S

Stoke Therapeutics

Develops targeted gene upregulation therapies

Vice President - Regulatory Strategy and CMC

Full-Time
$345k - $395k+ Annual bonus + Equity participation
Expert
Master's, PharmD, PhD
Bedford, MA, USA
HybridHybrid role; the position will move from Bedford to Waltham, Massachusetts by the end of 2026.
Biology & BiotechRegulatory & Quality (Life Sciences)

About the job

Requirements
  • A Ph.D., Pharm.D., M.S., or advanced scientific degree in Pharmaceutical Sciences, Chemistry, Biochemistry, Chemical Engineering, Biotechnology, or a related discipline is preferred.
  • At least 12 years of progressive experience in biotechnology, pharmaceutical, or cell and gene therapy industries, including at least 6 years in a leadership capacity.
  • Deep expertise in global Regulatory Affairs and CMC development.
  • Demonstrated success leading major regulatory submissions and health authority interactions.
  • Experience advancing products from early development through commercialization.
  • Significant experience managing multiple development programs.
  • Proven executive leadership experience building and managing multidisciplinary teams.
  • Executive presence and ELT-level communication.
  • Risk assessment and decision-making.
  • Cross-functional influence and collaboration.
  • Change management and organizational leadership.
  • Negotiation and stakeholder management.
  • Financial and operational planning.
Responsibilities
  • Provide oversight and guidance in developing and implementing integrated global regulatory strategies for Stoke programs.
  • Lead and manage teams responsible for INDs, CTAs, IMPDs, BLAs, NDAs, MAAs, and post-approval submissions.
  • Serve as the executive lead for interactions with FDA, EMA, MHRA, PMDA, Health Canada, and other global regulatory agencies.
  • Provide strategic regulatory guidance to executive leadership and program teams regarding development pathways, agency expectations, accelerated programs, and risk mitigation.
  • Monitor evolving regulatory requirements and assess impacts on development and commercial programs.
  • Support corporate business development, licensing, due diligence, and portfolio evaluation activities.
  • Provide oversight and guidance for enterprise-wide CMC strategies covering drug substance, drug product, analytical development, process development, manufacturing, supply, and combination drug-device development.
  • Ensure phase-appropriate CMC development plans align with regulatory expectations and business objectives.
  • Lead and manage teams responsible for CMC readiness activities supporting clinical studies, registration, launch, and lifecycle management.
  • Partner with Quality and Technical Operations to ensure compliance with cGMP, ICH, FDA, EMA, and global regulatory requirements.
  • Provide strategic regulatory guidance on long-range manufacturing and supply strategies to support global commercial expansion.
  • Build, mentor, and lead high-performing Regulatory Strategy and CMC teams.
  • Establish department goals, governance processes, resource planning, and budget management.
  • Foster a collaborative, science-driven, and compliance-focused culture.
  • Influence company strategy through participation on executive leadership committees and governance boards.
  • Manage external consultants, CROs, CDMOs, and strategic partnerships supporting regulatory and manufacturing activities.
  • Support launch readiness activities and commercial regulatory strategies.
  • Oversee post-approval changes, global lifecycle management activities, and market expansion initiatives.
  • Ensure regulatory compliance throughout the product lifecycle while enabling operational flexibility and business growth.
  • Identify regulatory and manufacturing risks and develop proactive mitigation strategies.
Desired Qualifications
  • An annual bonus and equity participation are offered as additional compensation.

About the company

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Stoke Therapeutics

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Website

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.

Company Size

201-500

Company Stage

IPO

Headquarters

Bedford, Texas

Founded

2014

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Simplify's Take

What believers are saying

  • June 30, 2026 EMPEROR enrolled 162 patients; readout is targeted for third quarter 2027.
  • August 8, 2026 management guided to early-2028 U.S. launch with about $420 million cash.
  • Four-year September 2026 extension data showed sustained cognition and seizure improvements with tolerability.

What critics are saying

  • EMPEROR failure in third quarter 2027 would destroy Stoke's only near-term value driver.
  • FDA discussions remain open after the January 11, 2026 request for additional information.
  • Zorevunersen caused elevated cerebrospinal fluid protein in 94% of patients by July 31, 2026.

What makes Stoke Therapeutics unique

  • Zorevunersen targets SCN1A haploinsufficiency; TANGO upregulates endogenous protein, not replacement.
  • Stoke and Biogen share zorevunersen commercialization; Stoke keeps U.S., Canada, Mexico rights.
  • September 2026 board adds Biogen-linked commercial operator Bo Cumbo before 2028 launch planning.

