Stoke Therapeutics, Inc. (Nasdaq: STOK) presented preclinical and early clinical data for its experimental treatment STK-002 at the American Academy of Ophthalmology 2026 Annual Meeting in New Orleans, according to a company press release.

STK-002 is an antisense oligonucleotide in development for autosomal dominant optic atrophy (ADOA), a rare inherited optic nerve disorder affecting approximately one in 25,000 people globally. ADOA causes progressive vision loss beginning in the first decade of life, and no approved treatments currently exist.

The condition is primarily caused by variants in the OPA1 gene, which result in reduced OPA1 protein expression. STK-002 is designed to increase functional OPA1 protein by targeting the non-mutated copy of the gene.

Data presented at the meeting included findings from the FALCON natural history study, which showed patients with ADOA exhibit higher levels of mitochondrial oxidative stress compared with healthy individuals, correlating with decreased visual acuity. Preclinical data showed dose-dependent increases in OPA1 protein expression and improved mitochondrial function in patient-derived cells.

Stoke also provided an update on the Phase 1 OSPREY study, a dose-escalation trial enrolling patients ages 6 to 55 across eight sites in the UK, Germany, Denmark, Italy, and Austria. Dosing in the first two cohorts is complete, with the third cohort expected to complete dosing by the end of 2026. Initial safety and efficacy data are anticipated in the first half of 2027.

"ADOA can cause severe progressive visual impairment often beginning in childhood, and there are currently no approved treatments," said Patrick Yu-Wai-Man, M.D., Ph.D., lead investigator on the OSPREY study and professor of ophthalmology at the University of Cambridge.

Barry Ticho, M.D., Ph.D., chief medical officer of Stoke Therapeutics, noted that dose cohorts three and four are designed to show measurable effects on visual function.

STK-002 has received orphan drug designation from the FDA. Stoke Therapeutics is headquartered in Bedford, Massachusetts.