Last updated: October 2026 SPK-10001 is an investigational gene therapy program from Spark Therapeutics designed to reduce huntingtin expression. Current status It remains in early clinical development, where researchers are evaluating delivery, safety, and biological activity. This candidate is investigational and is not FDA approved. Timing, eligibility, safety, effectiveness, availability, and cost remain uncertain. For…

Last updated: October 2026 SRP-1005 is an investigational small-interfering RNA treatment being developed by Sarepta Therapeutics to lower huntingtin expression. Current status Its first-in-human clinical program is focused on safety, tolerability, and evidence of target engagement. This candidate is investigational and is not FDA approved. Timing, eligibility, safety, effectiveness, availability, and cost remain uncertain. For…

Last updated: October 2026 VO659 is an investigational antisense oligonucleotide from Vico Therapeutics designed to target the expanded CAG repeat associated with Huntington’s disease. Current status A Phase 1/2a study is evaluating VO659 with dosing schedules and safety as key early questions. This candidate is investigational and is not FDA approved. Timing, eligibility, safety, effectiveness,…

Last updated: October 2026 WVE-003 is an investigational allele-selective treatment candidate from Wave Life Sciences, designed to target the mutant huntingtin gene while preserving the healthy copy. Current status It remains in early clinical development, and additional trials and data will be needed before any regulatory decision. It is not FDA approved, and timing, eligibility,…

Last updated: October 2026 Tominersen is an antisense oligonucleotide delivered by spinal injection and being studied to reduce huntingtin production. Current status Its development has continued through a revised clinical approach focused on earlier-stage patients. It is not FDA approved, and timing, eligibility, safety, effectiveness, availability, and cost remain uncertain. For the newest coverage across…

Last updated: October 2026 Votoplam (PTC518) is an oral huntingtin-lowering treatment candidate being developed for Huntington’s disease. Current status Its clinical program remains investigational and continues to be evaluated in clinical trials. It is not FDA approved, and timing, eligibility, safety, effectiveness, availability, and cost remain uncertain. For the newest coverage across the HD treatment…