Stoke Therapeutics Reports Early Data for STK-002 in ADOA, Eyes 2027 Efficacy Readout
Event summary
- Stoke Therapeutics presented preclinical and early clinical data for STK-002 at AAO 2026, supporting its disease-modifying potential in Autosomal Dominant Optic Atrophy (ADOA).
- Phase 1 OSPREY study dose escalation ongoing; third cohort expected to complete by year-end 2026.
- Initial safety and efficacy data from higher-dose cohorts anticipated in the first half of 2027.
- STK-002 aims to upregulate OPA1 protein expression, addressing the underlying cause of ADOA.
The big picture
Stoke Therapeutics' STK-002 represents a novel approach to ADOA, a rare inherited optic nerve disorder with no approved treatments. The company's RNA-based medicine targets the underlying OPA1 protein deficiency, aiming to slow or halt progressive vision loss. Success in the Phase 1 OSPREY study could validate the disease-modifying potential of antisense oligonucleotides in neurodegenerative eye diseases, positioning Stoke as a leader in RNA medicine for rare genetic disorders.
What we're watching
- Clinical Milestones
- Whether STK-002 demonstrates measurable effects on visual function in higher-dose cohorts by mid-2027.
- Regulatory Pathway
- The pace at which Stoke Therapeutics advances STK-002 toward later-stage trials based on Phase 1 results.
- Competitive Landscape
- How early data positions STK-002 against potential future therapies for ADOA.
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