At a Glance
- Medsafe approves INSIGHTT clinical trial in New Zealand
- SRP-1005 advances as first-in-human Huntington’s therapy
- Phase 1 study to assess safety in ~24 participants
- Trial initiation expected in Q2 2026
Written By: Samiksha Jadhav BPharm
Reviewed By: Pharmacally Editorial Team
Sarepta Therapeutics, Inc. received approval from New Zealand’s Medsafe to initiate its first-in-human clinical trial evaluating SRP-1005, an investigational small interfering RNA (siRNA) therapy for Huntington’s disease. The cleared clinical trial application (CTA) supports Study SRP-1005-101, known as INSIGHTT, with trial initiation planned for Q2 2026.
INSIGHTT is designed as a multi-center, dose-escalation Phase 1 study assessing the safety and tolerability of subcutaneous SRP-1005 in approximately 24 participants. The therapy leverages a transferrin receptor protein 1 (TfR1) delivery strategy utilizing a monovalent fragment antigen binding (fAb) approach, intended to enhance central nervous system penetration while minimizing receptor saturation. This subcutaneous administration model represents a notable departure from more invasive CNS delivery techniques.
Preclinical data suggest that SRP-1005 may enable meaningful huntingtin protein knockdown across brain regions heavily affected in Huntington’s disease, including the putamen, caudate, temporal cortex, and frontal cortex. These findings underpin the rationale for advancing the therapy into early clinical evaluation.
Huntington’s disease is caused by a mutation in the huntingtin (HTT) gene, resulting in progressive neurodegeneration that affects cognition, motor function, and behavior. The condition impacts an estimated 40,000 symptomatic individuals in the United States, with roughly 200,000 people carrying the genetic mutation that confers future risk. Despite decades of research, no approved disease-modifying therapies currently address the underlying genetic driver of the disease.
SRP-1005 forms part of Sarepta’s broader siRNA platform, which is focused on developing chronically administered therapies for neurodegenerative and pulmonary disorders. Beyond Huntington’s disease, the company is pursuing programs in facioscapulohumeral muscular dystrophy (FSHD), myotonic dystrophy type 1 (DM1), spinocerebellar ataxia (SCA), and idiopathic pulmonary fibrosis (IPF). Sarepta also maintains a strategic collaboration with Arrowhead Pharmaceuticals covering multiple muscle and CNS targets.
With regulatory clearance secured, SRP-1005 now advances into clinical testing as Sarepta explores a novel RNA-based approach for treating Huntington’s disease.
Reference
Sarepta Therapeutics Announces Approval of Clinical Trial Application for SRP-1005, Its Investigational Treatment for Huntington’s Disease, 04 February 2026, Sarepta Therapeutics Announces Approval of Clinical Trial Application for SRP-1005, Its Investigational Treatment for Huntington’s Disease | Sarepta Therapeutics, Inc.