Stoke Therapeutics Secures $165M to Ignite a New Era in Neuro and Ocular Disease Treatment

Stoke Therapeutics, a pioneering biotechnology company listed on Nasdaq as STOK, has successfully secured $165 million in its latest funding round, marking a significant milestone in its mission to address the underlying causes of severe diseases. The company is revolutionizing how we approach therapies by focusing not on replacing, repairing, or editing genes, but on enhancing the output of healthy genes. Utilizing its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform, Stoke is uniquely positioned to boost protein production from intact genes, restoring functional protein levels crucial for patients suffering from haploinsufficiencies—conditions resulting from the loss of about half the normal protein function. With nearly 6,500 genes identified to have a TANGO signature, the potential reach of this strategy spans a broad spectrum of severe disorders. The recent infusion of capital will accelerate the development and clinical testing of their promising pipeline, including STK-001, their lead candidate, which has shown positive Phase 1/2 results in treating Dravet syndrome—a severe genetic epilepsy associated with debilitating non-seizure comorbidities. Additionally, funding will support the advancement of STK-002, a candidate therapy for autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder, with clinical trials slated to commence in 2024. Beyond these programs, the investment will further bolster collaborative efforts with partners like Acadia Pharmaceuticals on treatments for neurodevelopmental disorders such as Syngap1 and Rett syndrome. Headquartered in Bedford, Massachusetts, with additional offices in Cambridge, Stoke Therapeutics is committed to transforming the treatment landscape for severe diseases, and this funding will catalyze its journey towards making a tangible difference in patients' lives.