Archive for the ‘Gene Therapy Research’ Category

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

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

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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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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.

The research study encompasses fundamental points of the global CNS 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 CNS Gene Therapy market. The CNS 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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key players and product offerings

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

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

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The global CNS 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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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.

The recent study on Cell and Gene Therapy market offers an all-inclusive analysis of this vertical, with emphasis on the growth driving factors as well as facets such as consumption and production. Constraints and potential threats expected to restrain the expansion along with solutions to overcome the challenges are discussed at length. Moreover, insights of the market share along with estimates reflecting the CAGRs of the listed segments are highlighted in the document.

In hindsight of the COVID-19 pandemic, the report investigates the prevalent business strategies employed by leading organizations and offers tactics for stakeholders to adapt to the industry changes over the forecast period.

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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

Or

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.

SOURCE Ampersand Capital Partners

http://www.ampersandventures.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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Top Key Players: Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene

By Product Type:Ex vivo, In vivo

By Application:Cancer Diseases, Monogenic Diseases, Infectious Diseases, Cardiovascular Diseases, Others

By Regions:

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:

North America (U.S., Canada)Europe (U.K., Germany, France, Italy)Asia Pacific (China, India, Japan, Singapore, Malaysia)Latin America (Brazil, Mexico)Middle East & Africa

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What is the current scenario of the Global Gene Therapy Market? How is the market going to prosper throughout the next 6 years?What are the emerging technologies that are going to profit the market?What is the historical and the current size of the Global Gene Therapy Market?Which segments are the fastest growing and the largest in the market? What is their market potential?What are the driving factors contributing to the market growth during the short, medium, and long term?What are the lucrative opportunities for the key players in the market?Which are the key geographies from the investment perspective?What are the major strategies adopted by the leading players to expand their market shares?Who are the distributors, traders and dealers of Global Gene Therapy market?What are sales, revenue, and price analysis by types and applications of market?

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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.

Reasons to Purchase this report:- The market estimate (ME) sheet in Excel format- 3 months of analyst supportRead the full report: https://www.reportlinker.com/p05974135/?utm_source=GNW

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

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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

Originally posted here:
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.

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

Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that it has submitted the Marketing Authorisation Application (MAA) for its lead product LUMEVOQ to the European Medicines Agency (EMA), seeking approval for the treatment of patients with vision loss due to Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene.

This first regulatory submission for GenSight is a major milestone in our progression from a pure research organization to one with commercial capabilities. It validates a technology platform that has the potential to address the high unmet medical needs of patients suffering from a range of rare diseases. I would like to thank all GenSight employees and partners whose motivation, focus and effort made this submission possible, said Bernard Gilly, Co-founder and Chief Executive Officer of GenSight Biologics.

LHON is a rare, mitochondrial genetic disease, mainly affecting young males. The ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There continues to be a high unmet medical need for the 800-1200 new LHON patients in Europe and the U.S. each year, particularly those who are struck blind in their prime working years.

Lenadogene nolparvovec (tradename: LUMEVOQ) is a recombinant adeno-associated viral vector, serotype 2 (rAAV2/2), containing a cDNA encoding the human wild-type mitochondrial NADH dehydrogenase 4 protein (ND4), which has been specifically developed for the treatment of LHON associated with mutation in the ND4 gene. It received orphan drug designation status for the treatment of LHON from the EMA in 2011 and from the U.S. Food and Drug Administration (FDA) in 2013.

GenSight submitted the MAA based on the benefit-risk balance established by results from a Phase-I/IIa study (CLIN-01), two pivotal Phase-III efficacy studies (CLIN-03A: RESCUE, and CLIN-03B: REVERSE) and the long-term follow up study of RESCUE and REVERSE (CLIN 06 readout at Year 3 post injection). To demonstrate the efficacy of LUMEVOQ in the context of a contralateral effect, the Company used a statistics-based indirect comparison methodology to assess the visual outcomes in LUMEVOQ-treated patients (from LUMEVOQ efficacy studies) against those in untreated patients from Natural History studies and GenSights REALITY Natural History Registry.

