Axovant Signs Three-Year Clinical Supply Agreement With Oxford Biomedica for Manufacturing and Supply of AXO-Lenti-PD – BioSpace

NEW YORK and BASEL, Switzerland, July 31, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (NASDAQ: AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, announced today that its subsidiary has signed a three-year Clinical Supply Agreement (CSA) with Oxford Biomedica plc (LSE:OXB), a leading gene and cell therapy group. 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 the Phase 2 SUNRISE-PD trial with AXO-Lenti-PD. Dosing of all patients in the second cohort has been completed with 6-month safety and efficacy data expected in the fourth quarter of 2020. Oxford Biomedica 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.

This Agreement with Oxford Biomedica means that together we can continue to advance the development of AXO-Lenti-PD in Parkinsons disease, said Pavan Cheruvu, Axovant Chief Executive Officer. 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.

John Dawson, CEO of Oxford Biomedica, added, 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 commercialization and we look forward to this next phase of our partnership.

About Axovant Gene Therapies

Axovant 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 target 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.

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 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 at http://www.oxb.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "might," "will," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovants management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of its gene therapy product candidates and platforms; Axovants scientific approach and general development progress; and the availability or commercial potential of Axovants product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovants most recent Annual Report on Form 10- K filed with the Securities and Exchange Commission on June 10, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media & Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523media@axovant.cominvestors@axovant.com

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Axovant Signs Three-Year Clinical Supply Agreement With Oxford Biomedica for Manufacturing and Supply of AXO-Lenti-PD - BioSpace

Gene Therapy for Inherited Genetic Disorders Market size in terms of volume and value 2019-2025 – Market Research Correspondent

Global Gene Therapy for Inherited Genetic Disorders market- Report defines the vital growth factors, opportunities and market segment of top players during the forecast period from 2019 to 2025. The report Gene Therapy for Inherited Genetic Disorders offers a complete market outlook and development rate during the past, present, and the forecast period, with concise study, Gene Therapy for Inherited Genetic Disorders market effectively defines the market value, volume, price trend, and development opportunities. The comprehensive, versatile and up-to-date information on Gene Therapy for Inherited Genetic Disorders market is provided in this report.

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The key players covered in this studyBioMarin Pharmaceutical Inc.bluebird bio Inc.Novartis AGOrchard Therapeutics PlcSpark Therapeutics Inc.

Market segment by Type, the product can be split intoEye DisordersHematological DisordersCentral Nervous System DisordersMuscular DisordersOthersMarket segment by Application, split intoHospitalClinicResearch InstituteOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

The study objectives of this report are:To analyze global Gene Therapy for Inherited Genetic Disorders status, future forecast, growth opportunity, key market and key players.To present the Gene Therapy for Inherited Genetic Disorders development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.To strategically profile the key players and comprehensively analyze their development plan and strategies.To define, describe and forecast the market by type, market and key regions.

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In continuation using earnings, this section studies consumption, and global Gene Therapy for Inherited Genetic Disorders market. This area also sheds light on the variance between ingestion and distribution. Export and Gene Therapy for Inherited Genetic Disorders significance data are provided in this part.

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Gene Therapy for Inherited Genetic Disorders Market size in terms of volume and value 2019-2025 - Market Research Correspondent

Greenwich swimmers take the plunge to fight cancer – Greenwich Time

By David Fierro and Matthew Brown

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of Greenwich's own Julian Fraser, who lost his life battling a rare form of cancer, raised over $45,000 for cancer research. Over 50 swimmers from Rocky Point Club and Greenwich High School's Swim and Water Pola teams participated in the 1-mile swim in the Long Island Sound. Several volunteers in kayaks and on paddle boards, Including Julian mother, Cristy Fraser, kept watch over the group during the benefit swim.

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of

Photo: Matthew Brown / Hearst Connecticut Media

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of Greenwich's own Julian Fraser, who lost his life battling a rare form of cancer, raised over $45,000 for cancer research. Over 50 swimmers from Rocky Point Club and Greenwich High School's Swim and Water Pola teams participated in the 1-mile swim in the Long Island Sound. Several volunteers in kayaks and on paddle boards, Including Julian mother, Cristy Fraser, kept watch over the group during the benefit swim.

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of

Greenwich swimmers take the plunge to fight cancer

GREENWICH A team of over 50 swimmers and 15 volunteers joined forces to raise more than $45,000 in the fight against cancer and to honor a 2014 Greenwich High graduate and standout swimmer who died of osteosarcoma.

The group known as Team Julian team gathered Saturday at Rocky Point Club in Old Greenwich for the endurance swim on Long Island Sound to benefit Swim Across America Fairfield County.

The event honored the legacy of Julian Fraser, who lost his battle with a rare form of bone cancer in 2017 at the age of 20.

The swimmers from Rocky Point Club and Greenwich High School's Swim and Water Pola teams participated in the 1-mile swim in the Long Island Sound. Several volunteers in kayaks and on paddle boards, including Julian Frasers mother, Cristy Fraser, kept watch over the group during the long benefit swim.

Julian Fraser earned All-America honors as a swimmer and water polo player. He competed on numerous championship teams for GHS coach Terry Lowe, then excelled on the Santa Clara University water polo team.

We are united to raise money for the fight against cancer and to honor Julian, said Lowe, who is one of the organizers of Team Julian.

Proceeds from the event supports the Stamford-based Alliance for Cancer Gene Therapy, the nations only foundation dedicated exclusively to funding cancer gene therapy research.

To learn more about Swim Across America Fairfield County or make a donation, visit swimacrossamerica.org/fc.

dfierro@greenwichtime.com

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Greenwich swimmers take the plunge to fight cancer - Greenwich Time

Gene Therapy Market Professional Industry Overview 2020 Segmentation and Development Analysis BY 2027 – Science Examiner

Impact Analysis of Covid-19

The complete version of the Report will include the impact of the COVID-19, and anticipated change on the future outlook of the industry, by taking into the account the political, economic, social, and technological parameters.

Get Sample PDF Including COVID19 Impact Analysis Of Gene Therapy, https://www.coherentmarketinsights.com/insight/request-pdf/1774

The Gene Therapy Market is expected to have a highly positive outlook for the next five years 2020-2027 according to a recently released Gene Therapy Market research report. The report has been added to his large database by Coherent Market Insights. This report is a guide that covers key strategic developments of the market including acquisitions & mergers, new technology launch, agreements, partnerships, collaborations & joint ventures, research & development, technology, and regional expansion of major participants involved in the market on the global and regional basis.

