Key Takeaways

  • Stoke Therapeutics will present data on investigational RNA medicine STK-002 in 2 presentations at AAO 2026
  • The Phase 1 OSPREY study is progressing through dose escalation, with cohorts 3 and 4 expected to reach therapeutic dosing levels
  • Initial safety and efficacy data from the higher-dose cohorts are anticipated in the first half of 2027

Stoke Therapeutics announced that data for its investigational RNA medicine STK-002 will be featured in 2 presentations at the 2026 American Academy of Ophthalmology (AAO) annual meeting, taking place October 9-12 in New Orleans.

STK-002 is being developed for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. The investigational therapy is designed to address the underlying cause of the disease by increasing OPA1 protein levels, with the goal of improving vision.

There are currently no approved treatments for ADOA, according to Stoke.

“ADOA causes progressive and irreversible vision loss in both eyes with 80% of patients symptomatic by age 10 and approximately half progressing to legal blindness,” Barry Ticho, MD, PhD, chief medical officer of Stoke Therapeutics, said in a statement. “We are encouraged by our preclinical data as well as emerging findings from the field demonstrating that upregulation of OPA1 protein may have disease-modifying potential."

STK-002 is currently being evaluated in the phase 1 OSPREY study in patients with ADOA. Dose escalation is ongoing, with the first 2 dose cohorts completed and the third cohort expected to be completed by the end of 2026. According to Stoke, cohorts 3 and 4 are expected to reach therapeutic dosing levels. Initial safety and efficacy findings from these higher-dose cohorts are anticipated during the first half of 2027.

The data being presented at AAO 2026 provide translational evidence supporting the potential of increasing functional OPA1 protein as a disease-modifying approach in ADOA, according to the company.

Details of the presentations are as follows:

Title: OSPREY: First-in-Human Phase 1 Study of the RNA-Based Therapy STK-002 in Autosomal Dominant Optic Atrophy
Podium Poster Date & Time: Sunday, October 11, 10:30-10:40 AM CT
Presenting Author: Patrick Yu-Wai-Man, M.D., Ph.D., Professor of Ophthalmology at the University of Cambridge and the UCL Institute of Ophthalmology, and Honorary Consultant Neuro-ophthalmologist at Addenbrooke’s Hospital and Moorfields Eye Hospital, United Kingdom
Poster Number: PO055

Title: RNA-Based OPA1 Upregulation in Autosomal Dominant Optic Atrophy: First-in-Human STK-002 Study in Mitochondrial Optic Neuropathy
Poster Panel Discussion Date & Time: Sunday, October 11, 3:15–3:21 PM CT
Presenting Author: Nancy J. Newman, M.D., Professor, Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine
Poster Number: PO222