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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, Ipsen, Merck, Sanofi, Takeda 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

Ipsen and Skyhawk Therapeutics announce RNA targeting research collaboration in rare neurological diseases

Option agreement for Ipsen to acquire exclusive license for the worldwide rights to develop two successful development candidates, and Skyhawk to receive an upfront payment and potential milestones up to $1.8 billion, plus potential for tiered royalties.

This promising platform technology created by Skyhawk allows for the exploration of previously undruggable RNA targets with small molecules, expanding the disease target landscape.

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 over $525 million to develop targeted drug candidates – driving growth in Skyhawk’s platform. 

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