrected Visual Acuity (BCVA) at 54 weeks. Secondary endpoints of the trial include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections. Patient selection criteria will include patients with wet AMD who are responsive to anti-VEGF treatment and will be independent of preexisting neutralizing antibody status. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314. The trial will be conducted at approximately 60 clinical sites based in the United States, with over 100 retinal surgeons.

AboutREGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Forward-Looking Statements

This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to accurately predict how long REGENXBIO's existing cash resources will be sufficient to fund its anticipated operating expenses, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at http://www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

SCS Microinjector is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

Contacts: Tricia TruehartInvestor Relations and Corporate Communications347-926-7709ttruehart@regenxbio.com

Investors:Brendan Burns, 212-600-1902brendan@argotpartners.com

Media:David Rosen, 212-600-1902david.rosen@argotpartners.com

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SOURCE REGENXBIO Inc.

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REGENXBIO Announces Additional Positive Interim Phase I/IIa and Long-Term Follow-Up Data of RGX-314 for the Treatment of Wet AMD - BioSpace

Dublin, Feb. 17, 2021 (GLOBE NEWSWIRE) -- The "I-O Therapy Combinations Spurring Growth of the Global Immuno-oncology Market" report has been added to ResearchAndMarkets.com's offering.

This study explores the global immuno-oncology (I-O) market's evolution from 2020 to 2025. An extensive analysis is provided on the growth opportunities envisioned by the analyst to help stakeholders leverage their momentum and disrupt the market by offering best-in-class solutions.

The study considers oncology agents for which the immune system is the basis of their actions, including monoclonal antibodies, cell and gene therapies, bioengineered vaccines, recombinant products, small molecules, and oncolytic viruses. Classified into 5 major therapy types - immunomodulators, adoptive cell therapy, cancer vaccine, oncolytic virus, and antibody-based targeted therapies - the analysis provides revenue forecasts by the global market and product category. The agents are classified into various therapy types based on the mechanism of action (MOA). Concerning pipeline products, we have considered industry-sponsored products under development.

The oncology therapeutic segment is expected to witness minimal impact from the COVID-19 pandemic because of the criticality of treating cancer patients and the segment's role as a major revenue-generator for pharmaceutical companies. The analyst have considered the impact of COVID-19 and two scenarios for the sales forecast: original scenario and COVID-19 impact scenario.

In the original scenario, the I-O market is expected to reach $132.66 billion by 2025 growing at a rate of 11.6% and witness minimal impact from the COVID-19 pandemic, with 2020 accounting for the majority of the decline. After 2022, the market will positively recuperate and experience a slight increase in revenue due to rebounding R&D budgets and accelerated developments of late-stage clinical candidates.

Research Highlights

Major Themes Covered in This Study:

Key Topics Covered:

1. Strategic Imperatives

2. Executive Summary

3. Market Overview-Immuno-oncology

4. Immuno-oncology Value Chain and R&D Expenditure-Immuno-oncology

5. Clinical Trial Overview-Immuno-oncology

6. Regulatory and Reimbursement Scenario-Immuno-oncology

7. Key Marketed and Pipeline Products Dashboard-Immuno-oncology

8. Growth Opportunity Analysis-Global Immuno-oncology Market

9. Growth Opportunity Analysis-Immunomodulators

10. Growth Opportunity Analysis-Adoptive Cell Therapy

11. Growth Opportunity Analysis-Antibody-based Targeted Therapies

12. Growth Opportunity Analysis-Cancer Vaccine and Oncolytic Virus

13. Growth Opportunity Universe-Immuno-oncology

14. Key Company Profiles-Immuno-oncology

15. Next Steps

For more information about this report visit https://www.researchandmarkets.com/r/x1ktob

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IO Therapy Combinations Spurring Growth of the Global Immuno-oncology Market, 2020 Report - Upcoming Product Launches in Adoptive Cell Therapy Segment...

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

DUBLIN--(BUSINESS WIRE)--The "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" report has been added to ResearchAndMarkets.com's offering.

Over time, several gene therapies have been developed for the treatment of both simple and complex genetic disorders. In fact, there are 10 approved gene therapies (recent examples include Zolgensma, ZyntegloT and Collategene) to date, and more than a thousand product candidates being evaluated in clinical trials, worldwide. 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 and genome editing and manipulation technologies, 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.

The 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.

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. GENE DELIVERY VECTORS

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

6. MARKET OVERVIEW

7. COMPETITIVE LANDSCAPE

8. MARKETED GENE THERAPIES

9. KEY COMMERCIALIZATION STRATEGIES

10. LATE STAGE GENE THERAPIES

11. EMERGING TECHNOLOGIES

12. KEY THERAPEUTICS AREAS

13. PATENT ANALYSIS

14. MERGERS AND ACQUISITIONS

15. FUNDING AND INVESTMENT ANALYSIS

16. CLINICAL TRIAL ANALYSIS

17. COST PRICE ANALYSIS

18. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES

19. DEMAND ANALYSIS

20. MARKET FORECAST AND OPPORTUNITY ANALYSIS

21. VECTOR MANUFACTURING

22. CASE STUDY: GENE THERAPY SUPPLY CHAIN

23. CONCLUSION

A Selection of Companies Mentioned Include:

For more information about this report visit https://www.researchandmarkets.com/r/rk0k5y

About ResearchAndMarkets.com

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

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Global Gene Therapy Market Industry Trends and Forecasts 2020-2030: Big Pharma Players and Analysis of Gene Therapy Related Initiatives -...

Global Gene Therapy Products Market Industry Trends and Forecast to 2027 Comprehensive Research Report to Added In Databridgemarketresearch.com directory. Report carrying 350 pages, 60 Figures And 220 Tables in it.

Global Gene Therapy Products Market Statistical Overview Report 2020 gives an outstanding tool for market Survey, openings, and Vital key and strategic basic leadership. This report perceives that in this quickly advancing and competitive scenario, up-coming data on the basis of Global Gene Therapy Products Market research execution and settle on basic choices for development and benefit. It gives data on market trends and advancements and sheds light on various sectors, limitations and advancements, and on the evolving structure of the market.

Global Gene Therapy Products Market By Product (Yescarta, Kymriah, Luxturna, Strimvelis, Gendicine), Application (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious diseases, Other Diseases), Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2026

Market Analysis:Global Gene Therapy Products Market

Global gene therapy products market is set to witness a substantial CAGR in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Rising cancer cases and unused potential for emerging markets are the major factors for the growth of this market.

Download FREE Sample (350 Pages PDF) Report: To Know the Impact of COVID-19 on this Industry@ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-gene-therapy-products-market&pm

Market Definition:Global Gene Therapy Products Market

Gene therapy or human gene therapy is a process which is used to modify gene for the treatment of any disease. Plasmid DNA, bacterial vector, human gene editing technology and viral vectors are some of the most common type of gene therapy products. The main aim of the gene therapy is to replace the dysfunctional genes. Somatic and germline are some of the most common type of the gene therapy.

