As an early funder of Avidity Biosciences, we are pleased to share that Novartis has announced that the FDA has accepted the Biologics License Application (BLA) for delpacibart zotadirsen (del-zota) for Priority […]
Capricor Therapeutics announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) target action date for its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy […]
Precision BioSciences has shared that the first participant in the FUNCTION_DMD clinical trial has received their investigational gene editing treatment, PBGENE-DMD. PBGENE-DMD delivers ARCUS nucleases via AAV9 to cut out […]
Capricor Therapeutics recently shared an update on the FDA review of their investigational therapy, deramiocel. The company shared that the Biologics License Application (BLA) for deramiocel remains under FDA review […]
FDA Will Review Dyne Application for Skipping Exon 51 CureDuchenne was an early funder of Dyne Therapeutics, and we are pleased to share that the FDA has accepted the BLA […]
Italfarmaco reported new data from the Phase 3 EPIDYS trial, showing that quantitative MRI measures of muscle mass and fat fraction were significantly different between the treatment and placebo groups. […]
Proposed text: CureDuchenne was an early funder of Sarepta Therapeutics, and we are pleased to share that the US FDA has accepted supplemental New Drug Applications (sNDAs) for AMONDYS 45 […]
Solid Biosciences has provided an update to the community about SGT-003, their AAV-delivered microdystrophin gene therapy. In the Phase 1/2 INSPIRE DUCHENNE clinical trial, 53 boys have been dosed, ranging […]
