FDA Delays Decision for Sarepta Therapeutics Duchenne Muscular Dystrophy Gene Therapy
According to Sarepta, the federal agency has indicated it may grant accelerated approval to SRP-9001 as a treatment for a subgroup of patients based on age.
Avidity Biosciences' Antibody Oligonucleotide Conjugates Progressing in Duchenne Muscular Dystrophy
AOC-1044, a lead drug in the company's pipeline, is undergoing a Phase I/II trial in DMD patients with mutations amenable to exon 44 skipping.
Locanabio Gets CureDuchenne Investment to Support Gene Therapy Development
The equity investment of an undisclosed amount will fund therapeutic development efforts that use AAV vectors to deliver snRNAs that promote exon skipping.
FDA Advisory Committee in Close Vote Backs Approval of Sarepta Therapeutics' DMD Gene Therapy
After weighing FDA and company experts' divergent interpretations of the evidence, the expert panel decided to recommend approval, eight to six.
MyoGene Bio Nets Additional Investment From CureDuchenne Ventures for CRISPR-Based Treatment
The Los Angeles-based biotech will use the funds to develop a gene editing approach targeting the DMD hotspot region where many disease-linked mutations occur.