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Skyhawk Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of novel small molecules that target RNA. The company's proprietary platform is designed to identify and advance drug candidates capable of modulating RNA expression or splicing. This innovative approach holds promise for addressing a wide array of serious diseases, including neurological disorders, various cancers, and fibrotic conditions, by correcting underlying genetic dysfunctions.
The company was co-founded in 2016 by Kathleen McCarthy, an expert in small molecules targeting RNA splicing, and Bill Haney, with Sergey Paushkin as a scientific co-founder. Their collective insight centered on the therapeutic potential of directly targeting RNA with small molecules, a strategy poised to expand the druggable genome. This foundational understanding laid the groundwork for Skyhawk's platform, aiming to create precise and effective treatments.
Skyhawk Therapeutics collaborates with major pharmaceutical partners, including Biogen, Merck, and Ipsen, to develop new treatments. These partnerships facilitate the advancement of their drug candidates toward clinical application, ultimately serving patients afflicted with debilitating diseases. The company’s vision is to bring forward a pipeline of first-in-class therapies that significantly improve outcomes by precisely modulating RNA.
Skyhawk Therapeutics has raised $40.0M across 1 funding round.
Skyhawk Therapeutics has raised $40.0M in total across 1 funding round.
Skyhawk Therapeutics is a clinical-stage biotechnology company based in Waltham, Massachusetts, specializing in small molecule therapies that modulate RNA expression to treat complex diseases, particularly in neurology.[1][2][3] Its proprietary SkySTAR® platform (Small molecule Therapies for Alternative splicing of RNA) enables the discovery and development of drugs targeting RNA splicing, with lead candidate SKY-0515 advancing in a Phase 2/3 FALCON-HD trial for Huntington's Disease (HD) after showing a 72% reduction in HTT mRNA and 62% in mutant HTT protein in Phase 1.[3] The company serves patients with intractable neurological disorders, addressing unmet needs where traditional protein-targeting drugs fall short, and has secured over $525 million in upfront payments from eight pharma partnerships, fueling a growing pipeline including SKY-0925, SKY-2100, and SKY-4100 for rare neuro indications.[3][4]
Founded in 2017 by Bill Haney, Skyhawk emerged from breakthroughs in RNA splicing modulation, leveraging expertise from a scientific advisory board of luminaries like Prof. Tyler Jacks (MIT Koch Institute Director, cancer genetics pioneer), Dr. Rob Hershberg (former Celgene CSO and Bristol-Myers Squibb executive), Prof. Jeannie Lee (epigenetics and lncRNA expert, co-founder of Translate Bio and Fulcrum Therapeutics), and HD specialist Dr. Ed Wild (mutant huntingtin researcher and HDBuzz co-founder).[5][6] The idea stemmed from recognizing RNA as a druggable target for diseases like HD, where protein aggregation resists conventional therapies; early traction came via pharma collaborations providing biology insights and funding, rapidly advancing the platform from discovery to clinic.[3][4]
Skyhawk rides the RNA therapeutics wave, shifting from protein-centric drugs to RNA modulation—splicing, mRNA, and editing—to tackle genetically driven diseases like HD, where timing aligns with CRISPR/RNAi advances and aging populations amplifying neuro demand.[3][4] Market forces favor it: high unmet needs in rare neuro (e.g., limited HD options), small-molecule advantages (oral bioavailability, brain penetration), and Big Pharma's push for next-gen modalities amid patent cliffs.[2][3] Skyhawk influences the ecosystem by validating RNA splicing as viable (via clinical data and partnerships), de-risking investments and inspiring platforms at firms like Fulcrum or Stoke, while its Merck KGaA tie-up accelerates neuro pipeline scaling.[4][5]
Skyhawk's clinic-ready SKY-0515 positions it for potential HD approval by late 2020s if Phase 2/3 succeeds, with pipeline expansion and partnerships driving milestones like IND filings for SKY-0925/others.[3] Trends like AI-accelerated target ID and combo RNA/protein therapies will amplify its platform, potentially evolving Skyhawk into a multi-asset neuro leader or acquisition target amid biotech M&A surge. This RNA pioneer, born from elite science, could redefine intractable disease treatment, fulfilling its mission to revolutionize patient care.[1][3][4]
Skyhawk Therapeutics has raised $40.0M in total across 1 funding round.
Skyhawk Therapeutics's investors include Good Growth Capital.
Skyhawk Therapeutics has raised $40.0M across 1 funding round. Most recently, it raised $40.0M Series A in May 2018.
| Date | Round | Lead Investors | Other Investors |
|---|---|---|---|
| May 1, 2018 | $40.0M Series A | Good Growth Capital |