It also going to elaborate the opportunities out there in micro niches for stakeholders to take a position, step by step investigation of the competitive landscape and even commodity professional services of famous players which include GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

The key features of the Gene Therapy Market report 2020-2027 are the organization, extensive amount of analysis, and data from previous and current years as well as forecast data for the next five years. Most of the report is made up of tables, charts, and figures that give our clients a clear picture of the market.

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The research report on the Gene Therapy market evaluates the growth trends of the industry through historical study and estimates future prospects based on comprehensive research. The report extensively provides market share, growth, trends, and forecasts for the period 2020-2027. The market size in terms of revenue (USD MN) is calculated for the study period along with the details of the factors affecting the market growth (drivers and restraints).

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Part 01: Executive SummaryPart 02: Scope Of The ReportPart 03: Research MethodologyPart 04: Market Landscape Market Ecosystem Market Characteristics Market Segmentation Analysis

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Part 11: Market TrendsPart 12: Vendor Landscape Overview Landscape Disruption Vendors Covered Vendor Classification Market Positioning Of Vendors

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Coherent Market Insights is a global market intelligence and consulting organization focused on assisting our plethora of clients achieve transformational growth by helping them make critical business decisions. We are headquartered in India, having office at global financial capital in the U.S. and sales consultants in United Kingdom and Japan. Our client base includes players from across various business verticals in over 150 countries worldwide. We pride ourselves in catering to clients across the length and width of the horizon, from Fortune 500 enlisted companies, to not-for-profit organization, and startups looking to establish a foothold in the market. We excel in offering unmatched actionable market intelligence across various industry verticals, including chemicals and materials, healthcare, and food & beverages, consumer goods, packaging, semiconductors, software and services, Telecom, and Automotive. We offer syndicated market intelligence reports, customized research solutions, and consulting services.

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Gene Therapy Market Professional Industry Overview 2020 Segmentation and Development Analysis BY 2027 - Science Examiner

Cell and Gene Therapy Market Overview, Growth & Advancement to (2020-2025) – Owned

Worldwide Cell and Gene Therapy Market report gives an in-depth review of the dynamics of the industries, which impacts on the increase of companies. The observe contains a mix of various segments inclusive of drivers, restraints, and possibilities. Special scenarios are examined on this document at the side of the highest driving elements which offers the approaches for enterprise growth. The effective strategies are implemented via the varied pinnacle-degree key gamers to shape the companies .

A comprehensive and elaborate primary analysis report highlights numerous facts like development factors, business improvement policies, analytical growth, economic profit or loss to assist readers and clients to know the market on a worldwide scale. Cell and Gene Therapy Market report gives details about the highest key players and makes that are driving the market. The report is completed after careful analysis of the collected information in various categories of the market that necessities innovative thoughts, hypothetical investigation, and its importance. The team of researchers and analysts presents the readers accurate statistics and analytical data within the report during a simple manner by means of graphs, diagrams, pie charts, and other pictorial illustrations.

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The global Cell and Gene Therapy Market report evaluates various factors related to growth, including pricing structure, production capabilities, demand-supply scenarios and profit margins. The whole research intelligence is predicated on an exhaustive primary industry research and in-depth proactive secondary research that aim to extract valued data points about Cell and Gene Therapy Market. The resultant data enables report readers to position themselves as potential market entrants and devise growth strategies to satisfy short- and long-term business goals.

Amgen Inc., bluebird bio, Inc., Dendreon Pharmaceuticals LLC., Fibrocell Science, Inc., Human Stem Cells Institute, Kite Pharma, Inc., Kolon TissueGene, Inc., Novartis AG, Orchard Therapeutics plc., Organogenesis Holdings Inc., Pfizer, Inc., RENOVA THERAPEUTICS

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Table of Contents:

Global Cell and Gene Therapy Market Research Report 2020

Chapter 1 About the Cell and Gene Therapy Market Industry

Chapter 2 World market Competition Landscape

Chapter 3 World Cell and Gene Therapy Market share

Chapter 4 Supply Chain Analysis

Chapter 5 Company Profiles

Chapter 6 Globalisation & Trade

Chapter 7 Distributors and Customers

Chapter 8 Import, Export, Consumption and Consumption Value by Major Countries

Chapter 9 World Cell and Gene Therapy market Forecast through during years

Chapter 10 Key success factors and market Overview REPORT HIGHLIGHTS :

KEY QUESTIONS ANSWERED IN THE REPORT:

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Cell and Gene Therapy Market Overview, Growth & Advancement to (2020-2025) - Owned

Movers & Shakers, July 31 | BioSpace – BioSpace

ASCO-Jonathan W. Friedberg has been appointed as the next editor-in-chief of theJournal of Clinical Oncology(JCO), the flagship journal of the American Society of Clinical Oncology (ASCO).JCOpublishes cutting-edge research on the diagnosis and treatment of patients with cancer and is one of the most highly cited oncology journals in the world. A hematologic oncologist for more than 20 years, Friedberg is currently director of the Wilmot Cancer Institute and Samuel Durand Professor of Medicine at the University of Rochester Medical Center in Rochester, New York. Friedbergs five-year term asJCOeditor-in-chief will begin in June 2021. He will succeed Stephen A. Cannistra, who will be completing his 10th year as the journals editor-in-chief.

Apic Bio Cambridge, Mass.-based Apic Bio formed a Scientific Advisory Board (SAB) which includes internationally recognized gene therapy experts. TheSAB will provide clinical and scientific expertise to help the company advance its pipeline and leverage its proprietary silence and replace THRIVE platform. The appointments to Apics Scientific Advisory Board include Robert H. Brown Jr., Leo P. and Theresa M. LaChance Chair in Medical Research, Professor of Neurology, Director of the Program in Neurotherapeutics, University of Massachusetts Medical School; R. Jude Samulski, president and chief scientific officer at AskBio; Barry Byrne, director of the Powell Gene Therapy Center at the University of Florida and Professor of Pediatrics and Molecular Genetics & Microbiology; Rob Kotin, adjunct professor at University of Massachusetts Medical School; Kevin Flanigan, Robert F. and Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research and Director of the Center for Gene Therapy in The Abigail Wexner Research Institute at Nationwide Childrens Hospital, and professor of Pediatrics and Neurology at The Ohio State University College of Medicine; Mark Kay, Dennis Farrey Family Professor in Pediatrics and Professor of Genetics at Stanford University School of Medicine.

I-Mab Shanghai-based I-Mab named Ivan Yifei Zhuas its chief commercial officer effectiveAug. 10. In this role, Zhu will focus on building and developing I-Mab's commercialization infrastructure and strategies and preparing the company for upcoming product launches. Before joining I-Mab, he served as vice president and General Manager of the sales division of Qilu Pharmaceutical Group where he managed the company's sales and marketing team. Zhu also served as the CCO of BeiGene where he played an instrumental role in the expansion of BeiGene's commercialization team and the implementation of its commercialization strategies.

