BOSTON, March 18, 2025 /PRNewswire/ — Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate critical RNA targets, today presents on its novel SKY-0515 small molecule RNA splicing modulator targeting Huntington’s Disease to members of the Huntington’s Disease Youth Organization (HDYO), at the biannual HYDO International Congress in Prague, Czech Republic.
SKY-0515 is Skyhawk’s investigational small molecule RNA splicing modifier developed through the company’s novel RNA-splicing platform. SKY-0515 is designed to reduce both HTT protein and PMS1 protein, an additional key driver of somatic CAG repeat expansion and HD pathology.
“We are excited to present on our Huntingtin-lowering clinical trials at the HDYO International Congress this month,” said Sergey Paushkin, Chief Scientific Officer and Head of R&D at Skyhawk, “with positive results including SKY-0515’s average HTT mRNA reduction of 72% at a daily oral dose of 9mg in trials thus far, and the drug’s predicted suppression of the PMS1 protein, SKY-0515, if approved, could make a meaningful difference in Huntington’s patients’ lives.”
Huntington’s disease is a rare hereditary neurodegenerative disease affecting over 40,000 patients in the United States and many more worldwide. There are no approved treatments that can reverse or slow its course of progression.
“We are pleased with the speed at which we’ve conducted our studies thus far and are focused on this patient community in great need”, said Bill Haney, Chief Executive Officer at Skyhawk. “SKY-0515’s compelling results thus far demonstrate the immense potential of our platform to target indications for which there are no approved disease modifying therapies.”