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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 fibrosis.

We have built collaborations with BMS, Biogen, Genentech, Merck, Sanofi and Vertex that leverage our novel SKYSTAR® platform to target challenging diseases in a variety of therapeutic areas. 

Skyhawk's novel RNA-targeting small molecule for Huntington's Disease, SKY-0515, is in clinical trials. A second Skyhawk-developed drug candidate in oncology is expected in the clinic by end of 2024 

Small Molecules that Modify RNA Expression

Latest News

Skyhawk Advances to Multiple Ascending Dose for its SKY-0515 RNA-Targeting Small Molecule for Huntington's Disease

SKY-0515 is a small molecule RNA splicing modifier developed through the company’s novel SKYSTAR® platform. SKY-0515’s Phase 1 study will include healthy volunteers before targeting Huntington’s patients. Huntington’s disease is a fatal neurological disease with no curative therapies.

Growing the
Pipeline for Patients

We have entered the clinic with, SKY-0515, targeting Huntington's Disease, and plan to file INDs for four additional drug candidates targeting high unmet need indications across neurology and oncology.

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Skyhawk’s platform has generated an expanding internal pipeline of neurology, oncology and fibrosis 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 nearly $500 million to develop targeted drug candidates – driving growth in Skyhawk’s platform. 

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