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STK-002

Autosomal Dominant Optic Atrophy (ADOA)

PreclinicalResearch/Discovery

Key Facts

Indication
Autosomal Dominant Optic Atrophy (ADOA)
Phase
Preclinical
Status
Research/Discovery
Company

About Stoke Therapeutics

Stoke Therapeutics is developing a new category of RNA medicines focused on upregulating protein expression from functional genes to treat severe genetic diseases. The company's lead program, zorevunersen (STK-001), is in Phase 3 for Dravet syndrome, with recent data published in The New England Journal of Medicine demonstrating potential disease modification. Stoke's strategy leverages its proprietary TANGO platform to address haploinsufficiency disorders, a large class of genetic conditions, and expand into other areas of high unmet medical need. Its $1.91B valuation reflects significant investor confidence in this novel therapeutic approach.

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Other Autosomal Dominant Optic Atrophy (ADOA) Drugs

DrugCompanyPhase
ABO-505Abeona TherapeuticsPreclinical