Analysis of COVID-19 Gene Therapy Market – Market Research Posts

Gene Therapy Market Snapshot

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

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

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

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

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

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Global Gene Therapy Market Snapshot

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

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

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

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

Yescarta to Dominate Global Gene Therapy Market over the Forecast Period

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

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

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

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

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

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Analysis of COVID-19 Gene Therapy Market - Market Research Posts

Gene Therapy Market is projected to grow at an annualized rate of 45%, till 2030 – Market Research Posts

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

The USD 11.6 billion (by 2030) financial opportunity within the Gene Therapy Market has been analyzed across the following segments:

Key therapeutic areas

Type of vector

Type of therapy

Type of gene modification

Route of administration

Key geographical regions

For more information, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

The Gene Therapy Market (3rd Edition), 2019-2030 report features the following companies, which we identified to be key players in this domain:

Table of Contents

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Spark Therapeutics Recognized for Fourth-straight Year as One of Philadelphia’s Best Places to Work by the Philadelphia Business Journal – BioSpace

PHILADELPHIA, Aug. 03, 2020 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced that it has been named one of Philadelphias Best Places to Work for the fourth year in a row by the Philadelphia Business Journal.

In seven years, we have grown to a team of more than 500 employees, who are truly what make Spark Therapeutics a great place to work, and achieved breakthroughs that have heralded a new era in medicine, said Lisa Dalton, chief people officer, Spark Therapeutics. It was here in Philadelphia that we brought the first gene therapy for a genetic disease in the U.S. to patients, and now, as a member of the Roche Group, we remain committed to mentoring the next generation of talent, investing in West Philadelphia and growing and fostering an engaged workforce that keeps us a best place to work for years to come.

ThePhiladelphia Business Journalselected winners based on employee responses to an engagement survey conducted by Quantum Workplace, an independent employee feedback software company. The Best Places to Work survey measures key areas such as work environment, professional development and embracing change and new ideas.

Spark remains fully dedicated to hiring and expanding its workforce to meet ambitious growth plans. The company strives to create a diverse workforce of unique viewpoints, experiences, and perspectives to break barriers in gene therapy and reflect the diversity of the world around us.

Interested candidates are encouraged to consider sparking a change in their careers and joining the company. Learn more and explore job openings by visiting http://www.sparktx.comor following Spark Therapeutics onLinkedIn.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanokevin.giordano@sparktx.com(215) 294-9942

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Spark Therapeutics Recognized for Fourth-straight Year as One of Philadelphia's Best Places to Work by the Philadelphia Business Journal - BioSpace

AGTC to Present at the Wedbush PacGrow Virtual Healthcare Conference on August 11, 2020 – BioSpace

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Aug. 04, 2020 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that Sue Washer, President & Chief Executive Officer, will present at the Wedbush PacGrow Virtual Healthcare Conference on Tuesday, August 11, 2020 at 10:20 am ET.

A live audio webcast of the presentation will be available by visiting http://ir.agtc.com/events-and-presentations. A replay will be available on the Company's website following the event.

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

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

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

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

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AGTC to Present at the Wedbush PacGrow Virtual Healthcare Conference on August 11, 2020 - BioSpace

Global Hemophilia Gene Therapy Market is estimated to Experience a Notable Rise in the coming era by Spark Therapeutics, Ultragenyx, Shire PLC,…

Global Hemophilia Gene Therapy Market presents comprehensive insights into the present and upcoming industry trends, enabling the readers to identify the products and services, hence driving the revenue increase and effectiveness. The research report presents a complete breakdown of all the major factors affecting the market on a global and regional scale, including drivers, constraints, threats, challenges, opportunities, and Hemophilia Gene Therapy industry-specific trends. Further, the report mentions global facts and figures along with downstream and upstream analysis of leading players.

The study gives answers to the following key questions:

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Major Players:

Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline Therapeutics

This research presents Hemophilia Gene Therapy market growth rates and the market value based on market dynamics, growth factors. The complete knowledge is based on the newest innovation in the business, opportunities, and trends. In adding up to SWOT analysis by key suppliers, the report contains an all-inclusive market analysis and major players landscape.

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The regional segmentation covers:

Segmentation by Type:

Hemophilia AHemophilia B

Segmentation by Application:

Hemophilia A Gene TherapyHemophilia B Gene Therapy

Any Query Or Specific Requirement? Ask to our Research expert @

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Report Objectives

Global Hemophilia Gene Therapy Market Size, Status and Forecast 2020-2024

Browse Detailed TOC & Table Of Figures

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Global Hemophilia Gene Therapy Market is estimated to Experience a Notable Rise in the coming era by Spark Therapeutics, Ultragenyx, Shire PLC,...

MEDIPAL HOLDINGS Partners with CRYOPORT to Bring Temperature-Controlled Supply Chain Solutions to the Japanese Market – PRNewswire

NASHVILLE, Tenn. and TOKYO, Aug. 4, 2020 /PRNewswire/ --Cryoport, Inc.(NASDAQ: CYRX) ("Cryoport"), a global leader in life sciences supply chain solutions, and MEDIPAL HOLDINGS CORPORATION (7459:JP), ("MEDIPAL") Japan's leading pharma wholesaler and distributor today announced a multi-year strategic business alliance agreement to create an integrated regenerative medicine supply chain solution in Japan.

Cryoport and MEDIPAL will partner to provide integrated, end-to-end distribution solutions for specialty cell and gene therapies that demand stringent temperature control, track and trace systems and global distribution. To meet demand from the increasing number of cell and gene therapies currently in development and expected to launch in coming years, this alliance will combine both companies' strengths to realize reliable and seamless distribution services for biopharmaceutical companies in Japan and the Asia-Pacific ("APAC") region / worldwide.

Cryoport supports over 465clinical trials, globally, and multiple cell and gene therapies in commercial distribution. Its suite of unique temperature-controlled solutions for the life sciences industry enables users to monitor their shipments and track the conditions, location, and courier handling of their biological commodities in transit around the clock. And should a problem occur that warrants intervention, Cryoport can deploy intervention capability to mitigate the situation.

MEDIPAL provides industry-leading efficient, reliable, and timely distribution solutions for specialty products in the healthcare industry across a broad range of temperatures, from -196to 37(-320to 99), with strict temperature control and traceability. With its high-quality logistics, including the original SDDU (Specialty Drug Distribution Unit: a long-life shipping unit using liquid nitrogen in liquid phase for ultra-low temperature logistics), MEDIPAL has positioned itself as a unique leader in cell and gene therapy product logistics in Japan. The goal of the partnership is to provide fully integrated solutions that reduce risk and improve certainty for the companies' Japan and global clients and build business revenue for both MEDIPAL and Cryoport.

Shuichi Watanabe, Representative Director, President and CEO said, "This alliance with Cryoport will enable our two companies to seamlessly provide an integrated, high-quality logistics service in the Japanese market, as well as overseas, complementing each other's strengths. We also believe that this alliance will accelerate our progress to satisfy unmet medical needs, both domestically and internationally, and it will help patients awaiting new treatment options, as well as the treating physicians."

