Stoke Therapeutics Advances STK-002 for Rare Optic Nerve Disorder…

Event summary

Stoke Therapeutics presented data on STK-002 at the AAO 2026 Annual Meeting, targeting Autosomal Dominant Optic Atrophy (ADOA).
Phase 1 OSPREY study dose escalation continues, with initial safety and efficacy data expected in the first half of 2027.
STK-002 aims to increase OPA1 protein levels, addressing the underlying cause of ADOA with no current approved treatments.
Presentations included translational evidence supporting STK-002's disease-modifying potential in ADOA.

The big picture

Stoke Therapeutics is advancing its RNA-based therapy STK-002 for ADOA, a rare inherited optic nerve disorder with no approved treatments. The company's proprietary TANGO approach aims to restore protein expression, addressing a significant unmet need in ophthalmology. The ongoing Phase 1 study and upcoming data presentations mark critical milestones in assessing the therapy's potential to modify disease progression and improve vision in ADOA patients.

What we're watching

Clinical Progress: Whether STK-002 can demonstrate meaningful efficacy in higher dose cohorts by mid-2027.
Regulatory Pathway: The pace at which Stoke can advance STK-002 toward potential regulatory approval given the orphan drug designation.
Market Opportunity: How Stoke positions STK-002 in the competitive landscape of rare disease treatments.