AAVMYO2

AAVMYO2 is an engineered adeno-associated viral (AAV) capsid designed for high muscle specificity and liver detargeting in gene therapy applications. Developed through a semirational, combinatorial approach, this peptide-displaying chimeric AAV capsid (also designated as AAVS1P1) is utilized as the delivery vehicle for the investigational gene therapy program CRD-003. It is primarily being developed by Cure Rare Disease to treat Limb-Girdle Muscular Dystrophy Type 2i/R9 (LGMDR9) by delivering a functional copy of the fukutin-related protein (FKRP) gene. Preclinical studies have demonstrated that AAVMYO2 provides superior muscle transduction and improved safety profiles compared to traditional serotypes like AAV8 in models of Duchenne muscular dystrophy and X-linked myotubular myopathy, showing prolonged survival and restored muscle function at lower doses.

Other names

AAVS1P1AAVS-1P1AAVS 1P1