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

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…

Companies - A Companies - B Companies - C Companies - E Companies - F Companies - G Companies - H Companies - I Companies - K Companies - L Companies - M Companies - N Companies - O Companies - P Companies - R Companies - S Companies - T Companies - U Companies - V Companies - W Companies - Y Information Companies Overview of biotechnology companies involved in gene therapy and/or cell therapy, a…

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…

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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…

Advertise on this site? Advertising opportunities on Gene Therapy Net include standard size banners as well as text ads. Click here for more information about advertising. You can also make a donation by PayPal and support the Gene Therapy Net website. Resources - Alliance for Cancer Gene Therapy - American Cancer Society - Bio-Medicine - Bioscience Explained - Gene Therapy and Children, KidsHeal…

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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…

Posted on: 15 January 2026, source: Nation Cymru The first facility in Wales is set to administer a revolutionary gene therapy to a patient with haemophilia. Cardiff and Vale University Health Board announced that Cardiff Haemophilia Centre based at the University Hospital of Wales will administer its first gene therapy infusion this week. This milestone marks a major breakthrough in the treatmen…

Posted on: 5 January 2026, source: EATRIS The Cell and Gene Therapy for Rare Diseases 2026 is a 5-day in-person workshop, scheduled for June 1-5, 2026, and is an intensive educational program focused on the development cycle of Advanced Therapy Medicinal Products (ATMPs). {fastsocialshare}