Archive for the ‘Gene Therapy Research’ Category

Manipal Academy of Higher Education (MAHE) in India and Esco Aster in Singapore have signed agreement to establish collaborative research unit at MAHE, Manipal

This is the second Esco Aster collaborative research unit set up globally after the firstCentre of Excellence (COE) with Bioprocessing Technology Institute (BTI), Singapore. Esco Aster hopes to continue to develop more collaborations in Asia Pacific in years to come to work on the booming cell and gene therapy.

As per the agreement, ESCO Aster and MAHE will jointly establish a research unit at MAHE Manipal campus to carry outactivities related to development and manufacturing in the biotherapeutics area.MAHE will provide cGMP compliant facility and necessary resources for carrying out the research activities.

-Lt. General Dr. Venkatesh M.D., MAHEs Vice Chancellor

Weare excited to sign this agreement with MAHE and we look forward for harnessing the skilled manpower and mentor pool available at MAHE to build our pipeline products and services to our clients. We envision showcasing this research unit to our potential clients and collaborators and also utilizing it as a training centre.

-Xiangliang Lin, Esco Aster Chief Executive Officer

Through this agreement, both parties aim to facilitate research, development, and commercial plans and activities relating to stem cells or extracellular vesicles. Other fields of study with potential advantages to scientific discoveries are also welcome. This collaboration opens up potential breakthrough discoveries aiming to drive the biotherapeutics industry.

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Singapore inks research collaboration with India for biotherapeutics - BSA bureau

LONDON, Sept. 24, 2020 (GLOBE NEWSWIRE) -- (Companies Included: Crispr Therapeutics, Thermo Fisher Scientific, Intellia Therapeutics, Horizon Discovery, and Synthego Corporation)

In another instance, in early May, the US Food and Drug Administration (FDA) granted Sherlock Biosciences an emergency use authorization (EUA) for its COVID-19 diagnostic assay, beating out other companies and academic groups trying to use the powerful gene-editing technology to figure out who is infected with the novel coronavirus. Sherlocks test is the first FDA-authorized use of CRISPR technology for anything. Sherlocks test is a molecular diagnostic, intended to identify people who have acute SARS-CoV-2 infection. It capitalizes on a CRISPR-based technology developed in the lab of Feng Zhang, a scientist at Broad Institute of MIT and Harvard and a cofounder of Sherlock.

The Business Research Companys report titled CRISPR Technology Global Market Report 2020-30: Covid 19 Growth And Change covers the CRISPR market 2020, CRISPR technology market share by company, global CRISPR technology market analysis, global CRISPR technology market size, and CRISPR technology market forecasts. The report also covers the global CRISPR technology market and its segments. The CRISPR technology market share is segmented by product type into Cas9 and gRNA, design tool, plasmid and vector, and other delivery system products. The CRISPR technology market share is segmented by end-user into biopharmaceutical companies, agricultural biotechnology companies, academic research organizations, and contract research organizations (CROs). By application, it is segmented into biomedical, agriculture, diagnostics, and others.

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The global CRISPR technology market value is expected to grow from $685.5 million in 2019 to $1,654.2 million in 2023 at a compound annual growth rate (CAGR) of 24.6%. The application of CRISPR technology as a diagnostic tool is expected to boost CRISPR technology market growth during the period. The Sherlock CRISPR SARS-CoV-2 kit is the first diagnostic kit based on CRISPR technology for infectious diseases caused due to COVID-19. In May 2020, the US FDA (Food and Drug Administration) announced emergency use authorization of Sherlock BioSciences Inc.s Sherlock CRISPR SARS-CoV-2 kit, which is a CRISPR-based SHERLOCK (Specific High-sensitivity Enzymatic Reporter unLOCKing) diagnostic test.

This test helps in specifically targeting RNA or DNA sequences of the SARS-CoV-2 virus from specimens or samples such as nasal swabs from the upper respiratory tract, and fluid in the lungs from bronchoalveolar lavage specimens. This diagnostic kit has high specificity and sensitivity, and does not provide false negative or positive results. Widening the application of CRISPR technology for the diagnosis of infectious diseases will further increase the demand for CRISPR technology products and services and drive the CRISPR market 2020.

Several advancements in CRISPR technology are trending in the market. Advancements in technology will help in reducing errors, limiting unintended effects, improving the accuracy of the tool, widening its applications, developing gene therapies, and more. Scientists, researchers and companies are increasingly developing advanced CRISPR technologies for more precise editing and to get access to difficult to reach areas of human genome. For instance, in March 2020, scientists at University of Toronto developed CHyMErA, a CRISPR-based tool for more versatile genome editing. Similarly, in March 2020, researchers at New York genome center developed a new CRISPR screening technology to target RNA, including RNA of novel viruses like COVID.

In November 2019, researchers at ETH Zurich, Switzerland, swapped CAS9 enzyme for Cas 12a, that allowed the researchers to edit genes in 25 target sites. It is also estimated that hundreds of target sites can be modified using the above method. In October 2019, a team from MIT and Harvard developed new CRISPR genome editing approach called prime editing by combining CRISPR-Cas9 and reverse transcriptase into a single protein. The prime editing has the potential to directly edit human cells with high precision and efficiency.

The CRISPR technology market share consists of sales of CRISPR technology products and services, which is a gene-editing technology that allows researchers to alter DNA sequences and modify gene function. The revenue generated by the market includes the sales of products such as design tools, plasmid & vector, Cas9 & gRNA, and libraries & delivery system products and services that include design & vector construction, screening and cell line engineering. These products and services are used in genome editing/genetic engineering, genetically modifying organisms, agricultural biotechnology and others, which include gRNA database/gene library, CRISPR plasmid, and human stem cell & cell line engineering.

CRISPR Technology Global Market Report 2020-30: Covid 19 Growth And Change is one of a series of new reports from The Business Research Company that provide market overviews, analyze and forecast market size and growth for the whole market, CRISPR technology market segments and geographies, CRISPR technology market trends, CRISPR technology market drivers, CRISPR technology marketrestraints, CRISPR technology market leading competitors revenues, profiles and market shares in over 1,000 industry reports, covering over 2,500 market segments and 60 geographies. The report also gives in-depth analysis of the impact of COVID-19 on the market. The reports draw on 150,000 datasets, extensive secondary research, and exclusive insights from interviews with industry leaders. A highly experienced and expert team of analysts and modellers provides market analysis and forecasts. The reports identify top countries and segments for opportunities and strategies based on market trends and leading competitors approaches.

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The Global CRISPR Technology Market Size Is Seeing Exponential Growth Due To The Application Of CRISPR Technology In Treating COVID-19 - GlobeNewswire

IRVINE, Calif.--(BUSINESS WIRE)--Applied Biology in collaboration with researchers at University Hospital Ramon y Cajal in Madrid, Spain, today announced results from their androgen receptor genetics study in hospitalized COVID-19 male patients (NCT04368897, https://clinicaltrials.gov/ct2/show/NCT04368897?term=androgen&cond=Covid19&draw=2&rank=4). The aim of the study was to confirm a direct association between androgens and COVID-19, potentially paving the way for a breakthrough therapy against SARS-CoV-2.

This prospective longitudinal study analyzed the length of a variable genetic repeat in the androgen receptor gene of hospitalized COVID-19 male patients. The results of the clinical study were published in the Journal of the European Academy of Dermatology and Venerology (doi: 10.1111/jdv.16956).

The study demonstrated that the proportion of male patients admitted to the ICU with the longer variant was statistically significantly higher compared to males with the shorter variant. Similarly, the risk of ICU admissions was significantly higher among males with the longer variant (OR 2.9143). In addition, COVID-19 patients with the shorter variant experienced a shorter duration of hospitalization compared to males with the longer variant (25 days vs 47.5 days). Overall, the longer variant of the androgen receptor was associated with higher disease severity among hospitalized male COVID-19 patients.

The team was led by Andy Goren, MD, Medical Researcher and Co-Investigator with the Department of Dermatology at the University Hospital Ramon y Cajal in Madrid, Spain; Sergio Va Galvn, MD, from the Department of Dermatology at the University Hospital Ramon y Cajal; Sabina Herrera, MD, from the Infectious Disease Department at the University Hospital Ramon y Cajal; Carlos G. Wambier, MD, PhD, from the Department of Dermatology at the Alpert Medical School of Brown University; and Flavio A. Cadegiani, MD, MSc, PhD, from Corpometria Institute in Brazil.

According to Dr. Sergio Va Galvn: To the best of our knowledge, this is the first study to directly confirm an association between androgens and COVID-19 disease severity.

According to Dr. Carlos Wambier: This is another piece of the puzzle for the scientific understanding of role of androgens in the pandemic.

Further, according to Dr. Andy Goren: The results of this study further support our research into the use of anti-androgens in the treatment of COVID-19. If randomized prospective trials demonstrate efficacy, anti-androgens may provide a breakthrough inexpensive treatment for COVID-19.

ABOUT APPLIED BIOLOGY

Founded in 2002, Applied Biology, Inc. (www.appliedbiology.com), headquartered in Irvine, California, is a biotechnology company specializing in hair and skin science. Applied Biology develops breakthrough drugs and medical devices for the treatment of androgen mediated dermatological conditions. Applied Biology's R&D pipeline includes a topically applied prophylactic treatment for chemotherapy induced alopecia; a novel diagnostic device that can aid dermatologists in identifying non-responders to topical minoxidil; an adjuvant therapy for non-responders to topical minoxidil; and a novel therapy for female pattern hair loss.

