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.
EU approves Hemgenix, one-time gene therapy for hemophilia B
Posted on: 7 March 2023, source: Hemophilia News Today
The European Commission has conditionally approved Hemgenix (etranacogene dezaparvovec), a one-time gene therapy to treat adults with hemophilia B. Hemgenix, originally developed by uniQure, is approved for adults with severe and moderately severe hemophilia B without a history of inhibitors. It’s the first gene therapy for hemophilia B available to patients across all European Union member states, plus Iceland, Liechtenstein, and Norway.
The European Commission has conditionally approved Hemgenix (etranacogene dezaparvovec), a one-time gene therapy to treat adults with hemophilia B. Hemgenix, originally developed by uniQure, is approved for adults with severe and moderately severe hemophilia B without a history of inhibitors. It’s the first gene therapy for hemophilia B available to patients across all European Union member states, plus Iceland, Liechtenstein, and Norway.
FDA officials offer advice on gene therapy trials
Posted on: 9 February 2023, source: Regulatory News
Officials from the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) asserted that the duration of clinical trials for gene therapies depends on the nature of the disease being treated, and that diseases that are more progressive and have a rapid onset may involve shorter trials. This was one of the learnings imparted by officials during a 7 February virtual town hall meeting to answer stakeholder questions on the clinical development of gene therapies for rare diseases outside the hematology and oncology space. The town hall is part of a series to address questions from stakeholders on topics under OTAT’s remit.
Officials from the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) asserted that the duration of clinical trials for gene therapies depends on the nature of the disease being treated, and that diseases that are more progressive and have a rapid onset may involve shorter trials. This was one of the learnings imparted by officials during a 7 February virtual town hall meeting to answer stakeholder questions on the clinical development of gene therapies for rare diseases outside the hematology and oncology space. The town hall is part of a series to address questions from stakeholders on topics under OTAT’s remit.
New gene therapy delivers treatment directly to brain
Posted on: 26 January 2023, source: PBS.org
When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age. A rare genetic disorder kept her from even lifting her head. Her parents took turns holding her upright at night just so she could breathe comfortably and sleep. Then, months later. doctors delivered gene therapy directly to her brain. Now the 4-year-old is walking, running, swimming, reading and riding horses — “just doing so many amazing things that doctors once said were impossible,” said her mother, Judy Wei.
When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age. A rare genetic disorder kept her from even lifting her head. Her parents took turns holding her upright at night just so she could breathe comfortably and sleep. Then, months later. doctors delivered gene therapy directly to her brain. Now the 4-year-old is walking, running, swimming, reading and riding horses — “just doing so many amazing things that doctors once said were impossible,” said her mother, Judy Wei.
FDA approves Ferring’s Adstiladrin as first gene therapy for bladder cancer
Posted on: 23 December 2022, source: FDA
The US Food and Drug Administration (FDA) has approved Ferring Pharma’s Adstiladrin (nadofaragene firadenovec-vncg) as the first gene therapy for the treatment of adult patients with non-muscle-invasive bladder cancer (NMIBC). The novel adenovirus vector-based gene therapy is specifically indicated for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumours and is to be administered into the patient's bladder once every three months. The FDA based its decision on a phase 3 multicentre clinical study in 157 patients with high-risk BCG-unresponsive NMIBC, 98 of whom had BCG-unresponsive CIS with or without papillary tumours and could be evaluated for response.
The US Food and Drug Administration (FDA) has approved Ferring Pharma’s Adstiladrin (nadofaragene firadenovec-vncg) as the first gene therapy for the treatment of adult patients with non-muscle-invasive bladder cancer (NMIBC). The novel adenovirus vector-based gene therapy is specifically indicated for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumours and is to be administered into the patient's bladder once every three months. The FDA based its decision on a phase 3 multicentre clinical study in 157 patients with high-risk BCG-unresponsive NMIBC, 98 of whom had BCG-unresponsive CIS with or without papillary tumours and could be evaluated for response.
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
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