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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 autoimmune disease.

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

Latest News

Skyhawk Therapeutics Announces Positive Topline Results from Parts A and B of its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease, Reaching 72% Huntingtin mRNA Reduction

Skyhawk’s SKY-0515 demonstrated dose-dependent huntingtin (HTT) mRNA reduction in healthy volunteers, with 72% reduction at the highest dose tested in the multiple ascending dose study

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.

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Skyhawk’s platform has generated an expanding internal pipeline of neurology, oncology and autoimmune assets

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Our Platform


Proprietary Platform

Skyhawk’s platform integrates four distinct and complementary technologies with rapid growing, machine learning models to accelerate the development of RNA targeting small molecule drugs across a range of targets.


Our Collaborations

Skyhawk’s seven Pharma partnerships 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. 

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