CIRM Awarded $7.4 Million Grant to Advance Novel AAV Gene Therapy for Rare Muscular Dystrophy

December 16, 2025

WOODBRIDGE, Conn. — Cure Rare Disease (CRD) announced it has been awarded a substantial $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to accelerate the development of a novel AAV (adeno-associated virus) gene therapy, CRD-003, for Limb-Girdle Muscular Dystrophy Type 2i/R9 (LGMD2i/R9).

LGMD2i/R9 is a progressive and debilitating neuromuscular disorder caused by mutations in the FKRP gene, for which there are currently no approved treatments. The CIRM funding is critical for advancing this investigational therapy toward clinical trials.

CRD-003: A Second-Generation AAV with Targeted Delivery

The AAV gene therapy, CRD-003, is designed to deliver a healthy copy of the FKRP gene to muscle cells. The program utilizes a second-generation AAV capsid known as AAVMYO2, which features a novel, dual design aimed at improving the safety and efficacy profile of the AAV therapy:

• Muscle-Tropic: The AAVMYO2 capsid is designed to specifically target and efficiently deliver the genetic cargo to muscle tissue.

• Liver-Detargeting: The design is intended to reduce the accumulation of the AAV vector in the liver, addressing a major safety concern associated with older-generation AAV gene therapies.

CRD CEO and founder Richard Horgan expressed excitement at the opportunity of bringing this “second-generation AAV capsid into the clinic.”

Clear Regulatory Path to Clinic

CRD and its collaborators have generated promising preclinical data for this specialized AAV gene therapy. The program has already successfully completed a pre-IND (Investigational New Drug) meeting with the U.S. Food and Drug Administration (FDA) and secured Orphan Drug Designation (ODD), establishing a clear regulatory pathway for clinical development.

The CIRM grant will fund several crucial steps for the AAV gene therapy program:

• Completion of late-stage preclinical development.

• GMP manufacturing of the AAV vector.

• IND submission to the FDA.

• Initiation of a Phase 1/2 clinical trial, which will be led by Principal Investigator Dr. Tahseen Mozaffar.

Advocates for the LGMD2i/R9 community hailed the investment, noting that the technology demonstrates how advances in one subtype can inform safer, more effective AAV approaches across multiple forms of LGMD.