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Applied Biology in Collaboration with Researchers at University Hospital Ramon y Cajal Hospital Announce Results from Study of Androgen Receptor...

Gene Therapy

A report by The Insight Partners on the Global Gene Therapy Market discusses the growth of the market in great detail. It discusses thoroughly all factors promoting and deterring the market. The report also focuses on the competitive dynamics in the market by monitoring the strengths and weaknesses of prominent contributors and their key products.

Gene Therapy provide the ability for remote medical diagnosis and remote medical care in the home, which is particularly important for the growing ageing society, which may require assisted living. Satellite connectivity can also assist in the gathering of data to predict and track disease progression and associated risks of outbreaks.

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Top leading companies operating in the global Gene Therapy market:-

Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure N.V., AveXis, Inc., Vineti, Solid Biosciences., Spark Therapeutics, Inc., CHIMERON BIO, RENOVA THERAPEUTICS, HORAMA S.A.

Valuable Data included in the report:

Our expert team is consistently working on updated data and information of key players related business processes which values the market. For future strategies and predictions, we provide special section regarding covid-19 situation.

Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Gene Therapy Market which would mention How the Covid-19 is Affecting the Disposable Incontinence Products (DIPs) Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Disposable Incontinence Products (DIPs) Players to fight Covid-19 Impact.

Important Queries Addressed in the report:

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The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We are a specialist in Technology, Healthcare, Manufacturing, Automotive and Defense.

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Gene Therapy Market 2020 Demand Analysis, Economic Stability and Investment Opportunity For Expansion 2027 - Crypto Daily

The research report, titled [Global Hemophilia Gene Therapy market 2020 by Manufacturers, Type and Application, Forecast to 2025], presents a detailed analysis of the drivers and restraints impacting the overall market. Analysts have studied the key trends defining the trajectory of the market. The research report also includes an assessment of the achievements made by the players in the global Hemophilia Gene Therapy market so far. It also notes the key trends in the market that are likely to be lucrative. The research report aims to provide an unbiased and a comprehensive outlook of the global Hemophilia Gene Therapy market to the readers.

The Hemophilia Gene Therapy market report offers a comprehensive analysis of this industry vertical, emphasizing on the key growth stimulants, opportunities, and constraints projected to shape the market dynamics over the forecast period.

As per industry experts, the market is anticipated to grow significantly, registering a CAGR of XX% over the analysis period of 2020-2025

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Instabilities in the supply & demand channels due to the stringent lockdown measures imposed to address the COVID-19 pandemic has left numerous companies in disarray. Speaking of the ambiguity of revenues in the near term, businesses are expected to face hindrance even once the economy recovers from the pandemic.

Additionally, the report offers a holistic assessment of various industry segments to infer the revenue projections for the market over the study duration.

Key inclusions of the Hemophilia Gene Therapy market report:

Hemophilia Gene Therapy Market segments covered in the report:

Regional analysis: North America, Europe, Asia-Pacific, South America and Middle East and Africa

Product spectrum: Hemophilia A and Hemophilia B

Applications spectrum: Hemophilia A Gene Therapy and Hemophilia B Gene Therapy

Competitive landscape:

Information including major players operating in the market along with the manufacturing facilities as well as competitors of each participant

Questions Answered by the Report:

What will be the size of the global Hemophilia Gene Therapy market in 2025?

What is the current CAGR of the global Hemophilia Gene Therapy market?

Which product is expected to show the highest market growth?

Which application is projected to gain a lions share of the global Hemophilia Gene Therapy market?

Which region is foretold to create the most number of opportunities in the global Hemophilia Gene Therapy market?

Will there be any changes in market competition during the forecast period?

Which are the top players currently operating in the global Hemophilia Gene Therapy market?

How will the market situation change in the coming years?

What are the common business tactics adopted by players?

What is the growth outlook of the global Hemophilia Gene Therapy market?

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Hemophilia Gene Therapy Market Overview, Environmental Analysis and Forecast to 2025 - Express Journal

A biotech company spun out of the University of Edinburgh will utilise research to develop new gene therapies to target different types of cancer.

The company, Cellinta Limited, was born out of research from scientists at the University of Edinburgh, and received funding through investments from SV Health Investors and Cancer Research UK.

The funding will be used to develop a pipeline of highly selective gene therapies targeting cancer stem cells in a variety of solid tumour types.

It will draw on research led by Professor Steven Pollard at the University of Edinburgh, which delved into stem cell biology, synthetic biology and cancer genomics.

Cellinta has entered into a collaborative research agreement with the University, and retains the option to license intellectual property developed from research conducted there.

Professor Pollard will join the Cellinta team as scientific founder and a senior adviser, while Dr Soraya Bekkali will take the helm as chief executive officer of the new company.

I am delighted to be leading such an exciting company, said Dr Bekkali.

Cellintas approach offers the opportunity to deliver combinations of therapies selectively to cancer stem cells, bypassing the limitations of traditional treatments which often target single genes or redundant signalling pathways, she added.

Meanwhile, Mike Ross managing partner at London-based SV Health Investors has been appointed chairman of Cellinta.

Cellinta represents a unique opportunity to build a leading company based on exciting and innovative science, with the potential to develop first-in-class therapies, said Ross.

We are delighted with the progress the company has made and extremely pleased to appoint Soraya, who has deep experience in gene therapy across various therapeutic areas, as CEO. We look forward supporting Cellinta in its progress as a pioneer in the field, he added.

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University of Edinburgh spinout Cellinta will develop gene therapies for cancer - PharmaTimes

Gene Therapy Market - Snapshot

Introduction of new production, availability of reimbursement together with high occurrences of cancer are estimated to propel growth of the global gene therapy market in the years to come. Gene therapy refers to an experimental technique, which utilizes genes for the prevention and treatment of various diseases. It is expected that in the near future, this technology could assist doctors to place a gene into the cells of a patient for the purpose of his treatment. This therapy could be used as an alternative to surgery or drugs. Scientists are examining various approaches to this therapy, which could comprise

Gene therapy has emerged as a promising treatment option for a large number of diseases such as certain viral infections, certain cancers, and inherited disorders. This factor is likely to work in favor of the global gene therapy market in the years to come.

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Progress in Gene-editing and Genomics Tools to Stoke Demand of Gene Therapy

CAR T-cell use has recently garnered considerable attention from the sponsors following the exhibition of its immense promise in the treatment of several diseases. The promising future of CAR T-cell is estimated to amplify the growth opportunities of the global gene therapy market. Sponsors hail CAR T-cell use as a brand new business model of the future.

In the pipeline of pharmaceutical industry, gene therapy account for a considerable share and this trend is likely to continue in the years to come. In addition, significant advancement has been made in the fields of cellular and molecular biology is likely to fuel growth of the global gene therapy market in the years to come. rapid technological progress made in the gene-editing and genomics tools are further estimated to drive the demand for gene therapy.

Global Gene Therapy Market Snapshot

Expanding at a stellar, double-digits CAGR (Compound Annual Growth Rate) of 40% over the forecast period of 2018 to 2026, the global gene therapy market is a dizzying trajectory, marking out a rosy landscape for players operating in the playfield. As per a Transparency Market Research report, based on extensive primary and secondary research, states that over the period states, the market would accrue a worth of USD 5164.03 million a steep and impressive increase from the USD 17 million worth noted in 2017.

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Fixing defective genes via introduction of new and healthy ones to fix severe genetic and chronic disorders is seeing an upward curve in demand for reasons of improved medical outcomes, major leaps in terms of technological advancement and minimal die-effects as compared to alternatives. Some of the most significant alternatives include surgery, and drug use.

Some of the most significant factors that the analysts of the report note include focused efforts towards marketing and commercialization, and a slew of approvals of new products hitting the global gene therapy market. Additionally, there are factors such as growing demand experienced for this treatment by a large pool of patients.

It is pertinent to note here that the global gene therapy market is consolidated and is dependent on clinincal research and development of the highest standards in order to chart growth. And, some of the players that operate the market landscape, and are into significant research projects include Gilead Sciences, Inc., Novartis AG, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., CELGENE CORPORATION, and Orchard Therapeutics Limited.

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Yescarta to Dominate Global Gene Therapy Market over the Forecast Period

The global gene therapy market report by TMR is segmented based on type, application, and region. The former includes the only five products that have been approved so far for commercial use. These include Gendicine, Yescarta, Strimvelis, Kymriah, and Luxturna. Riding the first mover benefits, Yescarta helf the dominant position in the market in 2017, and the trend will continue, adding more worth to the sub-segment. This is the product that brought out the initial CAR T therapy in the market for large B-cell lymphoma that relapse.

It is worth noting here that as per the global gene therapy market report, the high incidence of DLBCL and massive commercialization efforts directed towards Yescarta, particularly in Europe, will contribute positively and significantly to the overall growth of the global gene therapy market. The other sub-segment to make a mark over the global gene therapy market landscape will be Luxturna, owing to rising awareness levels and massive efforts towards comercialization.

