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 […]
Capricor Therapeutics has shared that the US FDA will hold an Advisory Committee meeting on July 29, 2026, to discuss the company’s Biological License Application (BLA) seeking approval of deramiocel, […]
Exon 44 Skipping CureDuchenne was an early funder of Avidity Biosciences, which is now a Novartis company. We are pleased to share that they have submitted a BLA to the […]
REGENXBIO has completed dosing in the confirmatory study of RGX-202, an AAV-delivered microdystrophin gene therapy for the treatment of Duchenne. The company is on track to initiate a Biologics License […]
As a founding partner and initial funder of the Collaborative Trajectory Analysis Project (cTAP), CureDuchenne is proud to support an important advancement for the Duchenne community: a novel prognostic score that helps […]
SonoThera press release CureDuchenne is pleased to announce that it has invested in SonoThera, supporting the advancement of its targeted, ultrasound-mediated gene delivery technology focused on the delivery of full-length […]
Read Avidity’s Community Letter:
RGX-202: Investigational Gene Therapy for Duchenne Muscular Dystrophy Pivotal Data Update from the AFFINITY DUCHENNE® Study Watch HERE Recorded Tuesday, May 19, 2026
Unlocking the therapeutic power of suppressor tRNAs for DMD Watch HERE Recorded Thursday, May 28, 2026
