CIRM Awards Cure Rare Disease $7.4 Million to Advance LGMD Gene Therapy

Rare Daily Staff

The California Institute for Regenerative Medicine has awarded Cure Rare Disease a $7.4 million grant to advance the development of a novel gene therapy for the progressive neuromuscular disorder limb-girdle muscular dystrophy Type 2I/R9.

Limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9) is caused by mutations in the FKRP gene. The disease is characterized by progressive muscle weakness and wasting. Over time, it can lead to significant mobility issues and respiratory complications. There are currently no approved treatments for LGMD2I/R9, making the development of effective genetic medicines a critical unmet need.

Cure Rare Disease’s CRD-003 is an experimental gene therapy designed with a novel, liver-de-targeting, muscle-tropic capsid, AAVMYO2, intended to improve safety and efficacy by directing the therapy specifically to muscle tissue.

In earlier developmental stages, the Cure Rare Disease team and its collaborators generated promising preclinical data. The organization has successfully completed a pre-IND meeting with the U.S. Food and Drug Administration and secured orphan drug designation for the therapy, establishing a clear regulatory pathway for future clinical development.

With CIRM funding support, Cure Rare Disease will advance the program through late-stage preclinical development, GMP manufacturing, IND submission, and Phase 1/2 clinical trial initiation.

“This program is a powerful example of what can be achieved when patients, organizations, researchers, and governing bodies come together with a shared mission,” said Cure Rare Disease CEO and founder Richard Horgan. “From the initial support of our community to our collaborations with leading scientists and regulatory guidance from the FDA, the development of a therapy for LGMD2I/R9 has been a truly unified effort.”

Photo: Rich Horgan, founder and CEO of Cure Rare Disease