Stoke Therapeutics

Stoke Therapeutics

Develops targeted gene upregulation therapies

Overview

Stoke Therapeutics develops therapies for severe genetic diseases caused by loss of function in a single gene, aiming to restore missing proteins by upregulating protein expression. Its main product approach relies on the TANGO (Targeted Augmentation of Nuclear Gene Output) platform, which delivers precise, durable, and controllable upregulation of gene expression across a range of tissues to re-create the missing protein. Compared with many biotech firms, Stoke centers on upregulating endogenous gene output rather than replacing genes or adding new ones, and focuses on disease-modifying treatments for rare disorders with unmet medical needs. Revenue comes from partnerships, licensing agreements, and, ultimately, commercialization of its therapies. The company’s goal is to provide targeted, durable treatments that address the underlying genetic deficiency and improve outcomes for patients with limited options.

About Stoke Therapeutics

Simplify's Rating
Why Stoke Therapeutics is rated
B-
Rated B on Competitive Edge
Rated B on Growth Potential
Rated C on Differentiation

Industries

Biotechnology

Healthcare

Company Size

201-500

Company Stage

IPO

Headquarters

Bedford, Texas

Founded

2014

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Simplify's Take

What believers are saying

  • Phase 3 EMPEROR enrollment completes Q2 2026, mid-2027 readout enables 2027 FDA filing.
  • $246.7M cash runway extends operations through mid-2028 post-Biogen $165M upfront.
  • NEJM publication validates multi-year seizure reductions and cognitive improvements in Dravet.

What critics are saying

  • Phase 3 failure to replicate Phase 1/2 efficacy erases $1.9B market cap existentially.
  • Biogen terminates collaboration, withdrawing $385M milestones needed for Phase 3 completion.
  • DNL343 small-molecule competitor demonstrates superior brain penetration and oral bioavailability.

What makes Stoke Therapeutics unique

  • TANGO platform enables durable, tissue-agnostic protein upregulation for genetic diseases.
  • Zorevunersen targets SCN1A loss-of-function, addressing Dravet syndrome's root cause.
  • Antisense oligonucleotide approach offers oral or feeding tube administration flexibility.

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Funding

Total Funding

$502.1M

Above

Industry Average

Funded Over

5 Rounds

Post IPO Equity funding comparison data is currently unavailable. We're working to provide this information soon!
Post IPO Equity Funding Comparison
Coming Soon

Benefits

Health Insurance

Dental Insurance

Vision Insurance

Life Insurance

Disability Insurance

Parental Leave

401(k) Retirement Plan

401(k) Company Match

Unlimited Paid Time Off

Employee Stock Purchase Plan

Stock Price

Growth & Insights and Company News

Headcount

6 month growth

-4%

1 year growth

-9%

2 year growth

-9%
Business Wire
Apr 7th, 2026
Stoke Therapeutics appoints Clare Kahn to board as Dravet syndrome treatment advances to Phase 3

Stoke Therapeutics has appointed Clare Kahn, PhD, to its Board of Directors. The Bedford, Massachusetts-based biotechnology company is developing zorevunersen, an investigational RNA medicine for Dravet syndrome, a severe genetic epilepsy. Dr Kahn brings over 30 years of pharmaceutical industry experience, most recently serving as R&D Strategy Officer at X-VAX Technology. She previously held senior positions at Pfizer and GlaxoSmithKline, with expertise in regulatory strategy and rare genetic disease development. The appointment comes as Stoke advances a Phase 3 clinical study of zorevunersen, which targets the underlying cause of Dravet syndrome. The company has a strategic collaboration with Biogen for the drug's development and commercialisation, retaining exclusive US, Canada and Mexico rights whilst Biogen holds rest-of-world rights.

Yahoo Finance
Mar 28th, 2026
Stoke Therapeutics chair sells $174K in shares as stock surges 340%

Stoke Therapeutics chair Arthur Tzianabos sold 4,355 shares of common stock for approximately $174,000 on 10th March 2026, according to an SEC Form 4 filing. The transaction resulted from exercising 3,955 stock options followed by an immediate sale. The sale reduced Tzianabos's direct common stock holdings by 12.20% to 31,339 shares. However, he continues to hold 118,696 stock options, maintaining significant potential future ownership. No indirect holdings through family trusts or LLCs were involved in the transaction. Stoke Therapeutics develops antisense oligonucleotide medicines targeting severe genetic diseases, with a market capitalisation of $1.9 billion. The company's lead clinical candidates include STK-001 for Dravet syndrome and STK-002 for autosomal dominant optic atrophy. The stock has risen 340% over one year.

Yahoo Finance
Mar 24th, 2026
Stoke Therapeutics CMO sells $457K in stock after 300% rally

Stoke Therapeutics chief medical officer Barry Ticho sold 14,311 shares of common stock for approximately $457,000 between 17 March and 19 March 2026, according to an SEC Form 4 filing. The sale price averaged $31.92 per share. The transaction reduced Ticho's direct holdings from 55,013 to 40,702 shares, a 26% decrease. He retains 83,035 stock options, giving him total beneficial interest of approximately 123,700 shares. The sale, which involved exercising options followed by immediate disposal, exceeded Ticho's historical median sale size of 7,073 shares. It occurred following a 300% one-year share price increase for the biotechnology company, which develops treatments for rare genetic neurological disorders. Stoke Therapeutics has a market capitalisation of $1.98 billion.

Yahoo Finance
Mar 7th, 2026
Stoke Therapeutics publishes Dravet syndrome trial data in NEJM, targets 2027 FDA filing

Stoke Therapeutics and Biogen announced that data on investigational Dravet syndrome treatment zorevunersen were published in The New England Journal of Medicine, showing multi-year seizure reductions and cognitive improvements. The company also updated its Phase 3 EMPEROR timeline, with enrollment completing by Q2 2026, mid-2027 readout, and plans to begin rolling FDA submission in the first half of 2027. The NEJM publication strengthens the biological rationale for zorevunersen's antisense platform. However, the key near-term catalyst remains whether the Phase 3 trial can confirm the long-term seizure and cognitive benefits observed thus far. Stoke's narrative projects $81.1 million revenue and $13.2 million earnings by 2028, implying a 26.7% yearly revenue decline. Optimistic analysts model 2028 revenue near $63.4 million.

ChartMill
Feb 4th, 2026
Stoke Therapeutics to Present at the Guggenheim Emerging Outlook: Biotech Summit 2026

Stoke Therapeutics to present at the Guggenheim Emerging Outlook: Biotech Summit 2026. Provided By Business Wire - Last update: Feb 4, 2026 Article Mentions: Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. Today, the Company announced that Chief Executive Officer Ian F. Smith will present at the Guggenheim Emerging Outlook: Biotech Summit 2026 on Wednesday, February 11, 2026, at 2:30 p.m. ET. A live audio webcast of the presentation, along with an archived replay, will be available in the Investors & News section of Stoke's website at https://investor.stoketherapeutics.com/. About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke's proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit https://www.stoketherapeutics.com/. About Zorevunersen Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing functional NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen (Nasdaq: BIIB) to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights.

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