Competitive Analysis:

Global gene therapy products market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of gene therapy products market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key Market Competitors:

Few of the major competitors currently working in the global gene therapy products market are Adaptimmune., Anchiano Therapeutics, bluebird bio, Inc., CELGENE CORPORATION, GlaxoSmithKline plc., Merck KGaA, Novartis AG, Achieve Life Sciences, Inc., Spark Therapeutics, Inc., Abeona Therapeutics, Inc, Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis, Inc., CRISPR Therapeutics, Intellia Therapeutics, Inc and Gilead Sciences,Inc. among others.

Market Drivers

Market Restraints

For More Insights Get FREE Detailed TOC @https://www.databridgemarketresearch.com/toc/?dbmr=global-gene-therapy-products-market&pm

Segmentation: Global Gene Therapy Products Market

By Product

By Application

Key Developments in the Market:

Reasons to Purchase this Report

Customization of the Report:

About Data Bridge Market Research:

An absolute way to forecast what future holds is to comprehend the trend today!Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

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Global Gene Therapy Products Market Study including Growth Factors, New Top Players, Competitive Analysis by regions from 2021 to 2027 - The Courier

ROCKVILLE, Md., Feb. 16, 2021 /PRNewswire/ --

RGX-314 using subretinal delivery continues to be generally well-tolerated at all dose levelsPositive interim update from Cohorts 4 and 5 at 1.5 years after RGX-314 administrationDurable treatment effect observed with stable visual acuity, decreased retinal thickness, and reductions in anti-VEGF injection burdenLong-term, durable treatment effect over three years demonstrated in Cohort 3Mean improvement in vision and stable retinal thickness50% of patients (3/6) remain anti-VEGF injection-free over three years; 67% of patients (4/6) are anti-VEGF injection-free from nine months to three yearsATMOSPHERE, the first of two planned pivotal trials for RGX-314, is active and enrolling

REGENXBIO Inc. (Nasdaq: RGNX) reported at the Angiogenesis, Exudation, and Degeneration 2021 conference additional positive interim data from Cohorts 4 and 5 of its RGX-314 Phase I/IIa trial for the treatment of wet age-related macular degeneration (wet AMD), and Cohort 3 of its Long-Term Follow-Up (LTFU) study. RGX-314 is a potential best-in-class, one-time gene therapy for the treatment of wet AMD.

"The continued durability of treatment effect up to three years after RGX-314 administration highlights the potential of RGX-314 as a one-time treatment option for patients with wet AMD. The results from the Phase I/IIa trial of RGX-314 using subretinal delivery have informed the key design elements of our pivotal program, in which we plan to conduct two randomized, well-controlled clinical trials, enrolling approximately 700 patients total," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO.

"I am excited about this data out to three years, which demonstrates that one-time treatment with RGX-314 has the potential to result in long-term stability to improvement of visual acuity outcomes and retinal anatomy, while alleviating treatment burden," said Allen C. Ho, M.D., Director of Retina Research at Wills Eye Hospital and Mid Atlantic Retina and investigator surgeon in the RGX-314 clinical trials. "In our practice, and as reported by multiple real-world studies, we see many patients losing vision due to lack of compliance with standard of care, which requires frequent anti-VEGF injections. I look forward to further evaluating the effects of RGX-314 in ATMOSPHERETM, the first pivotal trial of a gene therapy for the treatment of wet AMD."

Study Design and Safety Update from Phase I/IIa Trial of RGX-314 for the Treatment of Wet AMD Using Subretinal Delivery

In the Phase I/IIa trial of RGX-314, 42 patients with severe wet AMD requiring frequent anti-vascular endothelial growth factor (anti-VEGF) injections were treated across five dose cohorts, with doses ranging from 3x109 GC/eye to 2.5x1011 GC/eye.

As of January 22, 2021, RGX-314 continued to be generally well-tolerated across all cohorts, with 20 serious adverse events (SAEs) reported in 13 patients, including one possibly drug-related SAE of significant decrease in vision in Cohort 5. The most common nonserious adverse events in the eye were generally assessed as mild (87%). These included post-operative conjunctival hemorrhage (69% of patients), post-operative inflammation (36% of patients), eye irritation (17% of patients), eye pain (17% of patients), and post-operative visual acuity reduction (17% of patients). In 67% of patients across all cohorts, and in 83% of patients in Cohorts 3 through 5, retinal pigmentary changes were observed on imaging, the majority of which were in the peripheral inferior retina. Retinal hemorrhage was observed in 26% of patients and is an anticipated event in patients with severe wet AMD. There have been no reports of clinically-determined immune responses, drug-related ocular inflammation, or post-surgical inflammation beyond what is expected following routine vitrectomy.

Summary of Data for Cohorts 4 and 5

Today's update includes data from Cohorts 4 and 5 as of January 22, 2021. Each cohort enrolled 12 patients each at doses of 1.6x1011 GC/eye and 2.5x1011 GC/eye, respectively.

Patients in Cohorts 4 and 5 at 1.5 years after administration of RGX-314 demonstrated stable visual acuity with a mean Best Corrected Visual Acuity (BCVA) change of +1 letters and -1 letters from baseline, respectively, as well as decreased central retinal thickness (CRT), with a mean change of -46 m and -93 m, respectively.

There was a meaningful reduction in anti-VEGF treatment burden in both Cohorts 4 and 5 compared to the mean annualized injection rate during the 12 months prior to RGX-314 administration. Patients in Cohort 4 received a mean of 4.4 injections over 1.5 years following administration of RGX-314, a 58.3% reduction in anti-VEGF treatment burden. Patients in Cohort 5 received a mean of 1.7 injections over 1.5 years following administration of RGX-314, a reduction in anti-VEGF treatment burden of 81.2%.

In Cohort 4, four out of 12 (33%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of +2 letters at 1.5 years. Eight out of 11 (73%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of -2 letters at 1.5 years.

Summary of Long-Term Follow-Up (LTFU) Study Data

Following the Phase I/IIa trial, patients are encouraged to enroll in a LTFU study to assess safety and efficacy up to five years after RGX-314 administration. Patients in the LTFU study have scheduled visits every six months for the first year and then annual visits until the end of the study. Patient management is per physician discretion. Data collected during the scheduled study visits include safety, BCVA, and CRT. In addition, chart reviews are conducted at each scheduled study visit to collect the number of retina specialist visits and anti-VEGF injections each patient has received since the prior scheduled study visit.

As of January 22, 2021, RGX-314 continued to be generally well-tolerated in patients enrolled in the LTFU study, with no new drug-related ocular adverse events reported.

All six patients from Cohort 3 of the Phase I/IIa trial enrolled in the LTFU study, and long-term treatment effect was demonstrated over three years. These patients demonstrated a mean BCVA improvement of +12 letters from baseline at three years. Retinal anatomy as measured by machine-read CRT remained stable at three years compared to the two-year timepoint.

Patients also demonstrated long-term reductions in anti-VEGF treatment burden over three years with a mean annualized rate of 2.4 anti-VEGF injections after administration of RGX-314, which is a reduction of 66.7% from the mean annualized injection rate during the 12 months prior to administration of RGX-314. Three out of six (50%) patients received no anti-VEGF injections over three years following one-time administration of RGX-314. Four out of six (67%) patients have received no anti-VEGF injections from nine months to three years after RGX-314 administration. The four patients who did not receive anti-VEGF injections after nine months demonstrated a mean BCVA improvement from baseline of +11 letters at three years.