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Europe to be Ahead of the Global Gene Therapy Market Growth Curve over the Forecast Period

The global gene therapy markets regional segmentation includes incisive growth insights into some of the most significant areas that will shape up the overall growth in the market. These include North America, Europe, and Rest of the World. Researchers involved with the preparation of report claim that a massive chunk of about 40% would be accounted for by Europe over the forecast period. Some of the factors backing-up the market dominance of the region include high incidence of non Hodgkin lymphoma and increase in number of treatment centers into gene therapy.

This growth would be followed by North America region, owing to huge contributions from the United States of America which witnesses about 7500 cases of refractory DLBCL each year. These are ones that qualify for the CAR T therapy.

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Gene Therapy Market Progress in Gene-editing and Genomics Tools to Stoke Demand of Gene Therapy - BioSpace

The global Cancer Gene Therapy market study encloses the projection size of the market both in terms of value (Mn/Bn US$) and volume (x units). With bottom-up and top-down approaches, the report predicts the viewpoint of various domestic vendors in the whole market and offers the market size of the Cancer Gene Therapy market. The analysts of the report have performed in-depth primary and secondary research to analyze the key players and their market share. Further, different trusted sources were roped in to gather numbers, subdivisions, revenue and shares.

The research study encompasses fundamental points of the global Cancer Gene Therapy market, from future prospects to the competitive scenario, extensively. The DROT and Porters Five Forces analyses provides a deep explanation of the factors affecting the growth of Cancer Gene Therapy market. The Cancer Gene Therapy market has been broken down into various segments, regions, end-uses and players to provide a clear picture of the present market situation to the readers. In addition, the macro- and microeconomic aspects are also included in the research.

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Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech

Key points covered in the report

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The Cancer Gene Therapy market research covers an exhaustive analysis of the following data:

The Cancer Gene Therapy market research addresses critical questions, such as

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The global Cancer Gene Therapy market research considers region 1 (Country 1, country 2), region 2 (Country 1, country 2) and region 3 (Country 1, country 2) as the important segments. All the recent trends, such as changing consumers demand, ecological conservation, and regulatory standards across different regions are covered in the report.

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Increase in the Adoption of Cancer Gene Therapy to Propel the Growth of the Cancer Gene Therapy Market Between2020 - The Daily Chronicle

Management to Highlight Progress in Bone Marrow Transplant (BMT) and Chimeric Antigen Receptor T Cell (CAR T) Business Collaborations

Tel Aviv, Israel, Sept. 21, 2020 (GLOBE NEWSWIRE) -- Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of innovative technology which enables the functional selection of cells facilitating safer and more efficacious cell and gene therapies, today announced that the leadership will be presenting at the Cell & Gene Meeting on the Mesa, which is being held from October 12th 16th. In addition to providing an overview of the Company, the management team will provide a progress update on the clinical and development programs and an overview of the Companys business strategy. Management will be virtually meeting cell and gene therapy companies to support collaborations.

This is one of the most prestigious meetings for our industry, and despite it being virtual this year due to the COVID-19 pandemic, it will not dampen our enthusiasm commented Dr. Yarkoni. We have successfully adjusted our operations, and we continue to make significant progress with our Israel and U.S.-based clinical trials. We are also taking meaningful steps to accelerate nearer-term revenue opportunities as we are collaborating with several partners that are looking to leverage our technology platform to help improve their products, especially in high-growth areas such as CAR T, NK (natural killers) and MSCs. We are looking forward to sharing the progress we have made and also look forward to meeting with current and potential partners developing cell and gene therapies.

To schedule a meeting with the Companys Chief Executive Officer, Dr. Shai Yarkoni or Chief Operating Officer, Amos Ofer, please request a meeting through the meeting portal and/or contact the Company direct at shai@cellect.co or amoso@cellect.co. The presentation will be available on the Companys website prior to the commencement of the meeting. The Cell & Gene Meeting on the Mesa is the sectors foremost annual conference bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (APOP) has developed a breakthrough technology, for the functional selection of cells that aims to improve the robustness, safety and efficacy of a variety of cell and gene therapies. The Companys technology can be used by researchers, clinical community and pharma companies in a wide variety of applications including next generation Car T, NK, MSC and gene therapies.

The Company is also developing its own product that is an improved BMT which is in a current clinical trial for cancer treatment.

Forward Looking Statements

This press release contains forward-looking statements about the Companys expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as believe, expect, intend, plan, may, should, could, might, seek, target, will, project, forecast, continue or anticipate or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss Cellects expectations regarding timing of the commencement of its planned U.S. clinical trial and its plan to reduce operating costs. These forward-looking statements and their implications are based on the current expectations of the management of the Company only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: the Companys history of losses and needs for additional capital to fund its operations and its inability to obtain additional capital on acceptable terms, or at all; the Companys ability to continue as a going concern; uncertainties of cash flows and inability to meet working capital needs; the Companys ability to obtain regulatory approvals; the Companys ability to obtain favorable pre-clinical and clinical trial results; the Companys technology may not be validated and its methods may not be accepted by the scientific community; difficulties enrolling patients in the Companys clinical trials; the ability to timely source adequate supply of FasL; risks resulting from unforeseen side effects; the Companys ability to establish and maintain strategic partnerships and other corporate collaborations; the scope of protection the Company is able to establish and maintain for intellectual property rights and its ability to operate its business without infringing the intellectual property rights of others; competitive companies, technologies and the Companys industry; unforeseen scientific difficulties may develop with the Companys technology; the Companys ability to retain or attract key employees whose knowledge is essential to the development of its products; and the Companys ability to pursue any strategic transaction or that any transaction, if pursued, will be completed. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading Risk Factors in Cellect Biotechnology Ltd.s Annual Report on Form 20-F for the fiscal year ended December 31, 2019 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SECs website, http://www.sec.gov, and in the Companys periodic filings with the SEC.

ContactCellect Biotechnology Ltd.Eyal Leibovitz, Chief Financial Officerwww.cellect.co+972-9-974-1444

Or

EVC Group LLCMichael Polyviou(732) 933-2754mpolyviou@evcgroup.com

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Cellect Biotechnology to Present at the 2020 Cell & Gene Meeting on the Mesa - GlobeNewswire

Market Study Report, LLC, adds a thorough analysis of the ' Hemophilia Gene Therapy market', offering a comprehensive report emphasizing every vital aspect of the business vertical. The study has collectively presented refined data characterized by market valuation, SWOT analysis, market participants, regional segmentation, and revenue forecasts, enabling stakeholders to make logical business decisions.

The research report on Hemophilia Gene Therapy market comprises of driving factors and trends that will impact the industry growth during the forecast period. Thorough examination of market remuneration with reference to regional terrain is entailed in the report. It also mentions the challenges this business sphere will face as well as provide information regarding potential growth prospects. Besides, the report also includes COVID-19 case studies to deliver a better picture of this business sphere to all industry partakers.

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Pivotal pointers from COVID-19 effect assessment:

Overview of regional terrain:

Other important highlights from the Hemophilia Gene Therapy market report:

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Key features of this report are:

Table of Contents:

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Hemophilia Gene Therapy Market Comprehensive Analysis, Growth Forecast from 2020 to 2025 - AlgosOnline

A 2018 analysis of studies looking for genetic variants associated with disease found that under-representation [of minorities] persists: 78% of study participants were of European ancestry, compared to 10% of Asian ancestry and 2% of African ancestry. Other ancestries each represented less than 1% of the total. Several projects, such as H3Africa, are starting to increase participation of under-represented groups, both among participants and among researchers. Large biobanks assembled in Europe and North America, combining biological samples with health-related data, also set sampling targets to increase diversity.

But even when data from minority groups are available, many researchers discard them. Although there can be valid reasons to restrict analyses to a particular population, discarding such data by default is ethically problematic: it worsens under-representation and negates participants efforts to contribute to research.

Funding agencies have taken steps to improve the diversity of participants who are recruited for studies notably, this has led to better representation of women in clinical trials since the 1990s. But agencies have less control over researchers decisions of what to analyse. Scientists are pulled towards statistical convenience and publishing incentives, which can both conflict with the collective goal of greater equity.

There are good reasons to follow precedent: using standard analytical pipelines reduces development cost and the need for extensive validation and explanation. By omitting data, scientists squander an opportunity to build useful knowledge about minority populations.

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Asians and Blacks dramatically under-represented in medical research, distorting drug therapy effectiveness - Genetic Literacy Project

Xtalks Life Science Webinars

TORONTO (PRWEB) September 21, 2020

The International Organization for Standardization (ISO) has recently issued its eagerly awaited guidance on the transportation of cells for therapeutic use (ISO 21973). The ISO document is meant to provide general requirements and points to consider for transportation service providers, clients and senders to ensure cell quality, safety and efficacy during the transportation process. This webinar will discuss the key recommendations and changes that interested parties should review and evaluate when planning their clinical and commercial distribution strategies for cell and gene therapies. Some of the elements to be discussed are documentation, traceability, validation and qualification activities, temporary storage, as well as cleaning and disinfection.

Join Robert Jones, Vice President, BioServices, Cryoport Systems in a live webinar on Thursday, October 1, 2020 at 11am EDT (4pm BST/UK).

For more information or to register for this event, visit Addressing the Recent ISO 21973 Guidance: Considerations for Cell and Gene Therapy Distribution.

ABOUT CRYOPORT, INC.

