FDA approves Rett syndrome treatment  on June 14, 2024


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📺 Stoke Therapeutics' STK-001: A Potential Game-Changer For Dravet Syndrome

Science

Stoke Therapeutics (NASDAQ:STOK) is a biotechnology company developing RNA-based therapies for severe genetic diseases caused by dysfunctional gene copies that produce essential proteins. STOK’s proprietary Targeted Augmentation of Nuclear Gene Output [TANGO] platform enhances RNA output from healthy genes, compensating for deficiencies provoked by non-functional mutated genes. The company’s pipeline focuses on Dravet syndrome, autosomal dominant optic atrophy [ADOA], Rett syndrome, and SynGAP1 disorder. Its leading candidate is a phase ½ drug indicated for Dravet syndrome. I believe STOK could have a well-defined path toward FDA approval relatively soon, contingent on STK-001’s safety and efficacy in more rigorous phase 3 trials.

seekingalpha.com     Jun 14, 2024