Stoke Therapeutics Presents Data Supporting the Disease-Modifying Potential of STK-002 in Autosomal Dominant Optic Atrophy (ADOA) at the American Academy of Ophthalmology (AAO) 2026 Annual Meeting
Key Highlights
- ➤STK-002 OSPREY first two dose cohorts complete; third expected by end-2026
- ➤Initial STK-002 safety and efficacy data anticipated in H1 2027
- ➤STK-002 preclinical studies increased OPA1 expression and improved mitochondrial function
- ➤FALCON study linked higher mitochondrial oxidative stress with decreased visual acuity
- ➤All eight OSPREY clinical trial sites active across five European countries
Expert Statements
Patrick Yu-Wai-Man, Professor of Ophthalmology at the University of Cambridge and the UCL Institute of Ophthalmology; Honorary Consultant Neuro-ophthalmologist at Addenbrooke’s Hospital and Moorfields Eye Hospital; lead investigator on the Phase 1 OSPREY study
“ADOA can cause severe progressive visual impairment often beginning in childhood, and there are currently no approved treatments”
Patrick Yu-Wai-Man, Professor of Ophthalmology at the University of Cambridge and the UCL Institute of Ophthalmology; Honorary Consultant Neuro-ophthalmologist at Addenbrooke’s Hospital and Moorfields Eye Hospital; lead investigator on the Phase 1 OSPREY study
“These early data are an important step in evaluating whether an RNA-based medicine can address the underlying OPA1 deficiency and ultimately improve visual outcomes for people with ADOA.”
Barry Ticho, Chief Medical Officer of Stoke Therapeutics
“We’re encouraged by the progress in the Phase 1 OSPREY study of STK-002 in people with ADOA, with the first two dose cohorts now complete”
Barry Ticho, Chief Medical Officer of Stoke Therapeutics
“Dose cohorts three and four are designed to show measurable effects on visual function, and we look forward to safety and efficacy results from these latter cohorts in the first half of next year.”
Stoke Therapeutics Presents Data Supporting the Disease-Modifying Potential of STK-002 in 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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