Cryoport, Inc. is redefining temperature controlled supply chain support for the life sciences industry by providing a broad platform of temperature-controlled supply chain solutions, serving the Biopharma, Reproductive Medicine, and Animal Health markets. Our mission is to support life and health on earth by providing reliable and comprehensive solutions for the life sciences industry through our advanced technologies, global supply chain network and dedicated scientists, technicians and supporting team of professionals. Through our purpose-built, proprietary Cryoport Express Shippers; Cryoportal information technology; validated Global Logistics Centers; smart and sustainable temperature-controlled logistics solutions; and biostorage/biobanking services, Cryoport serves clients in life sciences research, clinical trials, and product commercializations. We support life-saving advanced cell and gene therapies and deliver vaccines, protein producing materials, and IVF materials in over 100 countries around the world. For more information, visit http://www.cryoport.com or follow @cryoport on Twitter at http://www.twitter.com/cryoport for live updates.

ABOUT XTALKS

Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit http://xtalks.comFor information about hosting a webinar visit http://xtalks.com/why-host-a-webinar/

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Addressing the Recent ISO 21973 Guidance: Considerations for Cell and Gene Therapy Distribution, Upcoming Webinar Hosted by Xtalks - PR Web

DetailsCategory: DNA RNA and CellsPublished on Tuesday, 22 September 2020 10:38Hits: 206

The comprehensive cancer center has also dosed its first patient in chlorotoxin CAR T cell therapy trial

DUARTE, CA, USA I September 21, 2020 I City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, today announced that it has licensed intellectual property relating to its pioneering chlorotoxin chimeric antigen receptor (CLTX-CAR) T cell therapy to Chimeric Therapeutics Limited, an Australian biotechnology company.

The therapy is currently being used in a phase 1 clinical trial at City of Hope to treat glioblastoma (GBM), a type of brain tumor. The first patient in the trial was recently dosed; Behnam Badie, M.D., chief of City of Hopes Division of Neurosurgery and The Heritage Provider Network Professor in Gene Therapy, is leading this innovative, first-of-its-kind trial.

Chimeric has acquired the exclusive worldwide rights to develop and commercialize certain patents relating to City of Hopes CLTX-CAR T cells, as well as to further develop the therapy for other cancers.

City of Hope is excited to enter into this agreement with Chimeric as it supports our innovative research in CAR T cell therapy and our commitment to extend these therapies to more patients, particularly those with GBM and other solid tumors that are difficult to treat, said Christine Brown, Ph.D., The Heritage Provider Network Professor in Immunotherapy and deputy director of City of Hopes T Cell Therapeutics Research Laboratory. Chimeric shares our goal of providing effective CAR T cell therapies to more patients with current unmet medical needs.

Led by Brown and Michael Barish, Ph.D., chair of City of Hopes Department of Developmental and Stem Cell Biology, and Dongrui Wang, Ph.D., a recent graduate of City of Hopes Irell & Manella Graduate School of Biological Sciences, the team developed and tested the first CAR T cell therapy using CLTX, a component of scorpion venom, to direct T cells to target brain tumor cells. The research was published this past March in Science Translational Medicine.

Chimeric is excited to join City of Hope in its quest to find more effective cancer therapies. This is an exceedingly rare opportunity to acquire a promising technology in one of the most exciting areas of immuno-oncology today, said Paul Hopper, executive chairman of Chimeric. Furthermore, the CLTX-CAR T cell therapy has completed years of preclinical research and development, and recently enrolled its first patient in a phase 1 clinical trial for brain cancer.

CARs commonly incorporate a monoclonal antibody sequence in their targeting domain, enabling CAR T cells to recognize antigens and kill tumor cells. In contrast, the CLTX-CAR uses a synthetic 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain.

In this recent study, City of Hope researchers used tumor cells in resection samples from a cohort of patients with GBM to compare CLTX binding with expression of antigens currently under investigation as CAR T cell targets. They found that CLTX bound to a greater proportion of patient tumors, and cells within these tumors.

CLTX binding included the GBM stem-like cells thought to seed tumor recurrence. Consistent with these observations, CLTX-CAR T cells recognized and killed broad populations of GBM cells while ignoring nontumor cells in the brain and other organs. The study team demonstrated that CLTX-directed CAR T cells are highly effective at selectively killing human GBM cells without off-tumor targeting and toxicity in cell-based assays and in animal models.

City of Hope, a recognized leader in CAR T cell therapies for GBM and other cancers, has treated more than 500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has 30 ongoing CAR T cell clinical trials, including CAR T cell trials for HER-2 positive breast cancer that has spread to the brain, and PSCA-positive bone metastatic prostate cancer. It was the first and only cancer center to treat GBM patients with CAR T cells targeting IL13R2, and the first to administer CAR T cell therapy locally in the brain, either by direct injection at the tumor site, through intraventricular infusion into the cerebrospinal fluid, or both. In late 2019, City of Hope opened a first-in-human clinical trial for patients with recurrent GBM, combining IL13R2-CAR T cells with checkpoint inhibitors nivolumab, an anti-PD1 antibody, and ipilimumab, blocking the CTLA-4 protein.

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"City of Hope is delighted to license this technology to Chimeric, said Sangeeta Bardhan Cook, Ph.D., City of Hope director of the Office of Technology Licensing. We are impressed with the ability of their executive team to push and bring therapies to market expeditiously. At City of Hope, our mission is to transform the future of health care. We believe Chimeric has the vision to offer innovative therapies to cancer patients.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nations Best Hospitals in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

SOURCE: City of Hope

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City of Hope Enters Licensing Agreement With Chimeric to Develop Its Pioneering Chlorotoxin CAR T Cell Therapy | DNA RNA and Cells | News Channels -...

The global CNS Gene Therapy market study encloses the projection size of the market both in terms of value (Mn/Bn US$) and volume (x units). With bottom-up and top-down approaches, the report predicts the viewpoint of various domestic vendors in the whole market and offers the market size of the CNS Gene Therapy market. The analysts of the report have performed in-depth primary and secondary research to analyze the key players and their market share. Further, different trusted sources were roped in to gather numbers, subdivisions, revenue and shares.

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CNS Gene Therapy Market Is Likely To Attain A Market Value Of US$ XX Mn/Bn In 2018 2028 - The Daily Chronicle

City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, today announced that it has licensed intellectual property relating to its pioneering chlorotoxin chimeric antigen receptor (CLTX-CAR) T cell therapy to Chimeric Therapeutics Limited, an Australian biotechnology company.

The therapy is currently being used in a phase 1 clinical trial at City of Hope to treat glioblastoma (GBM), a type of brain tumor. The first patient in the trial was recently dosed; Behnam Badie, M.D., chief of City of Hopes Division of Neurosurgery and The Heritage Provider Network Professor in Gene Therapy, is leading this innovative, first-of-its-kind trial.

Chimeric has acquired the exclusive worldwide rights to develop and commercialize certain patents relating to City of Hopes CLTX-CAR T cells, as well as to further develop the therapy for other cancers.

City of Hope is excited to enter into this agreement with Chimeric as it supports our innovative research in CAR T cell therapy and our commitment to extend these therapies to more patients, particularly those with GBM and other solid tumors that are difficult to treat, said Christine Brown, Ph.D., The Heritage Provider Network Professor in Immunotherapy and deputy director of City of Hopes T Cell Therapeutics Research Laboratory. Chimeric shares our goal of providing effective CAR T cell therapies to more patients with current unmet medical needs.

Led by Brown and Michael Barish, Ph.D., chair of City of Hopes Department of Developmental and Stem Cell Biology, and Dongrui Wang, Ph.D., a recent graduate of City of Hopes Irell & Manella Graduate School of Biological Sciences, the team developed and tested the first CAR T cell therapy using CLTX, a component of scorpion venom, to direct T cells to target brain tumor cells. The research was published this past March in Science Translational Medicine.

Chimeric is excited to join City of Hope in its quest to find more effective cancer therapies. This is an exceedingly rare opportunity to acquire a promising technology in one of the most exciting areas of immuno-oncology today, said Paul Hopper, executive chairman of Chimeric.

Furthermore, the CLTX-CAR T cell therapy has completed years of preclinical research and development, and recently enrolled its first patient in a phase 1 clinical trial for brain cancer.CARs commonly incorporate a monoclonal antibody sequence in their targeting domain, enabling CAR T cells to recognize antigens and kill tumor cells. In contrast, the CLTX-CAR uses a synthetic 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain.

In this recent study, City of Hope researchers used tumor cells in resection samples from a cohort of patients with GBM to compare CLTX binding with expression of antigens currently under investigation as CAR T cell targets. They found that CLTX bound to a greater proportion of patient tumors, and cells within these tumors.

CLTX binding included the GBM stem-like cells thought to seed tumor recurrence. Consistent with these observations, CLTX-CAR T cells recognized and killed broad populations of GBM cells while ignoring nontumor cells in the brain and other organs. The study team demonstrated that CLTX-directed CAR T cells are highly effective at selectively killing human GBM cells without off-tumor targeting and toxicity in cell-based assays and in animal models.

City of Hope, a recognized leader in CAR T cell therapies for GBM and other cancers, has treated more than 500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has 30 ongoing CAR T cell clinical trials, including CAR T cell trials for HER-2 positive breast cancer that has spread to the brain, and PSCA-positive bone metastatic prostate cancer. It was the first and only cancer center to treat GBM patients with CAR T cells targeting IL13R2, and the first to administer CAR T cell therapy locally in the brain, either by direct injection at the tumor site, through intraventricular infusion into the cerebrospinal fluid, or both. In late 2019, City of Hope opened a first-in-human clinical trial for patients with recurrent GBM, combining IL13R2-CAR T cells with checkpoint inhibitors nivolumab, an anti-PD1 antibody, and ipilimumab, blocking the CTLA-4 protein.

