Skyhawk Therapeutics

Skyhawk Therapeutics is a clinical-stage biotechnology company founded in 2016 and headquartered in Waltham, Massachusetts. The company develops small molecule therapeutics that modify RNA expression via its proprietary SkySTAR platform, targeting diseases including multiple sclerosis, spinal muscular atrophy, ALS, Huntington's disease, and cancers. Skyhawk's founders co-developed Evrysdi (risdiplam), the first approved small molecule to modulate RNA splicing, for the treatment of Spinal Muscular Atrophy. The company has established pharma collaborations contributing upfront payments of over $550 million, including a strategic research collaboration with Vertex Pharmaceuticals announced in December 2020.