CRD-003

CRD-003 is an adeno-associated virus (AAV) gene therapy developed by the nonprofit biotechnology company Cure Rare Disease for the treatment of Limb-Girdle Muscular Dystrophy Type 2i/R9 (LGMD2i/R9). The therapy utilizes a novel muscle-tropic capsid, AAVMYO2, which is engineered to selectively target skeletal and cardiac muscle while de-targeting the liver, thereby potentially improving safety and reducing the required therapeutic dose. LGMD2i/R9 is caused by mutations in the fukutin-related protein (FKRP) gene, which is essential for the proper glycosylation of alpha-dystroglycan to maintain muscle fiber integrity. CRD-003 delivers a functional copy of the FKRP gene to restore this biological process. The program has received Orphan Drug Designation from the FDA and is currently in late-stage preclinical development, supported by a grant from the California Institute for Regenerative Medicine (CIRM) to advance toward IND submission and Phase 1/2 clinical trials.

Other names

Cure Rare Disease-FKRPFKRP Gene TherapyAAVMYO2-FKRPAAVMYO-2-FKRPAAVMYO 2-FKRP