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"City of Hope is delighted to license this technology to Chimeric, said Sangeeta Bardhan Cook, Ph.D., City of Hope director of the Office of Technology Licensing. We are impressed with the ability of their executive team to push and bring therapies to market expeditiously. At City of Hope, our mission is to transform the future of health care. We believe Chimeric has the vision to offer innovative therapies to cancer patients.

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City of Hope enters licensing agreement with Chimeric to develop its pioneering chlorotoxin CAR T cell therapy - OncLive

Cell and Gene Therapy Market Research Report is a Proficient and In-Depth Study on the Existing State of Cell and Gene Therapy Industry. This Report Focuses on the Major Drivers, Restraints, Opportunities and Threats for Key Players. It also Provides Granular Analysis of Market Share, Segmentation, Revenue Forecasts and Regional Analysis till 2025.

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Cell and Gene Therapy Market Report 2020 Industry Capacity, Manufactur - News by aeresearch

LONDONandNEW YORK, Sept. 22, 2020 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc(Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced nine-month results from the ongoing Phase 1/2 clinical trial(NCT03252847) of AAV-RPGR, an investigational gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP), will be presented in an oral session at the EURETINA 2020 Virtual Meeting taking place October 2-4, 2020.

Details of the presentation are listed below. Data is embargoed until the date and time of presentation.

Title: Phase 1/2 Clinical Trial of AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 9-month ResultsPresenter: Michel Michaelides, BSc MB BS MD(Res) FRCOphth FACSDate and Time: Saturday, October 3, 10:45am EDT (4:45pm CEST)Session: EURETINA Session 11: Late Breaking & Reviews

MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

In July 2020, MeiraGTx announced six-month data from the ongoing Phase 1/2 MGT009 clinical trial, which demonstrated AAV-RPGR was generally well tolerated and produced significant improvement in vision in the dose escalation phase of the trial.

About AAV-RPGRAAV-RPGR is an investigational gene therapy for the treatment of patients with X-Linked Retinitis Pigmentosa (XLRP) caused by mutations in the eye specific form of theRPGRgene (RPGRORF15). AAV-RPGR is designed to deliver functional copies of theRPGRgene to the subretinal space in order to improve and preserve visual function.MeiraGTxand development partner Janssen are currently conducting a Phase 1/2 clinical trial of AAV-RPGR in patients with XLRP with mutations inRPGRORF15. AAV-RPGR has been granted Fast Track and Orphan Drug designations by theU.S. Food and Drug Administration(FDA) and PRIME, ATMP and Orphan designations by theEuropean Medicines Agency (EMA).

About the Phase 1/2 MGT009 Clinical TrialMGT009 is a multi-center, open-label Phase 1/2 trial (NCT03252847) of AAV-RPGR gene therapy for the treatment of patients with XLRP associated with disease-causing variants in theRPGRgene. MGT009 consists of three phases: dose-escalation, dose-confirmation, and dose-expansion. Each patient was treated with subretinal delivery of AAV-RPGR in the eye that was more affected at baseline. The patients other eye served as an untreated control. In dose-escalation (n=10), adults were administered low, intermediate, or high dose AAV-RPGR. The primary endpoint was safety. Visual function was assessed at baseline, three, six, nine and 12 months with Octopus 900 full-field static perimetry and mesopic fundus-guided microperimetry (MP); mean retinal sensitivity, visual field modeling and analysis (VFMA; Hill-of-vision volumetric measure), and pointwise comparisons were examined.

About X-Linked Retinitis Pigmentosa (XLRP)XLRP is the most severe form of retinitis pigmentosa (RP), a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. The most frequent cause of XLRP is disease-causing variants in theRPGRgene, accounting for more than 70% of cases of XLRP, and up to 20% of all cases of RP. There are currently no approved treatments for XLRP.

AboutMeiraGTxMeiraGTx(Nasdaq: MGTX) is a vertically integrated, clinical stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs.MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team,MeiraGTxhas taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTxs initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system and salivary gland,MeiraGTxintends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

For more information, please visitwww.meiragtx.com.

Forward Looking StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the development and efficacy of AAV-RPGR, plans to advance AAV-RPGR into Phase 3 clinical trial and anticipated milestones regarding our clinical data and reporting of such data and the timing of results of data, including in light of the COVID-19 pandemic, as well as statements that include the words expect, intend, plan, believe, project, forecast, estimate, may, should, anticipate and similar statements of a future or forward-looking nature. These forward-looking statements are based on managements current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, acquire additional capital, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID-19 pandemic on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter endedMarch 31, 2020, as such factors may be updated from time to time in our other filings with theSEC, which are accessible on the SECs website atwww.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Contacts

Investors:MeiraGTxElizabeth (Broder) Anderson (646) 860-7983elizabeth@meiragtx.com

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Media:W2O pureChristiana Pascale(212) 257-6722cpascale@purecommunications.com

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MeiraGTx Announces Data from Ongoing Clinical Trial of AAV-RPGR for the Treatment of X-Linked Retinitis Pigmentosa to be Presented at EURETINA 2020...

HEIDELBERG, Germany, Sept. 21, 2020 /PRNewswire/ -- GeneWerk GmbH, a cell and gene therapy testing laboratory focused on providing preclinical and clinical trial patient sample analysis services, today announced a majority investment from Ampersand Capital Partners, a private equity firm specializing in growth equity investments in the healthcare sector. Ampersand's growth investment will be used to expand GeneWerk's capabilities to meet rapidly growing demand for cell and gene therapy testing services.

GeneWerk provides cell and gene therapy sponsors with patient testing services in compliance with guidance by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The company is recognized as a leading provider of vector integration site analysis (ISA) services, a method that was developed by GeneWerk's founders and that the FDA and EMA recommend performing after the administration of both integrating and non-integrating cell and gene therapies. The company's test menu also includes vector persistence testing, gene edited off-target analysis, vector copy number (VCN), vector quality control, immune repertoire analysis, and dedicated bioinformatics studies. With a focus on vector safety, characterization, and functionality analysis, the company's 30 employees work in compliance with GCP and in line with GLP standards in a BSL-2 classified state-of-the-art genomics and bioinformatics laboratory in Heidelberg, Germany.

Annette Deichmann, Ph.D., Co-Founder and Co-CEO at GeneWerk commented, "With the benefit of Ampersand as our partner, GeneWerk will strengthen and expand its position in the U.S. and European markets while further investing in our testing capabilities to service cell and gene therapy sponsors and patients." Co-Founder and Co-CEO Manfred Schmidt, Ph.D., then added, "Our partnership with Ampersand solidifies GeneWerk's position in the space and will allow the company to continue to exceed our customers' expectations by facilitating the development of innovative cell and gene therapies. We are very pleased to have Ampersand on board as we take GeneWerk through its next phase of growth." Christof von Kalle, M.D., Co-Founder concluded, "This will greatly further GeneWerk's opportunities to contribute to medical breakthroughs and patient safety."

Marina Pellon-Consunji, Principal at Ampersand said, "GeneWerk is a leading company in its field. Given the exciting developments within the cell and gene therapy market and recent guidance by FDA and EMA, this is an excellent time for an investor with deep experience in cell and gene therapy to partner with the company. We are looking forward to working with the team at GeneWerk to accelerate and continue its success in delivering leading testing services to ensure the safety of patients receiving cutting edge cell and gene therapies."

About GeneWerk GmbHFounded in 2014 by Prof. Dr. Christof von Kalle, Dr. Manfred Schmidt, and Dr. Annette Deichmann with the participation of the German Cancer Research Center (DKFZ) Heidelberg, GeneWerk is a cell and gene therapy testing laboratory focused on providing preclinical and clinical trial patient sample analysis services. The company is recognized as a leading provider of vector integration site analysis (ISA) services, a method that was developed by the company's founders and that the FDA and EMA recommend performing after the administration of both integrating and non-integrating cell and gene therapies. For more information, please visit http://www.genewerk.com.

About Ampersand Capital PartnersFounded in 1988, Ampersand is a middle market private equity firm dedicated to growth-oriented investments in the healthcare sector. With offices in Boston and Amsterdam, Ampersand leverages its unique blend of private equity and operating experience to build value and drive superior long-term performance alongside its portfolio company management teams. Ampersand has helped build numerous market-leading companies across each of the firm's core healthcare sectors. Additional information about Ampersand is available at http://www.ampersandcapital.com.

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GeneWerk GmbH Announces Growth Equity Investment from Ampersand Capital Partners - PRNewswire

DetailsCategory: AntibodiesPublished on Tuesday, 22 September 2020 10:41Hits: 257

Adjuvant Opdivo doubled disease-free survival; is the first therapeutic option to show statistically significant and clinically meaningful disease-free survival benefit in these patients, regardless of tumor histology, following chemoradiation therapy and resection

Results from Phase 3 CheckMate -577 trial selected for presentation during a Presidential Symposium at the European Society for Medical Oncology Virtual Congress 2020

PRINCETON, NJ, USA I September 21, 2020 I Bristol Myers Squibb (NYSE: BMY) today announced first results from the Phase 3 CheckMate -577 trial in which adjuvant treatment with Opdivo (nivolumab) showed a statistically significant and clinically meaningful improvement in disease-free survival (DFS), the trials primary endpoint, compared to placebo in patients with esophageal or gastroesophageal junction (GEJ) cancer following neoadjuvant chemoradiation therapy (CRT) and tumor resection. The current standard of care for patients with esophageal or GEJ cancer following neoadjuvant CRT and tumor resection is surveillance. These results signify the first time an adjuvant therapeutic option has significantly prolonged DFS for patients in this setting.

