TEAM

Management

Edward M. Kaye, M.D. — Chief Executive Officer

Edward M. Kaye, M.D.

Chief Executive Officer

Ed Kaye is the Chief Executive Officer and a Director of Stoke Therapeutics. Ed served as President and Chief Executive Officer of Sarepta Therapeutics (NASDAQ: SRPT) from September 2016 to June 2017, interim Chief Executive Officer from March 2015 to September 2016, and Chief Medical Officer from June 2011 to April 2017. He also served on the Company’s Board of Directors. Previously, Ed was Group Vice President of Clinical Development at Genzyme Corporation from April 2007 to June 2011, where he supervised the clinical research in the lysosomal storage disease programs and in the genetic neurological disorders. Prior to this, Dr. Kaye held various roles at Genzyme Corporation since 2001, including Vice President of Medical Affairs for Lysosomal Storage Diseases, Vice President of Clinical Research and Interim Head of PGH Global Medical Affairs. Ed currently serves as a member of the Boards of Directors of Cytokinetics, Inc. and The Massachusetts Biotechnology Council.

Prior to entering the biotech industry, Ed trained in Pediatrics, Pediatric Neurology, and Biochemical Genetics. He was on the research staff of the Massachusetts General Hospital, Tufts University Medical Center and was the Chief of Biochemical Genetics at the Children’s Hospital of Philadelphia. He continues on staff in Pediatric Neurology at the Boston Children’s Hospital. Ed earned his B.S. in Biology from Loyola University and earned his M.D. at Loyola University Stritch School of Medicine.

Huw M. Nash, Ph.D. — Chief Operating Officer and Chief Business Officer

Huw M. Nash, Ph.D.

Chief Operating Officer and Chief Business Officer

Huw Nash is the Chief Operating Officer and Chief Business Officer of Stoke Therapeutics, and previously was the founding Chief Executive Officer. An Entrepreneur-in-Residence at Apple Tree Partners focused on novel therapeutics, Huw has 20 years of experience establishing, funding and growing start-up biotechnology companies. Prior to Stoke, Huw was a co-founder and Vice President of Corporate Development for Aileron Therapeutics (NASDAQ: ALRN). He was also a founding scientist of NeoGenesis Pharmaceuticals, where he served as Vice President of External Collaborations prior to the company’s acquisition by Schering-Plough. Huw received his B.A. in Biochemical Sciences from Harvard College and received his Ph.D. in Organic Chemistry from Harvard University.

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

Barry Ticho, M.D., Ph.D., FACC

Chief Medical Officer

Barry Ticho is the Chief Medical Officer at Stoke Therapeutics, where he is helping to lead the company’s efforts to develop first-in-class therapeutics to treat rare diseases. Prior to joining Stoke, Barry was Head of Development of mRNA treatments for Cardiovascular and Metabolic Diseases at Moderna Therapeutics. Previously, he was Head of External R&D Innovation for Cardiovascular and Metabolic Diseases at Pfizer, and prior to that he was Vice President of Clinical Development at Biogen. Barry obtained his M.D. and Ph.D. degrees from the University of Chicago and completed pediatrics training at Northwestern University and a cardiology fellowship at Children’s Hospital in Boston. He was on clinical staff at Harvard Medical School and Massachusetts General Hospital and conducted laboratory research on the regulation of cardiac development.

Gene Liau, Ph.D. — EVP, Head of Research & Preclinical Development

Gene Liau, Ph.D.

EVP, Head of Research & Preclinical Development

Gene Liau is the Executive Vice President; Head, Research and Preclinical Development at Stoke Therapeutics.  Gene previously served as Senior Vice President and Head of Gene Therapy R&D at Precision BioSciences from 2015-2017. From 2011-2015 he was at Pfizer where, as Executive Director, he led External R&D efforts for Rare Disease and Hematology and spearheaded the Pfizer Gene Therapy initiative.  Prior to Pfizer, he helped Shire build an innovative rare disease portfolio. Gene was at Novartis for twelve years, initially building a Research Unit focused on using gene therapy for cardiovascular and metabolic applications and eventually leading the cardiovascular/metabolic division at the Novartis Institutes for BioMedical Research in Cambridge where he was responsible for projects from target discovery to early clinical PoC. Prior to entering industry, Gene was a Professor at George Washington University Medical Center and also a Senior Scientist at the Jerome H. Holland Laboratory of the American Red Cross.  Gene received his Ph.D. in Biochemistry from Vanderbilt University and did his Postdoctoral Fellowship at the National Cancer Institute.

Isabel Aznarez, Ph.D. — Co-Founder and Vice President, Head of Biology

Isabel Aznarez, Ph.D.

