Cure Rare Disease (CRD) has achieved a significant regulatory milestone with the successful completion of a pre-Investigational New Drug (pre-IND) meeting with the U.S. Food and Drug Administration regarding its investigational gene therapy program for Limb-Girdle Muscular Dystrophy type 2I/R9 (LGMDR9). The nonprofit biotechnology organization announced that the FDA provided constructive feedback supporting the overall development approach for CRD-003, while offering guidance to strengthen the planned IND submission.
Novel Gene Therapy Approach
CRD-003 utilizes a novel, engineered AAVMYO2 capsid to deliver a functional copy of the FKRP gene to patients with LGMDR9. The therapy is designed for patients with mutations in the FKRP gene, a condition that leads to progressive muscle weakness and loss of ambulation. Murine and non-human primate studies demonstrate that AAVMYO2 de-targets the liver while offering enhanced muscle tropism, which allows for a reduction of the clinical dose.
The treatment is intended to be administered as a single intravenous injection and represents one of the first gene therapy candidates tailored to this rare neuromuscular disease population using a next-generation AAV capsid.
FDA Endorsement and Regulatory Pathway
The FDA provided written feedback across key domains including Chemistry, Manufacturing, and Controls (CMC), nonclinical development, and clinical trial design. Several notable regulatory agreements emerged from the meeting:
The FDA agreed that a 4-month murine GLP toxicology study, incorporating acute and chronic toxicity assessments, could be sufficient to support initiation of the first-in-human trial. The agency endorsed the proposed clinical trial design and patient selection strategy, and deemed the biomarker strategy—evaluating glycosylation changes and muscle MRI—appropriate.
Significantly, the agency suggested that CRD-003 may seek accelerated approval on the basis of results from the Phase I/II clinical trial, pending results.
Strategic Development Approach
"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 a cornerstone of our drug development strategy. It ensures that our programs are aligned with regulatory expectations from the outset, allowing us to prioritize both patient safety and operational efficiency."
Horgan emphasized that this collaborative dialogue helps navigate the complexities of developing therapies for rare diseases while furthering the organization's ability to bring much-needed treatments to patients who need them most.
Next Steps
With this milestone completed, CRD will continue executing its IND-enabling studies, incorporating FDA recommendations, and preparing for IND submission. The company's approach reflects its mission as a 501(c)(3) nonprofit biotechnology organization based in Woodbridge, Connecticut, focused on transforming possibilities for people with ultra-rare diseases by developing advanced therapeutics.