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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

Growth & Insights and Company News

Headcount

6 month growth

4%

1 year growth

7%

2 year growth

2%
Business Wire
Oct 16th, 2026
Stoke Therapeutics grants options on 245,920 shares to 18 new hires at $24.61 each

Stoke Therapeutics (Nasdaq: STOK) granted stock options to 18 new employees on October 15, 2026, as an incentive to join the company. The Bedford, Massachusetts, biotech said the grants comply with Nasdaq Listing Rule 5635(c)(4). Stephen Aschettino, its newly appointed senior vice president and chief information officer, received options to buy 70,000 shares. Seventeen other new hires received options for a combined 175,920 shares. The exercise price is $24.61 per share, the closing price on October 15, 2026. The options vest over four years, with a quarter vesting after one year and the rest monthly over the following 36 months. They run for 10 years under the 2023 Inducement Plan. Stoke is developing zorevunersen, an RNA medicine for Dravet syndrome, in a Phase 3 study.

Biotech Today
Oct 6th, 2026
Stoke brings STK-002 to AAO as evidence takes center stage.

Stoke brings STK-002 to AAO as evidence takes center stage. Stoke's STK-002 presentations will focus attention on the evidence for its investigational optic atrophy medicine, not establish a treatment breakthrough. Key facts. * Company and market snapshot: STOK: 24.36, currency and exchange unspecified in supplied data; down 3.79% as of October 6, 2026, 18:37:37 GMT. * Investigational medicine: STK-002, described by Stoke Therapeutics as an investigational treatment. * Intended condition: Autosomal dominant optic atrophy, abbreviated ADOA. * Scientific meeting: Presentations announced for the American Academy of Ophthalmology's 2026 Annual Meeting. Stoke Therapeutics announced presentations of STK-002, its investigational medicine for autosomal dominant optic atrophy, at the American Academy of Ophthalmology's 2026 Annual Meeting. Stoke Therapeutics (STOK) announced presentations of STK-002, an investigational medicine for autosomal dominant optic atrophy, at the American Academy of Ophthalmology's 2026 Annual Meeting. The development brings the candidate before an eye-care audience, but the announcement alone does not establish whether it improves vision or changes the course of the disease. The distinction matters for patients, clinicians and investors. A conference presentation can provide a setting for evaluating research; it is not itself a clinical success or a regulatory decision. The central question is what the presentations will show about STK-002 and how much that evidence can support. Stoke's announcement identifies the medicine, the intended condition and the meeting, according to Business Wire. The available details do not specify presentation findings, study design, patient results or a regulatory timetable. Those limits make this a research-development story rather than a report of demonstrated treatment benefit. What the AAO announcement establishes. STK-002 is being developed for autosomal dominant optic atrophy, abbreviated ADOA. The company describes it as investigational, meaning it remains under study rather than being established by this announcement as an approved treatment. Nothing in the supplied information supports a claim about its effectiveness, safety profile or readiness for commercial use. The American Academy of Ophthalmology meeting provides a specialist setting for the presentations. That is relevant because research aimed at an eye condition needs scrutiny not only of its scientific rationale but also of how its findings relate to visual function and clinical practice. Still, the setting should not be confused with the strength of the evidence. The announcement does not identify whether the presentations concern laboratory work, patient observations or another type of research. It would therefore be premature to describe them as a clinical-trial readout or to assign the program a development stage. For now, the concrete development is that Stoke plans to present work on the candidate. The next useful information will be the content of that work: what was investigated, how it was measured and what conclusions the results can reasonably support. The evidence questions that matter for STK-002. Any assessment should begin by distinguishing a biological effect from a benefit that matters to patients. Evidence that an investigational medicine influences a disease-related process can be important, but it does not automatically show that people see better, retain vision longer or experience improved daily functioning. If patient findings are presented, the methods will matter alongside the results. Readers should look for an explanation of who was studied, what outcomes were measured and whether the research included a comparison that helps separate a treatment effect from other influences. These are evaluation questions, not details confirmed for STK-002. The observation period would also affect interpretation. A result measured soon after treatment may answer a different question from one tracked over a longer interval. Without that information, it is difficult to assess whether an apparent effect is sustained or whether the research addresses the disease's course. A