Caladrius Biosciences Michael H. Davidson was named to the board of directors of Caladrius Biosciences. Davidson is the founder and chief scientific officer of Corvidia Therapeutics, which recently announced its sale to Novo-Nordisk for $2.1 billion. Davidson also serves as clinical professor and director of the Lipid Clinic at the University of Chicago Pritzker School of Medicine.

Cidara Therapeutics-Cidara Therapeutics announced the formation of a scientific advisory board (SAB) and the appointment of four leading experts in viral and fungal infections and hematology. TheSABwill work closely with the Cidara management team to help guide the direction and design of the companys development programs with an emphasis on the continued development and expansion of the companys Cloudbreak Antiviral Conjugate (AVC) program.Cidara TherapeuticsSABincludes Frederick G. Hayden,Stuart S. Richardson Professor Emeritus of Clinical Virology atUniversity of Virginia School of Medicine; Professor Emeritus of Medicine at the University of Virginia; Michael G. Ison, professor, Divisions of Infectious Diseases and Organ Transplantation atNorthwestern University Feinberg School of Medicine; Medical Director, Transplant & Immunocompromised Host Infectious Diseases Service at Northwestern University Comprehensive Transplant Center,Chicago; Johan A Maertens,professor of Internal Medicine and Hematology, Acute Leukemia and Transplantation Unit at University Hospitals Leuven;Department of Microbiology, Immunology and Transplantationat KULeuven, Leuven,Belgium; Kieren Marr, Professor of Medicine and Oncology Director, Transplant and Oncology Infectious Diseases Program and Vice-Chair for Innovation in Healthcare Implementation,Department of MedicineatJohns Hopkins University School of Medicine.

Alydia Health Following a $13.9 million Series C financing round, Alydia Health announced Rob Binney as its new chief executive officer. Binney joins the company from Intersect ENT where he served as chief commercial officer. Prior to Intersect ENT, Binney worked in leadership roles with both startups and large medical device companies, including Boston Scientific and AccessClosure, now part of Cardinal Health. Binney succeeds Anne Morrissey, who recently stepped down as the companys CEO. Colby Holtshouse, previously Alydias interim CEO and head of Marketing, will assume the role of chief operating officer. In association with the financing round, Zina Affas Besse, managing partner at Global Health Funds will join the companys board of directors.

Synlogic, Inc. Antoine Tony Awad was named chief operating officer of Synlogic. As COO, Awad will drive Synlogic's end to end development & manufacturing organization and supply chain as well as have broader corporate operations responsibilities, inclusive of laboratory operations, IT infrastructure, and the quality organization. Awad joined Synlogic in December 2018 as head of Technical Operations. Prior to joining Synlogic, he served as senior vice president of CMC and Operations at Abpro Therapeutics and L.E.A.F. Pharmaceuticals and served in roles of increasing responsibility at Ipsen Biosciences and Merrimack Pharmaceuticals. In addition to Awad, Synlogic also named Andrew March as head of Development Operations. Prior to joining Synlogic March served as Ra Pharmaceuticals' head of Clinical Development.

Engrail Therapeutics San Diego-based Engrail announced two key executive appointments. Kimberly Vanover has been named chief scientific officer, leading the building and advancement of the companys pipeline. Eve Taylor has been named vice president of clinical development, overseeing all facets of Engrails clinical development programs and operations. Stephen Cunningham, previously executive vice president, development and CSO, will assume the role of chief development officer. Vanover most recently served as senior vice president, early-stage clinical development and translational medicine at Intra-Cellular Therapies. Taylor most recently helped lead early-stage clinical development at Intra-Cellular Therapies. Previously, Taylor led clinical development activities at Cytori Therapeutics, Ceregene and ACADIA Pharmaceuticals. The company also expanded its board of directors to five members. Tony Ho was appointed to Engrails board of directors effective immediately. Ho served as CRISPR Therapeutics executive vice president, research and development since August 2017. Prior to joining CRISPR, Tony held a number of roles at AstraZeneca, most recently senior vice president and head of oncology integration and innovation.

CressetU.K.-based Cresset announced its SAB. The Cresset Scientific Advisory Board members are Stevan W Djuric, chairman of Discovery Chemistry and Technology Consulting LLC, USA;Richard Lewis of Novartis Pharma AG; Alexander Hillisch of Bayer; AstraZenecas Andreas Bender; Julien Michel of the University of Edinburgh; Paul Czodrowski of TU Dortmund University in Germany; and Kazuyoshi Ikeda of Keio University in Japan.

NeuBase Therapeutics William Mann was named chief operating officer of NeuBase Therapeutics. Mann will leverage his decades of experience in the biopharma industry to manage NeuBase operations and advance its strategic goals. Mann most recently served as the president and CEO of Helsinn Therapeutics. Before joining Helsinn, he held multiple positions at Sapphire Therapeutics, Inc.

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Movers & Shakers, July 31 | BioSpace - BioSpace

REGENXBIO To Host Conference Call on August 6 to Discuss Second Quarter 2020 Financial Results and Recent Operational Highlights – Herald-Mail Media

ROCKVILLE, Md., July 30, 2020 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAVTechnology Platform, today announced that it will host a conference call on Thursday, August 6, 2020, at 4:30 p.m. ET to discuss its financial results for the quarter ended June 30, 2020, and recent operational highlights.

To access the live call by phone, dial (800) 708-4539 (domestic) or (800) 619-6396 (international) and enter the passcode 49857700. To access a live or recorded webcast of the call, please visit the Investors section of the REGENXBIO website at http://www.regenxbio.com. The recorded webcast will be available for approximately 30 days following the call.

AboutREGENXBIO Inc.

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

Contacts:

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

Investors:Eleanor Barisser, 212-600-1902eleanor@argotpartners.com

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

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REGENXBIO To Host Conference Call on August 6 to Discuss Second Quarter 2020 Financial Results and Recent Operational Highlights - Herald-Mail Media

Catharpin mom helps raise national awareness for her young sons disease – WDVM 25

Neimann-Pick disease type C1 is a rare genetic condition that only has 500 known cases world wide.

by: Christy Matino

CATHARPIN, Va. (WDVM) Catharpin resident Meghann Ferguson is trying to bring national awareness to Neimann-Pick disease type C1, a rare genetic condition that only has 500 known cases world wide.

The disease affects the bodys ability to metabolize fat. This collection of fat can lead to enlargement of the liver and spleen and can cause difficulties with fine motor skills and cognitive impairment.

