Stoke Therapeutics Announces Presentations of STK-002, an Investigational Medicine for the Treatment of Autosomal Dominant Optic Atrophy (ADOA), at the American Academy of Ophthalmology (AAO) 2026 Annual Meeting
Business Wire•06/10/2026•08:00 ET
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Key Highlights
- ➤OSPREY dosing is complete in the first two patient cohorts
- ➤Third OSPREY cohort dosing expected to complete by year-end 2026
- ➤Initial STK-002 safety and efficacy data anticipated in H1 2027
- ➤STK-002 is designed to increase functional OPA1 protein in ADOA
- ➤AAO 2026 will feature two presentations of STK-002 clinical and preclinical data
Expert Statements
Barry Ticho, Chief Medical Officer of Stoke Therapeutics
“ADOA causes progressive and irreversible vision loss in both eyes with 80% of patients symptomatic by age 10 and approximately half progressing to legal blindness. We are encouraged by our preclinical data as well as emerging findings from the field demonstrating that upregulation of OPA1 protein may have disease-modifying potential. We continue to advance the Phase 1 OSPREY study of STK-002 in people with ADOA, with dose escalation progressing into higher cohorts expected to reach therapeutic levels. We anticipate initial results in the first half of next year to guide our next steps for development.”
Stoke Therapeutics Announces Presentations of STK-002, an Investigational Medicine for the Treatment of Autosomal Dominant Optic Atrophy (ADOA), at the American Academy of Ophthalmology (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–
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