About Wet AMD

Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

About RGX-314

RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

REGENXBIO is advancing two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

About the Phase I/IIa Clinical Trial of RGX-314 and Long-Term Follow-Up Study

RGX-314 is being evaluated in a Phase I/IIa, multi-center, open-label, multiple-cohort, dose-escalation study in adult patients with wet AMD in the United States. The study includes patients previously treated for wet AMD who are responsive to anti-VEGF therapy. The study is designed to evaluate five escalating doses of RGX-314, with six patients in the first three dose cohorts and 12 patients in the fourth and fifth dose cohorts. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic immune suppressive oral corticosteroid therapy before or after administration of RGX-314. The primary endpoint of the study is safety at 6 months following administration of RGX-314. Secondary endpoints include visual acuity, retinal thickness on SDOCT, ocular RGX-314 protein expression, and the need for additional anti-VEGF therapy. Following completion of the primary study period, patients enter a follow-up period and will continue to be assessed until week 106 for long-term safety and durability of effect. After completion of the Phase I/IIa clinical trial, patients are encouraged to enter a Long-Term Follow-Up study to continue to follow safety and efficacy for a total of 5 years following administration of RGX-314.

About ATMOSPHERE

ATMOSPHERE is a multi-center, randomized, active-controlled trial to evaluate the efficacy and safety of a single-administration of RGX-314 versus standard of care in patients with wet AMD. The trial is designed to enroll 300 patients at a 1:1:1 ratio across two RGX-314 dose arms (6.4x1010 genome copies (GC)/eye and 1.3x1011 GC/eye delivered subretinally) and an active control arm of monthly intravitreal injections of ranibizumab (0.5 mg/eye). The primary endpoint of the trial is non-inferiority to ranibizumab based on change from baseline in Best Corrected Visual Acuity (BCVA) at 54 weeks. Secondary endpoints of the trial include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections. Patient selection criteria will include patients with wet AMD who are responsive to anti-VEGF treatment and will be independent of preexisting neutralizing antibody status. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314. The trial will be conducted at approximately 60 clinical sites based in the United States, with over 100 retinal surgeons.

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Forward-Looking Statements

This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to accurately predict how long REGENXBIO's existing cash resources will be sufficient to fund its anticipated operating expenses, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at http://www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

SCS Microinjector is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

Contacts: Tricia Truehart Investor Relations and Corporate Communications 347-926-7709 ttruehart@regenxbio.com

Investors: Brendan Burns, 212-600-1902 brendan@argotpartners.com

Media: David Rosen, 212-600-1902 david.rosen@argotpartners.com

View original content to download multimedia: http://www.prnewswire.com/news-releases/regenxbio-announces-additional-positive-interim-phase-iiia-and-long-term-follow-up-data-of-rgx-314-for-the-treatment-of-wet-amd-301228344.html

SOURCE REGENXBIO Inc.

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REGENXBIO Announces Additional Positive Interim Phase I/IIa and Long-Term Follow-Up Data of RGX-314 for the Treatment of Wet AMD - The Baytown Sun

COLUMBUS, Ohio, Feb. 16, 2021 /PRNewswire/ --Forge Biologics Inc., a fully integrated clinical stage gene therapy manufacturing and development company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease (RPD) designations to FBX-101 for the treatment of patients with Krabbe disease. Forge is now actively recruiting patients for enrollment in the RESKUE phase 1/2 clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease. FBX-101 is the first intraveniousgene therapy program for patients with Krabbe disease and marks a major step forward in building out the company's hybrid model as a gene therapy manufacturing and development engine.

"FDA's decision to grant these designations to our first-in-human investigational gene therapy highlights the urgency of developing a treatment for Krabbe patients," said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics. "Krabbe is a devastating disease, and it is imperative to develop treatment options like FBX-101 that may address all manifestations of the disease."

Fast Track Designation is given when the FDA determines that a drug demonstrates the potential to address unmet medical needs for a serious or life-threatening disease or condition. This designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions, and may also allow for priority or rolling review of a company's Biologics License Application (BLA).

The FDA grants Orphan Drug designation to drugs and biological products intended for the treatment of patients with rare diseases that affect fewer than 200,000 people in the United States. RPD designation is granted by the FDA to encourage treatments for serious or life-threatening diseases primarily affecting children 18 years of age and younger and fewer than 200,000 people in the United States. On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended by Congress after it was scheduled to sunset in 2020. Under the newly extended RPD program, if FBX-101 is approved by the FDA, Forge Biologics will qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

"Infantile Krabbe is a progressive and devastating leukodystrophy," said Jessie Barnum, M.D., AssistantProfessor,Department of Pediatrics,Division of Blood and Marrow Transplantation and Cellular Therapies and Principal Investigator of the FBX-101 trial at UMPC. "FBX-101 is an AAV gene therapy that has shown promising preclinical efficacy in Krabbe animal models of disease by extending survival and improving neuromuscular function when administered early in the disease course."

"The FBX-101 preclinical data brings a new wave of hope to the Krabbe community," said Anna Grantham, Director of Leukodystrophy Care Network Programs at Hunter's Hope. "These FDA designations for FBX-101 underscore a beautiful and collective effort to accelerate the timelines of bringing this potential therapy to patients who urgently need them."

"To see a promising new treatment for Krabbe receive these designations so quickly brings us one step closer to what everyone in our disease community is ultimately working towards: an FDA-approved treatmentfor Krabbe disease to reach the beside of all patients impacted by this disease," said Stacy Pike-Langenfeld, Director of Programs and Administration at The Legacy of Angels Foundation. "Our mission has always been to promote research to develop and enhance treatments for Krabbe disease, so it's very encouraging to see that Forge and FBX-101 have made so much progress in such a short amount of time."

Patients and families can learn more about clinical trials for FBX-101 by visiting https://www.forgebiologics.com/science/#krabbe.

About Krabbe diseaseKrabbe disease is a rare, inherited leukodystrophy affecting approximately 1:12,500 - 100,000 people in the U.S.A. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase (GALC) gene, a lysosomal enzyme responsible for the breakdown of certain types of lipids such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells. The psychosine toxicity is most severe in the myelin cells surrounding the nerves in the brain and in the peripheral nervous system, eventually leading to the death of these cells. The disease initially manifests as physical delays in development, muscle weakness and irritability and advances rapidly to difficulty swallowing, breathing problems, cognitive, vision and hearing loss. Early onset or "Infantile", Krabbe disease cases usually results in death by age 2-4 years, while later onset or "Late Infantile" cases have a more variable course of progressive decline. There is currently no approved treatment for Krabbe disease.

About FBX-101Forge is developing FBX-101 to treat patients with infantile Krabbe disease. FBX-101 is an adeno-associated viral (AAV) gene therapy that is delivered after a hematopoietic stem cell transplant. FBX-101 delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral neuropathy and correct the behavioral impairments associated with Krabbe disease in animal models, and to drastically improve the lifespan of treated animals. This approach has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies.