These are the symptoms that Meghann noticed when her son Liam was born, just 24 hours after his birth. Liam was also in liver failure, and the Fergusons were looking into a liver transplant.

We heard the words that we were being told genetic, degenerative, fatal those types of words, and they really hit hard, she said.

Since Liams diagnosis, Meghann has been an advocate for Niemann-Pick disease nationally. She joined the board of the National Niemann-Pick Disease Foundation and has had multiple speaking engagements.

One was at the Georgetown University Hospital, and then we also spoke at a gene therapy company in North Carolina. I was also able to present his story at the NNPDF Conference, which is the national organization for this disease, Ferguson said.

The goal is to bring more awareness to the disease, since it is so easily misdiagnosed and difficult to recognize.

The more that people are aware that this condition exists, then I think that doctors, when they see it, will think about it as a possibility, she said.

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Catharpin mom helps raise national awareness for her young sons disease - WDVM 25

Spinraza-Zolgensma Combination Well-tolerated in Children with SMA… – SMA News Today

Combining Spinraza(nusinersen) with the gene therapyZolgensma(onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children withspinal muscular atrophy(SMA) type1, according to a case series study.

The data, which included children treated with Zolgensma at older ages than those reported in clinical trials, suggested that older patients may be at a higher risk of developing gene therapiesknown side effects, such as liver dysfunction and low platelet counts.

However, further studies are needed to better characterize combination therapies safety and determine whether it is more beneficial than single therapy, especially considering their high cost, researchers noted.

The case series study, Combination molecular therapies for type 1 spinal muscular atrophy, was published in the journal Muscle & Nerve.

Currently available SMA therapies Biogens Spinraza andNovartis Zolgensma both work to restore the levels ofSMN, the protein lacking in SMA patients due tomutations in theSMN1gene.

Spinraza, the firstapproved disease-modifying therapy for all SMA patients, targetsSMN2, a backup gene that can compensate partially for the loss ofSMN1-derived SMN. The therapy is given directly into the spinal canal every four months.

In contrast, Zolgensma is administered directly into the bloodstream and delivers a functional copy ofSMN1to cells. It is available to children up to age 2 in theU.S.andJapan,and to almost all SMA types in those weighing up to 21 kilograms (about 46 pounds) inEurope.

The gene therapy can be given only once, due to the bodys natural immune reaction and production of antibodies against the modified virus it uses to deliver the gene to cells.

Nevertheless, immune reactions can still occur after the single dose, which can raise the levels of liver enzymes an indicator of liver damage and drop those of platelets.For that reason, it is recommended that patients liver function and platelet counts are monitored before, and at regular periods after, treatment.

While treatment combination aiming at increasing or sustaining motor function improvements is likely to occur in these patients, there is limited data on its safety and effectiveness.

Researchers now have reported the effects of combination therapy in five children with type 1 SMA followed at the Arkansas Childrens Hospital of the University of Arkansas for Medical Sciences and the Ann and Robert H. Lurie Childrens Hospital of Chicago.

Patients, who were 17 to 29 months of age at last assessment, received first treatment between 1.5 and 7 months and second therapy between 9 and 23 months.

Four children were treated first with Spinraza and then with Zolgensma, and three of them continued Spinraza treatment after that. One child received Zolgensma first, followed by Spinraza, and was the only one to receive the gene therapy within the age range (6.5 months) studied in clinical trials.

After the combination therapy, all patients continued to show motor function improvements, as assessed by theChildrens Hospital of Philadelphia Infant Test of Neuromuscular Disorders and the Hammersmith Infant Neurological Examination Part 2.

Increased levels of liver enzymes, indicating liver dysfunction, were detected in the four children who received Zolgensma after Spinraza and were successfully normalized withprednisolone treatment (recommended when given gene therapy).

Prednisolone therapy was prolonged beyond the previously reported duration range with Zolgensma (one to four months) in two patients who were hospitalized due to liver problems.

The researchers suspected that this rise in liver enzymes was related mainly to Zolgensma and noted that the fast reintroduction of Spinraza after Zolgensma may result in cumulative liver toxicity.

Interestingly, the two children receiving the gene therapy the latest (23 months, nearly 2 years) also showed asymptomatic low levels of platelets, which could possibly be explained by a more experienced immune system posing a robust immune response, the researchers wrote.

The child who received Zolgensma after Spinraza experienced no side effects.

These findings suggest that combining Spinraza with Zolgensma is generally well-tolerated in type 1 patients, but prolonged prednisolone use and liver toxicity monitoring may be necessary, the team noted.

It is unclear whether combination therapy augments SMN expression levels above either monotherapy approach, and, if so, whether the augmented level is more beneficial that that achieved with either monotherapy, the researchers wrote.

Further studies involving more patients are needed to better understand the effects of combination therapy in liver function and to determine whether there are circumstances in which combination therapy would be more efficacious than either monotherapy, the them concluded.

Recently, Biogen announced plans to launch a Phase 4 clinical trial, called RESPOND, evaluating the benefits ofSpinrazain approximately 60 infants and children withSMA who were treated previously with Zolgensma.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

Total Posts: 85

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Spinraza-Zolgensma Combination Well-tolerated in Children with SMA... - SMA News Today

Vineti and EVERSANA form strategic alliance to advance patient-centered services and supply chain automation for regenerative medicine – PRNewswire

The Vineti/EVERSANA partnership looks at the cell and gene therapy lifecycle holistically and tracks both the product and patient journeys concurrently. EVERSANA's pre- and post-commercial launch services effectively plan market access, clinical trial recruitment, global pricing, patient and HCP engagement, patient and market access and adherence support, therapy storage and distribution needs, and outcomes measurement, with each step measured and optimized by a fully integrated data and analytics platform.

Vineti's Personalized Therapy Management platform automates the Chain of Identity, Chain of Custody and Chain of Condition while enabling the sophisticated scheduling of manufacturing and shipping to hospitals and labs worldwide with industry-leading user experience.

Under the alliance, clients will be able to view patient orders that originated from Vineti and are supported by EVERSANA's integrated commercial services, giving a full view of the patient and product journeys and saving time going back and forth between systems and multiple vendors.

"We're very excited to partner with EVERSANA to help overcome the inherent complexity of cell and gene therapies and provide efficient, integrated solutions for manufacturers and patients," said Vineti CEO and Co-founder Amy DuRoss. "Vineti is focused on industrializing advanced therapies so that all patients in need can benefit. This type of integrated solution with EVERSANA is essential to that goal."