Jerrell Shelton, CEO of Cryoport, added, "Cryoport's Chain of Compliance complete suite of technologies and informatics are trusted by biopharmaceutical companies worldwide. We use SmartPak Condition Monitoring System and Cryoportal technologies to monitor the conditions of cell and gene therapies in real-time from the time of packing through transit to unpacking thereby significantly mitigating potential risks. We look forward to this alliance with MEDIPAL and to growing together in the cell and gene therapy space in Japan and globally."

About Cryoport, Inc.Cryoport, Inc. (Nasdaq: CYRX) is redefining logistics for the life sciences industry by providing a platform of temperature-controlled 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 its purpose-built, proprietary Cryoport Express Shippers; Cryoportal information technology; validated Global Logistics Centers; smart and sustainable temperature-controlled logistics; and biostorage/biobanking services, Cryoport serves clients in life sciences research, clinical trials, and product commercialization. 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 MEDIPAL HOLDINGS CORPORATION

Name:

MEDIPAL HOLDINGS CORPORATION

Established:

May 1923

Business:

As a holding company, MEDIPAL controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale Business, and conducts business development for the MEDIPAL Group.

Head office:

Chuo-ku, Tokyo, Japan

Website:

https://medipal.co.jp/english

Representative:

Shuichi Watanabe, Representative Director, President and CEO

Forward Looking StatementsStatements in this news release which are not purely historical, including statements regarding Cryoport, Inc.'s intentions, hopes, beliefs, expectations, representations, projections, plans or predictions of the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. It is important to note that the Company's actual results could differ materially from those in any such forward-looking statements. Factors that could cause actual results to differ materially include, but are not limited to, risks and uncertainties associated with the effect of changing economic conditions, trends in the products markets, variations in the Company's cash flow, market acceptance risks, and technical development risks. The Company's business could be affected by a number of other factors, including the risk factors listed from time to time in the Company's SEC reports including, but not limited to, the Company's 10-K for the year ended December 31, 2019 filed with the SEC. The Company cautions investors not to place undue reliance on the forward-looking statements contained in this press release. Cryoport, Inc. disclaims any obligation, and does not undertake to update or revise any forward-looking statements in this press release.

SOURCE Cryoport, Inc.

http://www.cryoport.com

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Can progenitor cells go further than gene therapy in retinal disorders? A biotech’s answer shines in PhIIb – Endpoints News

By the time the FDA approved Luxturna the pioneering gene therapy for an inherited retinal disease Henry Klassen had been researching and treating the group of diseases known as retinitis pigmentosa for more than 20 years.

Spark Therapeutics success there had not just inspired a $4.3 billion takeover by Roche, it had also emboldened other biotechs pursuing a therapy that would deliver one of the genes tied to different variations of the disorder. Nightstar Therapeutics subsequently scored its own buyout with Biogen, and MeiraGTx recently posted early but exciting data on its J&J-partnered program.

But Klassen went a different way.

Catching on the stem cell craze right at the turn of the millennium, he took inspiration from scientists who transplanted neural progenitor cells into the retina and developed a method to grow retinal progenitor cells instead as a therapy. Starting out as the director of stem cell research at the Childrens Hospital of Orange County, he continued the work at the University of California, Irvine, eventually spinning out a biotech dubbed jCyte in 2012.

Over the weekend jCyte reported positive Phase IIb results from what they call one of the largest studies ever conducted in RP, suggesting that patients on the treatment saw improved functional vision compared to the placebo group.

The credit to the gene therapies is that theyre actively trying to fix the gene underlying the problem. Thats very commendable, and were not doing that, Klassen told Endpoints News. But our treatment as it stands should have impact across a variety of different genotypes.

The study enrolled a total of 84 patients, of whom 74 were included for the final analysis. For each patient on the primary endpoint of best corrected visual acuity (measured with glasses on), the mean change from baseline to month 12 for the sham, low dose and high dose arms were +2.81, +2.96, and +7.43 letters, respectively.

In a post hoc analysis for a target subgroup, the difference was even more prominent: +1.85, -0.15, and +16.27 letters, respectively.

There was one serious adverse event in the low dose arm, but jCyte said the grade 3 ocular hypertension resolved with treatment and other side effects were generally minor.

CEO Paul Bresge noted that the target subgroup analysis was intended to hammer out the criteria they might use to recruit patients into Phase III which would likely have a similar design and use the same primary endpoint of BCVA, the gold standard in the context of FDA. The late-stage trial is slated for 2021.

We did enroll a very wide patient population into our Phase IIb, including patients that had vision anywhere from 20/80 to 20/800, just to learn which patients would potentially be the best responders, he said.

The target subgroup is characterized by having reliable fixation on the study eye, and a study eye that does not have significantly worse BCVA (15 letters) than the fellow eye.

He added that investigators also observed encouraging results with the secondary endpoints such as low light mobility, contrast sensitivity kinetic visual fields and a vision function questionnaire, although the data werent disclosed.

Typically people think about the disease as a narrowing of this peripheral vision in a very nice granular way, but thats actually not what happens, he said about the visual fields finding. What happens in the disease is that patients lose like islands of vision. So what were doing in our tests is actually measuring [] islands that the patients have at baseline, and then what were seeing after treatment is that the islands are expanding. Its similar to the way that one would track, lets say a tumor, in oncology of course were looking for the opposite effect. Were looking for the islands of vision to expand.

The therapy works primarily by preserving photoreceptors, Klassen posits, not by generating new ones. But what he thinks is happening is that photoreceptors are regenerating the outer segment if photoreceptors are radios, these would be the antenna thereby regaining some function.

That could position it as a treatment for a different stage of the disease than Sparks or Nightstars. Klassen, whos also researching retinal reconstruction using stem cells, is happy to not view it through the competitive lens.

If you look into the future, one could imagine that gene therapies will be most effective very early in the course of a disease before photoreceptors are lost, he said. Then as photoreceptors begin to be lost anyway, if that happens, then a therapy like ours would become extremely valuable. And if ours starts to lose power late in the course of a disease, maybe cell transplantation under the retina could have a role.

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Can progenitor cells go further than gene therapy in retinal disorders? A biotech's answer shines in PhIIb - Endpoints News

Oxford Biomedica Signs Three Year Clinical Supply Agreement with Axovant Gene Therapies for Manufacture and Supply of AXO-Lenti-PD – GlobeNewswire

Oxford Biomedica Signs Three Year Clinical Supply Agreement with Axovant Gene Therapies for Manufacture and Supply of AXO-Lenti-PD

Oxford, UK 31st July, 2020: Oxford Biomedica plc (LSE:OXB) (Oxford Biomedica or the Group), a leading gene and cell therapy group, announced today that it has signed a three year Clinical Supply Agreement (CSA) with a wholly-owned subsidiary of Axovant Gene Therapies Ltd. (Axovant) (Nasdaq: AXGT). The CSA builds on the worldwide license agreement signed between the two companies in June 2018 for the Parkinsons disease gene therapy program OXB-102, now called AXO-Lenti-PD.

