Skyhawk's novel RNA-targeting small molecule for Huntington's Disease, SKY-0515, is in clinical trials and has demonstrated a 72% reduction in HTT mRNA in its Phase 1 Healthy Volunteer MAD study
SKYHAWK invents novel small molecules that modify RNA expression, targeting some of the world's most intractable diseases.
​
Our unique platform accelerates building RNA targeting small molecules across a range of fields including neurology, oncology and fibrotic diseases.
We have built collaborations with BMS, Biogen, Genentech, Ipsen, Merck, Sanofi, Takeda and Vertex that leverage our novel SKYSTAR® platform to
target challenging diseases in a variety of therapeutic areas.
Small Molecules that Modify RNA Expression
Skyhawk
Latest News
Skyhawk Therapeutics Presents at Huntington's Disease Youth Organization HDYO International Congress 2025
Skyhawk presents today on its novel SKY-0515 small molecule RNA splicing modulator targeting Huntington's Disease to members of the Huntington's Disease Youth Organization, at the annual HYDO International Congress in Prague, Czech Republic.
Growing the
Pipeline for Patients
SKYHAWK is the clinic with SKY-0515, targeting Huntington's Disease. Our next two internal neurology programs, SKY-1300 and SKY-1500, address Spinocerebellar Ataxia Type 3, and Frontotemporal Dementia (FTD) respectively, each an indication with no approved therapeutic agents.
Skyhawk’s platform has generated an expanding internal pipeline of neurology, oncology and fibrotic disease assets
Our Collaborations
Skyhawk’s eight Pharma partnerships have sparked rapid investment in our proprietary platform.
Skyhawk’s Pharma collaborations have contributed biology insights, commercial perspectives and upfront payments of over $525 million to develop targeted drug candidates – driving growth in Skyhawk’s platform.
