Sarepta Therapeutics filed a clinical trial application for its Huntington’s disease program, SRP‑1005‑101 (INSIGHTT), with Medsafe, the New Zealand Medicines and Medical Devices Safety Authority, on January 7, 2026. The submission marks the first regulatory step toward a first‑in‑human trial of the company’s siRNA‑based therapy, which is designed to knock down the mutant huntingtin protein that drives the neurodegenerative disease.
The siRNA platform that underpins SRP‑1005 was originally developed by Arrowhead Pharmaceuticals and licensed to Sarepta in a 2024 collaboration that granted the company exclusive global rights to multiple clinical, pre‑clinical, and discovery‑stage programs. The partnership has enabled Sarepta to leverage its expertise in precision genetic medicine and to accelerate the development of CNS‑targeted therapies.
SRP‑1005 uses a transferrin receptor‑1 (TfR1)–mediated delivery system that allows the siRNA to cross the blood‑brain barrier after subcutaneous injection. This approach is expected to provide robust central nervous system exposure while maintaining a convenient dosing schedule, a key advantage over other modalities such as antisense oligonucleotides.
Huntington’s disease affects roughly 40,000 people in the United States and an additional 200,000 carriers of the pathogenic mutation. With no approved disease‑modifying treatments, the market represents a significant unmet need. The launch of a first‑in‑human trial positions Sarepta to address a high‑impact therapeutic area and to diversify its revenue base beyond its flagship Duchenne muscular dystrophy gene therapy, Elevidys.
The submission signals a strategic expansion of Sarepta’s rare‑disease portfolio. By moving into a neurodegenerative indication, the company broadens the reach of its siRNA platform and opens new avenues for future approvals. The timing of the Medsafe review—typically around 45 days—means that, if approved, the trial could begin in the second quarter of 2026, aligning with Sarepta’s broader pipeline development schedule.
Louise Rodino‑Klapac, Ph.D., President of Research & Development and Technical Operations, said, “Huntington’s disease is a devastating, progressive condition with no approved treatments to slow or halt its course. SRP‑1005 represents a novel and potentially paradigm‑changing approach to targeting the underlying cause of this disease.”
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.