Splice time

Skyhawk Therapeutics Inc. is developing small molecules that manipulate the stability of the spliceosome during RNA processing to correct splicing defects that drive disease. Instead of focusing on genes in classic exon-skipping diseases, the newco is applying its strategy to otherwise “undruggable” targets in cancer, neurodegeneration and rare diseases.

CEO Kathleen McCarthy said Skyhawk’s compounds prevent RNA mis-splicing prior to protein translation, allowing the company to inhibit intracellular proteins considered undruggable with biologics or not readily targeted directly with small molecules...