11:41 AM
 | 
Mar 08, 2018
 |  BC Innovations  |  Emerging Company Profile

Splice time

How Skyhawk targets RNA splicing in cancer and exon-skipping diseases

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.

Following transcription, pre-mRNA undergoes RNA splicing within the spliceosome, which removes non-coding introns and joins together coding exons to form mRNA. Alternative splicing is a normal process by which multiple versions of a protein (isoforms) are created from the same pre-mRNA by including or excluding certain exons during RNA processing....

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