Stoke Therapeutics

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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.

TANGO (Targeted Augmentation of Nuclear Gene Output) is a proprietary RNA-based platform that uses antisense oligonucleotides to increase protein production from healthy genes, aiming to compensate for non-functioning mutated genes in haploinsufficiency disorders.

3 drugs in pipeline, 1 in Phase 3

Total raised: $345M

Stoke's main competitors in Dravet syndrome include companies developing gene therapies and other antisense approaches. Its core differentiation is the TANGO platform's unique mechanism of upregulating protein from healthy genes, setting it apart from most RNA-based therapies that focus on gene silencing, and positioning it as a pioneer in treating haploinsufficiency disorders.