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Sarepta pauses Duchenne gene therapy for non-ambulatory individuals after second death due to acute liver failure
Letter to the Community:
US leadership emphasizes support for rare disease drug development
CureDuchenne is pleased that one of the main themes at the recent US FDA Cell and Gene Therapy Roundtable was preserving incentives and leveraging regulatory flexibility and innovation to remove […]
Regenxbio reports positive interim data from Phase 1/2 microdystrophin gene therapy trial
Regenxbio shared positive data from the AFFINITY DUCHENNE trial of RGX-202. In addition to robust microdystrophin expression, Regenxbio reported functional benefits at 9 and 12 months after treatment for all […]
CureDuchenne Ventures Invests in Entos Pharmaceuticals for Development of a Redosable, Full-Length Dystrophin Gene Therapy
Newport Beach, Calif. and Edmonton, Canada (May 22, 2025) – CureDuchenne Ventures announced an initial $1M investment in Entos Pharmaceuticals Inc (Entos), a biotech company that develops genetic medicines utilizing […]
Wave Life Sciences: Update on 48-week Data from FORWARD-53 Exon Skipping Study
Wave Life Science Exon Skipping in Duchenne Wave Life Sciences: Update on 48-week Data from FORWARD-53 Exon Skipping Study: An update on Wave’s Phase 2 FORWARD-53 trial of WE-N531, Wave’s […]
Promising New Data from Wave Life Sciences in Duchenne Exon-skipping Trial
Wave Life Sciences Exon-skipping Trial Wave Life Sciences has shared new data from its ongoing FORWARD-53 clinical trial, evaluating the investigational exon-skipping therapy WVE-N531 for individuals with Duchenne muscular dystrophy […]
RegenXBio continues to report positive biomarker data from their AAV-microdystrophin gene therapy trial
Capricor announces the FDA will decide on approval of their experimental cell therapy in August 2025
CureDuchenne provided early funding to Capricor Therapeutics, and we are pleased to share that the FDA has accepted its Biologics Licenses Application (BLA) seeking approval for Deramiocel to treat cardiomyopathy in […]
Avidity plans to submit for FDA Accelerated Approval for Duchenne exon 44 skipping at the end of 2025; Open-label extension trial is still recruiting.
Avidity Biosciences, which received early funding from CureDuchenne, plans to submit for US FDA Accelerated Approval at the end of 2025 for Delpacibart zotadirsen (del-zota), an exon-skipping experimental therapeutic for […]