Co-Founder and Vice President, Head of Biology

Isabel Aznarez is a co-founder and Vice President of Biology of Stoke Therapeutics. Isabel has extensive experience in human genetics, RNA metabolism, and modulation of RNA processes using antisense oligonucleotides. Prior to founding Stoke Therapeutics, she was a Research Investigator with Prof. Adrian Krainer, whose lab she joined as a postdoctoral fellow in 2008. Previously, Isabel was a researcher at the Hospital for Sick Children with Prof. Lap-Chee Tsui, where she focused on the effect of cystic fibrosis mutations on the splicing of the CFTR gene. Isabel holds a Ph.D. in Medical & Molecular Genetics from the University of Toronto (2006), and a B.Sc. in Biology and Human Genetics from the University of Uruguay.

Charles R. Allerson, Ph.D. — Vice President of Chemistry

Charles R. Allerson, Ph.D.

Vice President of Chemistry

Chuck Allerson is the Vice President of Chemistry for Stoke Therapeutics. A career nucleoside/oligonucleotide chemist, Chuck has over 15 years’ experience in the design and development of oligonucleotide therapeutics for antisense, RNAi and microRNA targeting applications. Most recently, Chuck was Director of Chemistry for Regulus Therapeutics, and prior to that held roles of increasing responsibility at Ionis Pharmaceuticals and Variagenics. Chuck holds a Ph.D. in organic chemistry from Harvard University, where he studied with Professor Gregory Verdine, and was a post-doctoral researcher at the National Institutes of Health where he worked with Dr. Tracey A. Rouault, M.D.

Meena, Ph.D. — Vice President of Bioanalytical, DMPK and Biomarker Development

Meena, Ph.D.

Vice President of Bioanalytical, DMPK and Biomarker Development

Meena is the Vice President of Bioanalytical, DMPK and Biomarker Development at Stoke Therapeutics. Meena brings 20 years of experience in nucleic acids therapeutics to Stoke. Prior to joining Stoke, Meena served as Senior Director of Bioanalytical, Pharmacology and Biomarker Development at Wave Life Sciences. In her eight years at Wave (formerly Ontorii) and as its first employee, Meena played a pivotal role in building the company’s stereopure oligonucleotide chemistry platform and helping to guide the clinical entry of three antisense programs. Before joining Wave, Meena worked at Alnylam Pharmaceuticals on siRNA chemistry and targeted siRNA delivery. Meena received her Ph.D. in Chemistry with Dr. K.N. Ganesh at the National Chemical Laboratory in Pune, India and did her post-doctoral research on nucleic acids analogues with Prof. Larry W. McLaughlin at Boston College.

Shamim Ruff — Senior Vice President of Regulatory Affairs and Quality

Shamim Ruff

Senior Vice President of Regulatory Affairs and Quality

Shamim Ruff is the Senior Vice President of Regulatory Affairs and Quality at Stoke Therapeutics. Ruff has extensive knowledge of drug development with more than 25 years in the biopharmaceutical industry, working with a diverse range of therapeutics including small molecules, monoclonal antibodies, oncolytic viruses and oligonucleotides. Her expertise in both domestic and international regulatory affairs spans early and late development across multiple therapeutic areas. Prior to joining Stoke, she was at Sarepta Therapeutics, where she built the company’s regulatory affairs and quality organizations and was responsible for leading and defining the regulatory strategy for their rare and infectious disease pipelines. Prior to joining Sarepta, Ruff served as Vice President, Head of Regulatory Affairs Oncology at Sanofi-Genzyme, where she was responsible for leading the Global, European and CMC Regulatory Affairs teams. She previously held senior positions at Amgen, Abbott and AstraZeneca where she had global oversight for the development and filings of multiple successful regulatory approvals. Ruff holds a BSc. in chemistry & biology from the University of Leicester, UK, and an MSc. in analytical chemistry from the University of Loughborough, UK. Additionally, she is a Chartered Chemist and Member of the Royal Society of Chemistry (CChem MRSC), and is also a member of DIA, RAPS, ASCO and ASGCT.

Nancy Wyant — Vice President, Head of Clinical Operations

Nancy Wyant

Vice President, Head of Clinical Operations

Nancy Wyant is Vice President, Head of Clinical Operations at Stoke Therapeutics. Wyant has been an independent consultant since February 2018 and during that time has collaborated with Stoke on several key projects. Previously, she was Vice President, Clinical Operations at BeiGene USA, Inc, where she helped build the global clinical operations organization, implemented compliance standards and oversaw global clinical trial execution. Throughout her career, Wyant has led clinical trial development operations across the biotechnology industry, including as Vice President, Clinical Operations at Idera Pharmaceuticals, as Head of Clinical Operations at Sarepta Therapeutics and as Head of Global Clinical Operations at Shire Human Genetic Therapies. She also facilitated clinical operations and clinical compliance programs at EMD Serono, Therion Biologics Corporation and Vertex Pharmaceuticals. Wyant began her career at Massachusetts General Hospital as a Clinical Research and Program Coordinator. She is a member of Linking Leaders Boston and the American Epilepsy Society as well as the Society for Clinical Oncology and has a B.A. in psychology from Hartwick College.