About Forge BiologicsForge Biologics is a hybrid gene therapy contract manufacturing and therapeutic development company. Forge's mission is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft2 facility in Columbus, Ohio, "The Hearth", to serve as their headquarters. The Hearth is the home of a custom-designed cGMP facility dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

For more information, please visit https://www.forgebiologics.com.

Patient, Pediatrician, Genetic Counselors & Family InquiriesDr. Maria EscolarChief Medical OfficerForge Biologics Inc.medicalaffairs@forgebiologics.com

Media Inquiries:Dan SalvoDirector of Communications and Community DevelopmentForge Biologics Inc.media@forgebiologics.com

Investor Relations and Business DevelopmentChristina PerryVice President, Finance and OperationsForge Biologics Inc.Investors@forgebiologics.com

View original content:http://www.prnewswire.com/news-releases/forge-biologics-receives-fda-fast-track-orphan-drug-and-rare-pediatric-disease-designations-for-fbx-101-gene-therapy-for-patients-with-krabbe-disease-301228668.html

SOURCE Forge Biologics

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Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe...

DUBLIN--(BUSINESS WIRE)--The "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

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 by revenue is expected to grow at a CAGR of over 23% during the period 2021-2026.

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.

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 patient's 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.

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?

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

Other Prominent Vendors

Emerging Investigational Vendors In Europe

For more information about this report visit https://www.researchandmarkets.com/r/6gqw7e

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Europe Cell and Gene Therapy Market Industry Outlook and Forecast Report 2021-2026 with Data-driven Insights on the Impact of COVID-19 -...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a publication in Nature Biotechnology that demonstrates the use of artificial intelligence to generate an unprecedented diversity of adeno-associated virus (AAV) capsids towards identifying functional variants capable of evading the immune system, a factor that is critical to enabling all patients to benefit from gene therapies. The research was conducted in collaboration with Google Research, Harvards Wyss Institute for Biologically Inspired Engineering and the Harvard Medical School laboratory of George M. Church, Ph.D., a Dyno scientific co-founder. The publication is entitled Deep diversification of an AAV capsid protein by machine learning.

It is estimated that up to 50-70% of the human population have pre-existing immunity to natural forms of the AAV vectors currently being using to deliver gene therapies. This immunity renders a large portion of patients ineligible to receive gene therapies which rely upon these capsids as the vector for delivery. Overcoming the challenge of pre-existing immunity to AAV vectors is therefore a major goal for the gene therapy field.

The approach described in the Nature Biotechnology paper opens a radically new frontier in capsid design. Our study clearly demonstrates the potential of machine learning to guide the design of diverse and functional sequence variants, far beyond what exists in nature, said Eric Kelsic, Ph.D., Dynos CEO and co-founder. We continue to expand and apply the power of artificial intelligence to design vectors that can not only overcome the problem of pre-existing immunity but also address the need for more effective and selective tissue targeting. At Dyno, we are making rapid progress to design novel AAV vectors that overcome the limitations of current vectors, improving treatments for more patients and expanding the number of diseases treatable with gene therapies.

The Nature Biotechnology paper describes the rapid production of a large library of distinct AAV capsid variants designed by machine learning models. Nearly 60% of the variants produced were determined to be viable, a significant increase over the typical yield of <1% using random mutagenesis, a standard method of generating diversity.

The more we change the AAV vector from how it looks naturally, the more likely we are to overcome the problem of pre-existing immunity, added Sam Sinai, Ph.D., Dyno co-founder and Machine Learning Team Lead. Key to solving this problem, however, is also ensuring that capsid variants remain viable for packaging the DNA payload. With conventional methods, this diversification is time- and resource-intensive, and results in a very low yield of viable capsids. In contrast, our approach allows us to rapidly unlock the full potential diversity of AAV capsids to develop improved gene therapies for a much larger number of patients.

This research builds upon previous work published in Science in which a complete landscape of single mutations around the AAV2 capsid was generated followed by evaluation of the functional properties important for in vivo delivery. In parallel with these works, Dyno has established collaborations with leading gene therapy companies Novartis, Sarepta Therapeutics, Roche and Spark Therapeutics to develop next-generation AAV gene therapy vectors with a goal of expanding the utility of gene therapies for ophthalmic, muscle, central nervous system (CNS) and liver diseases.

About CapsidMap for Designing Optimized AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dynos proprietary CapsidMap platform overcomes the limitations of todays gene therapies on the market and in development. Todays treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery efficiency, immunity, payload size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology to engineer capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dynos massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the design of novel capsids optimized for gene therapy.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experiments to gene therapy. The companys proprietary CapsidMap platform rapidly discovers and systematically optimizes Adeno-Associated Virus (AAV) capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit http://www.dynotx.com for additional information.

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Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of...

Global Cancer Gene Therapy Market Report Provides Future Development Possibilities By Key Players, Key Drivers, Competitive Analysis, Scope, And Key Challenges Analysis. The Reports Conjointly Elaborate The Expansion Rate Of The Industry Supported The Highest CAGR And Global Analysis. This Report Providing An In Depth And Top To Bottom Analysis By Market Size, Growth Forecast By Applications, Sales, Size, Types And Competitors For The Creating Segment And The Developing Section Among The Global Cancer Gene Therapy Market . Market Expansion Worldwide With Top Players Future Business Scope and Investment Analysis Report

Global Cancer Gene Therapy Market Research Report Will Help To Take Informed Decisions, Understand Opportunities, Plan Effective Business Strategies, Plan New Projects, Analyze Drivers And Restraints And Give Vision On The Forecast. Report Is A Specialist And Broad Research Report On The Major Regional Market Conditions, Concentrating On The United States, China, Europe, Japan, Southeast Asia, And India Regions.

Cancer gene therapy market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 6407.88 million by 2027 growing with the CAGR of 32.54% in the above-mentioned forecast period. The high success rate of cancer gene therapy along with clinical trial and preclinical trial is gaining popularity among the patient which is leading towards the market.

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The major players covered in the cancer gene therapy market report are Adaptimmune, GlaxoSmithKline plc, bluebird bio, Inc, Merck & Co., Inc., CELGENE CORPORATION, Anchiano Therapeutics, Achieve Life Sciences, Inc among other domestic and global players.

Competitive Landscape and Cancer Gene Therapy Market Share Analysis

Cancer gene therapy market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to cancer gene therapy market.

Global Cancer Gene Therapy Market Scope and Market Size

Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period.

High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

This cancer gene therapy market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographical expansions, technological innovations in the market. To gain more info on Cancer gene therapy market contactData Bridge Market Researchfor anAnalyst Brief, our team will help you take an informed market decision to achieve market growth.

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Cancer Gene Therapy Market Country Level Analysis

Cancer gene therapy market is analysed and market size insights and trends are provided by country, therapy and end user as referenced above.

The countries covered in the cancer gene therapy market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.

The country section of the cancer gene therapy market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

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Healthcare Infrastructure Growth Installed Base and New Technology Penetration

Cancer gene therapy market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipment, installed base of different kind of products for cancer gene therapy market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the cancer gene therapy market. The data is available for historic period 2010 to 2018.