result measured soon after treatment may answer a different question from one tracked over a longer interval. Safety requires its own examination. A favorable biological finding cannot substitute for information about unwanted effects, tolerability or the practical demands of treatment. The available announcement supplies no basis for concluding that STK-002 is safe, unsafe or preferable to another approach. The most useful presentation would connect the scientific claim to the evidence supporting it while making the limitations clear. That means separating observed results from hypotheses and explaining what further work would be needed before a treatment claim could be established. Shares fall, but the cause is not established. Stoke's shares were down 3.79% on the day at the last-trade timestamp of October 6, 2026, at 18:37:37 GMT, according to the supplied licensed market data. The quote is an intraday snapshot, not a closing result. The decline occurred while the supplied broad-market benchmarks were positive. The S&P 500 benchmark was up 0.65%, the Nasdaq 100 benchmark was up 0.57%, and the Dow benchmark was up 0.50%. That contrast shows Stoke moving against the broader market direction at the stated time. It does not establish why the shares fell. The information supplied contains no investor commentary, trading-volume context or presentation results that would link the move to a particular scientific judgment. Calling the decline a rejection of STK-002 would go beyond the evidence. Investors should keep the trading signal and the research signal separate. A share-price change captures market activity at a moment in time. The candidate's scientific value depends on evidence that the presentation announcement does not yet describe. Neither the conference appearance nor the stock decline resolves the other. What patients and investors should watch next. For patients and families, the immediate distinction is between research visibility and treatment availability. This announcement does not establish access to STK-002 or provide a basis for changing care. Decisions about an investigational medicine require clinical information and guidance beyond a conference notice. For clinicians, the substantive material will be the methods and findings behind the presentations. For investors, it will also matter whether those findings clarify the program's next development steps. The available information does not support a launch forecast, a revenue estimate or a prediction about regulatory action. The next assessment should therefore focus on the presentation materials and any supporting evidence Stoke makes available. Until then, STK-002 has a defined intended use and an announced scientific forum, but not a demonstrated outcome in the facts supplied here. The meeting's value will rest on how much uncertainty the research can actually reduce. Frequently asked questions. What did Stoke Therapeutics announce about STK-002? Stoke Therapeutics announced presentations of STK-002 at the American Academy of Ophthalmology's 2026 Annual Meeting. The company describes the candidate as an investigational medicine for autosomal dominant optic atrophy. The supplied announcement details do not identify specific findings, patient outcomes, study methods or a regulatory timetable. Is STK-002 an approved treatment for ADOA? The announcement describes STK-002 as investigational, not as an approved treatment for autosomal dominant optic atrophy. A conference presentation does not itself establish regulatory approval, clinical benefit or treatment availability. The supplied information provides no basis for patients to change care or assume that the medicine is commercially available. Does the announcement show that STK-002 improves vision? No vision results are included in the supplied facts. The announcement establishes that presentations are planned, not that STK-002 improves vision or changes disease progression. Assessing those claims would require the actual findings, the measurements used, the study methods and an explanation of the research's limitations. What should readers look for in the presentations? Readers should look for the type of research presented, the methods used and the outcomes measured. If patient results are included, comparison methods, follow-up and safety information will be important. A biological effect should be distinguished from a demonstrated benefit in vision or daily functioning; neither is established by the announcement. How were Stoke shares trading after the announcement? The supplied licensed market data showed STOK down 3.79% on the day as of October 6, 2026, at 18:37:37 GMT. This was an intraday snapshot, not a closing result. The available information does not establish that the presentation announcement caused the decline or reveal investors' reasoning. What does the AAO presentation mean for investors? The presentation offers an opportunity to examine research on STK-002, but its investment significance depends on the evidence disclosed. Investors should assess whether the findings clarify benefit, safety and further development needs. The announcement alone does not support a commercial forecast, a regulatory prediction or a valuation conclusion.

Eyewire
Oct 6th, 2026
Stoke Therapeutics to present STK-002 data for Autosomal Dominant Optic Atrophy at AAO.