Under the terms of the CSA, Oxford Biomedica will manufacture GMP batches for Axovant to support the ongoing and future clinical development of AXO-Lenti-PD, a clinical-stage gene therapy product to treat moderate to severe Parkinsons Disease based on Oxford Biomedicas LentiVector platform. Axovant is currently conducting a Phase 2 SUNRISE-PD trial with AXO-Lenti-PD. Dosing of all patients in the second cohort is completed with 6-month safety and efficacy data expected in the fourth quarter of 2020. OXB expects to manufacture AXO-Lenti-PD in its commercial-scale GMP manufacturing facilities including Oxbox in the UK, and additionally in other OXB GMP facilities as required to ensure security of supply.

John Dawson, Chief Executive Officer of Oxford Biomedica, said: This new Agreement builds upon our existing worldwide licensing agreement with Axovant and highlights the strengths of Oxford Biomedica's commercial GMP manufacturing capabilities. We are pleased with how the partnership is progressing and excited by the clinical progress to date. The agreement today signals our commitment to the efficient ongoing development of this much needed product for patients with Parkinsons disease.

We are now at a stage in the partnership where we can determine the manufacturing activities and infrastructure required to support the mid and late-stage development of AXO-Lenti-PD in a way which is compatible with later commercialisation and we look forward to this next phase of our partnership.

Pavan Cheruvu, Chief Executive Officer at Axovant Gene Therapies, said: This Agreement with Oxford Biomedica means that together we can continue to advance the development of AXO-Lenti-PD in Parkinsons disease. We are pleased to extend our partnership with Oxford Biomedica, a world leader in lentiviral vector development and manufacturing, as we scale-up AXO-Lenti-PD production to support our Phase 2 and Phase 3 clinical studies and enable commercialization of the product. This marks another mutual accomplishment for our Parkinsons disease program where we expect to enroll the first subject in a randomized, sham-controlled trial in 2021.

-Ends-

Oxford Biomedica plc

John Dawson, Chief Executive OfficerStuart Paynter, Chief Financial OfficerCatherine Isted, Head of Corporate Development & IR

T: +44 (0)1865 783 000T: +44 (0)1865 783 000T: +44 (0)1865 954 161 / E: ir@oxb.com

Consilium Strategic Communications

Mary-Jane Elliott/Matthew Neal

T: +44 (0)20 3709 5700

About Oxford BiomedicaOxford Biomedica (LSE:OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford Biomedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology, CNS disorders, liver diseases and respiratory disease. The Group has also entered into a number of partnerships, including with Novartis, Bristol Myers Squibb, Sanofi, Axovant Gene Therapies, Orchard Therapeutics, Santen, Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium and Imperial Innovations, through which it has long-term economic interests in other potential gene and cell therapy products. Additionally the group has signed a Clinical and Commercial Supply Agreement with AstraZeneca for manufacture of the adeno based COVID-19 vaccine candidate, AZN1222. Oxford Biomedica is based across several locations in Oxfordshire, UK and employs more than 550 people. Further information is available atwww.oxb.com

About AXO-Lenti-PDAXO-Lenti-PD is an investigational gene therapy for the treatment of Parkinsons disease that is designed to deliver three genes (tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase) via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The investigational gene therapy aims to provide patient benefit for years following a single administration. The SUNRISE-PD Phase 2 trial is ongoing with dosing completed for all patients in cohort 2, with 6 month safety and efficacy data expected in Q4 2020. Axovant expects to dose the first patient in the Part B randomized, sham controlled study in 2021.

About Axovant Gene TherapiesAxovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

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Oxford Biomedica Signs Three Year Clinical Supply Agreement with Axovant Gene Therapies for Manufacture and Supply of AXO-Lenti-PD - GlobeNewswire

Cell and Gene Therapy Market 2020 | Enormous Growth with Recent Trends & Demand By Top Vendors JW CreaGene, Vericel, Tego Sciences, CHIESI…

Cell and Gene Therapy Market In-depth Analysis 2020-2026

The report include a thorough study of the global Cell and Gene TherapyMarket. It has successfully pointed out the key factors that have substantial impact on theCell and Gene Therapy market. This report is a result of a well-planned research methodology. The methodology employed both primary and secondary research tools.

These tools aid the researchers to gather authentic data and arrive at a definite conclusion. The prevailing competitors in the global Cell and Gene TherapyMarket has also been pictured in the report, offering an opportunity to theCell and Gene Therapy market players to measuring system their performance.

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The report has been prepared after studying the different parameters ruling the global Cell and Gene TherapyMarket and the forecast period has been estimated from 2020-2026. The forecast period is the time period when the key factors and parameters will help the market to flourish significantly. The estimated value of the market has been represented through a CAGR percentage. Additionally, the report represents the approximate revenue that can be generated over the forecast period. However, the report has also outlined the factors that can slowdown the growth of the global Cell and Gene TherapyMarket.

Key players in the global market covered, JW CreaGene, Vericel, Tego Sciences, CHIESI Farmaceutici, Spark Therapeutics, GC Pharma, MolMed, AnGes, Takeda Pharmaceutical Company, APAC Biotech, Gilead Sciences, Corestem, AVITA Medical, Novartis AG, JCR Pharmaceuticals, Dendreon, CO.DON, Medipost, Osiris Therapeutics, Amgen, Biosolution, CollPlant, Japan Tissue Engineering, Organogenesis, Orchard Therapeutics, Stempeutics Research, ,

on the basis of types, Cell Therapy, Gene Therapy, ,

on the basis of applications, Hospitals, Wound Care Centers, Cancer Care Centers, Ambulatory Surgical Centers, Others, ,

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The report includes the key driving forces prevailing in the global Cell and Gene TherapyMarket. This part of the report has been studied keeping in mind the political, economic, social, technological, geographical, and cultural scenario of the global Cell and Gene TherapyMarket. These factors can be projected to have their individual effects on the market, or they can have interconnected impacts. Besides, subtle change in the time framewithin which these factors are functioning might have ripple effects on the global Cell and Gene TherapyMarket.

Regional

Global Cell and Gene TherapyMarket has been segmented into Europe, the Americas, Asia Pacific, and the Middle East & Africa. This part of the report provides an exhaustive view of the regional scope existing in the global Cell and Gene TherapyMarket. The trends and preferences dominating each region has a direct impact on the industries. The report tries to exploit the trends and preferences prevailing in a region to offer the users with a clear picture of the business potential existing in that region.

Research Methodology

The primary research procedure conducted to arrive at the results includes panel of face to face interviews with industry experts and consumers. The secondary research procedure includes an intricate study of the scholarly journals and reports available online.

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Cell and Gene Therapy Market 2020 | Enormous Growth with Recent Trends & Demand By Top Vendors JW CreaGene, Vericel, Tego Sciences, CHIESI...

