By Jocelyn KaiserJun. 29, 2020 , 5:20 PM Two boys have died after receiving high doses of a gene therapy treatment for their rare muscle disease, Biopharma Dive reports. The patients, born with x-linked myotubular myopathy, developed liver problems that apparently led to sepsis, according to a 23 June letter to patient groups from trial sponsor Audentes Therapeutics
In recent months, even as our attention has been focused on the coronavirus outbreak, there have been a slew of scientific breakthroughs in treating diseases that cause blindness. Researchers at U.S.-based Editas Medicine EDIT, +1.45% and Ireland-based Allergan (now owned by AbbVie ABBV, +0.32% ) have administered CRISPR for the first time to a person with a genetic disease. This landmark treatment uses the CRISPR approach to a specific mutation in a gene linked to childhood blindness.
- Updated data will be presented in an oral presentation on the efficacy and safety of AAV gene therapy FL180a in the B-AMAZE study in severe haemophilia B patients - - Additional data will be presented in 5 poster presentations - LONDON, June 29, 2020 (GLOBE NEWSWIRE) -- Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announces that Professor Pratima Chowdary of the Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital UK and UCL Cancer Institute, and Chief investigator for the study, will present data in a late-breaking abstract session at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Congress on behalf of co-authors, which will be held virtually 12-14 July 2020.In addition, Freeline has five posters at the conference and will be presenting data on the Factor IX Padua variant and health economics from its Adeno-Associated Virus (AAV)-based haemophilia gene therapy platform. We are pleased to have an abstract on our novel investigational gene therapy treatment for haemophilia B accepted as a late-breaking abstract at the upcoming ISTH conference, said Theresa Heggie, CEO.
North America is the largest regional market for gene therapies This press release was orginally distributed by SBWire Northrook, IL -- (SBWIRE) -- 06/29/2020 -- [135 Pages Report] The Global Gene Therapy Market is estimated to grow from USD 3.8 billion in 2019 to USD 13.0 billion by 2024, at a CAGR of 27.8% The growth of this market is majorly driven by the high incidence of cancer and other target diseases, availability of reimbursements, and the increasing funding for gene therapy research. However, the high cost of gene therapies is expected to hamper market growth to a certain extent during the forecast period.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced the appointment of Kim Raineri, as chief manufacturing and technology officer
5pm on June 26, 2020 - Kawasaki/Japan: The Innovation Center of NanoMedicine (iCONM), the National Institute for Quantum Science and Technology (QST), and the University of Tokyo jointly announced that a reagent for the selective and safe coating of the liver sinusoidal walls to control the clearance of gene therapy drugs was successfully developed.
Fior Markets has announced a new report titledGlobal Gene Therapy Marketbegins with the market definition and the market scope. The report provides a market overview, regional market opportunity, sales, and revenue by region, manufacturing cost analysis, industrial chain, market effect factors analysis
Germantown, MD, US, June 29, 2020 Orgenesis Inc. (NASDAQ: ORGS)(Orgenesis or the Company), a pioneering, global biotech company committed to accelerating commercialization and transforming the delivery of cell and gene therapies (CGTs), today announced that it has been added to the Russell 3000(R) Index, following the annual Russell indexes reconstitution, effective after the market open on June 29, according to a final list of additions posted on June 26. Annual Russell indexes reconstitution captures the 4,000 largest US stocks, ranking them by total market capitalization
The Global Cancer Gene Therapy Market Research Report provides customers with a complete analytical study that provides all the details of key players such as company profile, product portfolio, capacity, price, cost, and revenue during the forecast period from 2020 to 2027. The report provides a full assessment.
A report on Gene Therapy for Age-related Macular Degeneration market compiled by Brand Essence Market Research provides a succinct analysis regarding the values and trends existing in the current business scenario.
The study on the Gene Therapy for Age-related Macular Degeneration Marketby Brand Essence Market Research is a compilation of systematic details in terms of market valuation, market size, revenue estimation, and geographical spectrum of the business vertical. The study also offers a precise analysis of the key challenges and growth prospects awaiting key players of the Gene Therapy for Age-related Macular Degeneration market, including a concise summary of their corporate strategies and competitive setting. In 2018, the Global Gene Therapy for Age-related Macular Degeneration Market size was xx million US$ and it is expected to reach xx million US$ by the end of 2025, with a CAGR of xx% during 2019-2025
The report examines the Gene Therapy market with respect to the industry trends, growth rate, prospects, drivers, restraints, threats, and lucrative opportunities, by means of distinguishing the high-growth segments of the market through the various stakeholders.
The same day Oxford Biomedica signed a 5-year deal to make AstraZenecas Covid-19 vaccine, the UK gene therapy company announced they received word Sanofi was sending a couple of decade-old experimental drugs back to them.
The Personalized Gene Therapy Treatment Market Research Report 2020 published by Prophecy Market Insights is an all-inclusive business research study on the current state of the industry which analyzes innovative strategies for business growth and describes significant factors such as top developers/manufacturers, production value, key regions, and growth rate. Impact of Covid-19 pandemic on the market will be completely analyzed in this report and it will also quantify the impact of this pandemic on the market.
DUBLIN, June 26, 2020 /PRNewswire/ -- The "Non-Viral Transfection Reagents and Systems Market, 2020-2030" report has been added to ResearchAndMarkets.com's offering.
DetailsCategory: AntibodiesPublished on Sunday, 28 June 2020 14:40Hits: 307 Reblozyl regulates late-stage red blood cell (RBC) maturation to potentially reduce or eliminate the need for regular RBC transfusions Reblozyl is the first and only erythroid maturation agent to be approved in the European Union, representing a new class of therapy PRINCETON, NJ & CAMBRIDGE, MA, USA I June 26, 2020 IBristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced that the European Commission (EC) has approved Reblozyl (luspatercept) for the treatment of: "Dependence on blood transfusions caused by anemia in hematologic malignancies like MDS can often mean frequent and lengthy hospital visits, which can pose additional health risks and affect patients quality of life, said Uwe Platzbecker, M.D., lead investigator of the MEDALIST study, Head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital. Todays approval of Reblozyl provides healthcare professionals with a new therapy that has been shown to significantly reduce the number of red blood cell transfusions needed by MDS patients and, in some cases, helped them to achieve transfusion independence.
In the race to produce a coronavirus vaccine, some companies are experimenting with a novel approach using gene therapy.
By Hemant Khanna, The Conversation In recent months, even as our attention has been focused on the coronavirus outbreak, there have been a slew of scientific breakthroughs in treating diseases that cause blindness. Researchers at U.S.-based Editas Medicine and Ireland-based Allergan have administered CRISPR for the first time to a person with a genetic disease.
XconomyNational A new playbook is needed to ensure consistent chemistry, manufacturing, and controls (CMC) reviews for gene therapy products, the lack of which is hindering the development of these products, asserted a top official at the US Food and Drug Administration . Now is the time to get things right asserted Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, who spoke at a 15 June virtual Drug Information Association annual meeting session on how innovation can help overcome hurdles for these products. The sessions moderator, Nancy Myers, president of Catalyst Healthcare Consultants, asked the panelists to describe some of their main CMC constriction points in developing gene therapy products, and to identify potential solutions
The Gene Therapy Market Research Report 2020 published by Prophecy Market Insights is an all-inclusive business research study on the current state of the industry which analyzes innovative strategies for business growth and describes significant factors such as top developers/manufacturers, production value, key regions, and growth rate. Impact of Covid-19 pandemic on the market will be completely analyzed in this report and it will also quantify the impact of this pandemic on the market