Stoke Therapeutics to present STK-002 data for Autosomal Dominant Optic Atrophy at AAO. Key Takeaways * Stoke Therapeutics will present data on investigational RNA medicine STK-002 in 2 presentations at AAO 2026 * The Phase 1 OSPREY study is progressing through dose escalation, with cohorts 3 and 4 expected to reach therapeutic dosing levels * Initial safety and efficacy data from the higher-dose cohorts are anticipated in the first half of 2027 Stoke Therapeutics announced that data for its investigational RNA medicine STK-002 will be featured in 2 presentations at the 2026 American Academy of Ophthalmology (AAO) annual meeting, taking place October 9-12 in New Orleans. STK-002 is being developed for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. The investigational therapy is designed to address the underlying cause of the disease by increasing OPA1 protein levels, with the goal of improving vision. There are currently no approved treatments for ADOA, according to Stoke. "ADOA causes progressive and irreversible vision loss in both eyes with 80% of patients symptomatic by age 10 and approximately half progressing to legal blindness," Barry Ticho, MD, PhD, chief medical officer of Stoke Therapeutics, said in a statement. "We are encouraged by our preclinical data as well as emerging findings from the field demonstrating that upregulation of OPA1 protein may have disease-modifying potential." STK-002 is currently being evaluated in the phase 1 OSPREY study in patients with ADOA. Dose escalation is ongoing, with the first 2 dose cohorts completed and the third cohort expected to be completed by the end of 2026. According to Stoke, cohorts 3 and 4 are expected to reach therapeutic dosing levels. Initial safety and efficacy findings from these higher-dose cohorts are anticipated during the first half of 2027. The data being presented at AAO 2026 provide translational evidence supporting the potential of increasing functional OPA1 protein as a disease-modifying approach in ADOA, according to the company. Details of the presentations are as follows: Title: OSPREY: First-in-Human Phase 1 Study of the RNA-Based Therapy STK-002 in Autosomal Dominant Optic Atrophy Podium Poster Date & Time: Sunday, October 11, 10:30-10:40 AM CT Presenting Author: Patrick Yu-Wai-Man, M.D., Ph.D., Professor of Ophthalmology at the University of Cambridge and the UCL Institute of Ophthalmology, and Honorary Consultant Neuro-ophthalmologist at Addenbrooke's Hospital and Moorfields Eye Hospital, United Kingdom Poster Number: PO055 Title: RNA-Based OPA1 Upregulation in Autosomal Dominant Optic Atrophy: First-in-Human STK-002 Study in Mitochondrial Optic Neuropathy Poster Panel Discussion Date & Time: Sunday, October 11, 3:15-3:21 PM CT Presenting Author: Nancy J. Newman, M.D., Professor, Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine Poster Number: PO222

Kalkine
Sep 28th, 2026
Stoke Therapeutics (NASDAQ:STOK) replaces departing director edward Kaye with Bo Cumbo.