Comprehensive Report on Gene Therapy for Ovarian Cancer Market 2020 | Trends, Growth Demand, Opportunities & Forecast To 2026 | Takara Bio, VBL…

Gene Therapy for Ovarian Cancer Market research is an intelligence report with meticulous efforts undertaken to study the right and valuable information. The data which has been looked upon is done considering both, the existing top players and the upcoming competitors. Business strategies of the key players and the new entering market industries are studied in detail. Well explained SWOT analysis, revenue share and contact information are shared in this report analysis.

Gene Therapy for Ovarian Cancer Market is growing at a High CAGR during the forecast period 2020-2026. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market.

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Top Key Players Profiled in This Report:

Takara Bio, VBL Therapeutics, CELSION, Targovax

The key questions answered in this report:

Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Gene Therapy for Ovarian Cancer market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Gene Therapy for Ovarian Cancer markets trajectory between forecast periods.

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Table of Contents:

Global Gene Therapy for Ovarian Cancer Market Research Report

Chapter 1 Gene Therapy for Ovarian Cancer Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Gene Therapy for Ovarian Cancer Market Forecast

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Comprehensive Report on Gene Therapy for Ovarian Cancer Market 2020 | Trends, Growth Demand, Opportunities & Forecast To 2026 | Takara Bio, VBL...

Gene therapy reverses memory loss from Alzheimer’s in mice – BioNews

3 August 2020

Memory loss has been reversed in mice with Alzheimer's disease following gene therapy.

A study led bybrothersProfessor Lars Ittner and Dr Arne Ittner of the Macquarie University Dementia Research Centre in Sydney Australia, has shown that the gene therapy not only halts the progression of memory loss, but it can also reverse the effects when applied to mice with advanced Alzheimer's disease.

Discussing the findings, ProfessorIttner said: 'We were completely surprised. They actually recovered their memory function and their ability to learn returned. So, two months after we treated the mice at very old ages, these mice suddenly behaved like their normal siblings.'

By introducing genetic material into the cells of affected mice, the researchers were able to activate an enzymeknown as p38gamma. Previous research by the team revealed that this enzyme, when activated, is protective against the development of Alzheimer's disease. Their latest research builds on this, using gene therapy to enhance the activity of p38gamma in mice with established memory loss.

Results from the study suggest that this gene therapy may be useful in treating other forms of dementia, such as frontotemporal dementia, which typically affects a younger population. As there were no adverse events reported in the mice, even those treated with high doses over a longer period, the team are planning to trial the therapy in humans.

'There is no comparable therapy out there and no other gene therapy either,' said ProfessorIttner. 'This provides hope, as there is a lot of therapy out there focussed on prevention, but not much for those already affected by the disease.'

'It will be exciting to see how over ten years of basic research to understand the mechanisms of Alzheimer's disease will finally transition intoclinical developmentto eventually benefit those most in need, people living with dementia' he added.

The study was published in the journal Acta Neuropathologica.

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Gene therapy reverses memory loss from Alzheimer's in mice - BioNews

Hemophilia Gene Therapy Industry Market Analysis by Size, Share, Growth, Application, Segmentation and Forecast to 2025 – Express Journal

Global Hemophilia Gene Therapy Industry market Report 2020 presents critical information and factual data about the Hemophilia Gene Therapy Industry market, providing an overall statistical study of this market on the basis of market drivers, market limitations, and its future prospects. The widespread Hemophilia Gene Therapy Industry market opportunities and trends are also taken into consideration in Hemophilia Gene Therapy Industry industry. with growth trends, various stakeholders like investors, traders, suppliers, SWOT analysis Opportunities and Threat to the organization and others.

The Hemophilia Gene Therapy Industry market report comprises of the key trends which influence the industry growth with respect to the regional terrain and competitive arena. The study highlights the opportunities that will support the industry expansion in existing and untapped markets along with the challenges the business sphere will face. Besides this, the report also offers an intricate analysis of case studies including those of COVID-19 pandemic, with the aim to provide a clear picture of this industry vertical to all shareholders.

Pivotal pointers from COVID-19 impact assessment:

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Analysis of the regional terrain:

Highlights of the Hemophilia Gene Therapy Industry market report:

Key Coverage of report:

Impact of the latest technological innovations on the Hemophilia Gene Therapy Industry market

Key growth strategies adopted by the prominent market players to address the challenges and restraints put forward by the COVID-19 pandemic

Historical and current trends likely to affect the overall market dynamics of the Hemophilia Gene Therapy Industry market

Growth assessment of the various market segments over the forecast timeline

Regional and global presence of major market players in the Hemophilia Gene Therapy Industry market

Table of Content:

1 Hemophilia Gene Therapy Industry market Introduction and Market Overview

1.1 Objectives of the Study

1.2 Overview of Hemophilia Gene Therapy Industry market

1.3 Scope of The Study

1.3.1 Key Market Segments

1.3.2 Players Covered

1.3.3 COVID-19's impact on the Hemophilia Gene Therapy Industry industry

1.4 Methodology of The Study

1.5 Research Data Source

2 Executive Summary

2.1 Market Overview

2.1.1 Global Hemophilia Gene Therapy Industry market Size, 2015 - 2020

2.1.2 Global Hemophilia Gene Therapy Industry market Size by Type, 2015 - 2020

2.1.3 Global Hemophilia Gene Therapy Industry market Size by Application, 2015 - 2020

2.1.4 Global Hemophilia Gene Therapy Industry market Size by Region, 2015 - 2025

2.2 Business Environment Analysis

2.2.1 Global COVID-19 Status and Economic Overview

2.2.2 Influence of COVID-19 Outbreak on Hemophilia Gene Therapy Industry Industry Development

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Hemophilia Gene Therapy Industry Market Analysis by Size, Share, Growth, Application, Segmentation and Forecast to 2025 - Express Journal

Dyne Therapeutics Appoints Susanna High as Chief Operating Officer – Business Wire

WALTHAM, Mass.--(BUSINESS WIRE)--Dyne Therapeutics, a biotechnology company focused on developing life-transforming therapeutics for patients with serious muscle diseases, today announced that it has appointed Susanna High, MBA, as its chief operating officer. Ms. High has more than two decades of experience leading corporate strategy, portfolio management, business planning and operations for biotechnology companies.

Susannas demonstrated success at innovative biotech companies will be an incredible asset to Dyne as we pursue our goal of building the worlds leading muscle disease company, said Joshua Brumm, president and chief executive officer of Dyne. I look forward to partnering with Susanna and drawing on her depth of experience, particularly in rare diseases, as we work to develop and commercialize modern oligonucleotide therapeutics for serious muscle diseases. I am thrilled to welcome Susanna to the Dyne family.

In her most recent position, Ms. High served as chief operating officer of bluebird bio where her numerous responsibilities included the advancement of the companys severe genetic disease portfolio, leading to the European filing and then approval of ZYNTEGLO, a gene therapy for the treatment of transfusion-dependent beta-thalassemia, as well as overseeing the build-out of its European organization. Before joining bluebird, Ms. High worked in roles of increasing responsibility at Alnylam Pharmaceuticals, including as senior vice president, strategy and business integration, where she led corporate and portfolio strategy, program and alliance management, business planning and information technology. Previously, she supported corporate strategy and business operations at Millennium Pharmaceuticals (now Takeda Oncology). Ms. High holds an M.S. in economics and business management from Bocconi University in Italy and an MBA from the MIT Sloan School of Management.

