New Zealand — January 7, 2026 — Leads & Copy — Sarepta Therapeutics, Inc. (NASDAQ:SRPT) has submitted a clinical trial application (CTA) for Study SRP-1005-101, known as INSIGHTT, to Medsafe, the New Zealand Medicines and Medical Devices Safety Authority. The company anticipates initiating the first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026, pending approval.
SRP-1005 is an investigational small interfering RNA (siRNA) therapeutic intended for Huntington’s Disease treatment.
Louise Rodino-Klapac, Ph.D., president of research & development and technical operations at Sarepta, emphasized the urgent need for new treatments for Huntington’s disease, a progressive condition with limited therapeutic options. She noted that SRP-1005 represents a potentially paradigm-changing approach by targeting the protein that causes Huntington’s disease with a subcutaneously dosed siRNA therapy designed to reach the deep brain.
The INSIGHTT study is a Phase 1, multi-center, dose escalation trial that will assess the safety and tolerability of SRP-1005, administered subcutaneously, in approximately 24 participants. The therapy utilizes an advanced TfR1 (transferrin receptor protein 1) approach, incorporating a monovalent fragment antigen binding (fAb) for efficient delivery to the central nervous system. Subcutaneous delivery aims to maintain the therapy below transferrin saturation, enabling consistent penetration across the blood-brain barrier. Preclinical data have indicated 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 gene, leading to the deterioration of nerve cells in the brain. This affects cognition, movement, and behavior. In the U.S., approximately 40,000 people have symptomatic HD, with an additional 200,000 at risk due to carrying the genetic mutation. Symptoms typically manifest between ages 30 and 50 and worsen over time. Currently, there is no known cure or approved treatments that modify the underlying cause of the disease.
Sarepta’s next-generation siRNA platform is focused on therapies for neurodegenerative and pulmonary diseases, including treatments for facioscapulohumeral muscular dystrophy (FSHD), myotonic dystrophy type 1 (DM1), spinocerebellar ataxia type 2 (SCA2), idiopathic pulmonary fibrosis (IPF), and 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.
Sarepta Therapeutics is focused on precision genetic medicine for rare diseases. The company holds leadership positions in Duchenne muscular dystrophy and is building a portfolio of programs across muscle, central nervous system, and cardiac diseases.
Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta.
Source: Sarepta Therapeutics