GenSight expects to submit the Biologics License Application (BLA) for LUMEVOQ to the FDA in H2 2021. First-in-human data from GenSights second clinical stage program, GS030, are expected to be available in H2 2021.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing 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), is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to the eye by intravitreal injection to offer patients a sustainable functional visual recovery.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200914005857/en/

GenSight BiologicsChief Financial Officer

Thomas Gidointgidoin@gensight-biologics.com+33 (0)1 76 21 72 20

LifeSci AdvisorsInvestor Relations

Guillaume van Renterghem

gvanrenterghem@lifesciadvisors.com+33 (0)6 69 99 37 83

RooneyPartnersMedia Relations

Marion Janic

mjanic@rooneyco.com+1-212-223-4017

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Excerpt from:
GenSight Biologics Submits EU Marketing Authorisation Application for LUMEVOQ Gene Therapy to Treat Vision Loss Due to Leber Hereditary Optic...

When a researchers talk about gene editing, theyre usually thinking about several steps. First you need to zero in on the defective gene; then, depending on the need, youd want to knock out, replace or insert genetic material.

CRISPR/Cas9 technologies have transformed the field by making a breakthrough for the first problem. Inducing double-stranded DNA breaks, or achieving single-letter changes as base editing allows, have promising applications in multiple diseases that are starting to get tested in humans.

But Versant Ventures, one of the pioneering investors in the space, still sees a gap. And theyre teaming up with a group of prominent Stanford researchers plus a seasoned biotech exec to fill it.

Achieving high-efficiency targeted gene integration has been a critical objective of gene editing for more than 15 years, but only now is this technologically possible, Jerel Davis, Versants Vancouver-based managing director, said.

Graphite Bio has $45 million to start things off, but the money matters little when compared to the rich brain bank its drawing from.

Matthew Porteus, an academic founder of CRISPR Therapeutics, is lending the tech platform to create this next-gen play alongside gene therapy expert Maria Grazia Roncarolo.

Drawing from research work led by Danny Dever while a postdoc at Porteus lab, Graphites big promise is to increase integration efficiency from less than 1% to greater than 50% across diverse genetic lesions in a wide range of cell types.

Specifically, Dever and Porteus showed that they could correct the sickle globin gene in patient-derived hematopoietic stem cells ex vivo by combining Cas9 ribonucleoproteins with a donor molecule that serves as a template, delivered in recombinant adeno-associated viral vectors of serotype 6 (rAAV6).

That could make for a much more scalable replacement for transplants, they wrote in a 2017 paper describing mouse studies.

Notably, we devise an enrichment paradigm to purify a population of HSPCs with >90% targeted integration, they wrote. We also show efficient correction of the SCD-causing E6V mutation in patient-derived HSPCs that after differentiation into erythrocytes, express adult -globin (HbA) mRNA, confirming intact transcriptional regulation of edited HBB alleles.

Chief executive Josh Lehrer is leading the charge to start Phase I for this program in early 2021, bringing all the sickle cell knowledge and experience from a six-year run as Global Blood Therapeutics, most recently as CMO. More preclinical therapies are in the works for unnamed serious diseases. Samsara BioCapital is joining Versant for the launch round and sending Abe Bassan to the board, which also features Davis and Carlo Rizzuto from Versant.

Read more here:
Versant teams up with Stanford gene editing experts on a $45M next-gen play marrying CRISPR and AAV to fix sickle cell - Endpoints News

David Dismuke, Ph.D., joins as Chief Technical Officer, Christopher Shilling as Vice President of Regulatory Affairs and Quality Assurance, and Christina Perry as Vice President of Finance and Operations.All three leaders will be in place in September and join Forge's executive leadership team.

"The addition of David, Chris, and Christina to the Forge leadership team brings immeasurable value to the Forge team and expands our team of gene therapy experts," said Timothy J. Miller, Ph.D., Forge's Co-Founder, President & CEO. "We believe that our accomplished and passionate team, with demonstrated experience in manufacturing and developing gene therapy products, is the foundation of fulfilling our mission to accelerate transformative medicines to reach those who need them most."

David Dismuke joins Forge with more than 15 years of experience in large-scale manufacturing. He is an authority in the bioprocessing and design of gene therapy vectors and has led CMC operations in the large-scale manufacturing of pre-clinical and clinical-grade AAV vectors for more than 10 years. Prior to joining Forge, David was the Vice President of Manufacturing at StrideBio where he directed the development of manufacturing and analytical processes for AAV vectors that utilize novel capsids. In addition, he led the design of therapeutic and reporter transgenes and innovative molecular enhancements to improve AAV production and therapeutic function.He was also previously the Head of Vector Production at Voyager Therapeutics where he led teams in the manufacturing and analytical testing of AAV using the baculovirus/Sf9 production system.Prior to Voyager, David was the Director of the UNC Vector Core, where he oversaw GMP operations as well as the production of research-grade vectors.He earned his PhD from Vanderbilt University, focusing on the molecular biology and lifecycle of HIV-1, and then performed his postdoctoral research at UNC Chapel Hill.

Christopher Shilling joins Forge as Vice President of Regulatory Affairs and Quality. He has over 15 years of experience in development of novel gene therapies for rare and severe disorders. Christopher is an experienced leader in gene therapy regulatory affairs, pharmacology, toxicology, and project management focused on developing strategies for early phase clinical trials in support of a variety of transformative therapeutics for pediatric and rare diseases. Prior to joining Forge, Christopher started the Drug and Device Development program at Nationwide Children's Hospital which was instrumental in gaining acceptance from regulators for over twenty first-in-human gene therapy clinical trials of novel biologic products, a dozen orphan drug designations, and two fast track designations. He received his Bachelor of Science degree in biology and a Master of Science degree in Pathology both from the Ohio State University.

Christina Perry joins Forge Biologics as the Vice President of Finance and Operations. She spent the last several years as the CFO of Drive Capital where she built out all accounting and finance operations, back-office needs, and investor relations and reporting. Christina managed complex entity legal structures and has had extensive exposure to equity and debt financings.Over the last 15+ years Christina has worked with public and private companies and startups across varying industries, developing operational processes to scale with high growth. She began her career at Deloitte, is a licensed CPA in the state of Ohio, earned her Bachelor of Business Administration at the University of Notre Dame and a Master of Accountancy from Miami University.

These additions to the Forge Biologics leadership team continue Forge's momentum within the biotechnology industry in Columbus Ohio, bringing positive impact to both Ohio and the global rare disease community.

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

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

Media Inquiries:Dan SalvoForge Biologics Director of Communications and Community Development[emailprotected]

Business Inquiries:Erandi De Silva, Ph.D.Forge BiologicsCo-Founder and Vice President of Product Development[emailprotected]

SOURCE Forge Biologics

https://forgebiologics.com

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Forge Biologics Strengthens Gene Therapy Leadership with Key Hires in AAV Manufacturing, Regulatory Affairs and Finance to Support Strategic Growth -...

DUBLIN--(BUSINESS WIRE)--The "Viral Vector & Plasmid DNA Manufacturing Market Research Report by Type, by Application, by End User - Global Forecast to 2025 - Cumulative Impact of COVID-19" report has been added to ResearchAndMarkets.com's offering.

The Global Viral Vector & Plasmid DNA Manufacturing Market is expected to grow from USD 551.56 Million in 2019 to USD 2,101.82 Million by the end of 2025 at a Compound Annual Growth Rate (CAGR) of 24.97%.

This research report categorizes the Viral Vector & Plasmid DNA Manufacturing to forecast the revenues and analyze the trends in each of the following sub-markets:

The FPNV Positioning Matrix evaluates and categorizes the vendors in the Viral Vector & Plasmid DNA Manufacturing Market on the basis of Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. The Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth.

Cumulative Impact of COVID-19:

COVID-19 is an incomparable global public health emergency that has affected almost every industry so far and the long-term effects projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlaying COVID-19 issues and potential paths forward. The report is delivering insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecast, considering the COVID-19 impact on the market.

The report provides insights on the following pointers:

1. Market Penetration: Provides comprehensive information offered by the key players

2. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets

3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments

4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players

5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments

The report answers questions such as:

1. What is the market size and forecast of the Global Viral Vector & Plasmid DNA Manufacturing Market?

2. What are the inhibiting factors and impact of COVID-19 shaping the Global Viral Vector & Plasmid DNA Manufacturing Market during the forecast period?

3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Viral Vector & Plasmid DNA Manufacturing Market?

4. What is the competitive strategic window for opportunities in the Global Viral Vector & Plasmid DNA Manufacturing Market?

5. What are the technology trends and regulatory frameworks in the Global Viral Vector & Plasmid DNA Manufacturing Market?

6. What are the modes and strategic moves considered suitable for entering the Global Viral Vector & Plasmid DNA Manufacturing Market?

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/w76mxp

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

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Global Viral Vector & Plasmid DNA Manufacturing Market Analysis and Forecasts to 2025 - Cumulative Impact of COVID-19 - ResearchAndMarkets.com -...

More than 1 million Americans are diagnosed with a chronic brain disorder each year, yet effective treatments for most complex brain disorders are inadequate or even nonexistent.

A major new research effort at the McGovern Institute for Brain Research at MIT aims to change how we treat brain disorders by developing innovative molecular tools that precisely target dysfunctional genetic, molecular, and circuit pathways.

The K. Lisa Yang and Hock E. Tan Center for Molecular Therapeutics in Neuroscience was established at MIT through a $28 million gift from philanthropist Lisa Yang and MIT alumnus Hock Tan 75. Yang is a former investment banker who has devoted much of her time to advocacy for individuals with disabilities and autism spectrum disorders. Tan is president and CEO of Broadcom, a global technology infrastructure company.This latest gift brings Yang and Tans total philanthropy to MIT to more than $72 million.

In the best MIT spirit, Lisa and Hock have always focused their generosity on insights that lead to real impact," says MIT President L. Rafael Reif. Scientifically, we stand at a moment when the tools and insights to make progress against major brain disorders are finally within reach. By accelerating the development of promising treatments, the new center opens the door to a hopeful new future for all those who suffer from these disorders and those who love them. I am deeply grateful to Lisa and Hock for making MIT the home of this pivotal research.

Engineering with precision

Research at the K. Lisa Yang and Hock E. Tan Center for Molecular Therapeutics in Neuroscience will initially focus on three major lines of investigation: genetic engineering using CRISPR tools, delivery of genetic and molecular cargo across the blood-brain barrier, and the translation of basic research into the clinical setting. The center will serve as a hub for researchers with backgrounds ranging from biological engineering and genetics to computer science and medicine.

Developing the next generation of molecular therapeutics demands collaboration among researchers with diverse backgrounds, says Robert Desimone, McGovern Institute director and the Doris and Don Berkey Professor of Neuroscience at MIT. I am confident that the multidisciplinary expertise convened by this center will revolutionize how we improve our health and fight disease in the coming decade. Although our initial focus will be on the brain and its relationship to the body, many of the new therapies could have other health applications.

There are an estimated 19,000 to 22,000 genes in the human genome and a third of those genes are active in the brain the highest proportion of genes expressed in any part of the body. Variations in genetic code have been linked to many complex brain disorders, including depression and Parkinsons disease. Emerging genetic technologies, such as the CRISPR gene editing platform pioneered by McGovern Investigator Feng Zhang, hold great potential in both targeting and fixing these errant genes. But the safe and effective delivery of this genetic cargo to the brain remains a challenge.

Researchers within the new Yang-Tan Center will improve and fine-tune CRISPR gene therapies and develop innovative ways of delivering gene therapy cargo into the brain and other organs. In addition, the center will leverage newly developed single-cell analysis technologies that are revealing cellular targets for modulating brain functions with unprecedented precision, opening the door for noninvasive neuromodulation as well as the development of medicines. The center will also focus on developing novel engineering approaches to delivering small molecules and proteins from the bloodstream into the brain. Desimone will direct the center and some of the initial research initiatives will be led by associate professor of materials science and engineering Polina Anikeeva; Ed Boyden, the Y. Eva Tan Professor in Neurotechnology at MIT; Guoping Feng, the James W. (1963) and Patricia T. Poitras Professor of Brain and Cognitive Sciences at MIT; and Feng Zhang, James and Patricia Poitras Professor of Neuroscience at MIT.

Building a research hub

My goal in creating this center is to cement the Cambridge and Boston region as the global epicenter of next-generation therapeutics research. The novel ideas I have seen undertaken at MITs McGovern Institute and Broad Institute of MIT and Harvard leave no doubt in my mind that major therapeutic breakthroughs for mental illness, neurodegenerative disease, autism, and epilepsy are just around the corner, says Yang.

Center funding will also be earmarked to create the Y. Eva Tan Fellows program, named for Tan and Yangs daughter Eva, which will support fellowships for young neuroscientists and engineers eager to design revolutionary treatments for human diseases.

We want to build a strong pipeline for tomorrows scientists and neuroengineers, explains Hock Tan. We depend on the next generation of bright young minds to help improve the lives of people suffering from chronic illnesses, and I can think of no better place to provide the very best education and training than MIT.

The molecular therapeutics center is the second research center established by Yang and Tan at MIT. In 2017, they launched the Hock E. Tan and K. Lisa Yang Center for Autism Research, and, two years later, they created a sister center at Harvard Medical School, with the unique strengths of each institution converging toward a shared goal: understanding the basic biology of autism and how genetic and environmental influences converge to give rise to the condition, then translating those insights into novel treatment approaches.

All tools developed at the molecular therapeutics center will be shared globally with academic and clinical researchers with the goal of bringing one or more novel molecular tools to human clinical trials by 2025.

We are hopeful that our centers, located in the heart of the Cambridge-Boston biotech ecosystem, will spur further innovation and fuel critical new insights to our understanding of health and disease, says Yang.

Originally posted here:
New molecular therapeutics center established at MIT's McGovern Institute - MIT News

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Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.AdvantageneGene Therap

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CancerMonogenicInfectious diseaseCardiovascular diseaseOther

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Ex vivoIn VivoGene Therap

By Geographical Regions

Asia Pacific: China, Japan, India, and Rest of Asia PacificEurope: Germany, the UK, France, and Rest of EuropeNorth America: The US, Mexico, and CanadaLatin America: Brazil and Rest of Latin AmericaMiddle East & Africa: GCC Countries and Rest of Middle East & Africa

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Executive Summary

Assumptions and Acronyms Used

Research Methodology

Gene Therapy Market Overview

Gene Therapy Supply Chain Analysis

Gene Therapy Pricing Analysis

Global Gene Therapy Market Analysis and Forecast by Type

Global Gene Therapy Market Analysis and Forecast by Application

Global Gene Therapy Market Analysis and Forecast by Sales Channel

Global Gene Therapy Market Analysis and Forecast by Region

North America Gene Therapy Market Analysis and Forecast

Latin America Gene Therapy Market Analysis and Forecast

Europe Gene Therapy Market Analysis and Forecast

Asia Pacific Gene Therapy Market Analysis and Forecast

Middle East & Africa Gene Therapy Market Analysis and Forecast

Competition Landscape

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Impact Of Covid-19 On Gene Therapy Market 2020 Analysis By Geographical Regions, Type And Application Till 2027 With Top Key Players: Bluebird Bio,...

NEW YORK--(BUSINESS WIRE)--As part of a two-day virtual Investor Day, Pfizer Inc. (NYSE: PFE) provided an extensive overview of pipeline advances and shared updates on the Companys efforts to battle the COVID-19 pandemic on multiple fronts, including new data on the BNT162b2 vaccine candidate being developed in collaboration with BioNTech SE. The pipeline updates contribute to the Companys expectation of at least a 6% revenue CAGR over the next five years, as well as delivery of longer-term topline growth beyond that period.

Pfizers goal of delivering up to 25 breakthroughs to patients by the year 2025 has 38 such opportunities to draw from as of today, including the companys 20-valent pneumococcal conjugate vaccine candidate (20vPnC). On a non-risk adjusted basis, these opportunities collectively represent more than $15 billion (excluding 20vPnC) in potential incremental revenue for Pfizer from 2020 to 2025, as well as aggregate peak annual sales potential of $35 billion to $40 billion (including 20vPnC). If successful, the Companys COVID-19 programs would be incremental to these estimates.

Pfizers purpose Breakthroughs that change patients lives has never been more relevant, and our R&D pipeline has never been more dynamic, said Dr. Albert Bourla, Pfizer Chairman and CEO. I am proud of the truly transformational science that our research and clinical teams are bringing to the fight against disease, as well as the unprecedented speed with which we are advancing our clinical programs in the battle against COVID-19. In the coming months and years, I look forward to the new Pfizer continuing to demonstrate the agility and innovative spirit of a biotech combined with the scale of Big Pharma. With the depth and breadth of our current portfolio, the tremendous potential of our pipeline and scientific engine, and the power of our culture of innovation, we are poised to continue delivering meaningful value to patients by addressing some of the worlds most difficult health challenges.

UPDATES ON COVID-19 DEVELOPMENT PROGRAMS

Pfizer announced several key advances in its efforts to protect humankind from the COVID-19 pandemic and prepare the pharmaceutical industry to better respond to future global health crises.

BNT162 mRNA-based Vaccine Program

Pfizer and BioNTech shared several updates from their BNT162 mRNA-based vaccine program against SARS-CoV-2, the virus that causes COVID-19 disease, including:

Protease Inhibitor Program

The company announced the initiation of its Phase 1b clinical trial to evaluate the safety of a novel investigational therapeutic for COVID-19, PF-07304814. Of note,

THERAPEUTIC AREAS OF FOCUS

Pfizer shared significant research advances across its various therapeutic areas including candidates with blockbuster potential expected to launch by 2025.

Vaccines

In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval. Updates on these late-stage clinical development programs include:

Rare Disease

Pfizers Rare Disease late-stage pipeline currently includes three gene therapy programs that, if successful, are expected to gain regulatory approval by the end of 2023, with an additional pipeline of 10 preclinical initiatives that are at various stages of maturity. Key updates include:

Oncology

Pfizers Oncology pipeline has the potential to deliver up to 14 approvals expected by the end of 2025 and the potential for 24 new molecular entities in the clinic by the end of 2021. Key updates included, for the first time, early-stage opportunities obtained from the 2019 acquisition of Array BioPharma:

Inflammation and Immunology

The Inflammation & Immunology pipeline is focused on patients with autoimmune and chronic inflammatory diseases across rheumatology, gastroenterology and dermatology, with five distinct immuno-kinases, in oral and topical formulations, studied for potential treatment of 10 diseases, and three additional novel biologics in Phase 2 studies. Key updates included:

Internal Medicine

The Internal Medicine pipeline addresses the increasing global burden of cardiometabolic disease, with nine investigational medicines in active clinical studies and additional therapies in the pre-clinical pipeline. Key updates included:

To access a replay of the webcast, including audio, video and presentation slides, visit our web site at http://www.pfizer.com/investors.

About Pfizer: Breakthroughs That Change Patients Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice: The information contained in this release is as of September 15, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release or the webcast as the result of new information or future events or developments.

This release and the webcast contain forward-looking information about Pfizers anticipated operating and financial performance, business plans and prospects, Pfizers pipeline portfolio (including anticipated regulatory submissions, data read-outs, study starts, approvals, revenue contributions and market opportunities), and our efforts to respond to COVID-19, including our investigational vaccine candidate against SARS-CoV-2 and our investigational protease inhibitor, including their potential benefits, among other things, that are subject to substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any drug applications, biologics license applications and/or emergency use authorization applications may be filed in any jurisdictions for any potential indication for Pfizers product candidates; whether and when any such applications that may be filed for any of Pfizers product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether any such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of Pfizers product candidates, including development of products or therapies by other companies; manufacturing capabilities or capacity; uncertainties regarding the ability to obtain recommendations from vaccine technical committees and other public health authorities and uncertainties regarding the commercial impact of any such recommendations; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

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Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas - Business Wire