Here are 1-3 brief analogies for Stoke Therapeutics (STOK):

• Like a Vertex Pharmaceuticals focused on severe neurological genetic disorders, but using advanced RNA technology.
• Similar to Alnylam Pharmaceuticals or Ionis Pharmaceuticals, but pioneering a novel RNA platform to treat genetic diseases by precisely regulating protein levels.

• STK-001 for Dravet Syndrome: An investigational RNA-based therapeutic designed to increase the expression of the NaV1.1 protein, aiming to restore proper function in patients with Dravet Syndrome.
• Programs for Opioid-induced Respiratory Depression (OIRD): Early-stage research and development efforts focused on an investigational RNA-based therapy targeting the *KCNT1* gene to address OIRD.
• Programs for Autosomal Dominant Optic Atrophy (ADOA): Early-stage research and development efforts focused on an investigational RNA-based therapy targeting the *OPA1* gene to address ADOA.

Stoke Therapeutics (STOK) is a clinical-stage biotechnology company focused on developing RNA-based medicines for severe genetic diseases. As a company in the research and development phase, Stoke Therapeutics does not currently have commercial products on the market that are being sold to major customers.

Therefore, Stoke Therapeutics does not have "major customers" in the traditional sense of companies purchasing their products or individuals directly buying therapies. Their primary activities involve research, preclinical studies, and clinical trials to develop potential treatments.

If and when Stoke Therapeutics successfully develops and gains regulatory approval for a therapeutic product, its "customers" would primarily be components of the healthcare system. These would typically include:

1. Healthcare Providers/Institutions: Hospitals, clinics, and specialized treatment centers that administer the therapies to patients.

2. Pharmaceutical Wholesalers/Distributors: Companies that purchase the drug from Stoke Therapeutics and distribute it to pharmacies and healthcare institutions. Examples of major public wholesalers include AmerisourceBergen (ABC), Cardinal Health (CAH), and McKesson Corporation (MCK).

3. Payers (Insurance Companies and Government Programs): While not direct purchasers of the drug from Stoke, these entities cover the cost of the therapies for eligible patients and significantly influence market access and demand. Examples include Aetna (part of CVS Health, CVS), UnitedHealthcare (part of UnitedHealth Group, UNH), and government programs like Medicare and Medicaid.

Ultimately, the patients suffering from the specific genetic diseases Stoke aims to treat would be the beneficiaries of any approved therapies, accessing them through the healthcare system.

Ian F. Smith Chief Executive Officer & Director

Ian F. Smith was appointed permanent Chief Executive Officer of Stoke Therapeutics in October 2025, after serving as interim CEO since March 2025. He has been a member of Stoke's Board of Directors since 2023. With over 30 years of experience in biotechnology leadership, Mr. Smith previously held executive roles at Vertex Pharmaceuticals from 2001 to 2019, including Executive Vice President, Chief Operating Officer, and Chief Financial Officer, where he helped transform the company into a global biopharmaceutical leader. He also serves as a senior advisor to Bain Capital Life Sciences and holds board positions at several biotechnology companies, including Executive Chairman at Solid Biosciences and Chairman at Rivus Pharmaceuticals.

Tommy Leggett Chief Financial Officer

Tommy Leggett is the Chief Financial Officer of Stoke Therapeutics, overseeing financial strategy, operations, and business development. He brings over 20 years of experience as a biotechnology executive, board member, and investment banker. Prior to joining Stoke, Mr. Leggett served as CFO of Affinia Therapeutics. He was also CFO at Black Diamond Therapeutics, where he led the company's Series C financing and initial public offering (IPO), and CFO at Axcella Health, where he helped raise capital through multiple financings, including an IPO. Before his roles in the biotechnology industry, Mr. Leggett spent more than a decade as an investment banker for healthcare groups at J.P. Morgan Securities, Lazard Frères & Company, and UBS Securities. He currently serves on the board of directors of Clover Biopharmaceuticals, Ltd.

Isabel Aznarez, Ph.D. Senior Vice President, Head of Research and Co-Founder

Isabel Aznarez is a co-founder of Stoke Therapeutics and serves as the Senior Vice President, Head of Research. She is a key figure in the company's scientific direction and its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) approach.

Barry Ticho, M.D., Ph.D., FACC Chief Medical Officer

Barry Ticho is the Chief Medical Officer at Stoke Therapeutics. He is a board-certified cardiologist with extensive experience in clinical development within the pharmaceutical industry. His career has involved leadership roles in various biotech companies, focusing on advancing treatments for a range of diseases.

