ENVISION Gene Therapy Sarepta Therapeutics, which received early funding from CureDuchenne, has provided a community letter outlining the status of ongoing research in their gene therapy and exon-skipping programs. Of […]
Satellos Bioscience, which received prior funding from CureDuchenne, has announced that after meeting with the FDA they have amended the primary endpoint in their Phase 2 BASECAMP trial to now […]
Becker muscular dystrophy Servier announced that topline results from the 18-month placebo controlled GRAND CANYON study in Becker muscular dystrophy did not meet the primary endpoint, with no statistically significant […]
Exon 45 and Exon 44 Skipping Program Updates As an early investor in Entrada Therapeutics, we are pleased to share updates on their Exon 45 and Exon 44 skipping programs […]
Capricor Therapeutics presented data from HOPE-3 Open Label Extension study at the 2026 World Muscle Society Congress indicating that deramiocel treatment is associated with a slower decline in upper limb […]
ELEVIDYS Data Read the press release and letter to the community:
Secretome Therapeutics has initiated an open label phase 2 study to evaluate the safety and potential skeletal muscle and cardiac effects of STM-01, an investigational cell therapy derived from cardiac progenitor cells. […]
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 […]