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An absolute way to forecast what future holds is to comprehend the trend today!Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

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Global Cancer Gene Therapy Market Insights, Size Estimation, Research Insights, COVID-19 Impact and Future Trends By 2028 KSU | The Sentinel...

LONDON and LAUPHEIM, Germany, Feb. 11, 2021 (GLOBE NEWSWIRE) -- The Cell and Gene Therapy Catapult (CGT Catapult), an independent centre of excellence in innovation advancing the UKs cell and gene therapy industry, and Rentschler Biopharma SE, a leading global contract development and manufacturing organisation (CDMO) for biopharmaceuticals, have announced today that Rentschler Biopharma will establish their manufacturing capability in Advanced Therapy Medicinal Products (ATMPs), including Adeno-Associated Virus (AAV) Vectors for clinical trial supply, at the CGT Catapult site in Stevenage.

Under the terms of the agreement, Rentschler Biopharma will make a significant investment at the site over the next five years to set up their manufacturing capabilities, benefitting from the expertise and unique collaborative model provided by the CGT Catapult. The companys investment is expected to make a major contribution to meeting the demand from UK and international researchers for suitable manufacturing capability. This development will further strengthen the UK ecosystem through the addition of Rentschler Biopharmas more than 40 years of experience and solid reputation in the development and manufacturing of biologics for both clinical and commercial supply. The company will leverage the CGT Catapults expertise in ATMP manufacturing setup and technology development, as well as the cell and gene therapy cluster and ecosystem that has developed around Stevenage and across the UK.

Dr. Frank Mathias, CEO of Rentschler Biopharma, said:We are excited to take this next big step in our evolution and address the growing industry demand for ATMP manufacturing capacity and viral vector supply. With the largest industry cluster for cell and gene therapies outside the US, the UK is an ideal location for us to establish our Center of Excellence for cell and gene therapy. We look forward to working with the CGT Catapult as we invest in this growing field. They are well established in this important market, enabling us to immediately tap into the organisations network and utilisethe UKs strong expertise and supply chain in cell and gene therapy manufacturing.

Matthew Durdy, CEO of the Cell and Gene Therapy Catapult, commented:We are very pleased that Rentschler Biopharma, a global CDMO, has chosen to build their ATMP capacity in the UK, bringing in their expertise and investment. This will build new capacity to benefit the international ATMP supply chain and meet growing academic and commercial demand across the industry. As more companies from around the globe come to the UK, it demonstrates and enhances the attractiveness of its cell and gene therapy ecosystem as a place to develop new technologies and capabilities.

The investment in the UK cell and gene therapy industry announced today is expected to further accelerate the development of the vital infrastructure and skilled jobs needed to meet the rising demand for manufacturing capacity in the UK and globally, as well as streamline the supply chain for these advanced therapies. Currently, 27% of European ATMP companies are operating in the UK, and there are more than 90 advanced therapy developers. The last year has also seen a 50% increase in the number of ATMP clinical trials being run in the UK, accounting for 12% of global ATMP clinical trials, and these numbers are predicted to increase further.

The CGT Catapult manufacturing centre has been backed by over 75m of funding, including investment from the UK Governments Industrial Strategy Challenge Fund, the Department for Business, Energy and Industrial Strategy, Innovate UK and from the European Regional Development Fund. Since it was announced, there has been over 1.1bn of investment in the ATMP industry in its vicinity.

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.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK.

For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About the European Regional Development Fund

This project has received 3.36m of funding from the England European Regional Development Fund as part of the European Structural and Investment Funds Growth Programme 2014-2020. The Ministry of Housing, Communities and Local Government (and in London the intermediate body Greater London Authority) is the Managing Authority for European Regional Development Fund. Established by the European Union, the European Regional Development Fund helps local areas stimulate their economic development by investing in projects which will support innovation, businesses, create jobs and local community regenerations. For more information visit https://www.gov.uk/european-growth-funding.

About the Industrial Strategy Challenge Fund

This project has received 12m of funding from the Industrial Strategy Challenge Fund, part of the governments modern Industrial Strategy. The Industrial Strategy Challenge Fund is a four-year, 1 billion investment in cutting-edge technology designed to create jobs and improve living standards, built on guidance from business and the academic community. Healthcare and Medicine is one of three core areas for investment under the programme.

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Rentschler Biopharma to build new cell and gene therapy capabilities in the UK - BioSpace

Part of Aldevrons expansion is a fermentation suite that will allow the company to expand its projects scope and scale. Submitted photo

From February through June, we will highlight the ways that UWMadison powers the states economy through research and innovation, educates the next generation and reaches out to Wisconsinites to improve their lives. Februarys theme is Economic Prosperity. Watch for more at #CantStopABadger and #UWimpact on social media.Your supportcan help us continue this work.

February 10, Aldevron hosted a virtual celebration of its lab facility expansion in Madison.

Aldevron produces the raw materials that allow clients to make groundbreaking therapies, and its protein business unit has operated within University Research Park since 2009. The facilitys growth from 8,000 to nearly 30,000 square feet includes a new 3,500-square-foot fermentation suite and will allow the company to expand its projects scope and scale.

Aldevron founders John Ballantyne and Michael Chambers partnered with Tom Foti to establish the companys protein business unit in Madison in 2009 to expand Aldevrons offerings.

Madison is rightly known around the world as a biotech hotspot, and the fact that there was a team out there ready to hit the ground running made it sort of a no-brainer, said Ballantyne, who serves as Aldevrons Chief Science Officer.

Speakers at the celebration pointed out that Aldevrons partnerships with UWMadison in research and education benefit both the company and the university.

I watched Aldevron partner with researchers at the University of WisconsinMadison, with other companies at the Park, and with partners all over the globe, and its these partnerships that are at the very center of Aldevrons business model, said Aaron Olver, the Managing Director of University Research Park, a UWMadison-affiliated nonprofit that creates neighborhoods where innovation can flourish. So with the newly expanded capacity at the Park, I know Aldevrons going to be able to find new partnerships and new opportunities, to push the boundaries of science even further.

Ballantyne envisions the Madison team continuing their collaborations with researchers and also expanding into RNP capabilities for gene editing and IVT enzymes. He praised the teams reputation and ability to transition new products into a manufacturing environment, saying theres an art to that tech transfer.

A lot of rigor goes into building our systems, especially with the complexity of protein, he said. Its very exciting what their ramp-up is going to look like out there.

The University of Wisconsin has created a dynamic hub of biotech companies, based on its amazing research companies and abundance of trained talent, said Aldevron CEO Kevin Ballinger, citing Aldevrons partnership with UWMadison as a compelling reason the company has continued to invest in its Madison site.

We made the investment ten years ago and doubled down on this site because we see the incredible return and endless possibilities in the following areas: scientific collaboration around gene editing, a supply chain for cell therapy manufacturing, partnership on workforce development, and talent recruitment to help staff our growing team.

