Skyhawk Therapeutics, Inc.
Skyhawk Therapeutics is dedicated to revolutionizing disease treatment by developing small molecule therapeutics that modify RNA expression. Their platform accelerates the creation of RNA-targeting small molecules across various fields including neurology, oncology, and fibrotic diseases, aiming to address some of the world's most intractable diseases with innovative solutions.
Industries
Nr. of Employees
medium (51-250)
Skyhawk Therapeutics, Inc.
Waltham, Massachusetts, United States, North America
Products
SKY-0515
Investigational oral small-molecule RNA splicing modulator targeting huntingtin (HTT) mRNA for Huntington's disease; evaluated in first-in-human SAD and MAD trials with reported blood mRNA reduction and tolerability results; progressed to patient cohorts and later-stage trials.
SKY-1214
Preclinical/IND-ready small-molecule RNA splicing modulator targeting FANCL and FANCI, developed for hematologic malignancies including multiple myeloma and non-Hodgkin's lymphoma; reported in vivo and in vitro anti-tumor activity and combination synergy.
Additional internally developed RNA-targeting programs (examples: SKY-1300, SKY-1500, SKY-1700, SKY-1800)
A portfolio of internally discovered small-molecule RNA splicing modulators in development across neurology, oncology, and fibrotic disease indications; most programs are at discovery or preclinical stages with specific targets undisclosed on public pages.
SKY-0515
Investigational oral small-molecule RNA splicing modulator targeting huntingtin (HTT) mRNA for Huntington's disease; evaluated in first-in-human SAD and MAD trials with reported blood mRNA reduction and tolerability results; progressed to patient cohorts and later-stage trials.
SKY-1214
Preclinical/IND-ready small-molecule RNA splicing modulator targeting FANCL and FANCI, developed for hematologic malignancies including multiple myeloma and non-Hodgkin's lymphoma; reported in vivo and in vitro anti-tumor activity and combination synergy.
Additional internally developed RNA-targeting programs (examples: SKY-1300, SKY-1500, SKY-1700, SKY-1800)
A portfolio of internally discovered small-molecule RNA splicing modulators in development across neurology, oncology, and fibrotic disease indications; most programs are at discovery or preclinical stages with specific targets undisclosed on public pages.
Services
Research collaborations and licensing
Collaborative discovery and development programs with pharmaceutical partners via option agreements and co-development arrangements; partnerships provide biology insights, commercial perspectives, and upfront funding to advance RNA-targeting candidates.
Research collaborations and licensing
Collaborative discovery and development programs with pharmaceutical partners via option agreements and co-development arrangements; partnerships provide biology insights, commercial perspectives, and upfront funding to advance RNA-targeting candidates.
Expertise Areas
- RNA-targeted small molecule discovery
- Clinical trial design and execution (SAD/MAD and Phase 2/3 progression)
- Preclinical pharmacology and combination oncology studies
- Biomarker development and PD assay implementation
Key Technologies
- Small-molecule modulation of RNA splicing
- RNA-targeting small molecule chemotypes
- Bioinformatics and structural computational biology
- Multiplex screening assays for RNA motifs