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TANGO Platform for SYNGAP1

SYNGAP1-related disorders

Research/DiscoveryPotential being explored

Key Facts

Indication
SYNGAP1-related disorders
Phase
Research/Discovery
Status
Potential being explored
Company

About Stoke Therapeutics

Stoke Therapeutics is developing first-in-class RNA medicines designed to address the underlying genetic causes of severe diseases by restoring protein expression. The company's core TANGO (Targeted Augmentation of Nuclear Gene Output) platform represents a distinct approach in RNA therapeutics, targeting haploinsufficiencies. With its lead program in Phase 3 for Dravet syndrome and promising preclinical data in autosomal dominant optic atrophy (ADOA), Stoke is positioned to potentially deliver disease-modifying therapies. The company is publicly traded and is advancing a pipeline focused on severe genetic disorders of the central nervous system and eye.

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