Stoke Therapeutics, Inc. (NASDAQ: STOK)

Stoke Therapeutics Inc is a late stage clinical company focused on developing RNA based medicines that increase protein expression to treat severe diseases.

The company uses its proprietary TANGO platform to design antisense oligonucleotides that target non productive splicing events and boost production of functional protein from the healthy gene allele.

Its lead product candidate zorevunersen is being evaluated in a global phase three study for Dravet syndrome a severe genetic epilepsy caused by haploinsufficiency of the SCN1A gene.

Stoke also advances a second clinical candidate STK 002 for autosomal dominant optic atrophy another condition caused by haploinsufficiency of the OPA1 gene.

The pipeline includes additional preclinical programs targeting haploinsufficiency disorders of the central nervous system and the eye.

Stoke Therapeutics does not yet have product sales and derives revenue primarily from collaboration and licensing agreements.

In January 2022 the company received an upfront payment of sixty million dollars from Acadia Pharmaceuticals under a collaboration to develop RNA based medicines for neurodevelopmental diseases.

In February 2025 Stoke entered into a collaboration with Biogen receiving an upfront payment of one hundred sixty five million dollars for the joint development and commercialization of zorevunersen outside the United States Canada and Mexico.

Both agreements include potential milestone payments and tiered royalties on future net sales if the products are approved and commercialized.

These arrangements constitute the company’s current source of revenue while it advances its clinical programs.

Stoke Therapeutics occupies a niche within the broader antisense oligonucleotide field by focusing on upregulating protein expression in haploinsufficiency disorders.

Most competitors in the nucleic acid therapeutics space aim to reduce toxic protein or edit genes whereas Stoke’s TANGO approach increases functional protein without altering the DNA.

Key competitors include Ionis Pharmaceuticals which develops antisense drugs for various targets and Encoded Therapeutics which explores gene regulation for Dravet syndrome.

Biogen although a partner for zorevunersen outside North America also represents a potential competitor in the neurology space.

Stoke’s competitive advantages stem from its proprietary TANGO mechanism its demonstrated tissue specificity and its ability to treat a wide range of genetic diseases with a single drug approach.

Stoke Therapeutics’ current customers are its collaboration partners Acadia Pharmaceuticals and Biogen who provide upfront payments and share development costs.

Looking forward the company expects to serve patients diagnosed with Dravet syndrome autosomal dominant optic atrophy and other haploinsufficiency conditions as well as the physicians and healthcare providers who treat them.

These future customers will rely on Stoke’s therapies to address the underlying genetic cause of their disease.