Also known as: ADOA
Autosomal dominant optic atrophy (ADOA) is an inherited optic neuropathy characterized by degeneration of optic nerves and progressive bilateral vision loss, typically beginning in childhood. Many cases are associated with variants in OPA1.
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Stoke Therapeutics presented long-term Dravet syndrome data showing up to 80% seizure reductions sustained through three years, outlined its Phase 3 EMPEROR trial design targeting mid-2027 data, and disclosed first patient dosing in its ADOA program.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06970106 |
Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle) |
RECRUITING | PHASE1/PHASE2 |
| NCT06461286 |
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew) |
ACTIVE_NOT_RECRUITING | PHASE1 |
| NCT06140329 |
Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation |
TERMINATED | |
| NCT01793168 |
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford |
RECRUITING |