Jonathan Allan, J.D. General Counsel

Jonathan Allan serves as the General Counsel of Stoke Therapeutics. In this role, he is responsible for the company's legal affairs and is also listed as Corporate Secretary.

For Stoke Therapeutics (symbol: STOK), the addressable markets for their main products are as follows:

• Zorevunersen (STK-001) for Dravet Syndrome:

  The estimated addressable market for Dravet syndrome is approximately 35,000 patients in the U.S. and EU regions, and potentially 300,000 patients worldwide. Another estimate suggests up to 38,000 people are living with Dravet syndrome in the U.S., UK, EU-4, and Japan. The global market size for Dravet Syndrome was valued at USD 376.17 million in 2023 and is projected to grow to USD 844.38 million by 2032.

• STK-002 for Autosomal Dominant Optic Atrophy (ADOA):

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Here are 3-5 expected drivers of future revenue growth for Stoke Therapeutics (STOK) over the next 2-3 years:

1. Successful Commercialization of Zorevunersen (STK-001) for Dravet Syndrome: Stoke Therapeutics' lead investigational medicine, zorevunersen, is currently in a pivotal Phase 3 EMPEROR study for Dravet syndrome, a severe and progressive genetic epilepsy. The anticipated readout for this Phase 3 study is in the second half of 2027. Zorevunersen has received Breakthrough Therapy designation from the FDA, and analysts hold an optimistic outlook regarding its potential commercial uptake following a successful Phase 3 trial. As a potential first-in-class, disease-modifying therapy that addresses the root cause of Dravet syndrome, its approval and market launch would represent a significant new revenue stream for the company.
2. Advancement and Potential Commercialization of STK-002 for Autosomal Dominant Optic Atrophy (ADOA): Beyond Dravet syndrome, Stoke is actively pursuing a second clinical program, STK-002, for Autosomal Dominant Optic Atrophy (ADOA), the most common inherited optic nerve disorder. A Phase 1 OSPREY study for STK-002 in ADOA treatment was initiated in the second quarter of 2025. The successful progression of this candidate through clinical trials and eventual commercialization would diversify Stoke's product portfolio and contribute to future revenue growth.
3. Expansion of the Pipeline through the TANGO Platform: Stoke Therapeutics utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform to develop RNA-based medicines to selectively restore protein levels, addressing the underlying cause of severe genetic diseases. The company has established earlier-stage programs for other haploinsufficiency disorders, including SYNGAP-1 disorders and Rett syndrome. Lead optimization is underway to identify a clinical candidate for SYNGAP-1 in 2026. The broad potential of the TANGO platform, with proof of concept demonstrated in various organs and tissues, suggests future pipeline expansion into additional rare genetic diseases, creating new product opportunities and revenue drivers.
4. Revenue from Existing and Future Strategic Collaborations: Stoke Therapeutics has already demonstrated substantial revenue generation from collaborations. The company's Q2 2025 revenue and Q3 2025 revenue saw significant increases driven by contractual obligations under agreements with partners such as Acadia Pharmaceuticals and Biogen. A collaboration with Biogen, established in February 2025, grants Stoke rights in the U.S., Canada, and Mexico, while Biogen holds rights for the rest of the world. These collaborations often involve upfront license fees, milestone payments upon achieving certain development or regulatory benchmarks, and potential future royalties on product sales, providing a consistent and growing revenue stream. Future strategic partnerships could further augment these revenues.

Share Issuance

• Stoke Therapeutics issued common stock amounting to approximately $10.66 million in the trailing twelve months ending June 30, 2025, $131.09 million in 2024, $53.01 million in 2023, and $46.41 million in 2022.
• The number of shares outstanding has steadily increased, from 0.033 billion in December 2020 to 0.058 billion by June 2025.
• The company utilized inducement stock grants, including stock options and shares, for key hires in March and July 2025, as a strategy for talent retention and aligning executive incentives with shareholder interests.

Inbound Investments

• Stoke Therapeutics has a license and collaboration agreement with Biogen Inc. for the development and commercialization of zorevunersen (STK-001), an investigational medicine for Dravet syndrome.

Capital Expenditures

• Capital expenditures were approximately -$0.45 million for the trailing twelve months ending June 30, 2025, and ranged from -$0.20 million in 2024 to -$3.96 million in 2022.
• These expenditures are relatively modest, consistent with an early-stage biopharmaceutical company focused on research and development activities for its RNA-based medicines and clinical programs.