Board of Directors

Edward M. Kaye, M.D. — CEO and Director

Edward M. Kaye, M.D.

CEO and Director

Ed Kaye is the Chief Executive Officer and a Director of Stoke Therapeutics. Ed served as President and Chief Executive Officer of Sarepta Therapeutics (NASDAQ: SRPT) from September 2016 to June 2017, interim Chief Executive Officer from March 2015 to September 2016, and Chief Medical Officer from June 2011 to April 2017. He also served on the Company’s Board of Directors. Previously, Ed was Group Vice President of Clinical Development at Genzyme Corporation from April 2007 to June 2011, where he supervised the clinical research in the lysosomal storage disease programs and in the genetic neurological disorders. Prior to this, Dr. Kaye held various roles at Genzyme Corporation since 2001, including Vice President of Medical Affairs for Lysosomal Storage Diseases, Vice President of Clinical Research and Interim Head of PGH Global Medical Affairs. Ed currently serves as a member of the Boards of Directors of Cytokinetics, Inc. and The Massachusetts Biotechnology Council.

Prior to entering the biotech industry, Ed trained in Pediatrics, Pediatric Neurology, and Biochemical Genetics. He was on the research staff of the Massachusetts General Hospital, Tufts University Medical Center and was the Chief of Biochemical Genetics at the Children’s Hospital of Philadelphia. He continues on staff in Pediatric Neurology at the Boston Children’s Hospital. Ed earned his B.S. in Biology from Loyola University and earned his M.D. at Loyola University Stritch School of Medicine.

Adrian Krainer, Ph.D. — Co-founder and Director

Adrian Krainer, Ph.D.

Co-founder and Director

Adrian R. Krainer is a co-founder and Director of Stoke Therapeutics, and is the St. Giles Professor of Molecular Genetics and Program Chair of Cancer and Molecular Biology at Cold Spring Harbor Laboratory. Prof. Krainer’s fundamental and applied research on RNA splicing for over three decades directly led to the invention and development of SPINRAZATM (nusinersen), the first approved therapeutic that corrects a splicing defect, and a life-saving drug for infants with spinal muscular atrophy. He is a member of the American Academy of Arts and Sciences, a Pew Scholar in the Biomedical Sciences, and a recipient of an NIH MERIT award and New York Intellectual Property Law Association’s 2017 inventor-of-the-year award.

Arthur A. Levin, Ph.D. — Director

Arthur A. Levin, Ph.D.

Director

Art Levin is a Director of Stoke Therapeutics and has an unparalleled track record and reputation in the field of nucleic acid-based therapeutics. He currently serves as Executive Vice President, Research and Development at Avidity Biosciences. Previously he held that position at miRagen Therapeutics. Prior to that he held senior drug development roles at Ionis Pharmaceuticals and Santaris Pharma. He has played key roles in the development of numerous of oligonucleotides including the first approved antisense NDAs, and the first microRNA-targeted therapeutic in clinical trials. He has a combined three decades of experience in all aspects of drug development from discovery through drug registration, both in large pharma and biotech companies. Art has published over 60 scientific articles and several of the most cited reviews in the field. He serves as a director of the Oligonucleotide Therapeutics Society and holds several additional scientific organization affiliations and honors. He received a doctorate in toxicology from the University of Rochester, and a bachelor’s degree in biology from Muhlenberg College.

Seth L. Harrison, M.D. — Chairman

Seth L. Harrison, M.D.

Chairman

Seth Harrison is the founder and managing partner of Apple Tree Partners (ATP) and is Chairman of the Board of Directors of Stoke Therapeutics. He has invested in life sciences since 1991. He is also currently Chairman of ATP companies Braeburn Pharmaceuticals, Elstar Therapeutics, Limelight Bio, and Syntimmune, and is a Director of Corvidia Therapeutics.

Seth’s prior investments include: Aileron Therapeutics, ArQule, Coelacanth, Cyrano Sciences, Gloucester Pharmaceuticals, HeartWare International, Informed Access, SGX Pharmaceuticals, Tokai Pharmaceuticals, Ultracision, and ViroPharma. From 2002 to 2010, he also served on the board of the International Partnership for Microbicides. Prior to founding ATP in 1999, Seth was a general partner at Oak Investment Partners, and before that a venture partner at Sevin Rosen Funds.

Seth received an A.B. from Princeton University, an M.D. and M.B.A. both from Columbia University, and completed a surgery internship at the Presbyterian Hospital in the City of New York.

Samuel W. Hall, Ph.D. — Director

Samuel W. Hall, Ph.D.

