huntington’s

Harness Therapeutics Nominates HRN001, a First-in-Class Drug Candidate for Huntingtons Disease and Establishes Clinical Advisory Board

HRN001 is a potent and specific antisense oligonucleotide designed to drive controlled upregulation of FAN1, a genetically validated target in…

2 weeks ago

Skyhawk Therapeutics Announces Nine Month Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease

Nine-month findings show mean improvement in Composite Unified Huntington's Disease Rating Scale from baseline of +0.64 points, compared to natural…

1 month ago

uniQure Provides Regulatory Update on AMT-130 for Huntingtons Disease

LEXINGTON, Mass. and AMSTERDAM, Dec. 04, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing…

3 months ago

Skyhawk Therapeutics Announces Positive First Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease

SKY-0515 achieves dose-dependent reductions of mutant huntingtin (mHTT) protein, with 62% lowering at Day 84 on the 9mg daily oral…

5 months ago