Cure Rare Disease Secures FDA Orphan Drug Designation for Investigational Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type R9 (LGMD2i/R9)

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WOODBRIDGE, Conn.--(BUSINESS WIRE)--Sep 24, 2025--

Cure Rare Disease (CRD), a 501(c)(3) nonprofit biotechnology company developing genetic therapies for ultra-rare neuromuscular diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for its investigational therapy, CRD-003, for the treatment of congenital muscular dystrophy caused by biallelic mutations in the FKRP gene (Limb-Girdle Muscular Dystrophy Type R9, LGMD2i/R9).

The investigational therapy uses an adeno-associated viral (AAV) vector (serotype: AAV MYO2) encoding the fukutin-related protein (FKRP) under the control of a muscle-specific promoter to restore glycosylation of alpha-dystroglycan, a critical process that maintains muscle integrity and function. Mutations in the FKRP gene prevent the proper glycosylation of alpha-dystroglycan, leading to progressive weakness in skeletal and cardiac muscles, as well as impacts in the central nervous system. LGMDR9 affects approximately 4.48 per million individuals worldwide.

“Receiving Orphan Drug Designation is a critical milestone for Cure Rare Disease as we advance our gene therapy program for LGMDR9,” said 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 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.”

The FDA’s Orphan Drug Designation program provides incentives to support the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S. Benefits include seven years of market exclusivity upon regulatory approval, tax credits for qualified clinical trials, and waiver of certain FDA fees.

About Cure Rare Disease

Cure Rare Disease, a 501(c)(3) nonprofit biotechnology company based in Woodbridge, CT, is dedicated to developing life-saving genetic therapies for ultra-rare diseases, transforming the lives of patients previously deemed untreatable. By fostering strategic collaborations among top researchers, clinicians, policy experts, and donors, we bring hope and advanced solutions to those awaiting a cure.

For more information, please visit www.cureraredisease.org.

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SOURCE: Cure Rare Disease

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PUB: 09/24/2025 07:05 AM/DISC: 09/24/2025 07:04 AM

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