Stoking Functional Copies of Genes to Compensate for Mutated Ones

RARECast

Stoke Therapeutics platform technology allows it to target genetic diseases where people have one functional copy of a gene and one mutated copy. As a result, they can only produce half as much protein as they need to maintain health. Stoke seeks to restore missing proteins by increasing the protein output from healthy genes to compensate for the non-functioning copy of the gene. The company’s lead experimental therapy is an antisense oligonucleotide to treat the rare and progressive genetic epilepsy Dravet syndrome. We spoke to Ed Kaye, CEO of Stoke, about the company’s platform technology, how it works, and its lead program in Dravet syndrome.