Director

Sam Hall is a principal at Apple Tree Partners (ATP) and is a Director of Stoke Therapeutics. Prior to joining ATP in 2013, Sam was a researcher at the University of Cambridge in the U.K., where his work focused on novel therapeutic strategies for autoimmune diseases. Previously, Sam was a member of the investment team at Symphony Capital, a private equity firm dedicated to investments in biopharmaceutical development. Prior to Symphony, he served as a member of the healthcare investment banking team at Citigroup, where he advised leading biotechnology, medical device and healthcare services businesses on numerous completed strategic and debt and equity financing engagements.

Sam holds an A.B. in Molecular Biology from Princeton University and completed his M.Phil. and Ph.D. research at the University of Cambridge, where he was a Taylor Research Scholar and was awarded an NSF Graduate Research Fellowship for his work.

In addition to Stoke Therapeutics, Sam is on the Board of Directors of Syntimmune, Elstar Therapeutics and Limelight Bio.

Arthur Tzianabos, Ph.D. — Director

Arthur Tzianabos, Ph.D.

Director

Dr. Arthur Tzianabos is the president and CEO of Homology Medicines. At Homology, Dr. Tzianabos leads efforts to develop genetic medicines by leveraging its in vivo gene therapy and nuclease-free gene editing platform for patients with rare genetic diseases. He previously served as president and CSO of OvaScience, where he translated early science into treatments for fertility patients. Dr. Tzianabos also spent nine years at Shire, where he worked on the development and launches of multiple treatments for patients with rare genetic disorders and worked closely with the business development team to build Shire’s product pipeline through investments and acquisitions. Earlier in his career, Dr. Tzianabos was a principal investigator and faculty member at Harvard Medical School for 15 years, reaching the rank of associate professor of medicine and maintaining laboratories at the Channing Laboratory, Brigham and Women’s Hospital and the Department of Microbiology and Molecular Genetics at Harvard Medical School. Dr. Tzianabos has published more than 80 scientific papers, reviews, book chapters and patents. He is a member of the board of directors for gene therapy company Akouos and serves on the development board for the University of New Hampshire’s College of Life Sciences and Agriculture. Dr. Tzianabos holds a B.S. in biology from Boston College and a Ph.D. in microbiology from the University of New Hampshire.

Investors

OPPORTUNITY

Gene-specific up-regulation is needed for many diseases

  • There are 7,000 orphan genetic diseases with 350,000,000 affected individuals worldwide
  • Most are due to loss/reduction of function of a single gene; many are due to loss of one allele (haploinsufficiencies)
  • ONLY 5% have approved treatments
  • Current treatments manage symptoms with little impact on outcomes & life expectancy

SOLUTION

Antisense up-regulation addresses an unmet drug space

APPROACH

Efficient target validation & hit ID pipeline

  • NextGen RNAseq yields whole transcriptome starting datasets for organs targetable by antisense oligonucleotides in humans & animals
  • Stoke’s bioinformatic engine delivers prioritized database of targetable genes
  • Cross-referencing with genetic disease databases identifies thousands of target gene opportunities for monogenic diseases caused by loss or reduction of function
  • Accelerated target to hit ID phase with focused antisense oligonucleotide arrays yields hits within 4 weeks

TECHNOLOGY

Targeted Augmentation of Nuclear Gene Output (TANGO)

There are two ways that Stoke’s TANGO technology works:

graphic_NMD_untreated-cell102218-01
graphic_NMD_aso-treated102218-01

TANGO exploits non-productive splicing events to effect targeted enhancement of gene expression

INTELLECTUAL PROPERTY

Dominant IP estate around technology & targets

  • Exclusively licensed foundational TANGO patents from Cold Spring Harbor Laboratory & University of Southampton
  • Core TANGO claims have been allowed by USPTO
  • Company has also filed patents around top drug targets amenable to up-regulation, providing composition of matter-level protection on these genes

DIFFERENTIATED PIPELINE

DISEASE FOCUS

Dravet Syndrome (DS)

  • An autosomal dominant severe epileptic encephalopathy affecting more than 30,000 patients in the U.S., EU and Japan
  • Characterized by febrile seizures within the first year of life, then frequent, uncontrolled afebrile seizures and intellectual development stagnation
  • Despite understanding the genetic mechanism, which is caused by haploinsufficiency of SCN1A, there are no genetic therapies in development for DS
  • Stoke’s technology could provide the first gene-specific, disease-modifying approach to prevent seizures and reduce developmental deficits

NEWS

Stay up to date with the latest news from Stoke Therapeutics.

