Stoke Therapeutics, Inc.

Stoke Therapeutics is a biotechnology company whose mission is to address the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines. Its proprietary research platform, TANGO (Targeted Augmentation of Nuclear Gene Output), aims to restore missing proteins by increasing protein output from healthy genes, compensating for a non-functioning copy of a gene. The company's initial applications target haploinsufficiencies, diseases in which one copy of a gene functions normally and the other is mutated. Clinical studies of zorevunersen (STK-001) for the treatment of Dravet syndrome are ongoing, and Stoke has demonstrated promise for a TANGO ASO to treat the underlying cause of autosomal dominant optic atrophy (ADOA). Stoke was co-founded in 2014 by Professor Adrian Krainer, Ph.D. and Isabel Aznarez, Ph.D.

Stoke was co-founded in 2014 by Professor Adrian Krainer, Ph.D. and Isabel Aznarez, Ph.D., who aimed to use their science targeting pre-mRNA splicing to develop precision medicines. The two founders began working together in 2008 at Cold Spring Harbor Laboratory in New York, focusing on applying antisense oligonucleotides to modulate RNA splicing and RNA metabolism pathways. Their early work led to the development of Stoke's proprietary TANGO platform, designed to deliver disease-modifying therapies to a wide range of relevant tissues in a highly precise, durable and controlled manner. Dr. Aznarez continues to guide research as Chief Technology Officer, and Professor Krainer remains closely involved as a member of the Board of Directors.

RNA-based medicines designed to restore missing proteins by upregulating protein expression from healthy genes, with an initial focus on haploinsufficiencies.

• zorevunersen (STK-001)

  Under clinical studies for the treatment of Dravet syndrome.

• TANGO ASO for autosomal dominant optic atrophy (ADOA)

  Stoke has demonstrated promise for a TANGO ASO to treat the underlying cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.

1. 1Home - Stoke Therapeuticsstoketherapeutics.com
2. 2Corporate Responsibility - Stoke Therapeuticsstoketherapeutics.com
3. 3About Stoke - Stoke Therapeuticsstoketherapeutics.com
4. 4Founders - Stoke Therapeuticsstoketherapeutics.com