Median DFS was doubled in patients receiving Opdivo [22.4 months; (95% Confidence Interval [CI]: 16.6 to 34.0)] compared to those receiving placebo after surgery [11.0 months; (95% CI: 8.3 to 14.3)] (Hazard Ratio [HR] 0.69; 96.4% CI: 0.56 to 0.86; p=0.0003). The median duration of treatment for patients in the Opdivo arm was just over 10 months [10.1 months (<0.1 to 14.2)] versus nine months for patients in the placebo arm [9.0 months (<0.1 to 15)]. The safety profile of Opdivo in CheckMate -577 was consistent with previously reported studies of Opdivo monotherapy.

While about 25% to 30% of patients with esophageal or gastroesophageal junction cancer achieve a complete response following chemoradiation therapy and surgery, the remaining 70% to 75% do not, and there is currently no adjuvant treatment option available for these patients with the potential to improve their outcomes, said Ronan J. Kelly M.D., MBA, Director, Charles A. Sammons Cancer Center at Baylor University Medical Center. Adjuvant treatment with nivolumab in the CheckMate -577 trial doubled patients time without disease recurrence, representing the first adjuvant treatment advancement for these patients with esophageal or gastroesophageal junction cancer.

Opdivo was well tolerated with an acceptable safety profile relative to placebo. The majority of patients in the Opdivo arm (89%) were able to receive a relative dose intensity of 90%. The incidence of any treatment-related adverse events (TRAEs), including any grade and Grade 3-4, was 71% and 13% among patients treated with Opdivo compared to 46% and 6% among patients receiving placebo. Serious TRAEs of any grade and Grade 3-4 occurred in less than 10% of patients treated with Opdivo (any grade in 8%, Grade 3-4 in 5%) compared to 3% and 1% of patients receiving placebo, with a low rate of any grade treatment-related discontinuations in both arms (9% for Opdivo vs. 3% in placebo).

These results make esophageal and gastroesophageal junction cancer the second cancer type following melanoma where Opdivo has demonstrated a benefit in the adjuvant setting, indicating the potential for Opdivo to become a new standard of care for these patients, said Ian M. Waxman, M.D., development lead, Gastrointestinal Cancers, Bristol Myers Squibb. This advancement showcases our commitment to evaluating our therapies in earlier stages of disease where we may be able to have a greater impact on preventing disease recurrence and improving patient outcomes. We look forward to discussing these encouraging results from CheckMate -577 with global health authorities in the coming months.

These data (Presentation #LBA9) will be featured in a Presidential Symposium at the European Society for Medical Oncology (ESMO) Virtual Congress 2020 on September 21 from 19:31-19:43 CEST.

About CheckMate -577

CheckMate -577 is a Phase 3 randomized, multi-center, double-blind study evaluating Opdivo as an adjuvant therapy in patients with resected esophageal or GEJ cancer who have received neoadjuvant CRT therapy and have not achieved a pathological complete response. The primary endpoint of the trial is DFS and the secondary endpoint is overall survival (OS). Following neoadjuvant CRT therapy and complete tumor surgical resection (also known as trimodality therapy), a total of 794 patients were randomized to receive placebo (n=262) or Opdivo (n=532) 240 mg by intravenous infusion every two weeks for 16 weeks followed by Opdivo 480 mg every four weeks until disease recurrence, unacceptable toxicity or withdrawal of consent, with a maximum of one year total treatment duration.

About Esophageal Cancer

Esophageal cancer is the seventh most common cancer and the sixth leading cause of death from cancer worldwide, with approximately 572,000 new cases and over 508,000 deaths in 2018. The two most common types of esophageal cancer are squamous cell carcinoma and adenocarcinoma, which account for approximately 85% and 15% of all esophageal cancers, respectively, though esophageal tumor histology can vary by region with the highest rate of esophageal adenocarcinoma occurring in North America (65%). The majority of cases are diagnosed in the advanced setting and impact a patients daily life, including their ability to eat and drink.

About Gastric Cancer

Gastric cancer, also known as stomach cancer, is the fifth most common cancer and the third leading cause of cancer death worldwide, with over 1,000,000 new cases and approximately 783,000 deaths in 2018. There are several cancers that can be classified as gastric cancer, including certain types of cancers that form in the GEJ, the area of the digestive tract where the esophagus and stomach connect. While GEJ cancer has a lower prevalence than gastric cancer, it continues to rise.

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early- to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

About Opdivo

Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the bodys own immune system to help restore anti-tumor immune response. By harnessing the bodys own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

Opdivos leading global development program is based on Bristol Myers Squibbs scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.

In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Companys Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.

INDICATIONS

OPDIVO (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

OPDIVO (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.

OPDIVO (nivolumab) is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with progression after platinum-based chemotherapy and at least one other line of therapy. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC).

OPDIVO (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.

OPDIVO (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

OPDIVO (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.

OPDIVO (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.

CheckMate Trials and Patient Populations

Checkmate 037previously treated metastatic melanoma; Checkmate 066previously untreated metastatic melanoma; Checkmate 067previously untreated metastatic melanoma, as a single agent or in combination with YERVOY; Checkmate 227previously untreated metastatic non-small cell lung cancer, in combination with YERVOY; Checkmate 9LApreviously untreated recurrent or metastatic non-small cell lung cancer in combination with YERVOY and 2 cycles of platinum-doublet chemotherapy by histology; Checkmate 017second-line treatment of metastatic squamous non-small cell lung cancer; Checkmate 057second-line treatment of metastatic non-squamous non-small cell lung cancer; Checkmate 032small cell lung cancer; Checkmate 025previously treated renal cell carcinoma; Checkmate 214previously untreated renal cell carcinoma, in combination with YERVOY; Checkmate 205/039classical Hodgkin lymphoma; Checkmate 141recurrent or metastatic squamous cell carcinoma of the head and neck; Checkmate 275urothelial carcinoma; Checkmate 142MSI-H or dMMR metastatic colorectal cancer, as a single agent or in combination with YERVOY; Checkmate 040hepatocellular carcinoma, as a single agent or in combination with YERVOY; Checkmate 238adjuvant treatment of melanoma; Attraction-3esophageal squamous cell carcinoma

About the Bristol Myers Squibb and Ono Pharmaceutical Collaboration

In 2011, through a collaboration agreement with Ono Pharmaceutical Co., Bristol Myers Squibb expanded its territorial rights to develop and commercialize Opdivo globally, except in Japan, South Korea and Taiwan, where Ono had retained all rights to the compound at the time. On July 23, 2014, Ono and Bristol Myers Squibb further expanded the companies strategic collaboration agreement to jointly develop and commercialize multiple immunotherapies as single agents and combination regimens for patients with cancer in Japan, South Korea and Taiwan.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

SOURCE: Bristol-Myers Squibb

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Opdivo (nivolumab) Demonstrated Superior Disease-Free Survival in Patients with Resected Esophageal or Gastroesophageal Junction Cancer Compared to...

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported that statistical analysis of pooled data from LUMEVOQ trials and natural history studies found a statistically significant and clinically meaningful difference between the visual outcomes in LUMEVOQ-treated patients and untreated patients.

Treated eyes showed progressive and sustained improvement from Month 12 to Month 52, in contrast to the absence of recovery over the same period for untreated eyes. At Month 18, the difference became statistically significant (p=0.01). By Month 48, the difference between the mean visual acuity in treated patients and that in untreated patients was both statistically significant (p<0.01) and clinically meaningful (-0.33 LogMAR, or +16.5 ETDRS letters equivalent, in favor of treated eyes).

Note: All patients had a confirmed G11778A mutation in the ND4 mitochondrial gene and were at least 15 years old. The diagram shows the Locally Estimated Scatterplot Smoothing (LOESS) curves for visual acuity in LUMEVOQ-treated patients and untreated patients. The shaded areas represent the 95% confidence interval for the mean BCVA. Treated eyes refer to all eyes (LUMEVOQ and sham) from the RESCUE, REVERSE and CLIN06 trials (N=76 patients / 152 eyes). Untreated eyes refer to patient-level data from the REALITY study and a matched data set from two prospective and eight retrospective natural history studies1 (N=208 patients / 408 eyes). LOESS curves were estimated using a non-parametric, local regression model that treated each eye as independent of the other. LOESS curves are shown from Month 12 to depict post-treatment progression among treated patients (93% of LUMEVOQ patients had already been treated within 12 months from onset). *Statistically significant difference between mean visual acuity of treated and untreated eyes at M18, M24, M36 and M48, as illustrated by the non-overlapping confidence intervals.

