Skyhawk’s small molecules that modify RNA expression

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Learn about Skyhawk’s Phase 2/3 FALCON-HD worldwide Pivotal trial

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FALCON-HD expands its North American sites

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Skyhawk appoints Aaron Deves to lead commercialization of SKY-0515 for Huntington’s disease

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Skyhawk to present at EHDN Clinical Research Congress in Krakow, October 2026

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Latest News

Skyhawk shares its fifteen-month final dataset for its Phase 1/2 SKY-0515 Huntington’s trial, including cUHDRS and its subcomponents, safety, and biomarker findings.

Skyhawk Publications

Skyhawk has shared its best-in-disease SKY-0515 data for Huntington’s at a number of congresses. Access the posters, presentations, and abstracts, here.

200+
SKY-0515 patients
enrolled
30+
Global clinical sites participating
9
Rare neuro pipeline drugs

Meet Skyhawk

We believe in a small team with a big impact for patients.

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What are RNA-targeting small molecules?

 We make orally-administered small molecule pills, taken daily, that bind to RNA and modify downstream protein production.

Helping More People

Skyhawk’s small molecule drugs are patient, clinician, and payer friendly – offering access to patients worldwide; other RNA modalities have therapeutic limitations – and are available to few

Small Molecules that Target RNA

Skyhawk’s small molecule drug candidates are taken orally, cross the blood brain barrier, and offer therapeutically advantaged, broad, biological distribution.

Skyhawk’s Proprietary AI Tools

Our machine learning tools integrate data from SKYSTAR, med chem, animal studies, tox data and clinical programs to drive the next generation of novel RNA splicing modulators.

“Our team has deep expertise in RNA splicing and structure, and we’re focused on using proprietary computational biology, machine learning, and rapid drug development for the benefit of patients with huge unmet need.”

Kathleen McCarthy
Co-Founder & Distinguished Senior Scientist