The progress Dyne has made in its lead programs underscores the potential of the FORCE platform and the companys commitment to transforming the treatment opportunities for individuals living with serious muscle diseases, said Ms. High. This is an exciting time for Dyne as their programs advance towards the clinic, and I am honored to join this committed leadership team to develop potentially life-changing therapies.

About Dyne Therapeutics

Dyne Therapeutics is building a leading muscle disease company focused on advancing innovative life-transforming therapeutics for patients with genetically driven diseases. The Company utilizes its proprietary FORCE platform to overcome the current limitations of muscle tissue delivery with modern oligonucleotide therapeutic candidates. Dyne is developing a broad portfolio of therapeutics for muscle diseases, including lead programs in myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Dyne was founded by Atlas Venture and is headquartered in Waltham, Mass. For more information, please visit http://www.dyne-tx.com, and follow us on Twitter, LinkedIn and Facebook.

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Dyne Therapeutics Appoints Susanna High as Chief Operating Officer - Business Wire

Cell and Gene Therapy Industry Market with manufacturers, Application, regions and SWOT Analysis 2025 – CueReport

Global Cell and Gene Therapy Industry market Report 2020 presents critical information and factual data about the Cell and Gene Therapy Industry market, providing an overall statistical study of this market on the basis of market drivers, market limitations, and its future prospects. The widespread Cell and Gene Therapy Industry market opportunities and trends are also taken into consideration in Cell and Gene Therapy Industry industry. with growth trends, various stakeholders like investors, traders, suppliers, SWOT analysis Opportunities and Threat to the organization and others.

The Cell and Gene Therapy Industry market report comprises of the key trends which influence the industry growth with respect to the regional terrain and competitive arena. The study highlights the opportunities that will support the industry expansion in existing and untapped markets along with the challenges the business sphere will face. Besides this, the report also offers an intricate analysis of case studies including those of COVID-19 pandemic, with the aim to provide a clear picture of this industry vertical to all shareholders.

Request Sample Copy of this Report @ https://www.cuereport.com/request-sample/27662

Pivotal pointers from COVID-19 impact assessment:

Request Sample Copy of this Report @ https://www.cuereport.com/request-sample/27662

Analysis of the regional terrain:

Highlights of the Cell and Gene Therapy Industry market report:

Key Coverage of report:

Impact of the latest technological innovations on the Cell and Gene Therapy Industry market

Key growth strategies adopted by the prominent market players to address the challenges and restraints put forward by the COVID-19 pandemic

Historical and current trends likely to affect the overall market dynamics of the Cell and Gene Therapy Industry market

Growth assessment of the various market segments over the forecast timeline

Regional and global presence of major market players in the Cell and Gene Therapy Industry market

Table of Content:

1 Cell and Gene Therapy Industry market Introduction and Market Overview

1.1 Objectives of the Study

1.2 Overview of Cell and Gene Therapy Industry market

1.3 Scope of The Study

1.3.1 Key Market Segments

1.3.2 Players Covered

1.3.3 COVID-19's impact on the Cell and Gene Therapy Industry industry

1.4 Methodology of The Study

1.5 Research Data Source

2 Executive Summary

2.1 Market Overview

2.1.1 Global Cell and Gene Therapy Industry market Size, 2015 - 2020

2.1.2 Global Cell and Gene Therapy Industry market Size by Type, 2015 - 2020

2.1.3 Global Cell and Gene Therapy Industry market Size by Application, 2015 - 2020

2.1.4 Global Cell and Gene Therapy Industry market Size by Region, 2015 - 2025

2.2 Business Environment Analysis

2.2.1 Global COVID-19 Status and Economic Overview

2.2.2 Influence of COVID-19 Outbreak on Cell and Gene Therapy Industry Industry Development

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Cell and Gene Therapy Industry Market with manufacturers, Application, regions and SWOT Analysis 2025 - CueReport

Investing in Advanced Manufacturing to Support Public Health – FDA.gov

By: Stephen M. Hahn, M.D., Commissioner of Food and Drugs, and Anand Shah, M.D., Deputy Commissioner for Medical and Scientific Affairs

Americans may be surprised to learn that many 21st century medical products are still being manufactured using technologies commonly employed since the middle of the last century. These manufacturing platforms are not dynamic and can increase the risk of shortages, limit flexibility during an emergency, and contribute to the high cost of medical products. For the past several years, the U.S. Food and Drug Administration has sought to encourage and facilitate the adoption of advanced manufacturing, which refers to new and emerging approaches for the production of medical technologies.

Advanced manufacturing approaches are applicable to different medical product areas. For example, process intensification methods, such as continuous manufacturing, can simplify and centralize the production of many essential medicines. Likewise, techniques such as 3D printing can help produce patient-specific medical devices. Furthermore, digital and smart design and manufacturing processes also promise to increase efficiency and reduce uncertainty.

The potential public health value of advanced manufacturing is even greater in the context of the ongoing COVID-19 pandemic, which has highlighted the strain on supply chains and the need for adaptive manufacturing systems to accelerate the production of medical countermeasures. The FDA has established a strong regulatory foundation to support the uptake of advanced manufacturing, and COVID-19 provides the unique impetus to spur further advancement of medical manufacturing.

FDA regulations cover both sides of the innovation equation: development (whether the product meets the appropriate statutory standard) and manufacturing (whether quality products can be produced for widespread use). Many manufacturers continue to use the same production techniques that were developed more than 50 years ago. Typical manufacturing processes that use long shipping lines or outsourced supply chains render U.S. manufacturing vulnerable to delays, disruptions, and quality control issues. These existing supply chain vulnerabilities have been exacerbated during the COVID-19 pandemic. Additionally, batch manufacturing lacks the flexibility needed to sustainably produce therapies for the personalized medicine era; this is a significant concern given that the FDA anticipates approving approximately 40 gene therapies in the next few years.

Advanced manufacturing often enables innovation, increases in efficiency, and improved supply chain resiliency for medical products that provide wide-ranging public health benefits. Over the past decade, the FDA made strategic, forward-looking investments in personnel, policies, and processes to create a clear regulatory pathway for innovators across the three medical product areas of drugs, biologics, and devices.

First, the agency recognized that innovators seeking to adopt advanced manufacturing technologies may be concerned about the technical and regulatory challenges associated with transitioning away from their existing platforms. To this end, the FDAs Center for Drug Evaluation and Research (CDER) created the Emerging Technology Program, which has a dedicated team available to provide pre-submission support on issues such as the development of process control measures for continuous manufacturing of drugs. To provide focused expertise for advanced manufacturing of biological products, the FDAs Center for Biologics Evaluation and Research (CBER) established the Advanced Technologies Team, which works with prospective developers on issues such as technical considerations for platform technologies in gene therapy.