Press Releases
Stoke Therapeutics to Present on First Precision Treatments for Genetic Epilepsies at J.P. Morgan Healthcare Conference
January 3, 2018

Stoke Therapeutics Expands Senior Leadership Team, Adding Deep Expertise in Regulatory Affairs and Clinical Operations
December 18, 2018

Stoke Therapeutics Presents Data Showing Single Dose of ASO Therapy Restores Normal Protein Levels in Animal Model of Genetic Epilepsy
December 1, 2018

Stoke Therapeutics to Present New Data on Lead Program at American Epilepsy Society’s 2018 Annual Meeting
November 27, 2018

Stoke Therapeutics Announces $90M Series B Financing to Advance Novel Oligonucleotide Medicines
October 23, 2018

Stoke Therapeutics to Present Research Supporting Advancement of Dravet Syndrome Program at Annual Meeting of the Oligonucleotide Therapeutics Society
September 27, 2018

Stoke Therapeutics Appoints Biotech Veteran Arthur Tzianabos, Ph.D. to Board of Directors
September 17, 2018

Stoke Therapeutics to Present Research Supporting Its Novel Approach to Oligonucleotide Mediated Gene Up-Regulation at TIDES and ASGCT Conferences
May 8th, 2018

Stoke Therapeutics Expands Leadership Team, Building on Deep Expertise in Oligonucleotide Chemistry and Rare Disease Research and Development
April 17th, 2018

Stoke Therapeutics Presents Early Research on its Novel ASO Pipeline for Severe Genetic Diseases at OPT
March 26th, 2018

Stoke Therapeutics Announces $40 Million Series A Financing to Create Pioneering New Medicines that Restore Gene Expression in Severe Genetic Disease
January 4th, 2018

In the News
Watson’s latest stumble, a glimpse into the crystal ball, and revisiting Sarepta (AUDIO)
STAT: The Readout LOUD
December 20, 2018

Baystate Business: Markets and Biotech (AUDIO)
Bloomberg
December 7, 2018

Stoke Unveils Dravet Data that Supported $90M Series B 
BioCentury
December 6, 2018

Episode 9 – From physician to CEO; always a passion for rare (AUDIO)
Rare in Common
November 15, 2018

Stoke stoked with $90M as TANGO platform leads, others follow in DS mode
BioWorld
October 24, 2018

Stoke CEO weighs clinical trials, IPO and partnerships after $90M round
Boston Business Journal
October 23, 2018

With $90M Round, Stoke Eyes a “Spinraza For Epilepsy” And a 2019 IPO
Xconomy
October 23, 2018

Disease-Modifying Treatment for Dravet Syndrome on the Horizon
Practical Neurology
October 5, 2018

Startup seeks answers to insidious form of epilepsy
The Boston Globe
May 16, 2018

Stoking Protein Production
BioCentury
April 5th, 2018

Gene Therapy 2.0: New Startups Aim to Fine-Tune Gene Delivery and Control
Xconomy
January 4th, 2018

Antisense player Stoke Therapeutics picks up $40M series A
Fierce Biotech
January 4th, 2018

Posters and Publications
TANGO – Targeted Augmentation of Nuclear Gene Output for the Treatment of Dravet Syndrome
American Epilepsy Society Annual Meeting
November 30 – December 4, 2018

TANGO – Targeted Augmentation of Nuclear Gene Output  for the Treatment of Genetic Diseases
American Society of Gene and Cell Therapy Annual Meeting
April 29 – May 2, 2018

CAREERS

 

Stoke is always looking for individuals with a passion to develop precision medicines for inherited diseases.
For more info, please contact – careers@stoketherapeutics.com

 

Chief Financial Officer
Stoke is seeking a Chief Financial Officer. The ideal candidate is an executive level finance professional with experience raising capital in the life sciences industry, preferably someone who has taken a venture-backed company public. The candidate will have recent biotech sector experience and have been the right hand to the CEO with a strong base in financial leadership and demonstrated success with strategic planning, designing and executing a financing strategy to support the company’s growth (IPO experience preferred), developing and maintaining strong relations with Board members and investors and transacting with strategic partners.

Primary Responsibilities:

  • Serve as an integral member of the executive management team and contributing strongly to strategic and operational decision making, with high quality financial advice, experience and analysis.
  • Act as a partner to the CEO and senior leadership team – appropriately debate and evaluate business and corporate development and financing strategies (including equity, debt and other non-dilutive funding).
  • Be central to evaluating, organizing and implementing major transaction including IPO/other financings, working with the executive leadership team, Board of Directors, advisors/banks, IR and PR firms, and current and new investors.
  • Facilitate investor and external relations; build relationships with and give presentations to analysts, investors and other audiences of relevance, working appropriately with the executive team and with advisers; act as a credible and effective representative of the company.
  • Bring rounded commercial financial acumen to help the company to accurately build business cases, interpret performance and anticipate the impact of decisions, ensuring that financial input to business decisions is of the highest quality and provided on a timely basis.
  • Ensure that the company identifies business issues before they escalate and assist the leadership team to timely interpret business performance and make financially informed business decisions.
  • Build and maintain a small finance and accounting staff to provide timely, accurate financial records and ensure an appropriate internal control structure is maintained and documented.

