SRP-6006

SRP-6006 is a next-generation, dual-vector adeno-associated virus (AAV) gene therapy developed by Sarepta Therapeutics for the treatment of limb-girdle muscular dystrophy type 2B/R2 (LGMD2B/R2). This ultra-rare neuromuscular disease is caused by mutations in the *DYSF* gene, which encodes dysferlin, a protein critical for muscle membrane repair. Due to the large size of the *DYSF* gene (~6.2 kb), which exceeds the packaging capacity of a single AAV vector (~5 kb), SRP-6006 utilizes a dual-vector system where the 5' and 3' portions of the transgene are delivered separately and reconstituted in vivo through homologous recombination. SRP-6006 employs a novel myotropic capsid, AAVrh74Myo, and the MHCK7 promoter to optimize muscle transduction and protein expression. Preclinical data indicates that SRP-6006 offers significantly enhanced potency and improved membrane repair at lower doses compared to the earlier clinical candidate SRP-6004.