The analysis compared data from the completed Phase III trials RESCUE and REVERSE studies and interim results from the long-term follow-up CLIN06 study to a matched sample created from the REALITY registry study and 10 other natural history studies1. The natural history studies were identified from an extensive review of the scientific literature and selected based on specific inclusion criteria for their patient-level data. In all, the visual outcomes in 76 treated patients could be compared to the visual outcomes of 208 untreated patients.

The extra granularity of this analysis down to individual patient data confirms that the natural history of visual outcomes in ND4-LHON patients is poor and provides the best comparison we have for assessing the therapeutic efficacy of gene therapy, commented Dr. Nancy J. Newman, MD, LeoDelle Jolley Professor of Ophthalmology and Neurology at the Emory University School of Medicine in Atlanta, GA, USA, and a global authority on LHON who recently completed a meta-analysis of the natural history of ND4-LHON.2

Separate analyses of patients enrolled in RESCUE and REVERSE demonstrated similarly favorable results compared to untreated patients. Full findings from the indirect comparison were included in the European Marketing Authorisation Application (MAA) for LUMEVOQ and are being prepared for publication in a peer-reviewed journal.

This indirect comparison represents a significant contribution to our understanding of LUMEVOQs therapeutic effect," said Dr. Jos-Alain Sahel, MD, Director of the Institut de la Vision (Sorbonne-Universit/Inserm/CNRS), Paris, France; Chairman of the Department of Ophthalmology at Centre Hospitalier National dOphtalmologie des XV-XX, Paris, France; Professor and Chairman of the Department of Ophthalmology at University of Pittsburgh School of Medicine and UPMC (University of Pittsburgh Medical Center), USA; and Co-Founder of GenSight Biologics. The use of a large external control group, including analyses of patient-level data, provides more evidence for a significant treatment-related visual improvement.

The findings are a gratifying outcome of our push to overcome a key challenge for assessing LUMEVOQs efficacy, namely the inability of sham eyes to act as a control group, commented Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. We are excited to take this evidence that LUMEVOQ modifies the disease outcome forward into our conversations with national and regional authorities.

The LUMEVOQ MAA was filed in September, and the decision is expected in H2 2021. The Company is also working towards submitting LUMEVOQs Biologics License Application (BLA) to the FDA in H2 2021.

GenSight will host a conference call today, September 21, 2020, at 10am CEST in French, and at 2.00pm CEST (8.00am EST) in English, to discuss these results.

Webcast & Conference call in French (10am CEST)

Dial-in numbers:

United States: +1 212 999 6659France: +33 (0)1 7037 7166United Kingdom: +44 (0)20 3003 2666Password: GenSight FR

Webcast link: https://bit.ly/3mAS0Vy

Webcast & Conference call in English (2.00pm CEST / 8.00am EST)

Dial-in numbers:

United States: +1 212 999 6659France: +33 (0) 1 7037 7166United Kingdom: +44 (0) 20 3003 2666Password: GenSight ENG

Webcast link: https://bit.ly/35RCfnl

A replay of the calls and webcasts will be available by using the above links.

Rationale for the Indirect Comparison

In both the REVERSE and RESCUE studies, as well as in the long-term follow-up study CLIN06, unilaterally injected patients experienced an unexpected visual improvement in their contralateral eye, which mirrored the sustained and clinically relevant gain in eyes treated with LUMEVOQ. This bilateral improvement eliminated the control group that was to consist of sham eyes in the original trial design. An indirect comparison methodology, based on formal statistical methods applied to an external control group, was needed to assess the magnitude of LUMEVOQ efficacy.

Methodology Highlights

The sample of LUMEVOQ-treated patients included all data from the two pivotal studies REVERSE and RESCUE and Year 3 data from the ongoing extension study CLIN06. This approach yielded a treated patient pool consisting of 76 patients (152 eyes), with LUMEVOQ-injected eyes and sham-treated eyes considered equivalent, based on the contralateral effect demonstrated in the studies.

The external control group included data from the REALITY Natural History registry and patient-level data from 10 published articles1 on ND4-LHON, which were identified from a systematic review of the literature. ND4-LHON studies were included in the indirect comparison only if they had individual patient data that would allow indirect comparison with LUMEVOQ-treated patients: confirmed ND4 genotype, at least 15 years of age, at least one BCVA measurement with associated time of vision loss. The final external control group included 208 patients (408 eyes).

The visual acuity curves of treated and untreated patients were defined using a Locally Estimated Scatterplot Smoothing (LOESS), non-parametric, local regression model. They were aligned in terms of time from vision loss for the statistical tests performed at M12, M18, M24, M36 and M48 from vision loss and at the last available observation. The starting time point for the statistical tests (12 months post-vision loss) was defined to enable assessment of LUMEVOQ efficacy, as 93% of LUMEVOQ patients were treated by that time.

1The 10 studies that passed the inclusion criteria were: Hotta 1995, Lam 2014, Nakamura 1993, Newman 1991, Qu 2007, Qu 2009, Romero 2014, Sadun 2004, Yang 2016, and Zhou 2010.

2Newman NJ, Carelli V, Taiel M and Yu-Wai Man P. Visual outcomes in Leber hereditary optic neuropathy patients with the m.11778G>A (MTDN4) mitochondrial DNA mutation. J Neuro-Ophthalmol. In Press. 2020.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQ (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About LUMEVOQ (GS010)

LUMEVOQ (GS010) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,400 to 1,500 new patients who lose their sight every year in the United States and Europe.

About RESCUE and REVERSE

RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint measures the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, will be used for statistical purposes. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.

The secondary endpoints involve the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis was evaluated, including the proportion of patients who maintain vision (< ETDRS 15L loss), the proportion of patients who gain 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, biodissemination and the time course of immune response. Readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials were conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were transferred to a long-term follow-up study that will last for three years.

ClinicalTrials.gov Identifiers:REVERSE: NCT02652780RESCUE: NCT02652767

About CLIN06 (RESCUE and REVERSE Long-term Follow-up)

CLIN06 is the long-term follow-up study of ND4 LHON subjects treated with LUMEVOQ (GS010) gene therapy in the RESCUE or REVERSE Phase III Clinical Trials. The total study period for an individual subject is 3 years, i.e., 5 years post-gene therapy administration. No study treatment is administered during CLIN06.

The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment. The secondary objective is to assess the long-term treatment efficacy of the therapy and the quality of life (QoL) in subjects up to 5 years post-treatment. The first subject was enrolled on January 9, 2018. 61 subjects have enrolled.

ClinicalTrials.gov Identifiers:CLIN06: NCT03406104

About REALITY

REALITY is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on subjects medical charts and the administration of surveys on Health-Related Quality of Life (HRQoL) and direct and indirect costs associated with the disease.

The study aimed to recruit at least 50 subjects (both adult and pediatric) chiefly in the following countries: Spain, Italy, France, United Kingdom and the United States.

The primary objectives for the REALITY study were: to describe the evolution of visual functional and structural changes and other associated symptoms in patients with LHON; understand the impact of LHON-related vision loss on the HRQoL; and understand the economic burden for patients and their families arising from direct and indirect costs associated with the disease. The secondary objective is to describe the relationship between genetic, lifestyle and/or environmental factors and the expression of the LHON phenotype.

The first subject was enrolled on 3 January 2018. Enrollment was completed in early Q2 2020.

ClinicalTrials.gov Identifiers:REALITY LHON Registry: NCT03295071

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GenSight Biologics Reports New Analysis Demonstrating Statistically Significant and Clinically Meaningful Difference Between Visual Outcomes in...

Garner Insights has added a new report titled, Global Gene Therapy Market Professional Report 2026 to its vast repository of research reports. This is a thorough report focused on the current and future prospects of the Global Gene Therapy Market. The report offers data of previous years along with an in-depth analysis from 2020 to 2026 on the basis of revenue (USD Billion). Besides, the report offers a comprehensive analysis about the factors driving and restraining the growth of the market coupled with the impact they have on the demand over the forecast period.

The market is segmented into different sections such as: by product type, by technology type, by application, by end-users, by deployment mode, and by key geography. The report then employs market breakdown and data triangulation procedures to complete the overall market engineering process and arrive at the exact statistics for all segments and sub-segments. The report on the Global Gene Therapy Markethas been curated by analyzing the top players functioning in the market. In order to get an in-depth analysis of the market, the report carried out SWOT analysis, Porters five forces analysis, and Pestel analysis.

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This report forecasts revenue growth at the global, regional, and local levels and provides an analysis of the most recent industry trends from 2020 to 2026 in each of the segments and sub-segments.In addition, the report highlights the impact of COVID-19 on the Global Gene Therapy Market. Some of the major geographies included in the market are given below:

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Impact of Covid-19 on Gene Therapy Market Trends by Countries, Type and Application | Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche...

New York, Sept. 22, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Autologous Stem Cell and Non-Stem Cell Based Therapies Market - Growth, Trends, and Forecast (2020 - 2025)" - https://www.reportlinker.com/p05974135/?utm_source=GNW

- Stem cell technology is found to be a speedily developing technology which plays a major role in regenerative medicine, as it also serves the disciplines of tissue engineering, developmental cell biology, cellular therapeutics, gene therapy, chemical biology, and nanotechnology. Stem cells offer the possibility of replacing the cells and tissues to treat various conditions including spinal cord injury, arthritis, and Parkinsons disease, among others.- The applications of stem cell technologies in the treatment of diseases have ultimately increased the overall adoption rate of these technologies across the world.- The advantage of autologous stem cell transplant is that one is getting ones own cells back. This means there is no risk that the immune system of the individual will reject the transplant or that the transplanted cells will attack or reject the individuals body.