"We're amazed when we look at the pipeline for cell and gene therapies and the hope that's been created for millions of patients worldwide. But as an industry, we have to make that hope a reality by tackling the access, affordability and logistics challenges in this sector," said Jim Lang, CEO, EVERSANA. "That's why we are partnering with best-in-class technology providers like Vineti who share our vision for an accelerated regenerative medicine pipeline that gets products to patients safely, effectively and in a value- and data-driven model. Innovators carry less risk and more financial predictability, while their patients have the promise of healthier lives."

The alliance ensures that EVERSANA clients will be able to view patient orders that originated from Vineti, giving them a full view of the patient journey and saving time going back and forth between systems. Vineti clients will be able to access EVERSANA's solutions through the Vineti Alliance Network.

Media

Stacy Henry Vineti[emailprotected]

Sarah Zwicky EVERSANA[emailprotected]

About Vineti

Vineti is the first commercial, configurable cloud-based platform to expand patient access to life-saving cell and gene therapies. Vineti was co-founded by GE and the Mayo Clinic to solve the key challenges that patients, medical providers, biopharmaceutical companies and regulators face in the delivery and commercialization of individualized therapies. Now a fully independent company, Vineti offers a digital platform of record to integrate logistics, manufacturing and clinical data for personalized therapies. The Vineti platform supports the full continuum of patient-specific therapies, including cancer vaccines and autologous and allogeneic therapies. The company is expanding rapidly, and Vineti's Personalized Therapy Management (PTM) platform was in use in hundreds of leading medical centers worldwide in 2019, on behalf of multiple biopharmaceutical partners. In 2019, the World Economic Forum selected Vineti as a global Technology Pioneer.

About EVERSANA

EVERSANA is the leading provider of global services to the life science industry. The company's integrated solutions are rooted in the patient experience and span all stages of the product lifecycle to deliver long-term, sustainable value for patients, prescribers, channel partners and payers. The company serves more than 500 organizations, including innovative start-ups and established pharmaceutical companies, to advance life science solutions for a healthier world. To learn more about EVERSANA, visit eversana.com or connect through LinkedIn and Twitter.

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Vineti and EVERSANA form strategic alliance to advance patient-centered services and supply chain automation for regenerative medicine - PRNewswire

Expression Therapeutics Announces Bill Swaney as VP of Manufacturing and the Construction of Cell and Gene Therapy Manufacturing – Yahoo Finance

ATLANTA, July 30, 2020 /PRNewswire/ --Expression Therapeutics has announced the appointment of Bill Swaney as the Vice President of Manufacturing. He comes to Expression Therapeutics from the Cincinnati Children's Hospital Medical Center where he was the Director of the Vector Production Facility & Viral Vector Core. Mr. Swaney is an internationally recognized expert in GMP cell and gene therapy manufacturing and has conducted over 70 GMP production runs for academic and commercial clients.

"We are pleased to welcome Bill, a pioneer in cell and gene therapy manufacturing, to Expression Therapeutics. Under his leadership, we now have a complete manufacturing team, making us one of the few biotechnology companies with its own therapeutic pipeline and in-house manufacturing, with the capability to provide CDMO services to commercial clients," said Mohan Rao, Ph.D., CEO of Expression Therapeutics.

Expression Therapeutics has also announced today the construction of a 43,000-sq ft manufacturing facility in Cincinnati, Ohio. The facility will manufacture lentiviral (LV), retroviral (RV), and adeno associated viral (AAV) GMP vectors,with a mix of 30-180 liter runs in cell stacks and up to 1,000 liter runs in bioreactors, and will have the capability to manufacture up to 100 GMP vector products per year. Additionally, it provides the necessary infrastructure to fulfill Expression Therapeutics' ex vivo cell processing and recombinant protein production needs. Expression Therapeutics is using prefabricated PODs from G-CON Manufacturing, Inc. for its ISO Class 7 cleanroom suites.

"We decided to utilize advanced pre-built modular cleanrooms from G-CON to accelerate our buildout and commence vector manufacturing this year. With vector GMP manufacturing backlogs today typically exceeding 18 months, we wanted to bring on additional capacity as soon as possible to serve clients," said Bill Swaney, Vice President of Manufacturing for Expression Therapeutics.

Expression Therapeutics is a biotechnology company based in Atlanta and Cincinnati. The current therapeutic pipeline includes advanced gene therapies for hemophilia, neuroblastoma, T-cell leukemia/lymphoma, acute myeloid leukemia (AML), and primary immunodeficiencies such as hemophagocytic lymphohistiocytosis (HLH).

For manufacturing inquiries, please contact Bill Swaney at wswaney@expressiontherapeutics.com.

For all other inquiries, please contact: Ashley Walsh Director of Corporate Development Expression Therapeutics 1860 Montreal Road Tucker, Georgia 30084 awalsh@expressiontherapeutics.com +1 312.637.2975

(PRNewsfoto/Expression Therapeutics)

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Expression Therapeutics Announces Bill Swaney as VP of Manufacturing and the Construction of Cell and Gene Therapy Manufacturing - Yahoo Finance

Orchard Therapeutics to Webcast Conference Call of Second Quarter 2020 Financial Results – GlobeNewswire

BOSTON and LONDON, July 30, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will host a conference call and live webcast on Thursday, August 6, 2020, at 8:00 a.m. ET to review business updates and its second quarter 2020 financial results.

A live webcast will be available under "News & Events" in the Investors & Media section of the company's website at http://www.orchard-tx.com. The conference call can be accessed by dialing (866) 987-6504 (U.S. domestic) or +1 (602) 563-8620 (international) and referring to conference ID 7392139. A replay of the webcast will be archived on the Orchard website following the presentation.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

Investors

Renee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

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Orchard Therapeutics to Webcast Conference Call of Second Quarter 2020 Financial Results - GlobeNewswire

REGENXBIO To Host Conference Call on August 6 to Discuss Second Quarter 2020 Financial Results and Recent Operational Highlights – PRNewswire

ROCKVILLE, Md., July 30, 2020 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAVTechnology Platform, today announced that it will host a conference call on Thursday, August 6, 2020, at 4:30 p.m. ET to discuss its financial results for the quarter ended June 30, 2020, and recent operational highlights.

To access the live call by phone, dial (800) 708-4539 (domestic) or (800) 619-6396 (international) and enter the passcode 49857700. To access a live or recorded webcast of the call, please visit the Investors section of the REGENXBIO website at http://www.regenxbio.com. The recorded webcast will be available for approximately 30 days following the call.

AboutREGENXBIO Inc.

