Skyhawk Therapeutics
Skyhawk Therapeutics develops novel small molecules that modify RNA expression to target challenging diseases, leveraging its proprietary SKYSTAR® platform and collaborating with major pharmaceutical companies.
Company Overview
Skyhawk Therapeutics focuses on inventing novel small molecules that modify RNA expression to target some of the world's most intractable diseases. The company is headquartered in Waltham, MA, and also maintains a European office in Basel, Switzerland. Skyhawk's unique platform accelerates the development of RNA-targeting small molecules across various fields, including neurology, oncology, and fibrosis. With a broad portfolio, their drug candidates are taken orally, cross the blood-brain barrier, and offer extensive biological distribution.
SKYSTAR® Platform
The SKYSTAR® platform at Skyhawk Therapeutics is instrumental in the exploration and development of small molecule drugs that target RNA. It integrates four distinct and complementary technologies: SKYSTAR®, SKYLIBRARY™, SKYSEQ™, and SKYAI™. This comprehensive platform has screened over 700 targets and has facilitated the development of drug candidates against high unmet need indications across neurology, oncology, and fibrosis. The platform is notable for its ability to address targets previously considered undruggable.
Collaborations and Partnerships
Skyhawk Therapeutics has formed strategic collaborations with major pharmaceutical companies, including BMS, Biogen, Genentech, Ipsen, Merck, Sanofi, Takeda, and Vertex. These partnerships leverage the SKYSTAR® platform to target challenging diseases. Pharma collaborations have also contributed valuable biology insights, commercial perspectives, and upfront payments totaling over $525 million. Notably, Ipsen and Skyhawk have an RNA-targeting research collaboration focusing on rare neurological diseases.
Drug Pipeline
The drug pipeline of Skyhawk Therapeutics includes both wholly-owned and partnered programs targeting a variety of diseases. SKY-0515, a novel RNA-targeting small molecule for Huntington's Disease, is currently in clinical trials and received HREC approval for a Phase 1 study. Additionally, a second drug candidate in oncology is expected to reach the clinic by the end of 2024. Skyhawk plans to file INDs for four additional drug candidates addressing high unmet need indications in neurology and oncology.
Technological Integration
Skyhawk Therapeutics' platform is distinguished by the integration of four distinct technologies: SKYSTAR®, SKYLIBRARY™, SKYSEQ™, and SKYAI™. These technologies work together, guided by rapidly growing machine learning models, to address complex biological challenges. This technological infrastructure supports the development of an expanding internal pipeline and allows for the efficient targeting of previously undruggable RNA targets with small molecules.