Second, the FDA recognizes that policy must keep pace with innovation. To expedite the development of newer technologies, the agency developed a series of leapfrog guidance documents, which the FDA uses to share initial thoughts regarding emerging technologies that are likely to be of public health importance. Such leapfrog guidance documents include the FDAs Center for Devices and Radiological Healths (CDRH) 2017 guidance on Technical Considerations for Additive Manufactured Medical Devices, which encompasses many technologies including 3D printing. The agency has provided further regulatory clarity as technologies mature and are commercialized; for example CDER issued guidance in 2019 on Quality Considerations for Continuous Manufacturing. The FDAs engagement in public dialogue supports the proactive identification and resolution of potential barriers for the transition to advanced manufacturing.

Third, as a science-based agency, the FDA supports research and partnerships that expand the knowledge base for advanced manufacturing. For example, the FDA has used authorities in the 21st Century Cures Act to award research grants to support investigators exploring key questions around monitoring and control techniques for advanced manufacturing platforms. To foster collaborations across the public, private, and non-profit sector, the FDAs Office of the Chief Scientist (OCS) launched a new program for advancing regulatory science in public health and formed partnerships with stakeholders such as America Makes, BioFab USA, the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and a number of industry organizations and clinical societies. OCS has also spearheaded multiple intramural research programs to help develop the regulatory expertise required to evaluate advanced manufacturing technologies. These initiatives will enable the FDA to identify and address cross-cutting scientific, technical, and regulatory challenges and opportunities for advanced manufacturing.

The COVID-19 pandemic has shown how conventional manufacturing practices and predominantly international supply chains may be a liability for Americas emergency response efforts. In addition to the policy and programmatic foundations described above, the FDA has also taken a number of actions to shore up manufacturing capacity specifically for public health preparedness. For example, CDER entered a multi-year partnership with BARDA, the Biomedical Advanced Research and Development Authority, to explore how continuous manufacturing techniques could improve Americas capacity to rapidly manufacture medical countermeasures during emergency situations. During COVID-19 specifically, OCS and CDRH helped develop a Memorandum of Understanding between the FDA, the National Institutes of Health, and the Department of Veterans Affairs to facilitate information sharing on 3D printing to help support manufacturing of essential medical supplies such as personal protective equipment and medical device parts.

Advances in regulatory science are not an end in themselves. True public health preparedness requires incentives and investments in the technologies that the agency has been promoting for years. Increasing emphasis on domestic manufacturing strengthens our response capability, yet it is not enough to bring supply chains back home. We must also ensure that the renewed focus on the importance of domestic manufacturing capacity is paired with a recognition of the capital requirements and scientific expertise needed to adopt more resilient and efficient platforms.

The FDA is committed to doing its part to foster the adoption of advanced manufacturing technologies. To reduce the burden on innovators, the agency is actively working to ensure international concordance on guidelines for continuous manufacturing as part of the International Council for Harmonisations Q13 proposal. To ensure that best practices are informed by the latest research, the agency is committed to monitoring ongoing grant programs, with the intent of fostering initiatives that can demonstrate tangible improvements in safety, quality, and efficiency. To promote communication about the adoption of best practices and innovative ideas between stakeholders, the FDA will continue to proactively provide forums for scientific discussion, participate in national and international workshops, and collaboratively engage stakeholders.

Because pandemics by nature are unpredictable, our approach to manufacturing must be adaptable. Advanced manufacturing provides an approach for protecting our supply chain and improving our response capacity during crisis situations. By establishing the regulatory foundation, the FDA has created a pathway for industry to continue adopting the needed improvements in manufacturing technology for the benefit of public health.

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Investing in Advanced Manufacturing to Support Public Health - FDA.gov

Genetic Studies Could Pave the Way to New Pain Treatments – Pain News Network

By Dr. Lynn Webster, PNN Columnist

Millions of Americans order DNA test kits to determine their ancestries. Knowing where you come from can be entertaining. However, DNA testing can also help identify your risk of developing some diseases, including chronic pain.

Prenatal testing for genetic disorders is common. But genetic testing is also increasingly used to determine the risk of developing certain diseases or potential responses to specific drugs.

Currently, little is known about how to use genes to make an individual more or less sensitive to pain, or to learn the likelihood that someone will respond in a particular way to an analgesic based on their genetics. The good news is that we are on the cusp of gaining more information about the genes that control pain and pain treatments, and that knowledge should allow us to develop targeted pain therapies.

Most physicians still believe that everyone experiences pain in the same way. Research recently published in Current Biology discovered a genethe so-called "Neanderthal gene"that is associated with increased sensitivity to pain. Recognizing that a mutation of a specific gene can influence pain perception may be illuminating for many members of the medical profession.

Pain specialists have known for a long time that given the same stimulus, some people feel more pain than others. The truth is, there are several genes besides the Neanderthal gene that determine how an individual experiences pain. Some genes increase our sensitivity to pain, while other genes decrease it. Some genes influence how pain is processed, while other genes determine an individual's response to an analgesic.

The ability for an analgesic to provide pain relief in an individual is partially determined by the genetics of the receptor to which the pain medication binds. These genes are different from pain-sensitivity genes. For example, oxycodone may be very effective in relieving pain for one individual, but only partially effective for another.

Optimal pain relief requires recognition that each individual responds uniquely to a given analgesic. Doctors are beginning to provide gene therapy for cancer patients. Advancements in research may someday allow us to do the same for patients with pain.

The array of pain responses to the same stimulus is a major reason why one-size-fits-all dosing of pain medications is flawed. A given dose may leave some patients undertreated and others over-treated. Unfortunately, regulators who set arbitrary dose limits fail to understand or consider this biologic variability.

Differing clinical responses to pain stimuli and medications underscore the need to individualize therapy. Knowing more about the biology of pain can help us to understand each individuals response to painful stimuli and the variable response to any therapy.

How we experience pain is a result of both environmental and genetic features. The genetic factors are what we inherit. Environmental factors which we develop rather than inherit include cultural attitudes, emotions, and individual responses to stress. Our personality and lifes experiences are included in the environmental factors that contribute to our experience of pain. Therefore, pain is a result of genetic and environmental interactions. Both can make an individual more or less sensitive to stimuli or analgesia. It is a complex and dynamic process.

The so-called Neanderthal gene is not a new discovery but was newly recognized in Neanderthals. The discovery is interesting, because it implies the gene has an evolutionary purpose. The gene is known as SCN9. There are several pain syndromes associated with the genetic mutations of the SCN9 gene, including some types of back pain and sciatica. Mutations of this gene can result in the total absence of pain or a heightened pain expression. The type of mutation determines the phenotype (or personal characteristics) of our response to a painful stimulus.

It is unclear how Neanderthals benefited biologically from increased pain sensitivity. As we know, acute pain elicits an alarm and is considered protective. It teaches us to avoid dangers that can threaten our life, and prevents us from walking on a broken leg until it heals sufficiently to bear our weight.