Qualifications & Experience:

  • Experience building and leading the finance organization in a biotech company that is based on high quality science and technology.
  • Experience raising capital, familiarity with and understanding of public company accounting and reporting requirements (U.S. GAAP and Sarbanes-Oxley) and ideally has experience successfully taking a company through a US listing within the last 3 years.
  • Evidence of a network with the relevant U.S. investor community members and advisors.
  • Knowledge of the U.S. tax rules
  • 10-15 years of experience in financial leadership roles including in small enterprises.
  • Demonstrated proactive initiative taking and leadership qualities and have operated at Board level.
  • Demonstrable strategic and commercial acumen, underpinned by robust financial, planning and analysis skills.
  • Driven, energetic, with strong interpersonal skills, communication ability and powers of persuasion

Primary Location
Cambridge, MA and Bedford, MA

Interested candidates should submit a CV and cover letter via e-mail to careers@stoketherapeutics.com.

Medical Director
Stoke Therapeutics is seeking a talented, independent, and motivated clinician to help design, implement and communicate the global clinical development plan for our Dravet Syndrome product candidate. The successful candidate will also be involved in advising preclinical development and ultimate clinical introduction of future antisense oligonucleotide products targeting other rare, orphan indications. This individual will have demonstrated a track record of success in his/her field, the capacity to lead with minimal supervision, plan strategically, achieve goals on time, and communicate effectively. This position also offers the opportunity to apply Stoke’s platform to other serious diseases (including other neurologic diseases and ophthalmic diseases) and to take on a major leadership role in a rapidly growing organization. This position reports to the Chief Medical Officer of the company.

Primary Responsibilities:

  • Aiding in developing the clinical strategy and integrated development plan for our Dravet Syndrome program and future programs in rare, orphan diseases, and working cross-functionally to implement, manage, interpret and communicate the results of clinical trials.
  • Being responsible for the clinical content of all clinical and regulatory documents, including protocols, INDs, CTAs, investigator brochures, CRF’s, CSRs, annual IND reports, and clinical expert reports.
  • Leading and supervising clinical trials, collaborating closely with the Clinical Operations team, and being accountable for all deliverables of clinical trials under his/her direct responsibility.
  • Responsible for analyzing clinical data, including safety monitoring in collaboration with PV.
  • Developing and maintaining relationships with key opinion leaders and PIs.
  • Organizing and presenting at relevant clinical advisory boards, DMCs and medical/scientific meetings.
  • Writing manuscripts, publications, or other documents intended for external audiences.
  • Helping to ensure Clinical Study Team compliance with FDA, EMA, PMDA, ICH and GCP guidelines and internal SOPs.
  • Contributing in an active and ongoing manner to the scientific, clinical, and commercial development of current and future product candidates.
  • Traveling, as needed, nationally and internationally.

Qualifications & Experience:

  • M.D. or M.D./Ph.D. required.
  • Board certified or eligible in a medical or pediatric subspecialty preferred.
  • Expertise in neurology or orphan diseases preferred.
  • Strong record of basic and/or translational research productivity.
  • At least 2 years of early clinical development experience in an industry setting. Substantial experience working on industry-sponsored trials will be considered.
  • Experience with oligonucleotide therapeutics is an advantage.
  • Experience with design, execution, and analysis of Phase 1-2a clinical studies.
  • Thorough understanding of Phase I-III drug development with proven ability to plan and conduct clinical trials, deliver high quality results within established timelines, interpret, analyze and communicate clinical/scientific data, and generate clinical/scientific documents.
  • Excellent written and verbal communication skills to meet the needs of varied audiences.  Strong interpersonal skills commensurate with the need to work closely with partners, investigators, contractors, consultants, and team members across functions.
  • Knowledge of FDA/EMA requirements, good clinical practices and pharmaceutical clinical development.
  • Excellent analytical, problem-solving and strategic planning skills.
  • Well organized with strong management, leadership, mentoring and motivational skills.
  • Ability to work independently and thrive in a fast-paced environment.
  • Attention to detail, demand for high-quality work, and sense of passion and urgency to achieve goals and improve the lives of patients.

Compensation: Compensation will be highly competitive for the industry and directly commensurate with experience.

Interested candidates should submit a CV and cover letter via e-mail to careers@stoketherapeutics.com.

Senior Staff Accountant
Reporting to the Director of Finance and Accounting, the Senior Staff Accountant will support the month-end close process, in addition to processing Accounts Payable and Payroll. The Senior Staff Accountant is a newly created position within the finance team and will be able to get involved in and be a part of various projects and tasks within the finance organization. This role is designed for long-term growth within the company.

