Sarepta Therapeutics Announces Pause of ELEVIDYS Shipments in the USA
Today, Sarepta Therapeutics notified the U.S. Food and Drug Administration (FDA) of its decision to voluntarily and temporarily pause all shipments of ELEVIDYS (delandistrogene...
Sarepta to Lay Off About 500 Employees After Duchenne Gene Therapy Setbacks
Sarepta stated that company intends to halt development on a number of medicinal products and lay off about 36% of its employees. By 2026,...
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...