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., The Massachusetts Biotechnology Council and Neurovia.

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.

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., The Massachusetts Biotechnology Council and Neurovia.

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.

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)

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

NEWS

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

Press Releases
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

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 Genetic Diseases
American Society of Gene and Cell Therapy Annual Meeting
April 29 – May 2, 2018

Conferences and Events
Post-Transcriptional Gene Regulation, Gordon Research Conference
July 15 – 20, 2018
Newry, ME

2018 Dravet Syndrome Foundation Biennial Family and Professional Conference
July 19-22, 2018
Aurora, CO

Mechanisms of Epilepsy and Neuronal Synchronization, Gordon Research Conference
August 19 – 24, 2018
West Dover, VT

International Roundtable on Nucleosides, Nucleotides & Nucleic Acids (IRT)
August 26-30, 2018
San Diego, CA

13th European Congress on Epileptology
August 26-30, 2018
Vienna, Austria

The 14th Annual Meeting of the Oligonucleotide Therapeutics Society
September 30 – October 3, 2018
Seattle, WA

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

Director of Accounting & Finance
STOKE-010
Director of Accounting & Finance
Stoke Therapeutics

Company Overview:

Stoke Therapeutics is a biotechnology company working to increase gene expression to treat a wide array of rare diseases, including genetic conditions affecting the central nervous system, eye, and liver. For more information visit www.stoketherapeutics.com.

Stoke Therapeutics is looking for an Accounting & Finance Director to develop and lead our Accounting & Finance function. This position will report to the COO/interim CFO. The selected applicant will have the opportunity to develop and implement sustainable financial accounting systems and contribute the company’s long term goals and financial success. The successful candidate will be collaborative, highly motivated, and possess the desire to constantly improve processes with an emphasis toward financial and operational best practices.

Responsibilities:

  • Lead and develop a dynamic financial organization that is dedicated to financial integrity and focused on supporting Stoke’s growth strategy
  • Orchestrate all financial reporting activities including but not limited to internal management reporting, independent auditing firms, tax preparers, and valuation firms
  • Ensure the financial organization delivers timely financials in accordance with GAAP, ensure all significant estimates and judgments have been fully documented.
  • Effective internal controls over financial reporting and up to date documentation of those controls
  • Implement and maintain financial and accounting systems that are robust, controls compliant, and support rapid future growth
  • Oversee and manage payroll, tax, and audit activities
  • Responsible for overseeing monthly and quarterly financial close, all elements of operational accounting, including accounts payable, accounts receivable and payroll and share based compensation
  • Work with senior leadership to develop budgets and cash forecasts
  • Analysis of budget vs. actual and developing robust internal financial reporting systems to guide strategy and resourcing
  • Work in concert with operations team to ensure resource efficient operation of the company

Minimum Qualifications:

  • Bachelor’s degree in accounting, finance, or other related field and CPA designation
  • 7+ years of accounting and financial leadership experience including time in public accounting
  • Experience in the pharmaceutical/biotechnology industry encompassing research, development and commercial functions demonstrating a keen understanding of critical accounting and financial reporting issues, risks and challenges specific to the industry
  • Excellent written and verbal communication skills
  • Strong technical and financial acumen and well versed in U.S. GAAP
  • The ability to assist other leaders with non- financial backgrounds
  • A proven leader with the ability to motivate and develop talent
  • Demonstrated ability to apply “best practice” business process improvement and financial controls
  • Ability to make sound and timely business judgments consistent with company objectives
  • Willingness to contribute to the team in any manner possible to reach company goals

Interested candidates should submit a CV and cover letter via e-mail to careers@stoketherapeutics.com. Please reference position STOKE-010.

Research Associate, Analytical Chemistry
STOKE-009
Research Associate, Analytical Chemistry
Stoke Therapeutics

Company Overview:

Stoke Therapeutics is a biotechnology company working to increase gene expression to treat a wide array of rare diseases, including genetic conditions affecting the central nervous system, eye, and liver. For more information visit www.stoketherapeutics.com.

 

Opportunity Overview

Stoke is seeking an enthusiastic and highly motivated analytical chemist to join the chemistry group. The successful candidate will be responsible for:

  • 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.
  • Will collaborate extensively with discovery and development project teams.

Qualifications & Experience:

  • Bachelors or Masters degree in chemistry or biochemistry with 2-5 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 recordkeeping skills.
  • Prior experience with oligonucleotides is ideal.

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. Please reference position STOKE-009.

Research Associate/ Sr. Research Associate, Biology
STOKE-005
Research Associate/Sr. Research Associate, Biology
Stoke Therapeutics 

Company Overview:

Stoke Therapeutics is a biotechnology company working to increase gene expression to treat a wide array of rare diseases, including genetic conditions affecting the central nervous system, eye, and liver. For more information visit www.stoketherapeutics.com.

Opportunity Overview:

Stoke is seeking to hire a Research Associate or Senior Research Associate with a strong record of success in molecular biology and cell biology research.  Candidates must hold a Bachelor’s or Master’s degree with a strong background in molecular or cell biology with at least three years’ post-graduate experience.  Experience in RNA handling techniques, RNA metabolism, and cell-based assays is required.  Research experience in human genetic diseases and/or molecular therapeutics development is highly desirable.  Knowledge of CNS or ocular diseases is a plus. The successful candidate will work in a fast-paced and dynamic environment.  He or she will work with a team of scientists to identify oligonucleotide-based drug candidates for the treatment of ocular and CNS diseases.  This involves screening assays to identify lead candidates and development of in vitro and in vivo systems to evaluate these candidates.

Qualifications & Experience:

  • Bachelor’s or Master’s degree in molecular or cell biology
  • At least 3 years’ post-graduate experience, ideally in industry
  • Expertise in RNA techniques, cell-based assays & RNA metabolism
  • Knowledge in human genetic diseases and/or molecular therapeutics development is desirable
  • Experience in antisense-based target validation and mouse handling is a plus

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. Please reference position STOKE-005.