Primary Responsibilities:

  • Preparing, recording, and processing of accounts payable and cash disbursements; interfacing with suppliers as needed.
  • Preparing monthly General Ledger journal entries including payroll, depreciation, accruals, prepaid expenses, reclassifications, and other standard entries.
  • Ensuring that project accounting is accurate.
  • Reconciling various general ledger accounts with sub-systems.
  • Maintaining all balance sheet reconciliations including cash and fixed assets.
  • Preparing and analyzing monthly financial statements and general ledger, comparing budget to actual activity and observing trends.
  • Active participation in the annual budgeting process.
  • Preparing annual audit schedules and supporting and interfacing with auditors.
  • Preparing annual tax schedules for tax accountants.
  • Ensuring compliance with GAAP principles.
  • Processing bimonthly payroll, including inputting employee maintenance, supporting various requests from Human Resources and external agencies. Other payroll activity as needed.
  • Developing and documenting business processes and accounting policies to streamline accounting tasks and to maintain and strengthen internal controls.
  • Assisting and owning tasks related to the implementation of processes pertaining to the newly implemented financial system.
  • Performing ad-hoc analysis and troubleshooting of matters as they arise.

Minimum Qualifications:

  • Bachelor’s degree in Accounting or Finance or related work experience.
  • 5+ years of previous accounting experience.
  • Fully understands GAAP and is able to articulate that information to others when needed.
  • Ability to do research to find solutions for accounting issues.
  • Experience working in the biotech industry.
  • ERP Systems Software Experience a plus.

Skills/Competencies:

  • Ideal candidate must be enthusiastic, self-motivated, results-oriented, and operate well in a team environment.
  • Must be detail-oriented and possess good analytical and problem-solving skills.
  • Excellent verbal and written communication skills and strong interpersonal skills.
  • Ability to communicate effectively with the accounting team, executive management and all departments across the organization.
  • Strong Microsoft Office and overall computer skills.
  • Able to function independently; proactive, capable of finding solutions.
  • Receptive to feedback, willing to learn, embrace continuous improvement, open and responsive to change.

Primary Location: Bedford, MA

Interested candidates should submit a CV and cover letter via e-mail to careers@stoketherapeutics.com.

Director / Senior Director, Regulatory Project Management and Operations
Stoke Therapeutics is currently seeking an experienced, dynamic and well-organized Director/Senior Director, Regulatory Operations to work in the regulatory affairs department reporting to the Senior Vice President, Regulatory Affairs. The successful candidate will oversee the planning and management of regulatory submissions and regulatory systems to ensure compliance, and will facilitate and manage planning by way of document management and workflow tools.

Primary Responsibilities:

  • Establish and implement systems to ensure regulatory compliance.
  • Oversee global regulatory operational strategy and submission activities.
  • Provide strategic vision and leadership to develop, create and implement global harmonized submission assembly processes to support business objectives.
  • Develop portfolio submission forecasts through partnership with stakeholders.
  • Provide leadership to the regulatory operations group and cross-functional teams.
  • Work closely with program/project management and cross-functional teams to effectively identify, plan for and manage timely completion of regulatory milestones.
  • Manage the successful delivery of regulatory submissions for all development programs.
  • Manage regulatory operations budgets to support all phases of development projects.
  • Represent regulatory on development teams to ensure submissions are compiled in accordance with document submission regulations.
  • Oversee the compilation and collaboration of all submissions.
  • Manage the regulatory information and document management system.

Qualifications & Experience: 

  • Bachelor’s degree with a minimum of 12 years of experience in regulatory operations.
  • Direct experience filing INDs, NDAs and preferably CTAs and MAAs.
  • Expertise in eCTD-compliant submission regulatory requirements and ICH requirements.
  • Expertise with the tools and processes associated with regulatory PM and submissions.
  • Excellent communication and presentation skills.
  • Experience with multiple phases of product development and lifecycle management.

Candidates must be authorized to work in the U.S.

Compensation: Stoke offers a competitive compensation and benefits package.

Location: This position is based in the Cambridge, MA office. This role will also require regular travel to the Bedford, MA office location.

Interested candidates should submit a CV and cover letter via e-mail to careers@stoketherapeutics.com.

Manager / Senior Manager, Clinical Operations
Stoke Therapeutics is seeking a highly motivated and enthusiastic clinical operations professional with hands on expertise in running rare disease clinical trials. The manager or senior manager will support Stoke’s clinical development pipeline and will be responsible for driving clinical operations activities within a development program and managing day-to-day study operations for one or more clinical studies to meet individual clinical trial objectives and corporate goals. For clinical studies assigned, this individual is responsible for overseeing all aspects of global and domestic clinical trials predominantly run through Contract Research Organizations to ensure delivery of company goals on time, within budget, and in compliance with SOPs and FDA and ICH/GCP guidelines.

