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.

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

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

Bedford, Mass., April 17, 2018 — Stoke Therapeutics, Inc., a biotechnology company developing therapeutics that increase gene expression to treat a wide array of diseases caused by genetic insufficiency, today announced the expansion of its leadership team to enhance its deep expertise in oligonucleotide chemistry and rare disease research and development.

Gene Liau, Ph.D., a veteran biopharma executive who has led research and development programs at Pfizer and Novartis, joins Stoke as executive vice president and head of research and preclinical development. Meena, Ph.D., an oligonucleotide chemist with 20 years of research experience in nucleic acids therapeutics, comes aboard as vice president of bioanalytical, DMPK and biomarker development.

“I am delighted to welcome Gene and Meena to the Stoke team. Their wealth of experience and their passion for bringing novel therapies to patients will accelerate our drive to develop the next generation of antisense oligonucleotide medicines,” said Edward M. Kaye, Stoke’s chief executive officer. “They share our mission of pioneering a new way to treat the underlying causes of severe genetic diseases by precisely up-regulating gene expression.”

Gene joins Stoke after three years as senior vice president and head of gene therapy R&D at Precision BioSciences in Durham, N.C. Before that, he led external R&D efforts in rare disease and hematology at Pfizer and spearheaded the Pfizer gene therapy initiative. Prior to Pfizer, Gene helped Shire build an innovative rare disease portfolio. Gene also spent 12 years at Novartis, eventually leading the cardio-metabolic division at the Novartis Institutes for BioMedical Research in Cambridge, where he was responsible for projects from target discovery to initial clinical proof of concept. Prior to entering the industry, Gene was a professor at George Washington University Medical Center. Gene received his Ph.D. in biochemistry from Vanderbilt University and did his postdoctoral fellowship with Dr. Benoit de Crombrugghe at the National Cancer Institute.

Meena comes to Stoke from Wave Life Sciences in Cambridge, Mass., where she was a founding employee and senior director of bioanalytical, pharmacology and biomarker development.  Meena played a pivotal role in building Wave’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.

About Stoke Therapeutics

Stoke Therapeutics is a biotechnology company working to increase gene expression to treat a wide array of severe genetic diseases, including genetic conditions affecting the central nervous system, eye and liver. Stoke was launched in 2018 with a $40 million Series A investment funded by Apple Tree Partners. For more information visit www.stoketherapeutics.com.

Media Contact

Stephanie Simon
Ten Bridge Communications
stephanie@tenbridgecommunications.com
617-581-9333

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

Stoke Therapeutics Presents Early Research on its Novel ASO Pipeline for Severe Genetic Diseases at OPT Boston

Bedford, Mass., March 26, 2018  — Stoke Therapeutics, Inc., a biotechnology company developing therapeutics that increase gene expression to treat a wide array of diseases caused by genetic insufficiency, will present research on its first-in-class medicines today at the Oligonucleotide & Peptide Therapeutics Boston Conference in Cambridge, Mass.

Charles R. Allerson, Ph.D., vice president of chemistry at Stoke Therapeutics, will present preclinical data on the company’s pipeline of novel antisense oligonucleotide (ASO) therapeutics, which are designed to upregulate gene expression and treat an array of severe genetic diseases. As an example, Stoke has been able to increase productive Scn1a mRNA and resulting Nav1.1 protein levels in mouse models without affecting closely related ion channels. Inherited deficiency of Scn1a gene dosage is known to cause Dravet Syndrome, a severe form of epilepsy. By directly and specifically increasing Scn1a gene expression, Stoke’s program represents a potential first-in-class disease-modifying strategy to treat Dravet Syndrome.

“We’re excited to share our early data as we work to develop a pipeline of innovative ASO therapeutics,” Allerson said. “Many human genetic diseases are due to loss or reduction of function of a single gene, yet there are no drugs on the market to address the underlying genetic causes of most of these conditions. At Stoke, we’re developing strategies to treat these monogenic diseases by directly increasing protein expression — without relying on viral or nanoparticle vectors.”

