What Stoke Therapeutics does

Stoke Therapeutics is a clinical-stage biotechnology company developing antisense oligonucleotide (ASO) medicines based on its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform, which modulates pre-mRNA splicing to increase protein output from healthy gene copies in autosomal dominant haploinsufficiency diseases. The company's lead candidate, zorevunersen (STK-001), targets Dravet syndrome by increasing SCN1A gene expression and is in Phase 3 development (EMPEROR study) under FDA Breakthrough Therapy Designation, with a rolling NDA submission planned for H1 2027 and data readout anticipated mid-2027. A second clinical candidate, STK-002, is in a Phase 1 dose-escalation study (OSPREY) for Autosomal Dominant Optic Atrophy, and an exploratory program targets SYNGAP1-related intellectual disability. The TANGO bioinformatics pipeline has identified approximately 1,200 monogenic diseases and approximately 6,500 additional genes as amenable to the platform, creating a broad pipeline optionality.

Stoke is a pre-commercial company that reported $184.42 million in FY2025 revenue, all derived from contractual arrangements with Biogen (global zorevunersen commercialization rights outside the US, Canada, and Mexico) and Acadia Pharmaceuticals, rather than product sales. The company retains exclusive US, Canada, and Mexico commercialization rights for zorevunersen and plans a direct field sales effort targeting specialized epilepsy treatment centers. Pricing for zorevunersen has not been publicly disclosed, though one analyst has modeled a $600,000 per year net price and projected peak annual US sales exceeding $1.5 billion.

The company is publicly traded on NASDAQ under ticker STOK, headquartered in Bedford, Massachusetts with a planned 2026 relocation to a triple-sized facility in Waltham, Massachusetts. It is led by CEO Ian F. Smith (appointed permanently in October 2024), with co-founders Adrian Krainer (Wolf Prize laureate, inventor of Spinraza) and Isabel Aznarez providing scientific leadership. As of December 31, 2025, Stoke held approximately $391.7 million in cash, expected to fund operations into 2028, and had a market capitalization of approximately $1.8 billion.

Name

Stoke Therapeutics

Legal name

Stoke Therapeutics, Inc.

Website

https://stoketherapeutics.com

Company type

Public

Founded year

2015

Operating status

Operating

Headcount range

101–250 employees

Short description

Stoke Therapeutics is a clinical-stage biotech developing antisense oligonucleotide medicines using its proprietary TANGO platform to treat genetic haploinsufficiency diseases, with its lead candidate zorevunersen in Phase 3 for Dravet syndrome and partnered globally with Biogen.

Ownership category

akta.pro rank

Product category

RNA-based antisense oligonucleotide therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown) (HLAAADAC)

akta.pro secondary industries

RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Therapeutics Safety & Immunogenicity (innate immune activation, tox, off-target) (HLAAADAI)

Keywords

• Antisense oligonucleotide therapeutics
• RNA-based medicines
• Rare disease therapeutics
• Genetic disease treatments
• Haploinsufficiency drug development

Headquarters

HQ city

Bedford

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure, Others

Revenue model

1. Contract Revenue: Stoke Therapeutics is a development-stage company with no approved products or current commercial revenue. It recognizes revenue from contractual partnerships with Biogen and Acadia Pharmaceuticals — in Q3 2025, the company reported $10.6 million in revenue from these arrangements.
2. Public Offering (Equity Financing): Stoke raised $125 million through an upsized public offering of common stock and pre-funded warrants in March/April 2024 to fund research, clinical development, and manufacturing of its RNA-based medicines.
3. Future Product Revenue (Projected): If zorevunersen is approved, Stoke retains US, Canada, and Mexico commercialization rights while Biogen holds rest-of-world rights. Peak annual sales are projected by analysts to exceed $1.5 billion in the US market alone.

Go-to-market motion2 records

Distribution channels3 records

Marketing channels6 records

Core offering

Stoke Therapeutics develops antisense oligonucleotide (ASO) medicines that use its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform to restore missing proteins by upregulating protein output from healthy gene copies in diseases caused by haploinsufficiency. Its lead candidate zorevunersen (STK-001) is in Phase 3 development for Dravet syndrome, with a second candidate STK-002 in Phase 1 for Autosomal Dominant Optic Atrophy (ADOA).

Product overview

Stoke Therapeutics is developing a portfolio of antisense oligonucleotide (ASO) medicines based on its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform. The lead product is zorevunersen (STK-001), an ASO for Dravet syndrome currently in Phase 3 development with Breakthrough Therapy Designation, developed in partnership with Biogen for global commercialization rights outside the US, Canada, and Mexico. The second product candidate is STK-002, an ASO for Autosomal Dominant Optic Atrophy (ADOA) in Phase 1 trials. The company is also exploring TANGO for SYNGAP1 and other diseases. The TANGO platform enables selective upregulation of protein expression to address genetic diseases caused by haploinsufficiency.