Evolution may not have been concerned about the effects of chronic pain. The Neanderthals' limited life expectancy, and the fact that their survival depended on strong physical conditioning, may have made chronic pain a non-issue. Chronic pain may have made survival difficult, or even impossible, for the Neanderthals.

The recent discovery that Neanderthals had the SCN9 gene should not be surprising, given the fact that modern humans shared a common ancestor with Neanderthals. The Neanderthal gene study is of particular interest to me, because I am working with several companies that are exploring potential drugs to affect the function of the SCN9 gene. The companies have different approaches, but they all are trying to find a way to dial down an individual's sensitivity to painful stimuli.

Since the SCN9 gene can be responsible for the total absence of all pain, as well as several extreme forms of pain, it may be reasonable to target the SCN9 gene to modulate pain.

My hope is that manipulation of the SCN9 gene will reduce pain sensitivity, making it easier to control pain by adjusting the dose and type of drug we prescribe.

It is possible one or more drugs that target the SCN9 gene will be available within the next 4-6 years. If that occurs, it could be game changer for people in pain. We can then thank our Neanderthal ancestors for the evolutionary gift.

Lynn R. Webster, MD, is a vice president of scientific affairs for PRA Health Sciences and consults with the pharmaceutical industry. He is author of the award-winning book, The Painful Truth, and co-producer of the documentary, It Hurts Until You Die. You can find Lynn on Twitter: @LynnRWebsterMD

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Genetic Studies Could Pave the Way to New Pain Treatments - Pain News Network

Oxford Biomedica Signs Development, Manufacture & License Agreement with Beam Therapeutics Inc., for LentiVector Platform for Next Generation…

Oxford Biomedica Signs Development, Manufacture & License Agreement with Beam Therapeutics Inc., for LentiVector Platform for Next Generation CAR-T Therapeutics

Oxford, UK 3 August 2020: Oxford Biomedica plc (LSE:OXB) (Oxford Biomedica or the Group), a leading gene and cell therapy group, announced today that it has signed a new Development, Manufacture & License Agreement (DMLA) with Beam Therapeutics Inc. (Beam) (Nasdaq: BEAM), a Cambridge, Mass.-based biotechnology company developing precision genetic medicines through the use of base editing. The DMLA grants Beam a non-exclusive license to Oxford Biomedicas LentiVector platform for its application in next generation CAR-T programmes in oncology and puts in place a three year Clinical Supply Agreement.

Under the terms of the DMLA, Oxford Biomedica will receive an undisclosed upfront payment, as well as payments related to development and manufacturing of lentiviral vectors for use in clinical trials, and certain development and regulatory milestones for products sold by Beam that utilise Oxford Biomedicas LentiVector platform and an undisclosed royalty on the net sales of products sold by Beam that utilise the Groups LentiVector platform.

Oxford Biomedica is currently working on one pre-clinical programme with Beam, and the Agreement allows for the Parties to initiate additional projects in the future.

John Dawson, Chief Executive Officer of Oxford Biomedica, said: Beam Therapeutics is one of the leading next-generation CAR-T developers who deploy a wide range of innovative technologies to bring innovative CAR-T products into development. We are proud to be working with a leader in the field of gene editing technologies, including base editing, and this provides us another valuable opportunity for our LentiVector platform to support innovative product development of CAR-T products.

This is our third announced partnership with leaders in the CAR-T field, building on our longstanding partnership with Novartis and our more recently announced partnership with Bristol Myers Squibb earlier this year. We look forward to supporting the next generation CAR-T programmes at Beam.

-Ends-

Oxford Biomedica plc

John Dawson, Chief Executive OfficerStuart Paynter, Chief Financial OfficerCatherine Isted, Head of Corporate Development & IR

T: +44 (0)1865 783 000T: +44 (0)1865 783 000T: +44 (0)1865 954 161 / E: ir@oxb.com

Consilium Strategic Communications

Mary-Jane Elliott/Matthew Neal

T: +44 (0)20 3709 5700

About Oxford BiomedicaOxford Biomedica (LSE:OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford Biomedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology, CNS disorders, liver diseases and respiratory disease. The Group has also entered into a number of partnerships, including with Novartis, Bristol Myers Squibb, Sanofi, Axovant Gene Therapies, Orchard Therapeutics, Santen, Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium and Imperial Innovations, through which it has long-term economic interests in other potential gene and cell therapy products. Additionally the group has signed a Clinical and Commercial Supply Agreement with AstraZeneca for manufacture of the adeno based COVID-19 vaccine candidate, AZD1222. Oxford Biomedica is based across several locations in Oxfordshire, UK and employs more than 550 people. Further information is available atwww.oxb.com

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Oxford Biomedica Signs Development, Manufacture & License Agreement with Beam Therapeutics Inc., for LentiVector Platform for Next Generation...

AZ partners with Daiichi on ADC therapy – BioPharma-Reporter.com

AstraZeneca has returned to commit approximately $6bn (5.1bn) on Daiichi Sankyos antibody drug conjugate (ADC), after completing another separate multi-billion dollar deal last year with the company.

Yesterdays agreement sees AZ pay Daiichi $1bn upfront, due in three intervals, a further $1bn tied to regulatory approvals, and up to $4bn in sales-related milestones. In return, AZ gets access to DS-1062, a trophoblast cell-surface antigen 2-directed ADC.

The drug candidate is being lined up as a treatment for multiple tumor types, with Daiichi already running Phase I trials against non-small cell lung cancer and exploring the possibility to target breast cancers.

Both companies will jointly develop and commercialize the treatment candidate, except in Daiichis home market, Japan, where it will retain exclusive rights. Daiichi will also be responsible for the production and supply of DS-1062.

With this latest deal, AZ has strengthened its existing collaboration with Daiichi, after the two companies announced a potential $7bn deal last year for another ADC, which the partners recently commercialized as Enhertu (trastuzumab deruxtecan).

The treatment was the lead drug in Daiichis ADC pipeline, with it being approved initially in HER2-positive metastatic breast cancer but going through additional Phase III trials to expand its indication.

As a result of both deals, AZs CEO, Pascal Soriot, was able to point to six potential blockbusters that the company has in its portfolio, whilst also noting the potential of its early- and late-stage pipeline.

The company has been working to bolster its pipeline in oncology through a number of smaller partnership and acquisition deals, which saw AZ acquire the rights to Innates potential first-in-class treatment, currently in Phase II trials, and agree a deal to develop oncolytic virus candidate alongside Transgene.

For Daiichi, the deal means that it has the capital for the further development of its ADC programs and to make deals to expand its own pipeline, such as bolstering its expansion into the gene therapy area.

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AZ partners with Daiichi on ADC therapy - BioPharma-Reporter.com

Getting gene therapy to the brain | Penn Today – Penn Today

A lone genetic mutation can cause a life-changing disorder with effects on multiple body systems. Lysosomal storage diseases, for example, of which there are dozens, arise due to single mutations that affect production of critical enzymes required to metabolize large molecules in cells. These disorders affect multiple organs including, notably, the brain, causing intellectual disability of varying degrees.

