Skyhawk Therapeutics, Inc.

Skyhawk Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Waltham, Massachusetts, USA. The company focuses on the discovery and development of novel small molecule therapies designed to modulate RNA expression. Its mission is to revolutionize patient treatment for a range of intractable diseases by correcting underlying genetic causes.

Utilizing its proprietary SKYSTAR® platform, Skyhawk Therapeutics develops RNA-targeting small molecules across various therapeutic areas, including neurology, oncology, fibrotic diseases, neurodegenerative diseases, and autoimmune conditions. Its lead product candidate, SKY-0515, is an RNA splicing modulator currently in Phase 2/3 clinical trials for Huntington's Disease. The company also has an expanding internal pipeline of other neuro programs, such as SKY-0925, SKY-2100, and SKY-4200, targeting rare neurological indications. Skyhawk operates primarily through a B2B model, engaging in collaborations with major pharmaceutical firms including BMS, Biogen, Genentech, Ipsen, Merck, Sanofi, Takeda, Vertex, and Merck KGaA, to co-develop RNA-targeting therapeutics.

In recent news, Skyhawk Therapeutics announced positive twelve-month interim results from its Phase 1/2 clinical trial of SKY-0515 for Huntington's Disease in June 2026, demonstrating significant reductions in mutant huntingtin protein and improvements in cUHDRS scores. The global Phase 2/3 FALCON-HD pivotal study for SKY-0515 is actively expanding. Leadership changes in June 2024 saw Clint Musil promoted to Chief Executive Officer, with co-founder Bill Haney transitioning to Executive Chairman. The company remains privately held, having secured substantial funding through venture capital and strategic partnerships.