J&J gene therapy treatment fails primary endpoints

Data from the LUMEOS phase 3 clinical trial demonstrated a rare disease gene therapy treatment has failed to improve the vision-guided mobility of patients with X-linked retinitis pigmentosa (XLRP).1 Botaretigene sparoparvovec (bota-vec) is an …

05.05.25 -- Navigating Cell And Gene Therapy Clinical Trials

The Advanced Therapies Congress is America's most exciting cell and gene therapy conference and exhibition. The event is for the leaders of pioneering ATMP companies and their most senior executives in charge of the latest tech and strategies that are …

Chengdu Origen and Vanotech dose first subject in gene therapy trial for wet AMD

Chengdu Origen Biotechnology and Vanotech have initiated the first subject dosing in the open-label Phase I trial, VAN-2401 of the gene therapy KH658, as a single suprachoroidal space administration for treating individuals with neovascular wet age-related …

First Patient to Receive Gene Therapy ‘Cure’ for Beta-Thalassemia Living Pain-Free

Rahemeen Nabeel – credit, Childrens Hospital of Philadelphia, released In 2020, GNN reported that CRISPR gene editing technology was being used to develop a medicine for beta-thalassemia, a genetically-inherited blood disorder. Now though, another medicine …

Vertex Abandons AAV as Gene Therapy Space Weathers Difficulties

Vertex Pharmaceuticals will no longer work on the adeno-associated virus vector technology commonly used to package and deliver gene therapies, according to a Friday report from Endpoints News. BioSpace has reached out to Vertex for independent …

J&J eye disease gene therapy bota-vec fails Phase 3

Matica Biotechnology and Texas A&M's NCTM Form Strategic Partnership to Deliver End-to-End Support for Cell and Gene ...

COLLEGE STATION, Texas, May 5, 2025 /PRNewswire/ -- Matica Biotechnology, a specialized contract development and manufacturing organization (CDMO) for cell and gene therapies (CGT), has entered into a strategic partnership with the National Center …

Opus Genetics Announces Presentation of OPGX-LCA5 Gene Therapy Data at ARVO; 12 Month Phase 1/2 Results Support ...

Improvements in subjective and objective measures of efficacy observed at six months persisted for one year in patients with severe vision impairment from inherited retinal degeneration due to mutations in the LCA5 gene Administration of OPGx-LCA5 by …

FDA will ask outside experts to review Capricor’s therapy for Duchenne muscular dystrophy

Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox. Today, we explore how an ambitious nonprofit is taking control of a shelved gene therapy, as it seeks to create a new model for …

Genezen and Optieum Biotechnologies Partner to Support cGMP Manufacturing of Groundbreaking CAR-T Therapy for ...

Collaboration supports manufacture of the OPTF01 product to Drive Next-Generation Chimeric Antigen Receptor T Cell (CAR-T) Development INDIANAPOLIS and EHIME, Japan, May 5, 2025 /PRNewswire/ — Genezen, a leading viral vector Contract Development …