Gene therapy holds promise to address these conditions, but the brains own protective mechanismthe blood-brain barrierhas been a formidable challenge for researchers working to develop one.

In a new study published in the journal Brain, a team led by John H. Wolfe, a researcher with Penns School of Veterinary Medicine and Perelman School of Medicine and the Childrens Hospital of Philadelphia, successfully applied a gene therapy platform to completely correct brain defects in a large animal model of a human genetic disease.

This is the first example of a large-brain mammal with a bona fide human genetic disease that has intellectual disability as part of the human syndrome where weve been able to correct the biochemistry and pathologic lesions in the whole brain, says Wolfe.

Wolfe has worked on models of human genetic diseases that impact the brain for many years. With gene therapy, a delivery vehicletypically a viral vectoris used to provide the normal version of a mutated gene to correct a condition. Wolfe and other scientists working in this area have made steady progress to treat neurogenetic diseases in rodents. However, applying the same treatment to the much larger brain of higher mammals has only been able to produce partial corrections.

Theres been a lot of excitement for the last 10 years or so that specific vectors can be injected into the blood and enter the brain, says Wolfe. They do cross the blood-brain barrier. One such treatment with restricted distribution has been effective in treating a disease that primarily affects the spinal cord.

And while scientists have shown these therapies can reverse the pathology throughout the brains of mice, its been hard to judge what effect it would have in patients, as the rodent brains have a much smaller cerebral cortex than larger mammals, like humans.

In the current study, the team used an animal model with a brain more similar to humans, cats, to assess the effectiveness of a gene-correcting therapy for one type of lysosomal storage disease: a condition called alpha-mannosidosis, which naturally occurs in cats and results from a mutated copy of the alpha-mannosidase gene.

Having refined the gene-delivery technique during many years of work, the researchers selected a specific vector that they showed, in mice, was capable of crossing the blood-brain barrier to reach sites throughout the brain.

They next delivered the vector, containing a reporter gene, to normal cats. Several weeks later, they were able to find evidence that the corrected gene had distributed to various parts of the brain, including the cerebral cortex, hippocampus, and mid-brain.

Finally the research team assessed the therapy in cats with alpha-mannosidosis, using either a low or high dose of the vector. They injected the therapy into the carotid artery, so that it would go directly to the brain before traveling to other parts of the body. Compared to untreated cats, treated animals had a significant delayed onset of certain neurological symptoms and a longer lifespan; those that received the higher dose of the vector delivered through the carotid artery lived the longest.

Its a big advance, says Wolfe. Nobody has been able to treat the whole brain of a large-brained animal before. Were hopeful that this will translate into clinical use in humans.

Wolfe cautions, however, the findings dont amount to a cure.

These were significant improvements, but they were only just improvements on a serious condition, Wolfe says. The cats werent cured, and we dont know what impact this has on mental ability. However, since the pathology is found throughout the brain, it is thought that complete correction will be necessary.

As alpha-mannosidosis is a childhood-onset disease with no cure, however, any improvements that lessen the severity of symptoms are welcome. The approach the researchers developed may potentially be employed to treat many other diseases that affect the whole central nervous system.

In future work, Wolfe and his collaborators hope to refine their methods to achieve the same outcomes with a lower dose, making an effective treatment safer as well as more affordable. And they will continue to work to understand the details of why their treatment works, including precisely how the vector travels through the brain, a line of investigation that could shed light on additional strategies to address these serious disorders.

John H. Wolfe is a Stokes Investigator of the Childrens Hospital of Philadelphia Research Institute, director of the Walter Flato Goodman Center for Comparative Medicine Genetics at the School of Veterinary Medicine, and a professor of pathology and medical genetics in Penn Vets Department of Pathobiology and in the Perelman School of Medicines Department of Pediatrics.

Wolfes coauthors on the work were Sea Young Yoon, Jacqueline E. Hunter, Sanjeev Chawla, Dana L. Clarke, Caitlyn Molony, Patricia A. ODonnell, Jessica H. Bagel, Manoj Kumar, Harish Poptani, and Charles H. Vite.

The work was supported by the National Institutes of Health (grants DK063973, OD010939, TR001878, and NS007180).

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Getting gene therapy to the brain | Penn Today - Penn Today

Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative…

MALVERN, Pa., July 27, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced the U.S. Food and Drug Administration (FDA) granted the third Orphan Drug Designation (ODD) for OCU400 in the treatment of RHO mutation-associated retinal degeneration. The RHO mutation is part of the Retinitis Pigmentosa (RP) group of rare, genetic disorders that involve a breakdown and loss of cells in the retina and can lead to visual impairment and blindness. This is one of the larger mutations within the RP class, representing about 12% of RP patients in the US.

A novel gene therapy product candidate, OCU400 has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor (NHR) gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis and potentially offer longer benefit, stabilizing cells and rescuing photoreceptor degeneration and vision loss.

Adding to ODDs for OCU400 for NR2E3 and CEP290 mutation-associated retinal degeneration, RHO gene mutation-associated retinal degeneration further supports Ocugens breakthrough modifier gene therapy platforms potential to treat multiple blindness diseases with a single product. RP is a group of heterogenic inherited retinal diseases associated with over 150 gene mutations, affecting over 1.5 million individuals worldwide. In addition, ~40% of RP patients cannot be genetically diagnosed, confounding the ability to develop personalized RP therapies. Traditional gene therapy or gene editing approaches may require more than 150 products to rescue these patients from vision loss. OCU400, a single product candidate, has potential to address broad-spectrum RP.

OCU400, comprising the nuclear hormone receptor geneNR2E3, has the potential to help modulate numerous biological pathways that function in maintaining the health of the retina. A recent preclinical study published in Nature Gene Therapy demonstrated the potency of NR2E3 to elicit broad-spectrum therapeutic benefits in early and intermediate stages of RP in five unique mouse models, said Dr. Mohamed Genead, acting Chief Medical Officer of Ocugen and Chair of Ocugens Retina Scientific Advisory Board. We believe OCU400 has the potential to address multiple genetic mutations associated with RP and, therefore, help a broader pool of patients, Dr. Genead continued.

Our third ODD for OCU400 from the FDA is an important step towards developing a broad-spectrum treatment for RP and getting a therapy faster to patients who are in desperate need of rescue, said Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen. Orphan designation for this indication supports the goal of our Modifier Gene Therapy Platform to treat a variety of inherited retinal diseases with a single gene therapy product. There are currently no approved treatments which slow or stop the progression of multiple forms of RP, which is why were excited to have a platform that can potentially address multiple mutations, including mutations in the Rhodopsin gene, with one therapy.

The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. Orphan designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including a seven-year period of U.S. marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding and waiver of Prescription Drug User Fee Act filing fees.

About OCU400OCU400 (AAV-hNR2E3) is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration and vision loss.

About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. For more information, please visithttps://ocugen.com/

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact: LaVoieHealthScienceEmmie Twomblyetwombly@lavoiehealthscience.com+1 857-389-6042

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Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative...