February 4, 2026 — Leads & Copy —
Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced that Medsafe, the New Zealand Medicines and Medical Devices Safety Authority, has approved its clinical trial application (CTA) for Study SRP-1005-101, known as INSIGHTT.
Sarepta plans to begin the first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026. SRP-1005 is an investigational small interfering RNA (siRNA) therapeutic for Huntington’s Disease treatment.
The INSIGHTT study is a Phase 1, multi-center, dose escalation study to assess the safety and tolerability of subcutaneous dosing of SRP-1005 in about 24 participants. SRP-1005 uses an advanced TfR1 (transferrin receptor protein 1) approach with monovalent fragment antigen binding (fAb) for efficient delivery to the central nervous system. Subcutaneous administration is designed to avoid saturating the transferrin receptor and achieve constant penetration across the blood brain barrier. Preclinical data for SRP-1005 has demonstrated the potential for significant protein knockdown in key deep brain regions, including the putamen and caudate, as well as the temporal and frontal cortexes.
Huntington’s Disease (HD) is a rare, inherited neurodegenerative disorder caused by a mutation in the huntingtin, or HTT, gene. This mutation leads to progressive deterioration of nerve cells in the brain, impacting cognition, movement, and behavior. Approximately 40,000 people in the U.S. have symptomatic HD, with an additional 200,000 at risk of developing symptoms due to carrying the genetic mutation. Symptoms typically emerge between ages 30 and 50 and worsen over time, with each child of an affected parent having a 50% chance of inheriting the condition. Currently, there is no known cure or approved disease-modifying treatments addressing the underlying cause.
Sarepta’s next-generation siRNA platform focuses on therapies for neurodegenerative and pulmonary diseases, including investigational treatments for:
- Facioscapulohumeral muscular dystrophy (FSHD)
- Myotonic dystrophy type 1 (DM1)
- Spinocerebellar ataxia type 2 (SCA2)
- Idiopathic Pulmonary Fibrosis (IPF)
- Huntington’s disease (HD)
Sarepta is also pursuing preclinical programs for Spinocerebellar ataxia type 1 (SCA1) and Spinocerebellar ataxia type 3 (SCA3) and has an exclusive collaboration with Arrowhead Pharmaceuticals to develop therapies for skeletal muscle diseases, with plans to pursue up to six discovery targets in muscle or central nervous system disorders.
Sarepta Therapeutics engineers precision genetic medicine for rare diseases. It holds a leadership position in Duchenne muscular dystrophy (Duchenne) and is developing a portfolio of programs across muscle, central nervous system, and cardiac diseases.
Source: Sarepta Therapeutics
