Cure Rare Disease Advances AAV Gene Therapy for LGMDR9 Following Positive FDA Pre-IND Meeting

WOODBRIDGE, Conn. – Cure Rare Disease (CRD), a nonprofit biotechnology organization developing genetic therapies for ultra-rare diseases, today announced a successful pre-Investigational New Drug (pre-IND) meeting with the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy program for Limb-Girdle Muscular Dystrophy type 2I/R9 (LGMDR9).

The program, CRD-003, utilizes a novel, engineered AAVMYO2 capsid to deliver a functional copy of the FKRP gene. This innovative AAV design allows for a reduced clinical dose by de-targeting the liver and enhancing muscle tropism, as supported by murine and non-human primate studies. CRD-003 is designed for LGMDR9 patients with FKRP gene mutations, a condition causing progressive muscle weakness and loss of ambulation. Administered as a single intravenous injection, CRD-003 is among the first AAV gene therapy candidates for this rare neuromuscular disease using a next-generation AAV capsid.

The FDA provided constructive guidance across key areas, including Chemistry, Manufacturing, and Controls (CMC), nonclinical development, and clinical trial design, while generally supporting CRD’s overall development approach. Notably, the FDA agreed that a 4-month murine GLP toxicology study could suffice for initiating the first-in-human trial, endorsed the proposed clinical trial design and patient selection, and deemed the biomarker strategy (evaluating glycosylation changes and muscle MRI) appropriate. The agency also suggested CRD-003 may seek accelerated approval based on Phase I/II clinical trial results.

“Receiving FDA feedback is a critical milestone as we advance CRD-003 toward the clinic,” said Rich Horgan, Founder and CEO of Cure Rare Disease. “Early and proactive engagement with the FDA is paramount to our drug development strategy, ensuring our programs align with regulatory expectations from the outset. This collaborative dialogue helps us navigate the complexities of rare disease therapy development and brings us closer to delivering much-needed treatments to patients.”

CRD will now proceed with IND-enabling studies, incorporating FDA recommendations, and preparing for IND submission.