Stoke Therapeutics Inc. has announced the dosing of the first patient in the Phase 1 OSPREY study of STK-002, an investigational medicine for the treatment of Autosomal Dominant Optic Atrophy (ADOA). The OSPREY study is a dose-escalating, open-label clinical trial recruiting children and adults aged 6 to 55 with a confirmed diagnosis of ADOA and a disease-causing variant in the OPA1 gene. The primary objectives are to assess the safety and tolerability of single ascending doses of STK-002, as well as drug exposure in blood. Secondary objectives include evaluating changes in visual function, ocular structure, and quality of life. The study is currently recruiting in the United Kingdom and Germany, with additional European sites expected to be added. Dose escalation is planned to continue through 2026 and early 2027, pending safety and tolerability assessments. Clinical results have not yet been presented.

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