Stoke Therapeutics Presents Data For STK-002 Supporting Disease-Modifying Potential In Autosomal Dominant Optic Atrophy At AAO 2026 Annual Meeting

–ADOA is the most common inherited optic nerve disorder and is primarily caused by variants in the OPA1 gene that result in insufficient levels of OPA1 protein, leading to progressive and irreversible vision loss–

–STK-002 represents a novel approach to ADOA by targeting the underlying disease biology, with the potential to be the first disease-modifying medicine for people living with ADOA–

–ADOA natural history findings and preclinical proof-of-concept data for STK-002 support the ongoing Phase 1 OSPREY study in people with ADOA–

–Dosing is now complete in first two cohorts of OSPREY study, with dosing of third cohort expected to complete by the end of 2026; initial safety and efficacy data anticipated in H1 2027–

Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced two presentations of data for STK-002 at the American Academy of Ophthalmology (AAO) 2026 Annual Meeting, taking place October 9-12 in New Orleans. STK-002 represents a novel approach to Autosomal Dominant Optic Atrophy (ADOA), the most common inherited optic nerve disorder, by targeting the underlying disease biology. ADOA natural history findings and preclinical data for STK-002 support the ongoing Phase 1 OSPREY study and the disease-modifying potential of STK-002 in ADOA.

Dose escalation is ongoing in the OSPREY study, with the first two dose cohorts now complete and the third cohort expected to complete by the end of 2026. Cohorts three and four have the potential to reach therapeutic levels of dosing, with initial safety and efficacy data from these latter cohorts anticipated in the first half of 2027.