Differentiator

Problem solved

Functional benefit

Products and services

• Zorevunersen (STK-001) Investigational antisense oligonucleotide (ASO) medicine designed to target the underlying genetic cause of Dravet syndrome by increasing protein expression from the SCN1A gene. Currently in Phase 3 EMPEROR clinical study with FDA Breakthrough Therapy Designation, and being co-developed with Biogen (which holds ex-US/Canada/Mexico commercialization rights) for use in patients with this severe developmental and epileptic encephalopathy.
• STK-002 Investigational antisense oligonucleotide therapy targeting OPA1 gene variants for Autosomal Dominant Optic Atrophy (ADOA), a rare genetic disease causing progressive and irreversible vision loss. Currently in Phase 1 OSPREY study evaluating safety and tolerability in patients ages 6 to 55 with confirmed OPA1 gene variants.

Quantifiable outcome

• Up to 85% seizure reduction observed in zorevunersen-treated patients in Phase 1/2a studies
• +4 more outcomes

Named customers1 record

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Four partnerships are on record, tiered flagship, minor and core.

• BiogenflagshipStrategic or Co-development Partner · 1 January 2020Strategic collaboration for zorevunersen (STK-001) for Dravet syndrome. Stoke retains exclusive US, Canada, and Mexico commercialization rights; Biogen holds commercialization rights for the rest of the world. The partnership includes co-development, shared clinical costs, and milestone payments. Biogen has participated in data presentation (NEJM publication March 2026) and jointly presents at scientific conferences.
• Acadia PharmaceuticalsminorStrategic or Co-development PartnerStoke recognized $10.6 million in revenue from contractual obligations with Acadia Pharmaceuticals in Q3 2025, suggesting a separate collaboration or licensing arrangement, though details are not extensively disclosed.
• Cold Spring Harbor LaboratorycoreTechnology or IntegrationExclusively licensed foundational TANGO patents from Cold Spring Harbor Laboratory. Adrian Krainer serves as St. Giles Foundation Professor at Cold Spring Harbor Laboratory Cancer Center and is a co-founder of Stoke Therapeutics.
• University of SouthamptoncoreTechnology or IntegrationExclusively licensed foundational TANGO patents from the University of Southampton, alongside Cold Spring Harbor Laboratory patents, forming the core IP estate for the TANGO platform.

Scale indicators9 records

Recent moves8 records

Expansion highlights5 records

Direct peers

• Ionis Pharmaceuticals: Ionis is the pioneer and leader in antisense oligonucleotide therapeutics, with approved drugs including Spinraza (developed by Stoke co-founder Krainer). Both companies target CNS and rare genetic diseases using ASO chemistry, making Ionis the closest direct comparable.
• Biogen: Biogen is Stoke's commercial partner for zorevunersen ex-US and the originator of Spinraza, the first approved CNS ASO. Biogen shares deep CNS/neurology rare disease expertise and is the natural benchmark for ASO commercialization economics.
• Sarepta Therapeutics: Sarepta develops genetic medicines for rare neuromuscular diseases including Duchenne muscular dystrophy. Both companies pursue disease-modifying therapies for severe genetic conditions via novel modalities and rare disease commercial models.
• Wave Life Sciences: Wave develops stereopure oligonucleotide therapeutics (ASOs and siRNA) targeting rare genetic neurological diseases including Dravet syndrome and Huntington's disease, directly competing with Stoke's TANGO platform in adjacent CNS indications.
• Alnylam Pharmaceuticals: Alnylam is the leading RNAi therapeutics company developing siRNA drugs for rare genetic diseases. While chemically distinct from ASOs, both companies use oligonucleotide-based platforms to silence or modulate gene expression in genetically defined patient populations.
• Avidity Biosciences: Avidity develops antibody-RNA conjugates for rare neuromuscular diseases, with deep ties to Stoke through board member Arthur Levin (former Avidity CSO) and Stoke co-founders. Both target rare genetic conditions with RNA-based modalities.

Emerging players

• Solid Biosciences: Solid Biosciences develops genetic medicines for Duchenne muscular dystrophy and other rare diseases. Board overlap (Julie Anne Smith, Ian Smith) and shared rare disease focus make Solid a comparable emerging rare disease genetic medicine player.
• Voyager Therapeutics: Voyager develops gene therapies for neurological diseases including CNS indications. Stoke's Chief Regulatory Officer Beth Mellen previously served at Voyager. Both are early-stage CNS genetic medicine companies competing for talent and partnerships.

Broad incumbents

• Moderna: Moderna is a leading mRNA therapeutics company with Stoke CMO Barry Ticho previously serving as Head of R&D. While broader in scope, Moderna represents an incumbent RNA therapeutics platform competing for talent, partnerships, and pipeline assets in the genetic medicine space.
• Pfizer: Pfizer is a broad pharmaceutical incumbent with active ASO and rare disease programs, including board member Clare Kahn's prior leadership roles. Pfizer represents the large-cap benchmark for rare disease commercialization, pricing, and competitive dynamics.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/StokeTx
• LinkedInlinkedin.com/company/stoke-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles19 records

Funding overview

Funding rounds4 records

Investors9 records

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M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Stoke Therapeutics