CRD-DYSF

CRD-DYSF is an investigational gene therapy being developed by Cure Rare Disease for the treatment of Limb-Girdle Muscular Dystrophy Type 2B (LGMD2B), also known as dysferlinopathy. LGMD2B is an autosomal recessive muscle-wasting disease caused by mutations in the *DYSF* gene, which encodes the dysferlin protein. Dysferlin is essential for the calcium-dependent repair of muscle cell membranes (sarcolemma); its deficiency leads to progressive muscle weakness and degeneration. Because the *DYSF* cDNA is approximately 6.5 kb, exceeding the standard 4.7 kb packaging capacity of a single adeno-associated virus (AAV) vector, the program explores advanced delivery strategies such as dual-AAV vector systems or CRISPR-based gene editing to restore functional dysferlin expression. The program is currently in the IND-enabling phase, focusing on manufacturing and safety studies required for clinical trial initiation.

Other names

DYSF gene therapyCure Rare Disease LGMD2B program