Biotech company Skyhawk Therapeutics has developed a novel SKY-0515 small molecule RNA splicing modulator targeting Huntington’s Disease.
SKY-0515 is an investigational small molecule RNA splicing modifier developed through the Skyhawk’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.
Skyhawk will present the therapy at the Huntington’s Disease Youth Organization International Congress 2025 in Prague.
“We are excited to present on our Huntingtin-lowering clinical trials at the HDYO International Congress this month,” said Sergey Paushkin, CSO and head of R&D at Skyhawk.
“With positive results including SKY-0515’s average HTT mRNA reduction of 72 per cent 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, CEO 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.”
