- STK-002 is an antisense oligonucleotide designed to upregulate OPA1 protein from the wild-type gene copy.
- ADOA is caused by OPA1 haploinsufficiency in 65-90% of cases.
- Phase 1 OSPREY study: open-label, dose-escalating, ages 6-55 with confirmed OPA1 variant.
- First two dose cohorts complete; third cohort expected to complete by year-end 2026.
- Cohorts three and four expected to achieve therapeutic dosing levels.
- Initial safety and efficacy data from cohorts three and four anticipated H1 2027.
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