The University of Wisconsin has created a dynamic hub of biotech companies, based on its amazing research companies and abundance of trained talent. Aldevron CEO Kevin Ballinger

Aldevrons Madison team makes CRISPR proteins like Cas9 that are essential to the field of gene editing and its potential to treat thousands of previously untreatable medical conditions. Aldevron Madison also makes the IVT enzymes for research use that support mRNA technology. Later this year, Aldevron will be making GMP IVT enzymes for clinical applications, all of which will be supported by the companys pending ISO1345:2016 registration. The company is currently pursuing ISO 1345:2016, a standard for FDA compliance.

Aldevrons RNP services turn CRISPR reagents into therapies, and by the middle of this year, well be the only company to launch a RNP manufacturing service to provide GMP reagents to clients developing gene therapies, says Ballinger.

Tom Foti, President of Aldevrons protein business unit since its 2009 Madison launch, said he will likely increase his staff by about 40% with the facilities expansion.

Diversity is super important in high-performing teams, said Foti. We are investing in talent to build a strong culture centered around problem-solving and continuous improvement.

Both Foti and Katie Rogers, Aldevrons Senior Manager of Upstream Processing, described the expanded main lab area and vibrant communal spaces bathed in natural light.

More scientists and equipment mean Aldevron can serve more clients, said Rogers.

Local expansion partners include companies based in the Madison area: J.H. Findorff & Son Inc.; Strang, Inc.; M&M Office Interiors; Fearings Audio Video Security; Capitol Mechanical; Faith Technologies; Livesey Painting, Inc.; Lake City Glass; Monona Plumbing and Fire Protection; Sergenians Floor Coverings; and Badger Acoustics, Inc.; as well as Wausau-based Graphic House.

Dr. Richard Moss, Senior Associate Dean for Basic Research, Biotechnology and Graduate Studies at the University of WisconsinMadison School of Medicine and Public Health, emphasized the strength of Aldevrons collaboration with UWMadison.

Our relationship with Aldevron over time has evolved into a partnership that is not only collaborative but very effective, said Moss. Ongoing conversations between UW and Aldevron are really focused on expanding learning opportunities for students at the UW in the health professions.

Aldevron supports UWMadisons Masters program in biotechnology, offering real-world educational opportunities for students in the program and for graduate students and postdocs across campus. UWMadisons Department of Industrial Engineering has collaborated with Aldevrons quick response manufacturing team on biological manufacturing processes, and Aldevron has partnered with professor Kris Saha in the Department of Biomedical Engineering on gene editing approaches to genetic medicine.

We hope we will be able to expand our partnership in the near future to our program in advanced cellular therapies, with the idea of moving these new therapies to cell therapy manufacturing, said Moss. We see these partnerships as a win for translating discoveries from the bench to the bedside. Its a win for learners; its a win for researchers; and most importantly, its a win for advancing the health of the patients in the communities that all of us serve.

UW-Madison contributes $20.8 billion per year to the Wisconsin economy, and UWMadison related start-ups contribute an additional $10 billion. Read morehere.

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Aldevron expands manufacturing capabilities in Madison - University of Wisconsin-Madison

The global rare disease gene therapy is expected to witness a significant growth in the forecast period, 2020-2030 on account of the increasing cases of genetic diseases worldwide. Gene therapy is relevant to rare disease patients and has improved the wellbeing and personal satisfaction of more seasoned kids and youthful grown-ups with X-SCID. These kids are expected to experience complex clinical issues in the wake of getting live-sparing bone marrow transfers to treat the condition.

Impact of COVID-19 on the Healthcare Industry

The COVID-19 pandemic has caused severe impacts on the global economy at various levels and which can be seen on the Healthcare industry as well. The thriving market of health care research and development is expected to exhibit a steep decline in the sales during the lockdown period owing to the shutdown of the manufacturing units, acute shortage in the supply of raw materials and absence of potential manpower. It can be deduced from the current situations brought about by the pandemic that the production, and supply chain activities have experienced minor hurdles. However, the market is projected to gradually recover post-COVID-19, which will present attractive opportunities for sales across various regions of the world in the following years.

Future Market Insights (FMI) adopted a multidisciplinary approach during the pandemic-era to focus on the growth and development of theRare Disease Gene Therapy Market. The study features insights on the current growth dynamics and the major revenue reforms prevailing in the market as of 2019 along with the key takeaways over the forecast period 2020 to 2030.

The team of analysts at Future Business Insights are focussing on research and market study to produce different Rare Disease Gene Therapy Market forecasts and predictions at both national and international levels. They have considered several leads of information pertaining to the industry like market figures and merger estimations to assess and produce reliable and informative insights on the Rare Disease Gene Therapy Market.

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Key Players

The writer will create content on the general strategies of market players. And then will write the key players in the market are: Pfizer Inc., Amgen Inc., Bristol-Myers Squibb Company, AbbVie Inc., Novartis AG F. Hoffmann-La Roche Ltd., Teva Pharmaceutical Industries Ltd and Others

Segmentation

The report provides insights on the important highlights and current trends prevailing in the market. This helps the readers to gain a deeper understanding and form an unbiased opinion on the market. Numerous segmentations have been provided for this market based on:

Gene Therapy

Indication

Administration

Distribution Channel

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Product Segmentation

The investigation offers a top to bottom evaluation of different clients journeys pertinent to the market and its segments.The study endeavours to assess the current and future development possibilities, undiscovered roads, factors that shapes their income potential in the global market by breaking it into di such as its types, applications, and region-wise assessment.

By Regional Analysis Covered

Full in-depth analysis of the parent market

The analysts at FMI are dedicated to provide insights after extensive research and study. The study also includes estimations, projections and evaluation of the market dynamics.

Important changes in market dynamics

The report has been created after detailed and exhaustive studies by the analysts at FMI taking several factors into consideration like monetary, ecological, social, mechanical, and political status of a particular demography. They study the key data to assess the revenue and production of manufacturers across various regions. The report also covers an in-depth analysis of the key changes in market dynamics in the recent past and the near future.

Segmentation details of the market

Queries Solved

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Table of Content

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About FMI

Future Market Insights (FMI) is a leading provider of market intelligence and consulting services, serving clients in over 150 countries.FMIis headquartered in Dubai, the global financial capital, and has delivery centers in the U.S. and India. FMIs latestmarket research reportsand industry analysis help businesses navigate challenges and make critical decisions with confidence and clarity amidst breakneck competition. Our customized and syndicated market research reports deliver actionable insights that drive sustainable growth. A team of expert-led analysts at FMI continuously tracks emerging trends and events in a broad range of industries to ensure that our clients prepare for the evolving needs of their consumers.

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Rare Disease Gene Therapy Market: Increasing cases of genetic diseases to drive the market - PharmiWeb.com

Feb. 15, 2021 19:00 UTC

MONTPELLIER, France--(BUSINESS WIRE)-- Regulatory News:

Sensorion (Paris:ALSEN)(FR0012596468 ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, announces the addition of a new gene therapy target, GJB2 coding for the Connexin 26 protein, to its development portfolio. The target is the third candidate to emerge from Sensorions R&D collaboration with Institut Pasteur. The GJB2 program will focus on major new markets with an estimated patient population (prevalence) of 300,000 children and adults in Europe and in the United-States alone.

