Inherited Disease
News on Mendelian diseases, hereditary cancer, variant classification, cystic fibrosis testing and more.
Coave Therapeutics Preparing for Retinitis Pigmentosa Gene Therapy Registration Trial
In an ongoing Phase I/II trial, the gene therapy improved visual function in patients with early stages of the rare, inherited visual disorder.
FDA Delays Decision for Sarepta Therapeutics Duchenne Muscular Dystrophy Gene Therapy
According to Sarepta, the federal agency has indicated it may grant accelerated approval to SRP-9001 as a treatment for a subgroup of patients based on age.
Vertex, CRISPR Therapeutics Share Promising Exa-Cel Data Readout in SCD, Beta-Thalassemia at ASGCT
The gene therapy improved hemoglobin levels in transfusion-dependent beta-thalassemia patients and hindered severe vaso-occlusive crises in those with sickle cell disease.
Locanabio Gets CureDuchenne Investment to Support Gene Therapy Development
The equity investment of an undisclosed amount will fund therapeutic development efforts that use AAV vectors to deliver snRNAs that promote exon skipping.
FDA Advisory Committee in Close Vote Backs Approval of Sarepta Therapeutics' DMD Gene Therapy
After weighing FDA and company experts' divergent interpretations of the evidence, the expert panel decided to recommend approval, eight to six.