Gene Therapy Net - News, Conferences, Vectors, Literature, Clinical Trials and more

Posted on: 8 August 2026, source: fabrydiseasenews A leading Chinese scientist is under investigation by his university after a six-year-old girl reportedly died following experimental gene-editing therapy. The girl, named Mei, died days after receiving a treatment designed to correct a genetic mutation affecting her neurodevelopment in March last year, according to a report published by Science …

Posted on: 26 July 2026, source: South China Morning Post A leading Chinese scientist is under investigation by his university after a six-year-old girl reportedly died following experimental gene-editing therapy. The girl, named Mei, died days after receiving a treatment designed to correct a genetic mutation affecting her neurodevelopment in March last year, according to a report published by S…

Posted on: 3 July 2026, source: medpagetoday The FDA on Wednesday expanded the approval for exagamglogene autotemcel (exa-cel, Casgevy) in severe sickle cell disease with recurrent vaso-occlusive crises and transfusion-dependent beta thalassemia to include children 2 years and up. Exa-cel, the first FDA-approved therapy utilizing CRISPR/Cas9 gene editing technology, was previously only approved f…

Posted on: 15 June 2026, source: news-medical.net An eight-month-old baby from Israel has become the first human ever to receive an experimental gene therapy designed to replace a missing gene responsible for a devastating neurological disease that until now offered no hope of survival. The treatment was performed at Clalit-Schneider Children’s Medical Center, part of Clalit Health Services, Isra…

Posted on: 12 May 2026, source: CHOSUNBIZ U.S. biotech company Fractyl Health is launching the world's first clinical trial of a glucagon-like peptide-1 (GLP-1) gene therapy. As GLP-1 class treatments that mimic the action of the hormone GLP-1, which is naturally secreted in the gut when eating, have emerged, the paradigm for treating diabetes and obesity has changed dramatically. Current GLP-1 t…

Posted on: 13 April 2026, source: National Today A groundbreaking gene regulation treatment developed by Dr. Linda Laux at Ann & Robert H. Lurie Children's Hospital of Chicago has demonstrated significant reductions in seizures and improvements in developmental and cognitive symptoms for patients with Dravet syndrome, a severe form of epilepsy. The treatment targets the root cause of the conditio…

Gene Therapy Research Institutes - US & Canada - Center for Cell and Gene Therapy, Baylor College of Medicine, Houston - Center for Gene Therapy, University of Michigan Medical Center, Ann Arbor - Dep. of Gene and Cell Medicine, Mount Sinai School of Medicine, New York - Gene Therapy Center, University of North Carolina School of Medicine - Harvard Gene Therapy Initiative, Harvard University, Bos…

Gene Therapy Legislation in the US Another DHHS agency, the National Institutes of Health (NIH), oversees the conduct of federally funded clinical trials through a series of guidelines that add additional requirements to those specified in the CFR. NIH - NExTRAC The Novel and Exceptional Technology and Research Advisory Committee (NExTRAC) is a federal advisory committee that provides recommendat…

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Gene Therapy Jobs Info Selection of latest gene therapy jobs found at NewScientist Jobs by using the keywords: "gene therapy" OR "gene targeting" OR "gene delivery" OR "viral vector" To advertise your vacancies here, please use the contact form. More gene therapy jobs and career oppertunities: To advertise your vacancies here, please use the contact form. More gene therapy jobs and career oppertu…

Gene Therapy Societies - American Society of Gene and Cell Therapy (ASGCT) - Australasian Gene and Cell Therapy Society (AGCTS) - Brazilian Association of Cell and Gene Therapy (ABTCel-Gen) - British Society of Gene and Cell Therapy (BSGCT) - European Society of Gene and Cell Therapy (ESGCT) - Finnish Gene Therapy Society (FGTS) - French Society of Cellular and Gene Therapy (SFTCG) - Gene Therapy…

- 06 April 2026Conditions: Vestibular Schwannoma Sponsors: Akouos, Inc.; Eli Lilly and Company Enrolling by invitation - 19 March 2026Conditions: Neovascular Age-Related Macular Degeneration (nAMD); Wet AMD Interventions: Genetic: Ixo-vec; Drug: Aflibercept Sponsors: Adverum Biotechnologies, Inc. Recruiting - 18 March 2026Conditions: GM1 Gangliosidoses; GM1 Gangliosidosis, Type I; GM1 Gangliosido…

Latest Articles on Gene Therapy Overview of latest articles and publications on gene therapy in PubMed, including Human Gene Therapy, Journal of Molecular Medicine and Journal of Gene Medicine. PubMed is a service of the US National Library of Medicine that includes over 18 million citations from MEDLINE and other life science journals. - Multimodal gene and targeted drug therapy for chronic myel…

New and Updated Clinical Gene Therapy Trials - 06 April 2026Conditions: Vestibular Schwannoma Sponsors: Akouos, Inc.; Eli Lilly and Company Enrolling by invitation - 19 March 2026Conditions: Neovascular Age-Related Macular Degeneration (nAMD); Wet AMD Interventions: Genetic: Ixo-vec; Drug: Aflibercept Sponsors: Adverum Biotechnologies, Inc. Recruiting - 18 March 2026Conditions: GM1 Gangliosidoses…

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 spo…

Posted on: 13 March 2026, source: Bezinga The 36-week data from the phase 3 study of DTX301 showed promising results, with a significant increase in urinary orotic acid levels. At week 36, DTX301-treated patients (n=18) demonstrated a 18% reduction in 24-hour plasma ammonia compared to placebo (n=19) and maintained average ammonia AUC0-24 in the normal range through week 36. Eight of nine patient…

Posted on: 13 March 2026, source: Bezinga The 36-week data from the phase 3 study of DTX301 showed promising results, with a significant increase in urinary orotic acid levels. At week 36, DTX301-treated patients (n=18) demonstrated a 18% reduction in 24-hour plasma ammonia compared to placebo (n=19) and maintained average ammonia AUC0-24 in the normal range through week 36. Eight of nine patient…

Posted on: 6 March 2026, source: CNBC UniQure needs to run another study to prove that its gene therapy “actually helps people with Huntington’s disease,” a senior U.S. Food and Drug Administration official said on a call with reporters Thursday. The official, who requested anonymity before discussing sensitive information, confirmed the agency has asked the company to run a placebo controlled tr…

Posted on: 16 February 2026, source: Nature Patients with esophageal cancer (EC) face intensive treatments with limited success and severe side effects, including severe swallowing difficulties. A novel drinkable gene therapy foam coats the esophagus and delivers mRNA treatment directly to the tumor site. In laboratory models, it achieved 110-fold greater tumor regression than standard treatment …

Gene Therapy Conferences 2026 - Evolution Summit 2026 - Gene and Cell Therapies - 5th mRNA-Based Therapeutics Summit Europe - 7th RNAi-Based Therapeutics Summit - Advanced Therapies Week - 5th Annual mRNA Analytical Development & Quality Control Summit - 9th CAR-TCR Summit Europe - 4th Viral Vector Process Development & Manufacturing Summit - Drug Discovery Innovation Programme 2026 - Bioprocessi…