CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the submission of its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT, to Medsafe, the New Zealand Medicines and Medical Devices Safety Authority. Pending approval, Sarepta expects to initiate this 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 the treatment of Huntington’s Disease.
“Huntington’s disease is a devastating, progressive condition with extremely limited therapeutic options and no approved treatments to slow or halt its course. Families carry the burden across generations, and the need for new approaches is urgent,” said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta. “SRP-1005, a subcutaneously dosed siRNA therapy intended to target the deep brain and knock down the protein that causes Huntington’s disease, represents a novel and potentially paradigm changing approach to the treatment of this devastating disease.”
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 delivery aims to allow the therapy to stay below transferrin saturation and achieve constant and robust penetration across the blood brain barrier. Preclinical data with SRP-1005 have 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.
About Huntington’s Disease
Huntington’s Disease (HD) is a rare and ultimately fatal inherited neurodegenerative disorder which is passed down from generation to generation within affected families. HD is caused by a mutation in the gene for a protein called huntingtin, or HTT, which leads to progressive deterioration of nerve cells in the brain, affecting cognition, movement, and behavior. In the U.S. about 40,000 people are living with symptomatic HD, and an additional 200,000 individuals carry the genetic mutation and are at risk of developing symptoms. Symptoms typically appear between ages 30 and 50 and worsen over time, with every child of an affected parent having a 50% chance of inheriting the condition. There is currently no known cure or approved disease-modifying treatments that address the underlying cause.
About Sarepta’s siRNA Platform
Sarepta’s next-generation siRNA platform is focused on chronically administered therapies for neurodegenerative and pulmonary diseases and includes 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.
About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.
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