New research (Boucher et al., Proc Natl Acad Sci U S A. 2020;117(49):31278-3128) published by scientists at the Institut Pasteur shows that anomalies in GJB2 are not only the most common cause of congenital deafness (prevalence of around 200,000 individuals in the US and in Europe alone) but also occur in adult cases of severe age-related hearing loss (around 100,000 adults in the same geographies). Although the types of GJB2 mutations in children and adults may differ, gene therapy could potentially provide a solution to both.

Sensorion's GJB2 gene therapy program has the potential to target three pathologies related to GJB2 mutations: age-related hearing loss in adults, progressive forms of hearing loss in children, and pediatric congenital deafness. Initially, the focus will be on the first two populations with GJB2-associated hearing loss, making Sensorion the first company to address these important medical needs in adults and potentially large market opportunities.

Current scientific understanding suggests that mutations in GJB2 alter a gap junction protein widely expressed in the inner ear, disturbing intercellular exchanges of molecules and leading to hearing loss that is severe-to-profound in a majority of cases.

The emergence of a new gene therapy target candidate validates our conviction that long-term solutions for restoring hereditary hearing loss will arise from an in-depth analysis of the "genetic landscape" of hearing loss," said Nawal Ouzren, CEO of Sensorion. "It was clear that mutations in the GJB2 gene are important in severe to profound childhood hearing loss. However, the new discovery made by our collaborators at Institut Pasteur shows that alteration of this gene in adults offers new opportunities for Sensorion. It marks significant potential expansion of our pipeline and supports our goal of becoming a global leader in the field of gene therapies for hearing loss disorders.

"Until now, the genetics of late forms (age-related deafness or presbycusis) was considered to involve multiple variants in each individual," said Professor Christine Petit, Director of the French Hearing Institute, an Institut Pasteur Center. "We have shown that the same genes underlying congenital or childhood deafness are also involved in severe forms of early presbycusis. The presence of these very rare genetic variants makes these forms of presbycusis appear to be monogenic types of hearing loss which can therefore be potentially treated by gene therapy."

Sensorions collaboration with Institut Pasteur initiated in 2019 has already led to gene therapy candidate programs in two other indications - Otoferlin deficiency and Usher Syndrome Type 1. GJB2-GT is the third program under this collaboration and represents the largest gene therapy opportunity for Sensorion to date.

Considering its broad and rich pipeline, Sensorion may have to consider a reallocation of some resources in the future to focus on the most attractive development programs.

Sensorion will host a webcast on the expansion of its gene therapy pipeline on Tuesday, February 16 at 2:00pm CET (8:00am ET). Please register for the webcast here.

About Sensorion

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. Its clinical-stage portfolio includes one Phase 2 product: SENS401 (Arazasetron) for sudden sensorineural hearing loss (SSNHL). Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases enabling it to select the best targets and modalities for drug candidates. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. In the second half of 2019, Sensorion launched two preclinical gene therapy programs aimed at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. The Company is potentially uniquely placed, through its platforms and pipeline of potential therapeutics, to make a lasting positive impact on hundreds of thousands of people with inner ear related disorders, a significant global unmet medical need.

http://www.sensorion.com

Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN

Disclaimer

This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2020 Half-Year financial report published on October 21, 2020 and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.

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Sensorion Announces Third Gene Therapy Collaboration With Institut Pasteur Targeting Important Pediatric and Adult Deafness Segments - BioSpace

LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the Company), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced that the Company will host an Expert Perspectives call on February 22, 2021 at 12:00pm EST. The call will feature a discussion between Aniz Girach, MD, Chief Medical Officer of ProQR Therapeutics and Paul Yang, MD, PhD about disease education and endpoints in Usher syndrome and non-syndromic Retinitis Pigmentosa (nsRP). Areas of focus for the session will include which vision measures are most informative in the context of this disease setting, the role of patient baseline and disease progression, and an overview of the objectives of the Phase 1/2 Stellar trial of QR-421a.

Event Details

Date/Time: February 22, 2021, 12:00pm EST

To register, please follow this link.

Following the discussion, a portion of the call will be dedicated to Q&A. The archived presentation will be available on the Companys website for approximately 30 days following the presentation date.

Paul Yang, MD, PhD, Casey Eye Institute, Oregon Health & Science University

Dr. Paul Yang received doctorates in medicine and neurophysiology at Dartmouth Medical School, which was funded by an MD/PhD pre-doctoral award from the National Institutes of Health. He completed residency and fellowship in ophthalmology at the Moran Eye Center in Salt Lake City, during which he first developed an interest in inflammatory eye diseases and degenerative retinal disorders. Thereafter, he pursued a fellowship in ocular immunology and uveitis at the Massachusetts Eye Research and Surgery Institution in Cambridge, Massachusetts, as well as a fellowship in ophthalmic genetics and inherited retinal degenerations at Casey Eye Institute in Portland, Oregon. He was funded by the Foundation Fighting Blindness (FFB) Clinical Research Fellowship Award, FFB Career Development Award, and NIH K08 to evaluate the effectiveness of mycophenolate as a neuroprotective agent in inherited retinal degenerations. For his pioneering work, he was awarded the 2015 ARVO/Alcon Early Career Clinician-Scientist Research Award. Dr. Yang is an assistant professor in ophthalmic genetics and immunology at the Casey Eye Institute (Oregon Health & Science University) where he specializes in patients with inherited retinal degenerations, autoimmune retinopathy, and gene therapy associated uveitis. He is a principal investigator and sub-investigator on numerous gene therapy and neuroprotection clinical trials for inherited retinal degenerations. Dr. Yang continues to conduct translational research in his lab with the goal of bringing new medical treatments to the clinic for patients with inherited retinal degenerations.

About Usher Syndrome Type 2a and Non-Syndromic Retinitis Pigmentosa

Usher syndrome is the leading cause of combined deafness and blindness. People with Usher syndrome type 2a are usually born with hearing loss and start to have progressive vision loss during adulthood. The vision loss can also occur without hearing loss in a disease called non-syndromic retinitis pigmentosa. Usher syndrome type 2a and non-syndromic retinitis pigmentosa can be caused by mutations in the USH2A gene. To date, there are no pharmaceutical treatments approved or in clinical development that treat the vision loss associated with mutations in USH2A.

About QR-421a

QR-421a is being evaluated in the Phase 1/2 Stellar trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and rare pediatric disease designations from the FDA.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

Learn more about ProQR at http://www.proqr.com.

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding this Expert Perspectives event. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.

Investor Contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorHans VitzthumLifeSci AdvisorsT: +1 617 535 7743hans@lifesciadvisors.com

Media Contact:Cherilyn Cecchini, MDLifeSci CommunicationsT: +1 646 876 5196ccecchini@lifescicomms.com

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ProQR Announces Expert Perspectives Call on Disease Education and Endpoints in Usher Syndrome - GlobeNewswire

Videos showing for the first time how small circles of DNA adopt dance-like movements inside a cell have been developed by researchers in Yorkshire.

The footage, created by a team of scientists from the Universities of Leeds, Sheffield and York, is based on the highest resolution images of a single molecule of DNA ever captured.