Primary Responsibilities:

  • Lead all aspects of assigned clinical trial(s) (Natural History, Phase 1-3) and demonstrate a high level of knowledge of clinical operations, including methodologies, organization, project management and leadership capabilities.
  • Actively participate in the development of department initiatives, including acting as lead on the development/definition of SOPs and processes.
  • Utilize project management skills to ensure consistency in processes and drive clinical operations performance.
  • Manage and oversee all study vendors (CROs) to ensure that Stoke’s clinical trials are getting the necessary resources and attention the vendor has committed to; ensure trial deliverables and performance goals are met and quality is maintained.
  • Oversee the development of and adherence to clinical trial project timelines and ensure line function heads are informed of and understand risks/challenges; includes putting risk mitigation plans in place, monitoring risk metrics, and communicating those risks.
  • Author clinical study protocols, informed consent forms (ICFs) and other clinical documents.
  • Coordinate the relevant and timely exchange of information/materials (e.g. subject enrollment, site selection/feasibility, and data collection) with other Stoke functions to support clinical trials delivery within the drug development process.
  • Coordinate with CMC regarding drug forecasting and supply of drug products to sites.
  • Support timely and efficient database lock by ensuring monitoring plans are in place and by participating in data review/reconciliation efforts.
  • Adhere to clinical trial budgeting process and provide input to finance on budget accruals and forecasts.
  • Participate in clinical trial scenario planning (estimate time and cost).

Qualifications & Experience:

  • Bachelor’s degree or higher in a scientific or healthcare discipline preferred with at least 3-5 years of progressive experience in clinical operations within biotech, pharmaceutical, or CRO industry.
  • Experience monitoring clinical studies and conducting PSVs, SIVs, IMVs, and COVs are required. Rare disease and neurology trial experience is highly desirable.
  • Efficient in Microsoft Word, Excel, Outlook, PowerPoint and MS Project.
  • Strong written and verbal communication skills.

Location: Cambridge, MA

Interested candidates should submit a CV and cover letter via e-mail to careers@stoketherapeutics.com.

Associate Director, Oligonucleotide Chemistry
Stoke is seeking an enthusiastic and highly motivated oligonucleotide chemist to join the chemistry group.

Primary Responsibilities:

  • Establishing internal oligonucleotide synthesis capability, including the set-up of new synthesis and purification equipment.
  • Synthesis and purification of oligonucleotides from milligram to multigram scale.
  • Synthesis of oligonucleotide conjugates and oligonucleotides containing novel chemistries.
  • Sourcing of raw materials and all other reagents needed for oligonucleotide synthesis.
  • Maintenance of all oligonucleotide synthesis and purification equipment.
  • Recruiting and managing personnel to support oligonucleotide synthesis activities.
  • Cross-training of other chemistry personnel in oligonucleotide synthesis and purification methodology.
  • Interacting with external oligonucleotide synthesis CMOs to manage orders, assist with troubleshooting, and facilitate technology transfer as needed.
  • Collaborating extensively with discovery and development project teams.

Qualifications & Experience:

  • Ph.D. degree in chemistry or biochemistry with at least 10 years of post-graduate experience in oligonucleotide chemistry.
  • Previous experience with ÄKTA Oligopilot and/or MerMade oligonucleotide synthesizer platforms.
  • Previous experience with oligonucleotide conjugation chemistry.
  • Strong organizational and recordkeeping skills.
  • Strong presentational skills to be able to communicate to both internal and external audiences.
  • Ability to work as a team player in a dynamic environment.
  • Ability to lead from the bench.

Compensation: Compensation will be highly competitive for the industry and directly commensurate with experience.

Location: Bedford, MA

Interested candidates should submit a CV and cover letter via e-mail to careers@stoketherapeutics.com.

Senior Research Associate, Analytical Chemistry
Stoke is seeking an enthusiastic and highly motivated analytical chemist to join the chemistry group.

Primary Responsibilities: 

  • Characterization of oligonucleotides using LC-MS.
  • Biophysical characterization of oligonucleotides, including Tm, viscosity, and protein binding.
  • Formulation and distribution of oligonucleotides.
  • Characterization of oligonucleotide formulations, including endotoxin and osmolality measurement.
  • Physical and database management of internal compound inventory.
  • Maintenance of the analytical lab and instrumentation.
  • Working closely with other members of the chemistry team, and collaborate extensively with discovery and development project teams.

Qualifications & Experience:

  • Bachelor’s or master’s degree in chemistry or biochemistry with 4+ years of relevant experience.
  • Previous experience with LC-MS and other relevant analytical methodologies
  • Ability to work as a team player in a dynamic environment.
  • Strong organizational and record-keeping skills.
  • Prior experience with oligonucleotides is ideal.

Compensation: Compensation will be highly competitive for the industry and directly commensurate with experience.

Location: Bedford, MA

Interested candidates should submit a CV and cover letter via e-mail to careers@stoketherapeutics.com.