Stoke Therapeutics

STK-002 Clin1 autosomal dominant optic atrophy; undisclosed RD SYNGAP1 syndrome; undisclosed RD Rett syndrome; undisclosed RD CNS condition

Stoke Therapeutics is using new insights in RNA biology to address the underlying cause of severe diseases. Many diseases arise when one copy of a gene functions normally and the other is mutated, called haploinsufficiency. Using their Targeted Augmentation of Nuclear Gene Output (TANGO) platform, Stoke is identifying ways to restore missing proteins by increasing protein output from healthy genes, thereby compensating for the non-functioning copy of the gene. The platform involves the design of antisense oligonucleotides (ASOs) that bind to pre-mRNA and help the target genes produce more protein. Stoke is currently applying their technology to patients with Dravet syndrome, a severe and progressive genetic epilepsy.