They show in unprecedented detail how the stresses and strains that are placed on DNA when it is crammed inside cells can change its shape.

Previously scientists were only able to see DNA by using microscopes limited to taking static images. But now the Yorkshire team has combined advanced atomic force microscopy with supercomputer simulations to create videos of twisted molecules of DNA.

The images are so detailed it is possible to see the iconic double helical structure of DNA, but when combined with the simulations, the researchers were able to see the position of every single atom in the DNA and how it twists and writhes.

Credit: University of Sheffield

Dr Alice Pyne, Lecturer in Polymers & Soft Matter at the University of Sheffield, who captured the footage, said: "Seeing is believing, but with something as small as DNA, seeing the helical structure of the entire DNA molecule was extremely challenging.

"The videos we have developed enable us to observe DNA twisting in a level of detail that has never been seen before."

The study, Base-pair resolution analysis of the effect of supercoiling on DNA flexibility and major groove recognition by triplex-forming oligonucleotides, is published in Nature Communications.

Every human cell contains two metres of DNA. In order for this DNA to fit inside our cells, it has evolved to twist, turn and coil a process called supercoiling. That means that loopy DNA is everywhere in the genome, forming twisted structures which show more dynamic behaviour than their relaxed counterparts.

To investigate how this process works, the research team studied small "packets" of genetic information called DNA minicircles, engineered and isolated from bacteria. DNA minicircles are special becausethe molecule is joined at both ends to form a loop. This loop enabled the researchers to give the DNA minicircles an extra added twist, making the DNA "dance" more vigorously.

When the researchers imaged relaxed DNA, without any twists, they saw that it did very little. But when they gave the DNA an added twist, it suddenly became far more dynamic and could be seen to adopt some very exotic shapes.

These exotic "dance moves" were found to be the key to finding binding partners for the DNA.

Gene therapy is the use of nucleic acids such as DNA to repair, replace, or regulate genes to prevent or treat human disease. In the past few decades, hundreds of gene therapy candidate genes have been uncovered, yet very few of these have turned into target therapies because of the challenge of delivering the gene therapy.

Professor Lynn Zechiedrich from Baylor College of Medicine in Houston Texas, who made the minicircles used in this study, has found a way to design supercoiled minicircles or, minivectors for use in gene therapy by inserting short genetic messages.

Professor Zechiedrich said: "The research team in Yorkshire have developed a technique that reveals in remarkable detail how wrinkled, bubbled, kinked, denatured, and strangely shaped they are!

"We have to understand how supercoiling, which is so important for DNA activities in cells, affects DNA in hope that we can learn how to mimic or control it someday."

Dr Massa Shoura from Stanford University, who has detected DNA minicircles in human cells, said: "Very little is currently understood about the function of circular DNAs in cells, but there is a chance they could be used as markers for early detection of disease."

Dr Sarah Harris, Associate Professor in the School of Physics and Astronomy at the University of Leeds, who supervised the research, said: "The laws of physics apply just as well to the molecules that make up living systems as to sub-atomic particles and galaxies. The synergy between our experiments and computer models shows we are beginning to understand the physics underlying supercoiled DNA. This insight should help researchers such as Professor Zechiedrich design bespoke minicircles for future therapies."

Further information

Top image: A visualisation of a DNA minicircle

For media enquiries, please contact David Lewis in the University of Leeds press office: d.lewis@leeds.ac.uk

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News > Science > Visualisation of 'dancing DNA' - University of Leeds

Research from the Sheffield Institute for Translational Neuroscience (SITraN) suggests that routine genetic testing may be appropriate for all MND patients and could impact disease subclassification and clinical care.

Research from the Sheffield Institute for Translational Neuroscience (SITraN) suggests that routine genetic testing may be appropriate for all MND patients and could impact disease subclassification and clinical care.

The new study, published today (15 February 2021) in the Journal of Neurology, Neurosurgery and Psychiatry (JNNP), performed targeted genetic sequencing of MND-relevant genes on 100 patients.

Researchers found higher than expected genetic changes in the group of patients. The paper recommends that genetic testing could be appropriate for all MND patients whether or not they have a family history of the disease.

While the majority of MND cases are considered sporadic, five per cent to 10 per cent have been shown to be familial. Currently only patients with a family history of MND, dementia, or who experience disease onset at a young age are routinely offered genetic screenings in the UK. However, with the development of new therapies targeting specific genetic forms of the disease, researchers are recommending that all MND patients are offered a screening.

Prof Janine Kirby, Professor of Neurogenetics at the University of Sheffield, said Our study found that 42 per cent of patients involved in the screening showed variants in known MND-linked genes. This doesnt mean that 42 per cent of MND cases are familial - but shows that some familial and sporadic cases can share the same genetic cause of disease.

We found that 21 per cent of patients had a clinically reportable genetic alteration that has been proven to increase the likelihood of developing MND. Of these, 93 per cent had no family history of MND and 15 per cent met the inclusion criteria for a current MND gene therapy clinical trial.

As future studies expand the number of verified genetic causes of MND, we will continue to see if they are also found in cases without a family history.

Professor Dame Pamela Shaw, Director of SITraN and the NIHR Sheffield Biomedical Research Centre said Our study suggests that all patients with MND should, with careful counselling, be offered genetic testing.

We hope that by screening all MND patients for gene mutations that are a known factor in MND, we can further our knowledge on subclassification of the disease, but also ensure that patients have access to clinical trials that are relevant for them personally."

This is increasingly important in light of the new personalised medicine treatments in development for MND that target a specific gene mutation to ensure that patients have access to potential treatments that could be beneficial to them.

Dr Brian Dickie, Director of Research Development at the Motor Neurone Disease Association said MND is a complex disease involving a complex mix of genetic and environmental factors. This latest research sheds more light on the genetic component and will hopefully lead to greater availability of genetic testing to aid earlier diagnosis and more tailored treatments in the future.

This study was supported by funds raised through the Ice Bucket Challenge and will be widened to include analysis of additional samples from two other clinics collaborating on this MND Association funded project. This will provide an even clearer picture of the UK MND genetic landscape.

MND - also known as amyotrophic lateral sclerosis (ALS) - is an adult-onset neurodegenerative disease characterised by progressive injury and cell death of upper and lower motor neurons. This leads to progressive failure of the neuromuscular system with death, usually from respiratory failure, within 25 years of symptoms in most cases.

Currently, there is no cure for MND and no effective treatments to halt or reverse the progression of this devastating disease.

The National Institute for Health Research (NIHR) is the nations largest funder of health and care research. The NIHR:

The NIHR was established in 2006 to improve the health and wealth of the nation through research, and is funded by the Department of Health and Social Care. In addition to its national role, the NIHR commissions applied health research to benefit the poorest people in low- and middle-income countries, using Official Development Assistance funding.

This work uses data provided by patients and collected by the NHS as part of their care and support and would not have been possible without access to this data. The NIHR recognises and values the role of patient data, securely accessed and stored, both in underpinning and leading to improvements in research and care. http://www.nihr.ac.uk/patientdata

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New study suggests genetic testing could be appropriate for all motor neuron disease (MND) patients whether or not they have a family history of the...