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

Contacts:

Tricia TruehartInvestor Relations and Corporate Communications347-926-7709[emailprotected]

Investors:Eleanor Barisser, 212-600-1902[emailprotected]

Media:David Rosen, 212-600-1902[emailprotected]

SOURCE REGENXBIO Inc.

http://www.regenxbio.com

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REGENXBIO To Host Conference Call on August 6 to Discuss Second Quarter 2020 Financial Results and Recent Operational Highlights - PRNewswire

Global Gene Therapy Market Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2025 – Research Newspaper

The Gene Therapy Market market report provides a detailed analysis of global market size, regional and country-level market size, segmentation market growth, market share, competitive Landscape, sales analysis, impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunities analysis, strategic market growth analysis, product launches, area marketplace expanding, and technological innovations.

It incorporates Gene Therapy Market market evolution study, involving the current scenario, growth rate (CAGR), and SWOT analysis. Important the study on Gene Therapy Market market takes a closer look at the top market performers and monitors the strategies that have enabled them to occupy a strong foothold in the market. Apart from this, the research brings to light real-time data about opportunities that will completely transform the trajectory of the business environment in the coming years to 2025. Some of the key players in the global Gene Therapy Market market is Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

Download Sample Copy of the Report to understand the structure of the complete report (Including Full TOC, Table & Figures) @ https://brandessenceresearch.com/requestSample/PostId/417

Gene Therapy Market Market: Competitive Landscape:

The competitive landscape further includes details about different players and their position on a global and a local level is also explained in detail in this compiled study. These insights were prepared through mapping business strategies and products that offer high revenue generation capacities. An overview of the Gene Therapy Market market Comprehensive analysis of the market Analyses of recent developments in the market Events in the market scenario of the past few years Emerging market segments and regional Gene Therapy Market markets Segmentations up to the second and/or third level Historical, current, and estimated market size in terms of value and volume Competitive analysis, with company overview, products, revenue, and strategies. An impartial assessment of the market Strategic recommendations to help companies increase their Gene Therapy Market market presence.

The Key Manufacturers covered in this Report:- Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

By Therapy Type:-SomaticGermlineBy Vector Type:-

non-viral Vectorsviral vectorsBy Application:-

Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular Diseases Others

The key regions covered in the Gene Therapy Market market report are:

North America (U.S., Canada, Mexico)

South America (Cuba, Brazil, Argentina, and many others.)

Europe (Germany, U.K., France, Italy, Russia, Spain, etc.)

Asia (China, India, Russia, and many other Asian nations.)

Pacific region (Indonesia, Japan, and many other Pacific nations.)

Middle East & Africa (Saudi Arabia, South Africa, and many others.)

Request Customization of the Report: https://brandessenceresearch.com/requestSample/PostId/417

Table of Contents

Report Overview: It includes major players of the global Gene Therapy Market Market covered in the research study, research scope, and Market segments by type, market segments by application, years considered for the research study, and objectives of the report.

Global Growth Trends: This section focuses on industry trends where market drivers and top market trends are shed light upon. It also provides growth rates of key producers operating in the global Gene Therapy Market Market. Furthermore, it offers production and capacity analysis where marketing pricing trends, capacity, production, and production value of the global Gene Therapy Market Market are discussed.

Market Share by Manufacturers: Here, the report provides details about revenue by manufacturers, production and capacity by manufacturers, price by manufacturers, expansion plans, mergers and acquisitions, and products, market entry dates, distribution, and market areas of key manufacturers.

Market Size by Type: This section concentrates on product type segments where production value market share, price, and production market share by product type are discussed.

Market Size by Application: Besides an overview of the global Gene Therapy Market Market by application, it gives a study on the consumption in the global Gene Therapy Market Market by application.

Production by Region: Here, the production value growth rate, production growth rate, import and export, and key players of each regional market are provided.

Consumption by Region:This section provides information on the consumption in each regional market studied in the report. The consumption is discussed on the basis of country, application, and product type.

Company Profiles:Almost all leading players of the global Gene Therapy Market Market are profiled in this section. The analysts have provided information about their recent developments in the global Gene Therapy Market Market, products, revenue, production, business, and company.

Market Forecast by Production:The production and production value forecasts included in this section are for the global Gene Therapy Market Market as well as for key regional markets.

Market Forecast by Consumption:The consumption and consumption value forecasts included in this section are for the global Gene Therapy Market Market as well as for key regional markets.

Value Chain and Sales Analysis:It deeply analyzes customers, distributors, sales channels, and value chain of the global Gene Therapy Market Market.

Key Findings: This section gives a quick look at important findings of the research study.

Get access to full summary @ https://brandessenceresearch.com/healthcare/gene-therapy-market-global

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We publish market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students.

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Global Gene Therapy Market Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2025 - Research Newspaper

Cell and Gene Therapy Tools, and Reagents: Global Markets – PRNewswire

NEW YORK, July 27, 2020 /PRNewswire/ -- Report Scope: The scope of this study encompasses an investigation of the markets cell and gene therapy tools such as GMP proteins, media, cell separation and activation reagents, viral and non-viral, cytokine release syndrome monitoring products, GMP antibodies, leukapheresis instrumentation, immunoassays (multiplex and singleplex) and bioreactors.The analyst examines each tool type, determines its current market status, examines its impact on future markets, and presents forecasts of growth over the next five years.

Read the full report: https://www.reportlinker.com/p05938974/?utm_source=PRN

Technological issues, including the latest trends, are discussed. The analyst examined the industry on a worldwide basis, from both application and demand perspectives, in the major regions of the world.

Report Includes: - 77 data tables - An overview of the global market for immunoassay-based cell and gene therapy tools, and reagents within the life sciences industry - Analyses of the global market trends, with data corresponding to market size for 2017-2019, and projections of compound annual growth rates (CAGRs) through 2024 - Information about in vitro diagnostics (IVD), its background and importance of IVD testing - Discussion on prevalence of infectious disease, metabolic disorders and chronic ailments as drivers of immunoassay testing market - Description of recent technologies such as chemiluminescent immunoassays (CLIA), fluorescent immunoassays, multiplex assays and rapid diagnostic technologies - Evaluation of market potential for immunoassay-based cell and gene therapy tools and reagents, current market size and market forecast, and market share analysis of the leading players engaged - Patent review and new developments, R&D efforts, industrial changes with emphasis on recent investments, and current state of the immunoassay technology - Company profiles of the key market participants, including Bio-Rad Laboratories, Cytiva Life Sciences, Luminex Corp., Millipore Sigma, Thermo Fisher Scientific and STEMCELL Technologies

Reasons for Doing this Study: Gene and cell therapy are emerging as important tools to treat human health.Techniques such as CAR-T therapy have emerged as key ways of treating many different types of cancers.

The promise of gene therapy using technologies such as CRISPR is starting to be realized in clinical trials, and markets are scaling up to treat other diseases as well, particularly rare gene- based diseases.As these therapies are coming to the fore, a new market for tools to develop these therapies using standard methodologies is emerging.