Key Market TrendsCancer Holds Significant Share in the Autologous Stem Cell and Non-Stem Cell Based Therapies Market

- Cancer rates could further increase by 50%, to 15 million new cases by 2020, as per World Cancer Report. It also provides clear evidence that healthy lifestyles and public health action by governments and health practitioners could stem this trend, and prevent as many as one-third of cancers, worldwide.- The American Cancer Society, the leading body in cancer stats and figures, reports 1,685,210 estimated new cancer cases and 595,690 deaths due to cancer in 2016, in the United States.- The National Cancer Institute reports that more than 60% of the worlds new cancer cases occur in Africa, Asia, and Central and South America; 70% of the worlds cancer deaths also occur in these regions. The International Agency for Research on Cancer (IARC) predicts that by 2030, the global burden is expected to rise to 21.7 million new cancer cases and 13 million cancer deaths, simply due to growth and aging of population, leaving aside factors, such as smoking, poor diet, physical inactivity, and fewer childbirths in economically developing countries.- According to WHO, almost 70% of deaths from cancer occur in low and middle-income countries, and only one in five low- and middle-income countries has the necessary data to drive cancer policy. This global and extensive threat of cancer remains a major market driver for new cancer therapies that help in risk assessment, early diagnosis, and effective monitoring of the treatment.

North America Dominates the Autologous Stem Cell and Non-Stem Cell Based Therapies Market

- North America dominated the overall stem cell market with the United States contributing to the largest share in the market.- The ease in the US government regulations and availability of funds from various organizations, like the National Institute of Health, have provided the potential for researchers to invest more in the use of biomarkers in drug discovery, drug development, detection of specific tumors, monitoring biological response to cancer therapy, and genetic studies for the identification of predisposed candidates of cancer.- In 2014, the Sanford Stem Cell Clinical Center at the University of California, San Diego (UCSD) Health System, announced the launch of a clinical trial, in order to assess the safety of neural stem cellbased therapy in patients with chronic spinal cord injury.- Researchers hoped that the transplanted stem cells may develop into new neurons that could replace severed or lost nerve connections, and restore at least some motor and sensory functions. Such numerous stem cell studies across the United States have helped in the growth of the stem cell market.

Competitive LandscapeThe global Autologous Stem Cell and Non-Stem Cell-Based Therapies market is competitive and consists of a few major players. The companies includes Novartis AG, BrainStorm Cell Limited, Caladrius, Cytori Therapeutics Inc., Dendreon Pharmaceuticals LLC, Gilead Sciences Inc., Regeneus Ltd, U.S. Stem Cell, Inc, among others, hold the substantial market share in the growth of overall market.

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Autologous Stem Cell and Non-Stem Cell Based Therapies Market - Growth, Trends, and Forecast (2020 - 2025) - GlobeNewswire

Helixmith said Wednesday that it has submitted a phase 3-3 extension study protocol of its gene therapy Engensis (VM202) for diabetic peripheral neuropathy (DPN), to the U.S. Food and Drug Administration.

The company set a one-year follow-up period to confirm the pain reduction and safety of VM202 in treating DPN. DPN is one of the most common complications of diabetic diseases. About 30 million Americans have diabetes, and 28.5 percent of them develop DPN. Among the DPN patients, 40 to 50 percent experience painful symptoms.

The study's primary endpoint is the average pain reduction effect measured and recorded in the pain diary over the last week of the sixth month from the first injection.

The study will be carried out with patients who had not taken Gabapentinoids, such as Pregabalin and Gabapentine, in 15 research laboratories across the U.S., including Northwestern University in Chicago.

"Existing painkillers for DPN patients are not a fundamental treatment for the disease as they only relieved pain while often accompanying serious side effects and high addiction," Helixmith CEO Kim Sun-young said. "We will try our best for the success of phase 3-3 clinical trials as well as the ongoing phase 3-2 study."

FDA recognized the clinical results of Engennsis and designated it as an advanced regenerative medicine advanced therapy (RMAT) in 2018, the company said. RMAT is a new system designed to speed up the development and approval of innovative regenerative therapies. It gives special privileges of the U.S. fast track and priority or accelerated screening.

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Helixmith eyeing P3-3 clinical trials of gene therapy Engensis - Korea Biomedical Review

BOSTON, Sept. 15, 2020 /PRNewswire/ --GreenLight Bioscienceshas received a $3.3 million grant from the Bill & Melinda Gates Foundation to develop new mRNA-based gene therapies for Sickle Cell Disease and other global health challenges.

The funding will support GreenLight's research and testing of affordable therapies using the company's novel messenger RNA (mRNA) approach to gene editing. mRNA technology is already being used to develop vaccine candidates for infectious diseases, including the COVID-19 pandemic.

While initial research will focus on a cure for Sickle Cell Disease, GreenLight plans to develop a versatile gene editing platform to address a variety of diseases affecting underserved patient populations, such as treating HIV in developing countries.

Sickle Cell Disease is a group of inherited blood disorders in which red blood cells develop abnormally, causing pain and anemia. More than 4 million people currently suffer from the disease, with another 40+ million having the sickle cell trait, which can be passed on to future generations. The disease primarily targets people of African, Hispanic, or Middle Eastern descent. Current treatment regimens including blood transfusions and bone marrow transplants are costly, invasive, and impractical for treating large segments of affected patient populations.

"Funders are recognizing the potential of our innovative approach to gene editing that, in combination with our proprietary RNA manufacturing capability, has the potential to deliver accessible gene therapies and improve human health globally," said Marta Ortega-Valle, senior vice president of Human Health and Corporate Development at GreenLight Biosciences. "Finding a safe and effective therapy is critical, but equally important is the ability to produce it affordably for broader access. We are grateful for the Gates Foundation's support to advance novel gene editing approaches for populations in which those therapies are currently out of reach."

Gene editing therapies hold significant promise in the treatment of Sickle Cell Disease since it is a disorder caused by gene mutation. Using RNA as its core, GreenLight Biosciences is working to develop an in vivo gene therapy that could ultimately offer a cure to the disease.

Once the therapy candidate is validated and moves into clinical use, GreenLight Biosciences' biomanufacturing platform will accelerate production of affordable treatments at scale. "Manufacturing sufficient quantities of high-quality RNA at an accessible cost is critical for achieving the full potential of new therapies that aim to reach a global patient population. That capability does not yet exist in the market, but GreenLight's end-to-end, self-contained manufacturing platform aims to make that possible for all mRNA-based therapies and vaccines," Ortega-Valle added.

About GreenLight Biosciences, Inc.GreenLight is a bio-performance company with a unique, cell-free production platform that delivers high-performing RNA solutions to human, plant and animal challenges. GreenLight develops RNA products for plant and life science applications, and collaborates with industry leaders to advance vaccine development, pandemic preparation, crop management, and plant protection. The cutting-edge, natural platform delivers higher-quality RNA at a lower cost and higher speed than was ever before possible. The GreenLight team values diversity, inclusion, and equality and promises to use collaboration to remain scientifically imaginative and passionately focused on making a difference in the world. For more information, visithttps://www.greenlightbiosciences.com/.

SOURCE GreenLight Biosciences

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GreenLight Biosciences Receives $3.3 Million Grant to Develop Sickle Cell Disease Cure Using mRNA Gene Therapy - PRNewswire

Moderna has agreed to separate deals with Vertex and Chiesi for research and development spanning cystic fibrosis (CF) therapies and pulmonary arterial hypertension (PAH).

In its agreement with Vertex, Moderna will receive $75m to collaborate on the research and development into gene therapies for CF. As part of the deal, Moderna will discover and develop lipid nanoparticles (LNPs) and mRNAs for the delivery of gene-editing therapies for the treatment of CF.

Under the terms of the deal, Moderna will receive $75m upfront to conduct research activities to discover and develop LNPs for gene-editing CF therapies. The US-based biotech company will also be eligible to receive up to $380m in further development, regulatory and commercial milestone payments, as well as tiered royalties on any products that come out of the collaboration.

While Moderna will be responsible for the initial discovery and manufacturing of LNPs and mRNA constructs for the gene-editing treatments, Vertex will be responsible for providing other components of the therapies to be formulated into LNPs, as well as the preclinical and clinical development and commercialisation efforts of any potential candidates.

This is the second agreement reached between Moderna and Vertex, with the previous collaboration aimed at the discovery and development of mRNA therapeutics for CF having been recently extended.

Our first collaboration with Vertex to deliver mRNA coding for cystic fibrosis protein in lung cells is advancing well and this second collaboration aims at using Modernas technologies to explore the use of gene editing in lung cells, said Stphane Bancel, chief executive officer of Moderna.

Prior to announcing the Vertex deal, Moderna revealed that it had come to a separate agreement with Italian-domiciled pharma company Chiesi, for the development of mRNA treatments for PAH.

As part of that deal, Moderna will receive $25m upfront as well as a potential $400m in milestone payments to conduct research and development activities in this therapy area.

PAH is a rare and progressive disorder, which occurs when arteries in the lungs constrict, which forces the heart to work harder, often causing heart failure. The condition affects around two to five million adults across the globe, and remains an area of high unmet medical need.

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Moderna inks R&D agreements with Vertex and Chiesi - PMLiVE