SKY-1300

SKY-1300 is a preclinical, orally administered small molecule RNA splicing modulator being developed by Skyhawk Therapeutics for neurological indications, specifically targeting spinocerebellar ataxia type 3 (SCA3) via modulation of ATXN3 pre-mRNA splicing.[7][14] Built on Skyhawk’s RNA-targeting small-molecule platform, the drug is designed to alter the splicing of the ATXN3 transcript, with the goal of reducing expression or pathogenic forms of ataxin-3 protein that drive SCA3 pathogenesis.[7][14] The program is currently in the preclinical stage with no publicly disclosed clinical trials or detailed pharmacology beyond its RNA splicing modulation of ATXN3.