@mmillington Active 2 years, 8 months ago Home Activity Profile Friends Groups Gene Therapy NewsUltragenyx's Gene Therapy Study Shows Ammonia Reduction In Rare Metabolic Disorder March 13, 2026Posted 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 […] FDA official calls UniQure’s gene therapy a ‘failed’ treatment for Huntington’s disease March 6, 2026Posted 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 […] Drinkable gene therapy foam for the treatment of constrictive esophageal carcinoma February 16, 2026Posted 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 […] First patient in Wales receives ground-breaking gene therapy treatment January 15, 2026Posted 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 […] Workshop: Cell and Gene Therapy for Rare Diseases 2026 January 5, 2026Posted on: 5 January 2026, source: EATRISThe 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} Groundbreaking gene therapy gives hope to kids with Hunter syndrome November 24, 2025Posted on: 24 November 2025, source: tbsnews.netA three-year-old boy from California has become the first patient with Hunter syndrome to receive an experimental gene therapy developed in Manchester, marking a potential advance for treating the rare and progressively disabling disorder. Hunter syndrome mostly affects boys and causes the body to slowly fill up with substances […] Gene therapy delivers lasting immune protection in children with rare disorder October 17, 2025Posted on: 17 October 2025, source: UCLAAn experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained immune system function in 59 of 62 children born with ADA-SCID, a rare and deadly genetic immune disorder. Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, […] Welcome to Gene Therapy Net December 31, 1999Gene 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 […]
