Cure Rare Disease Receives FDA Feedback on Limb-Girdle 2i/R9 Gene Therapy Program Following Successful Pre-IND Meeting

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

The LGMDR9 program, known as CRD-003, utilizes a novel, engineered AAVMYO2 capsid to deliver a functional copy of the FKRP gene. Murine and non-human primate studies support that AAVMYO2 de-targets the liver while offering enhanced muscle tropism which allows for a reduction of the clinical dose. CRD-003 is designed for patients with LGMDR9 caused by mutations in the FKRP gene, a condition that leads to progressive muscle weakness and loss of ambulation. CRD-003 is intended to be administered as a single intravenous injection and is among the first gene therapy candidates tailored to this rare neuromuscular disease population using a next-generation AAV capsid.

Following the pre-IND interaction, the FDA provided written feedback across key domains including Chemistry, Manufacturing, and Controls (CMC), nonclinical development, and clinical trial design. The agency supported CRD’s overall development approach while offering constructive guidance to further strengthen the planned Investigational New Drug (IND) submission. Notably:

• 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.
• The agency suggested that CRD-003 may seek accelerated approval on the basis of results from the Phase I/II clinical trial, pending 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 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. This collaborative dialogue helps us navigate the complexities of developing therapies for rare diseases while furthering our ability to bring much-needed treatments to the individuals who need them most.”

With this milestone completed, CRD will continue executing its IND-enabling studies, incorporating FDA recommendations, and preparing for IND submission.

About Cure Rare Disease

Cure Rare Disease, a 501(c)(3) nonprofit biotechnology company based in Woodbridge, CT, is transforming possibilities for people with ultra-rare diseases by developing advanced therapeutics in time to save lives. Through a collaborative community of forward-thinking families, patients, scientists and supporters, Cure Rare Disease has enabled an ecosystem of innovation and discovery to overcome the obstacles inherent in existing models of medicine and to expedite life-saving genetic technologies from research to the clinic.

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

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