 has granted Orphan Drug Designation (ODD) to CRD-003, an investigational gene therapy developed by the nonprofit biotechnology organization Cure Rare Disease (CRD), for the treatment of congenital muscular dystrophy caused by biallelic mutations in the FKRP gene, also classified as Limb-Girdle Muscular Dystrophy Type R9 (LGMDR9).){kind=link}
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CRD-003, an investigational gene therapy developed by the nonprofit biotechnology organization Cure Rare Disease (CRD), for the treatment of congenital muscular dystrophy caused by biallelic mutations in the FKRP gene, also classified as Limb-Girdle Muscular Dystrophy Type R9 (LGMDR9).
CRD-003 employs an adeno-associated viral (AAV) vector, specifically the AAV MYO2 serotype, to deliver a functional copy of the fukutin-related protein (FKRP) gene. Expression is driven by a muscle-specific promoter with the aim of restoring glycosylation of alpha-dystroglycan, a key biochemical modification required to maintain muscle fiber stability and function. Disruption of this pathway, due to mutations in FKRP, underlies the progressive muscle degeneration characteristic of LGMDR9. In addition to skeletal muscle weakness, the condition frequently involves cardiac dysfunction and can extend to the central nervous system. Epidemiological estimates suggest a global prevalence of ~4.48 cases per million individuals.
Receiving Orphan Drug Designation is a critical milestone for Cure Rare Disease as we advance our gene therapy program for LGMDR9. Patients and families affected by FKRP-related muscular dystrophies currently face a devastating lack of treatment options. This designation not only supports our approach, but also underscores the urgent need for innovative genetic medicines that can meaningfully change the lives of patients and their loved ones. Using a liver de-targeting, second-generation AAV vector, we believe this may be a safer approach compared to first-generation capsids.
Richard Horgan, Founder and CEO of Cure Rare Disease.
The FDA’s ODD program is designed to encourage therapeutic development in diseases affecting fewer than 200,000 people in the United States. Incentives include up to seven years of market exclusivity following approval, eligibility for federal tax credits on clinical testing costs, and exemption from certain regulatory fees.