Stoke Therapeutics, Inc. (NASDAQ: STOK) reported that it dosed the first patient in its Phase 1 OSPREY study of STK‑002 on February 11, 2026. STK‑002 is an antisense oligonucleotide designed to upregulate OPA1 protein in patients with autosomal dominant optic atrophy (ADOA). The dose‑escalating, open‑label study will enroll children and adults aged 6 to 55 with confirmed OPA1 variants and will evaluate safety, tolerability and exposure across sequential cohorts.
The first‑patient dosing marks the transition of STK‑002 from preclinical development to human testing, validating Stoke’s TANGO platform for haploinsufficiency diseases. STK‑002 has received orphan drug designation from the U.S. FDA, positioning the company to benefit from regulatory incentives and a clear pathway to approval for a disease that currently has no approved therapies.
The OSPREY study’s design—single‑ascending doses with planned escalation through 2026 and into early 2027—provides a structured framework to assess safety and early signals of efficacy. The program complements Stoke’s broader pipeline, which includes STK‑001 for Dravet syndrome, and could open a new therapeutic area for the company. Competitors such as PYC Therapeutics and Idebenone are also exploring OPA1‑targeted approaches, underscoring the unmet medical need and the strategic importance of STK‑002’s progress.
Dr. Patrick Yu‑Wai‑Man, lead principal investigator, noted that “there are currently no medicines available for people living with ADOA, and there is a lot of interest in this study from the ADOA community given the potential for STK‑002 to restore vision by addressing the root cause of the disease.” Barry Ticho, Chief Medical Officer, added that “ADOA is a haploinsufficient disease, one of many that we believe are ideally suited for our ASOs that are designed to increase naturally occurring protein levels to improve health.”
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