Cure Rare Disease Secures FDA Orphan Drug Designation for AAV Gene Therapy Targeting Muscular Dystrophy

WOODBRIDGE, Conn. – September 25, 2025–  Cure Rare Disease (CRD), a non-profit biotechnology organization focused on developing genetic therapies for ultra-rare neuromuscular diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational therapy, CRD-003.

CRD-003 is a potential treatment for congenital muscular dystrophy caused by mutations in the FKRP gene, specifically known as Limb-Girdle Muscular Dystrophy Type R9 (LGMDR9). LGMDR9 is an ultra-rare condition, affecting approximately 4.48 per million people worldwide, highlighting the severe lack of therapeutic options for this patient population.

AAV Vector Design Aims for Safer, Targeted Delivery

The investigational therapy, CRD-003, is an adeno-associated viral (AAV) vector designed to address the genetic cause of the disease. It utilizes a second-generation AAV serotype (AAV MYO2) to deliver a functional FKRP gene. This vector is engineered with a muscle-specific promoter to target skeletal and cardiac muscles, and crucially, features liver de-targeting to potentially enhance safety compared to first-generation AAV capsids.

The goal of the therapy is to restore the essential process of glycosylation of alpha-dystroglycan. Mutations in the FKRP gene prevent this proper glycosylation, leading to progressive muscle weakness and affecting both the skeletal and cardiac muscles, as well as the central nervous system.

“Receiving Orphan Drug Designation is a critical milestone for Cure Rare Disease as we advance our gene therapy program for LGMDR9,” stated Richard Horgan, Founder and CEO of Cure Rare Disease. “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.”

Orphan Status Provides Development Incentives

The FDA’s Orphan Drug Designation program offers key incentives for developing treatments for conditions affecting fewer than 200,000 people in the U.S. These benefits include tax credits for qualified clinical trials, waivers of certain FDA fees, and, most importantly, seven years of market exclusivity upon receiving regulatory approval.