This report will cover what those tools are, how they impact the larger life science tools market, and how they will evolve over the next five years.

Read the full report: https://www.reportlinker.com/p05938974/?utm_source=PRN

About Reportlinker ReportLinker 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.

Contact Clare: [emailprotected] US: (339)-368-6001 Intl: +1 339-368-6001

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Cell and Gene Therapy Tools, and Reagents: Global Markets - PRNewswire

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

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.

About Oxford Biomedica

Oxford 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-PD

AXO-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 Therapies

Axovant 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 - PharmiWeb.com

Expression Therapeutics Announces Bill Swaney as VP of Manufacturing and the Construction of Cell and Gene Therapy Manufacturing – BioSpace

ATLANTA, July 30, 2020 /PRNewswire/ --Expression Therapeutics has announced the appointment of Bill Swaney as the Vice President of Manufacturing. He comes to Expression Therapeutics from the Cincinnati Children's Hospital Medical Center where he was the Director of the Vector Production Facility & Viral Vector Core. Mr. Swaney is an internationally recognized expert in GMP cell and gene therapy manufacturing and has conducted over 70 GMP production runs for academic and commercial clients.

"We are pleased to welcome Bill, a pioneer in cell and gene therapy manufacturing, to Expression Therapeutics. Under his leadership, we now have a complete manufacturing team, making us one of the few biotechnology companies with its own therapeutic pipeline and in-house manufacturing, with the capability to provide CDMO services to commercial clients," said Mohan Rao, Ph.D., CEO of Expression Therapeutics.

Expression Therapeutics has also announced today the construction of a 43,000-sq ft manufacturing facility in Cincinnati, Ohio. The facility will manufacture lentiviral (LV), retroviral (RV), and adeno associated viral (AAV) GMP vectors,with a mix of 30-180 liter runs in cell stacks and up to 1,000 liter runs in bioreactors, and will have the capability to manufacture up to 100 GMP vector products per year. Additionally, it provides the necessary infrastructure to fulfill Expression Therapeutics' ex vivo cell processing and recombinant protein production needs. Expression Therapeutics is using prefabricated PODs from G-CON Manufacturing, Inc. for its ISO Class 7 cleanroom suites.

"We decided to utilize advanced pre-built modular cleanrooms from G-CON to accelerate our buildout and commence vector manufacturing this year. With vector GMP manufacturing backlogs today typically exceeding 18 months, we wanted to bring on additional capacity as soon as possible to serve clients," said Bill Swaney, Vice President of Manufacturing for Expression Therapeutics.

Expression Therapeutics is a biotechnology company based in Atlanta and Cincinnati. The current therapeutic pipeline includes advanced gene therapies for hemophilia, neuroblastoma, T-cell leukemia/lymphoma, acute myeloid leukemia (AML), and primary immunodeficiencies such as hemophagocytic lymphohistiocytosis (HLH).

For manufacturing inquiries, please contact Bill Swaney at wswaney@expressiontherapeutics.com.

For all other inquiries, please contact:Ashley WalshDirector of Corporate DevelopmentExpression Therapeutics1860 Montreal RoadTucker, Georgia 30084awalsh@expressiontherapeutics.com+1 312.637.2975

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SOURCE Expression Therapeutics

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Expression Therapeutics Announces Bill Swaney as VP of Manufacturing and the Construction of Cell and Gene Therapy Manufacturing - BioSpace

CPI, the Cell and Gene Therapy Catapult and MicrofluidX announce development of technology to rapidly scale up cell and gene therapy manufacture -…

London UK, 27th July 2020 CPI, MicrofluidX and the Cell and Gene Therapy Catapult (CGT Catapult) announced today their collaboration in a project to develop bioprocessing technology for the rapid scale-up of cell and gene therapies.

The technology has the potential to overcome a major bottleneck in the development of these novel treatments, and reduce the time and costs associated with bringing such therapies to market.

Significant advances have been made in recent years in the use of cell and gene therapies in disease areas with unmet clinical needs. However, reliable methods are still required to scale up the manufacture of these complex technologies to commercial levels. Cell and gene therapies are produced from inherently variable living cells. As such, it is highly challenging to achieve consistent performance at different scales, which can lead to prolonged development times. In turn, this can significantly raise the cost of these therapies upon entry to the market.

The microfluidic cell culture technology being developed by MicrofluidX will provide manufacturers of cell and gene therapies with fine process control throughout development, ensuring consistent quality. The technology can be used to optimise cell characteristics and growth conditions early in development, before rapidly transitioning to large-scale manufacturing without the need to invest time in redesigning processes. Crucially, using microfluidics greatly reduces the consumption of expensive reagents, cutting down overall development costs. MicrofluidX recently closed an initial funding round raising 1.4 million for its microfluidic platform. The company is now working with CPI and CGT Catapult to secure further funding and validate its prototypes.

CPI is an independent technology innovation centre and founding member of the UK Governments High Value Manufacturing Catapult. CPIs state-of-the-art facilities provide expertise and advanced equipment for developing GMP-compliant bioprocessing technology. In addition, CPI has drawn from its extensive experience in securing public funding to help MicrofluidX secure 500k of grant funding from Innovate UK for development of the microfluidic bioprocessing platform.

CGT Catapult have more than 230 experts in the industrialisation, manufacturing and clinical adoption of cell and gene therapies, and support industry and academic partners to develop and commercialise these transformative medicines. In this collaboration, the organisation will support MicrofluidX to implement, test and optimise the microfluidics platform, utilising the technology and expertise at the CGT Catapult development centre laboratories in London.

Dr Antoine Espinet, Founder and CEO, MicrofluidX, said:

We are excited to be working with CPI and the Cell and Gene Therapy Catapult on the development of our innovative microfluidics platform. Our technology will cut the time and costs required to bring cell and gene therapies to market, improving access to these novel treatments.

Lucy Foley, Director of Biologics, CPI, said:

We look forward to working with MicrofluidX, following our successful grant application. This technology could play an important role in the future development of cell and gene therapies, and our expertise in the manufacturing of biologics will help to ensure that the technology is market-ready as quickly as possible.

Matthew Durdy, CEO, CGT Catapult, commented:

We are delighted to be supporting the development of this microfluidic bioprocessing platform, technology which shows significant promise for scaling up cell and gene therapy manufacture. The Cell and Gene Therapy Catapult is looking forward to working with MicrofluidX and CPI, innovating together to overcome barriers in the UK industry.

