Our RNA modifiers

Skyhawk’s RNA splicing modulators can address a broad range of diseases.

Skyhawk's modalities

  • Huntington’s disease: SKY-0515
  • Rare neuro: SKY-2100
  • Rare neuro: SKY-4200
  • Rare neuro: SKY-1300

Our wholly-owned pipeline includes programs across neurodegenerative, neuromuscular, oncology, fibrotic and other disease types. Many of our programs address targets formerly considered “undruggable” using traditional protein-targeting mechanisms.

Therapeutic areas

RNA mis-expression is central to a host of diseases. Very few treatments are available for these challenging conditions, many of which have been considered "undruggable".

Want to learn more?

Skyhawk’s potent small molecule drug candidates target RNA across a broad range of diseases, from orphan and neurological conditions to major cancers.

Publications

SKY-0515

June 26, 2026
2026 Congress of the European Academy of Neurology (EAN)
Investigation of SKY-0515 in patients with Huntington's disease. This poster covers interim results of a Phase 1/2 study.
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Skyhawk Presents at HDYO

March 15, 2025
HDYO International Congress 2025
SKY-0515: A Daily Pill Designed To Target The Cause Of Huntington's Disease With A Potential To Help Slow Its Progression Skyhawk presents on its novel SKY-0515 small molecule RNA splicing modulator targeting Huntington's Disease, to...
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SKY-0515

February 24, 2026
Skyhawk Poster at CHDI’s 21st Annual Huntington’s Disease Therapeutics Conference
Skyhawk shares data and information printed at CHDI’s conference relating to SKY-0515 small molecule targeting the cause of Huntington’s disease: Investigation of SKY-0515 in Patients with HD.
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SKY-1214

April 25, 2026
22025 American Association for Cancer Research (AACR) Annual Meeting
SKY-1214, a small molecule splicing modulator of members of the Fanconi anemia pathway, exerts anti-cancer activity both as single agent and in combination in multiple myeloma and non-Hodgkin's... 
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