Stoke Therapeutics (NASDAQ:STOK) replaces departing director edward Kaye with Bo Cumbo. 28 September 2026 11:22 AM PDT Summarize with AI You are reading a free article with opinions that may differ from the recommendation given by Kalkine in its paid research reports. Become a Kalkine member today to get access to its research reports, in-depth technical and fundamental research. Learn more Key Highlights Dr. Edward M. Kaye notified Stoke Therapeutics' board on September 22, 2026 of his intent to resign as a Class III director, effective September 23, 2026, with the filing stating the resignation was not the result of any disagreement with the company. Kaye also stepped down from the board's Research and Development Committee effective September 23, 2026, and the filing does not disclose his reasons for leaving beyond ruling out a disagreement. The board appointed Alexander Bo Cumbo on September 23, 2026 to Fill the vacant Class III seat, with a term extending through the company's 2028 Annual Meeting of stockholders. Cumbo's compensation includes a $45,000 annual cash retainer and an initial stock option grant covering 39,674 shares, valued at approximately $724,000 under ASC 718, vesting in twelve equal quarterly installments over three years. Stoke Therapeutics, Inc. (NASDAQ:STOK) disclosed in an 8-K that Dr. Edward M. Kaye notified the board on September 22, 2026 of his intent to resign as a Class III director, effective the following day, and that the board appointed Alexander Bo Cumbo to fill the vacancy with a compensation package that includes an option grant valued at roughly $724,000. Kaye's Departure The filing states plainly that Dr. Kaye's resignation was not the result of any disagreement with the Company, the standard disclosure companies use to rule out any conflict over strategy or governance. Kaye also stepped down from his seat on the board's Research and Development Committee as part of his resignation, effective September 23, 2026. The filing does not disclose Kaye's reasons for leaving beyond confirming the absence of any disagreement, and it does not detail his prior tenure length on the board. Cumbo's Appointment The board appointed Cumbo on September 23, 2026 to fill the vacant Class III seat, with a term extending through the company's 2028 Annual Meeting of stockholders. The filing confirms Cumbo qualifies as an independent director under both SEC rules and Nasdaq listing standards, and states that no arrangements or understandings influenced his selection to the board. It further discloses that Cumbo has no material transactions with the company requiring disclosure under applicable SEC regulations governing related-party transactions. Compensation for the New Director Cumbo's pay package follows Stoke Therapeutics' standard non-employee director compensation program. He will receive an annual cash retainer of $45,000, along with an initial stock option grant covering 39,674 shares. The filing values that grant at approximately $724,000 under the accounting methodology specified in ASC 718, the standard used for Equity compensation. The Options vest in twelve substantially equal quarterly installments measured from the grant date, meaning full vesting will take three years assuming continued board service. Cumbo also executed the company's standard director indemnification agreement, a form previously filed as part of the company's 2019 registration statement on Form S-1. What the Filing Does Not Say The 8-K does not provide a biography or professional background for Cumbo beyond confirming his independence and lack of conflicting transactions, details on his prior career, education, or other board memberships are absent from this filing. It also does not disclose whether Kaye's Research and Development Committee seat has been filled by Cumbo or another existing director, and it does not explain why the board moved so quickly, filling the vacancy the day after Kaye's resignation took effect. Company Background Stoke Therapeutics is a Nasdaq-listed biotechnology company; the filing does not restate details of its drug pipeline or clinical programs, focusing solely on the Item 5.02 director transition. Investors seeking Business context on the company's therapeutic focus would need to consult the company's periodic reports rather than this filing. Vesting Timeline for Cumbo's Board Compensation Key dates to track include the 2028 Annual Meeting, when Cumbo's term will come up for renewal or election by shareholders, and the quarterly vesting dates for his option grant over the coming three years. Any further committee assignments for Cumbo, including a possible seat on the Research and Development Committee vacated by Kaye, would also be worth watching in future filings. Because Cumbo's option grant vests in equal quarterly installments over three years rather than immediately, his full compensation from this appointment will only be realized if he remains on the board through that entire vesting schedule, giving the company's board composition additional stability at least through the middle of the vesting period. The filing does not disclose any acceleration provisions that would apply if a future change of control or similar event were to occur before vesting completes. FAQs. Q: why did Dr. Edward Kaye resign from Stoke Therapeutics' board? A: The filing states the resignation was not the result of any disagreement with the company; no further reason is disclosed. Q: Who was appointed to replace Kaye? A: Alexander Bo Cumbo was appointed on September 23, 2026 to fill the vacant Class III director seat, with a term through the 2028 Annual Meeting. Q: What is Cumbo's compensation package? A: A $45,000 annual cash retainer plus an initial stock option grant covering 39,674 shares, valued at approximately $724,000 under ASC 718, vesting quarterly over three years. Q: Did Kaye's committee seat transfer to Cumbo? A: The filing does not disclose whether Cumbo or another existing director filled the Research and Development Committee seat that Kaye vacated. Download Free Report - Explore 3 Stock Ideas & Industry Insights Unlock 3 stock ideas and key industry insights in its free report. This information is general in nature and does not consider your personal objectives, financial situation, or needs. It is not financial advice. All investments involve risk - consider independent advice before making any investment decisions. Disclaimer:

Business Wire
Sep 23rd, 2026
Stoke Therapeutics adds Bo Cumbo to board as company prepares for zorevunersen FDA decision in early 2028

Stoke Therapeutics has appointed Bo Cumbo to its board of directors as the biotechnology company prepares for potential FDA approval of zorevunersen in early 2028. Zorevunersen is being developed as a first-in-class treatment for Dravet syndrome, a severe epileptic condition. Cumbo brings over 30 years of biopharmaceutical experience, currently serving as president and CEO of Solid Biosciences. He previously held commercial leadership positions at Sarepta Therapeutics, where he helped bring the first FDA-approved treatments for Duchenne muscular dystrophy to patients. His appointment coincides with the retirement of Edward Kaye from the board. Kaye served as a director since 2017 and was CEO until March 2025. Stoke has a strategic collaboration with Biogen to develop and commercialise zorevunersen, retaining exclusive US rights whilst Biogen holds rest-of-world commercialisation rights.