Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.
FDA Approves First Gene Therapy for Hemophilia B
Posted on: 29 November 2022, source: FDA
The FDA has approved etranacogene dezaparvovec for the treatment of adults with Hemophilia B who are currently using Factor IX prophylaxis therapy, have a history of life-threatening hemorrhaging, or have serious spontaneous bleeding episodes. The adeno-associated virus vector-based therapy is the first-of-its-kind for the treatment of Hemophilia B. “Gene therapy for hemophilia has been on the horizon for more than two decades,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release. “Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life.”
The FDA has approved etranacogene dezaparvovec for the treatment of adults with Hemophilia B who are currently using Factor IX prophylaxis therapy, have a history of life-threatening hemorrhaging, or have serious spontaneous bleeding episodes. The adeno-associated virus vector-based therapy is the first-of-its-kind for the treatment of Hemophilia B. “Gene therapy for hemophilia has been on the horizon for more than two decades,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release. “Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life.”
FDA finalizes neurodegenerative disease gene therapy guidance
Posted on: 2 November 2022, source: Regulatory Affairs Professionals Society
The US Food and Drug Administration (FDA) last week finalized guidance to assist sponsors developing human gene therapy (GT) products for neurodegenerative diseases. In the final version, FDA acceded to industry’s requests to modify its language on the use of crossover trials, provided more detail on the use of comparability studies in assessing the effect of post-approval manufacturing changes on products and eliminated limits for residual product-related impurities.
The US Food and Drug Administration (FDA) last week finalized guidance to assist sponsors developing human gene therapy (GT) products for neurodegenerative diseases. In the final version, FDA acceded to industry’s requests to modify its language on the use of crossover trials, provided more detail on the use of comparability studies in assessing the effect of post-approval manufacturing changes on products and eliminated limits for residual product-related impurities.
Gene Therapy Rapidly Improves Night Vision in Adults with Congenital Blindness
Posted on: 11 October 2022, source: Penn MedicineDNA
Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania. The patients had Leber Congenital Amaurosis (LCA), a congenital blindness caused by mutations in the gene GUCY2D
Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania. The patients had Leber Congenital Amaurosis (LCA), a congenital blindness caused by mutations in the gene GUCY2D
Codex DNA's BioXp 9600 System debuting at CAR-TCR Summit
Posted on: 20 September 2022, source: Codex DNA
Increasing the power of optimized synthetic biology workflows with expanded throughput and scale: Introducing the BioXpTM 9600 System. Codex DNA – a San Diego-based biotechnology company, and pioneer in automated benchtop synthetic biology systems – will be debuting their next-generation fully-automated synthetic biology benchtop workstation, the BioXp 9600 System, at this year’s CAR-TCR summit.
Increasing the power of optimized synthetic biology workflows with expanded throughput and scale: Introducing the BioXpTM 9600 System. Codex DNA – a San Diego-based biotechnology company, and pioneer in automated benchtop synthetic biology systems – will be debuting their next-generation fully-automated synthetic biology benchtop workstation, the BioXp 9600 System, at this year’s CAR-TCR summit.
BioMarin reports cancer case in hemophilia gene therapy trial
Posted on: 14 September 2022, source: Biopharmadive
Drug regulators have not ordered a trial hold after an analysis suggested leukemia was naturally occurring, company says. BioMarin Pharmaceutical, a California-based biotechnology company, said a patient in a clinical trial of its hemophilia gene therapy Roctavian has developed leukemia, although testing suggests that the case may be naturally occurring..
Drug regulators have not ordered a trial hold after an analysis suggested leukemia was naturally occurring, company says. BioMarin Pharmaceutical, a California-based biotechnology company, said a patient in a clinical trial of its hemophilia gene therapy Roctavian has developed leukemia, although testing suggests that the case may be naturally occurring..
Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma
Posted on: 15 August 2022, source: genengnews.com
Novartis has acknowledged that two patients have died of acute liver failure following treatment with its Zolgensma® (onasemnogene abeparvovec-xioi), a one-time gene therapy indicated for some forms of spinal muscular atrophy (SMA). As a result, the company said, it will revise Zolgensma’s label to specify that fatal acute liver failure has been reported.
Novartis has acknowledged that two patients have died of acute liver failure following treatment with its Zolgensma® (onasemnogene abeparvovec-xioi), a one-time gene therapy indicated for some forms of spinal muscular atrophy (SMA). As a result, the company said, it will revise Zolgensma’s label to specify that fatal acute liver failure has been reported.
First patient in the Netherlands successfully treated with stem cell gene therapy
Posted on: 21 July 2022, source: LUMC
Researchers from the Leiden University Medical Center (LUMC) have successfully used stem cell gene therapy to treat a baby with the severe congenital immune disorder SCID. An important milestone: it is the first time stem cell gene therapy of Dutch origin has been administered to a patient, and also the first time it has been used to treat this particular form of SCID worldwide. The treatment was effective and the patient is doing well.
Researchers from the Leiden University Medical Center (LUMC) have successfully used stem cell gene therapy to treat a baby with the severe congenital immune disorder SCID. An important milestone: it is the first time stem cell gene therapy of Dutch origin has been administered to a patient, and also the first time it has been used to treat this particular form of SCID worldwide. The treatment was effective and the patient is doing well.
Gene Therapy News
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CTAD 2022 – Lexeo takes baby steps towards an Alzheimer’s gene therapy - Evaluate Pharma
Dec 3, 2022 | 13:00 pm
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Alzheimer’s APOE4 Genetic Risk Targeted in Promising Trial - The New York Times
Dec 2, 2022 | 22:55 pm
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Despite Eye-Popping $3.5 Million Price Tag For Gene Therapy Hemgenix, Budget Impact For Most Payers Will Be Relatively Small - Forbes
Dec 2, 2022 | 17:11 pm
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Gene Therapy Startup Partnered with Jim Wilson's U Penn Program Nets $65M - Inside Precision Medicine
Dec 2, 2022 | 16:32 pm
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Mainstreaming cell and gene therapy – Realizing its potential - BioPharma Dive
Nov 28, 2022 | 11:11 am
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