Texas Children’s provides new breakthrough treatment for patient with AADC deficiency [caption id="" align="alignnone" width="700"] Texas Children's neurosurgeon Dr. Daniel Curry leads first-ever FDA-approved gene therapy treatment for AADC deficiency. ©Texas Children's Hospital[/caption] On August 19, 2025, Texas Children's, a non-profit healthcare organization, has announced that a three-year-old girl has…..
Gene therapy improves blood flow in the brain in patients with sickle cell disease [caption id="attachment_11887" align="aligncenter" width="700"] Akshay Sharma, MBBS, MSc, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy talks to St. Jude patient enrolled on the reported clinical trial, Corey Luellen. ©St. Jude Children's Research Hospital[/caption]…..
GNT0004 product confirms its clinical efficacy with stabilization of motor functions in patients treated at the effective dose for up to 2 years Genethon has unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American Society…..
Novel gene therapy trial for sickle cell disease launches in the US The study uses CRISPR to correct sickle cell mutation and aims to free patients of the disease. UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The…..
Preclinical data for AAV-FGF21 gene therapy shows durable reversal of liver fibrosis UAB researchers have cured liver disease MASH in mouse models by a one-time gene therapy . The results were obtained with a single intramuscular administration of the therapeutic viral vectors AAV-FGF21. The research has also determined that most…..
Genethon Using AI to Improve Effectiveness of Gene Therapies and Patient Access CEO Frederic Revah discusses how Artificial Intelligence is creating next-generation gene therapy vectors and reducing bioproduction costs in the latest issue of Genethon’s Newsletter Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy…..
Broken String Biosciences Closes $15M Series A Funding Round Broken String Biosciences, a UK-based genomics company building a technology platform to drive the development of cell and gene therapies that are safer by design, has closed a $15 million Series A investment round, co-led by Illumina Ventures and Mérieux Equity…..
Efficacy of gene therapy demonstrated in patients suffering from Crigler-Najjar Syndrome Crigler-Najjar syndrome is characterized by hyperbilirubinemia. If it is not treated quickly, the build-up of bilirubin, due to a deficiency in the liver enzyme UGT1A1, can cause significant neurological damage and become fatal. At the current time, the only…..
Novartis expands Zolgensma® manufacturing capacity with approval of multi-product North Carolina facility The FDA has granted commercial licensure approval for Novartis' Durham, N.C. site, a multi-product gene therapy manufacturing facility. This approval allows the state-of-the-art, 170,000 square-foot facility to make, test and release commercial Zolgensma®, as well as produce…..
Intellia Therapeutics and SparingVision Announce Strategic Collaboration to Develop Novel Ocular Therapies Using CRISPR/Cas9 Technology Collaboration combines Intellia’s proprietary genome editing technology platform with SparingVision’s significant ophthalmology expertise Intellia will grant SparingVision exclusive rights to its leading in vivo CRISPR/Cas9 technology for the development of ocular therapies directed to three…..
