As an early funder of Entrada Therapeutics, we are pleased to share that the FDA has removed the clinical hold on ENTR-601-44, the experimental therapeutic designed for individuals with Duchenne […]
Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003 Solid Biosciences, announced positive initial results from INSPIRE DUCHENNE, their next-generation gene therapy clinical study of […]
As an early funder of Entrada, CureDuchenne is pleased to share that Entrada received authorization in the United Kingdom (UK) to start a Phase 1/2 trial for individuals with Duchenne […]
Sarepta Therapeutics shared positive topline results from Part 2 of the EMBARK study, showing that treatment with the microdystrophin gene therapy, Elevidys, is associated with sustained benefits and disease stabilization […]
Amenable to skipping exon 44 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), […]
Dyne Therapeutics, which received early funding from CureDuchenne, plans to pursue US Accelerated Approval from the FDA in 2026 for DYNE-251, an exon-skipping experimental therapeutic for individuals with Duchenne amenable […]
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 […]
Capricor Therapeutics, which received early funding from CureDuchenne, has completed a Biologics License Application (BLA) with the US FDA, seeking full approval of deramiocel for individuals with Duchenne cardiomyopathy. Deramiocel […]
Australian company Percheron Therapeutics released topline 6-month data from its Phase 2 study of avicursen in non-ambulatory individuals with Duchenne. The trial did not meet its primary endpoint, and there were […]
