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Stoke Therapeutics
Edward Kaye took the helm at Stoke Therapeutics after the company's 2014 founding by Cold Spring Harbor Laboratory scientist Adrian Krainer.
Stoke Therapeutics
Edward Kaye took the helm at Stoke Therapeutics after the company's 2014 founding by Cold Spring Harbor Laboratory scientist Adrian Krainer. Krainer had spent decades on antisense oligonucleotides; Stoke was formed to commercialize his discovery that certain molecules could augment protein output from a single functional gene copy. The company went public on Nasdaq in 2019, raising $142 million (per its S-1 filing, 2019). Its lead program targets Dravet syndrome, a catastrophic pediatric epilepsy caused by a mutation in the SCN1A gene. Stoke operates exclusively in neurology and rare disease. Its pipeline centers on antisense oligonucleotides that upregulate protein expression, not the typical knockdown approach. Stage coverage spans preclinical through Phase 3: the pivotal program zorevunersen (STK-001) is in late-stage trials for Dravet syndrome, and STK-002 targets autosomal dominant optic atrophy. The company has strategic collaborations, including a 2021 royalty monetization agreement with RTW Investments, which provided additional development capital without selling equity (per company press release, January 2021). Stoke's geographic footprint is a single headquarters in Bedford, Massachusetts, with clinical trial sites across North America, Europe, and Japan. Stoke's scale is that of a mid-clinical-stage biotech. The company's public float and market capitalization have fluctuated alongside trial milestones. In May 2024, Stoke reported that it had enrolled the first patient in a Phase 3 registrational study for zorevunersen in Dravet syndrome (per the firm, May 2024). As of late 2023, the company held roughly $200 million in cash and equivalents. Kaye, a former CEO at Sarepta Therapeutics, brought deep rare-disease development experience. Stoke has not formed a family-office-style club or multi-strategy vehicle; it is a pure-play public biotech company. Stoke's structural differentiator lies in its TANGO (Targeted Augmentation of Nuclear Gene Output) platform. Unlike nearly all antisense therapies that silence problematic genes, TANGO molecules are designed to increase the amount of functional protein made by healthy copies of a gene, potentially addressing haploinsufficiencies in a way gene therapy or silencing cannot. That precision mechanism, mapped directly from Krainer's CSHL lab, positions the company as one of a handful pursuing mRNA upregulation rather than inhibition, making it a project shaped by a specific Nobel-caliber laboratory lineage.
General information
Firm type
Asset Manager
Year founded
2014
AUM
Undisclosed
Location
Region
North America
Country
United States
City
Bedford
Corporate office
Bedford, MA, United States
Principals
Edward M. Kaye
Chief Executive Officer
Adrian R. Krainer
Scientific Co-Founder
Sector focus
Frequently asked questions
Who runs investment decisions at Stoke Therapeutics?
Stoke Therapeutics is a public company — investment decisions for the pipeline are made by Chief Executive Officer Edward Kaye and the executive leadership team, under the oversight of a board of directors. Capital allocation and financing decisions are ultimately approved by the board, which includes venture and public-market biotech investors. As a listed entity, any large transaction would also involve a shareholder vote where required by Nasdaq rules.
How does Stoke's TANGO platform differ from standard antisense approaches?
Most antisense oligonucleotides work by binding to mRNA and blocking translation or promoting degradation. Stoke's TANGO platform does the opposite: it binds to pre-mRNA and increases the production of protein from a functional copy of a gene. The core insight, developed by co-founder Adrian Krainer, exploits naturally occurring non-productive splicing events to boost protein output for haploinsufficiencies — diseases where one gene copy produces too little protein (per the company's scientific publications, 2018-2020).
What is Stoke's lead asset, and what is its regulatory status?
The lead asset is zorevunersen, formerly known as STK-001, an antisense oligonucleotide targeting the SCN1A gene for Dravet syndrome. As of mid-2024, the company had advanced zorevunersen into a global Phase 3 registrational study, with the first patient enrolled in May 2024. The FDA has granted both Orphan Drug and Rare Pediatric Disease designations to the program.
Does Stoke Therapeutics have any partnership or funding arrangements beyond equity issuance?
In January 2021, Stoke entered a royalty monetization agreement with RTW Investments, receiving development capital in exchange for a percentage of future zorevunersen royalties. This arrangement allowed Stoke to fund its pipeline without diluting shareholders. The company has also accessed the public equity markets since its 2019 IPO to fund research and development.
What is the scientific origin of Stoke Therapeutics?
The company's technology originates from the laboratory of Adrian Krainer at Cold Spring Harbor Laboratory. Krainer is a leading authority on RNA splicing and antisense therapeutics, best known for co-developing the antisense drug Spinraza for spinal muscular atrophy. Stoke was founded in 2014 to translate his work on RNA-splicing upregulation into treatments for severe genetic diseases.
What indications beyond Dravet syndrome is Stoke pursuing?
Stoke's pipeline includes STK-002 for autosomal dominant optic atrophy, a rare inherited condition causing vision loss, which is currently in preclinical or early clinical development. The company's TANGO platform is broadly applicable to haploinsufficiencies, but Stoke has publicly committed to focus near-term resources on its SCN1A-related epilepsy programs.
How is Stoke Therapeutics financed, given it is not a typical investment fund?
Stoke is a publicly traded biotech company listed on Nasdaq, so it finances its operations through equity offerings, strategic partnerships, and royalty-monetization structures rather than through limited partner commitments. Its investor base includes biotech-focused funds, institutional investors, and the public market. There is no AUM or deployable blind-pool capital in the asset-management sense.
Profile maintained by Altss using OSINT (open-source intelligence), regulatory filings, licensed data partners, and verified direct submissions. Read the methodology. Last updated: . Continuous refresh with full update cycles at least every 30 days.
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