Stoke Therapeutics is a late-stage clinical biopharmaceutical company developing RNA-based medicines for severe genetic diseases caused by haploinsufficiencies — conditions where one copy of a gene is mutated, leaving the body with roughly half the normal protein output. Stoke's core platform, TANGO (Targeted Augmentation of Nuclear Gene Output), uses antisense oligonucleotides (ASOs) to block a naturally occurring non-productive splicing event in the healthy gene copy, boosting functional protein production. Because TANGO targets the wild-type allele rather than the mutation itself, a single drug can address virtually all loss-of-function mutations in a given gene. Stoke's lead candidate, zorevunersen, targets Dravet syndrome — a severe childhood-onset genetic epilepsy caused by SCN1A haploinsufficiency — by restoring Nav1.1 protein levels in the brain. Zorevunersen is administered intrathecally and has shown durable reductions in seizure frequency and improvements in cognition through three years of treatment in open-label extension data. The pivotal Phase 3 EMPEROR trial enrolled its first patient in August 2025, with pivotal data expected mid-2027 and a rolling NDA submission beginning in early 2027. Stoke retains North American commercial rights and has partnered with Biogen for ex-North American rights, receiving $165M upfront. Stoke also has a second clinical candidate, STK-002, targeting autosomal dominant optic atrophy, with a Phase 1 study initiated in early 2026. As a pre-revenue company, Stoke funds operations through equity raises and partnership proceeds.