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Excitement over targeting RNA with small molecules continues with the launch of Skyhawk Therapeutics. The Waltham, Mass.-based start-up raised $8 million in seed funding to make compounds that target binding pockets in RNA splicing machinery to treat conditions caused by exon skipping, a process in which shortened versions of RNA are produced. Drugs that fix exon skipping and restore production of RNA have gained attention in clinical trials for spinal muscular atrophy. Skyhawk plans to file an Investigational New Drug Application for a cancer compound by the end of 2018.
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