About CPI

CPI works with partners to translate inventions into products and processes that enhance health and wellbeing, protect and improve our environment and increase productivity across industries. With a deep understanding of technology fore-sighting, innovation processes and funding, outstanding technical expertise and industry-relevant assets, we enable the accelerated development of transformational products and processes that have the potential to disrupt and revolutionise markets. We also engage in incremental technological innovation that allows established products and processes to be optimised for better performance and efficient manufacture. Through the breadth of our technology platforms, we support our partners across many diverse markets, including pharmaceuticals, speciality chemicals, food and drink, electronics and transportation. http://www.uk-cpi.com

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 230 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About MicrofluidX

MicrofluidX is tackling the problems of process control, scalability, and cost associated with cell bioprocessing, in particular for autologous cell and gene therapy. Its automated, closed technology allows biologists to easily carry out process development by running dozens of cell culture conditions in parallel with extreme process control, and to scale up seamlessly up to several billion cells for manufacturing at a fraction of the current costs. https://www.microfluidx.co.uk/

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CPI, the Cell and Gene Therapy Catapult and MicrofluidX announce development of technology to rapidly scale up cell and gene therapy manufacture -...

Navigating Complexity in Oncology Cell and Gene Therapy Clinical Trials – Clinical OMICs News

As more knowledge is revealed about the genetic underpinnings of cancers, cell and gene therapies (CGT) are playing an increasingly important role in treating oncology patients.

The development of chimeric antigen receptor T cell (CAR-T cell) therapiessuch as Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel)and their success in combating hematological malignancies, has paved the way for a surge in investment in CGT, especially adoptive cellular transfer (ACT), in oncology. While in the beginning, we were somewhat limited to CAR-T monotherapies anchored in single antigen targets for CD19-expressing lymphomas and leukemias, research has grown to include expanded and multi-targeting modalities, including additional adoptive cell therapy immune cell vehicles (e.g. NK cells, macrophages, dendritic cells, CD 4+ T cells, etc.). Therefore, it is likely CAR-T cells may be used as both monotherapy, or in combination with novel agents.

Estimates as of February 2020 suggest there are more than 1,000 CGT trials globally, with nearly 600 of those in oncology. Whats more, recent market intelligence projects the global CGT market will continue to rise at a compound annual growth rate of 36.25 percent between 2019 and 2025. The investment in oncology emphasizes the importance of developing innovative and accelerated ways to deliver CGT trials and address the challenges of scaling to commercialization. As such, the demand for understanding how to solve these challenges will continue to increase over the next decade.

The success of CGT has transformed the delivery of clinical trial services, requiring the design of new workflows, processes, and tools to address the nuances and complexities of how CGT trials are executed compared to traditional trials. As the industry evolves, the next generation of adoptive cellular transfer products will move closer to becoming a front-line treatment, as opposed to being limited to relapsed or refractory patients, and will have improved safety and durability of response. Developing improved investigational products requires the use of laboratory services to better identify targets and biomarkers, such as advancing polymerase chain reaction (PCR) and liquid biopsy techniques.

Here, we outline the challenges in designing and executing oncology CGT clinical trials and potential solutions to those complexities.

Challenges in designing and executing CGT clinical trials

Cell therapies are living medicines, with many being autologous, requiring each patient sample to have its own process. This personalization creates a more complex development cycle than other biologic drugs. One of the main challenges in this approach is developing well-defined characteristics or attributes (i.e., cell phenotype of functional measurements) that are well correlated with outcomes for these investigational products, as there are many causes of variability. For example, there may be differences in cells from donor to donor, which can make it difficult to determine which factors drive efficacy and safety. Notably, with CAR-T therapies, significant side effects include cytokine release syndrome (caused by the production of a large number of inflammatory molecules by CAR-T cells) and neurotoxicity, both of which are related to therapeutic efficacy. New characteristic safety profiles could emerge, which need specific considerations on how to manage patients.

Moreover, as biotech companies start to move toward solid tumor targets, the treatment of patients with advanced solid tumors has become more dependent on tissue biomarkers to help guide management decisions. As a result, the ability to obtain tissue for the production or expansion of cell therapies adds another burden. Further, targeted therapies are associated with superior clinical outcomes, but to obtain updated biomarkers and targets, large biopsy specimens are often needed. This is not always feasible, either because lesions are not safely accessible to a surgeon or an interventional radiologist, or because the patient declines further invasive procedures.

Allogeneic therapies, or those using cells from healthy donors, may avoid some complexities of modifying a patients own cells. For example, Gracell Biotechnologies recently presented preliminary results of a first-in-human, universal CAR-T therapy, GC027which targets CD7 on malignant T cellsfor treating adult patients with relapsed/refractory T-cell acute lymphoblastic leukemia. The ability to produce hundreds of viable cells for multiple infusions can overcome issues related to the time it takes to manufacture T-cells, costs to generate a product for each patient, failures in the ability to generate a product from a patients immune cells, and the heterogeneity of T-cell products. However, allogeneic approaches will need to circumnavigate challenges in tissue-matching, which means that human leukocyte antigen typing becomes important in many cases, possibly limiting the target population.

Gene editing and other engineered products, such as T-cell receptor (TCR)-engineered T cells, to treat cancer, are entering into safety and feasibility trials. For example, the first clinical trial using CRISPR-Cas 9 editing to engineer T cells in patients with refractory cancer launched in the fall of 2019. These experimental technologies will raise similar challenges as they move forward through early phase clinical trials.

Conclusion

Over the next 10 years, we will continue to witness significant advances and excitement as CGT saves the lives of patients. First, the industry will conquer the challenges created by the tumor microenvironment, opening opportunities to successfully tackle a broader range of solid tumors. Second, new strategies will emerge to improve the manufacturing and delivery of autologous and allogeneic therapies, improving cost-efficiency. These future advances will be assisted by enhanced diagnostic assays and other tests to improve our understanding of targeting and treating cancer.

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Navigating Complexity in Oncology Cell and Gene Therapy Clinical Trials - Clinical OMICs News

Orchard Therapeutics to Webcast Conference Call of Second Quarter 2020 Financial Results – BioSpace

BOSTON and LONDON, July 30, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will host a conference call and live webcast on Thursday, August 6, 2020, at 8:00 a.m. ET to review business updates and its second quarter 2020 financial results.

A live webcast will be available under "News & Events" in the Investors & Media section of the company's website at http://www.orchard-tx.com. The conference call can be accessed by dialing (866) 987-6504 (U.S. domestic) or +1 (602) 563-8620 (international) and referring to conference ID 7392139. A replay of the webcast will be archived on the Orchard website following the presentation.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

Investors

Renee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

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Orchard Therapeutics to Webcast Conference Call of Second Quarter 2020 Financial Results - BioSpace