Sarepta Therapeutics Announces Approval of Clinical Trial for Huntington's Disease Treatment

The first-in-human study of SRP-1005 is expected to begin in Q2 2026.

Feb. 5, 2026 at 2:15am

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Sarepta Therapeutics announced that Medsafe, the New Zealand Medicines and Medical Devices Safety Authority, has granted approval for its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT. Sarepta expects to initiate this first-in-human clinical trial of SRP-1005, an investigational small interfering RNA (siRNA) therapeutic for the treatment of Huntington's Disease, in the second quarter of 2026.

Why it matters

Huntington's Disease is a rare and fatal inherited neurodegenerative disorder that affects cognition, movement, and behavior. There is currently no known cure or approved disease-modifying treatments that address the underlying cause. The approval of this clinical trial for SRP-1005 represents a significant step forward in the development of potential new treatments for this devastating disease.

The details

INSIGHTT is a Phase 1, multi-center, dose escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants. SRP-1005 leverages an advanced TfR1 (transferrin receptor protein 1) approach that uses monovalent fragment antigen binding (fAb) designed for efficient delivery to the central nervous system. Subcutaneous administration is intended to avoid saturating the transferrin receptor and achieve constant and robust penetration across the blood brain barrier.

  • Sarepta expects to initiate the first-in-human clinical trial of SRP-1005 in the second quarter of 2026.

The players

Sarepta Therapeutics

A biopharmaceutical company focused on precision genetic medicine for rare diseases, including Duchenne muscular dystrophy and Huntington's Disease.

Medsafe

The New Zealand Medicines and Medical Devices Safety Authority, which granted approval for Sarepta's clinical trial application for SRP-1005.

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What’s next

Sarepta expects to initiate the INSIGHTT study, the first-in-human clinical trial of SRP-1005, in the second quarter of 2026.

The takeaway

The approval of the clinical trial application for SRP-1005 represents a significant milestone in the development of potential new treatments for Huntington's Disease, a rare and devastating neurodegenerative disorder with no known cure or approved disease-modifying therapies.