Precision BioSciences Highlights New Preclinical Data for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with...
Sarepta Therapeutics Halts ELEVIDYS Shipments for Non-Ambulatory DMD Patients
Due to two documented incidents of severe liver failure that resulted in death, Sarepta Therapeutics has stopped shipping its gene treatment ELEVIDYS for individuals...
JCR Pharmaceuticals Proprietary JUST-AAV Capsids May be Used in the Development of Duchenne Cures
Alexion, AstraZeneca’s rare disease subsidiary, is licensing specialized gene therapy vehicles from the Japanese firm JCR Pharmaceuticals.The deal, announced on 8th June 2025, gives...
Taiho Pharmaceutical Announced TAS-205 Phase 3 Clinical Study Did Not Meet Its Primary Endpoint
Taiho Pharmaceutical announced that the results of a phase III, randomized, placebo-controlled, double-blind and open-label, extension study of TAS-205 (Pizuglanstat) in patients with Duchenne...
Edgewise Therapeutics Reports Positive Results on Sevasemten Program for Becker and Duchenne Muscular Dystrophies
Edgewise Therapeutics, a leading muscle disease biopharmaceutical company, today unveiled positive results in its sevasemten program for Becker and Duchenne muscular dystrophies.What is Sevasemten?Sevasemten:...