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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.

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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 to Present Additional Positive Topline Data from Parts A and B of Phase 1 Clinical Trial of SKY-0515 at European Huntington's Disease Network and Enroll-HD 2024

The presentation highlights additional positive topline results from SAD/MAD study and preclinical data of SKY-0515, a novel small molecule designed to reduce both HTT and PMS1 proteins, two key drivers of Huntington's disease

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 fibrotic disease assets

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Our Platform

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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.

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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. 

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