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.

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).

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.

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.

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.

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.

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.