Allerson’s presentation, called “Targeted Augmentation of Nuclear Gene Output,” will be held today at 4:45 p.m. For the full conference agenda, please see http://optcongress.com/Oligonucleotide-Discovery/

About Stoke Therapeutics

Stoke Therapeutics is a biotechnology company working to increase gene expression to treat a wide array of severe genetic diseases, including genetic conditions affecting the central nervous system, eye and liver. Stoke was launched in 2018 with a $40 million Series A investment funded by Apple Tree Partners. For more information visit www.stoketherapeutics.com.

Media Contact

Stephanie Simon
Ten Bridge Communications
stephanie@tenbridgecommunications.com
617-581-9333

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

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

Edward M. Kaye, M.D., industry leader in rare disease drug development, named Chief Executive Officer

Company leveraging groundbreaking work on RNA splicing from Scientific Founder, Cold Spring Harbor Laboratory Professor Adrian Krainer, Ph.D.

Bedford, Mass., January 4, 2018 — Stoke Therapeutics, Inc, a new company working to increase gene expression to treat a wide array of diseases caused by genetic insufficiency, today announced that it has completed a $40 million Series A financing to support the further advancement of multiple pre-clinical development programs.  The Series A round was provided by founding investor Apple Tree Partners.

Edward M. Kaye, M.D., an industry leader in the development of gene-based medicines for rare diseases and most recently the Chief Executive Officer of Sarepta Therapeutics, has joined Stoke as Chief Executive Officer. He leads a deeply experienced management team with a track record of successfully translating novel biology into groundbreaking new therapies for patients.

Stoke is pioneering a unique therapeutic approach using antisense oligonucleotides to increase the expression of proteins whose function is reduced in genetic diseases. The company is focused specifically on modulating RNA splicing, a critical step in gene expression, to increase the production of messenger RNA that can be translated into protein.

“Stoke Therapeutics represents a bold step forward in opening up a vast new area of drug development focused on up-regulation of gene expression,” said Edward M. Kaye, Stoke Therapeutics Chief Executive Officer. “By restoring gene dosage using target-specific antisense approaches, we have the opportunity to create a new way of treating diseases that are not amenable to enzyme replacement, gene therapy or other existing modalities.”

The technological foundation of Stoke’s approach was developed in close collaboration with Scientific Founder Adrian Krainer, Ph.D., of Cold Spring Harbor Laboratory, an expert in RNA splicing and an inventor of the recently approved antisense therapy SPINRAZA® (nusinersen), a life-saving drug for children with Spinal Muscular Atrophy (SMA).

Since its inception, Stoke has validated hundreds of disease target genes that could be up-regulated with its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform and is prioritizing therapeutic programs that target antisense-addressable tissues including the central nervous system, eye, and liver. While Stoke’s initial focus is on inherited diseases caused by the reduced function of a single mutated gene, the TANGO approach is also applicable to diseases whereby targeted augmentation of a non-mutated gene could reverse or prevent disease progression.

“We are tremendously impressed by the broad potential of this approach to address so many debilitating diseases and have assembled the team, platform and funding to thoroughly exploit this opportunity,” said Sam Hall, Ph.D., Principal of Apple Tree Partners and member of the Stoke Board of Directors.

Stoke Therapeutics is led by a team with strong scientific and clinical experience in the identification and development of novel therapies for patients. In addition to Dr. Kaye, the company’s leadership includes:

  • Huw M. Nash, Ph.D., is Chief Operating Officer and Chief Business Officer and was Founding CEO of Stoke; an Entrepreneur-in-Residence at Apple Tree Partners with 20 years’ experience establishing, funding and growing start-up biotechnology companies.
  • Barry Ticho, M.D., Ph.D., FACC, is Chief Medical Officer; he previously held senior positions at Moderna Therapeutics, Pfizer and
  • Isabel Aznarez, Ph.D., is co-founder and VP, Head of Biology; she was previously a member of the Krainer lab and has extensive experience in human genetics, RNA metabolism, and modulation of RNA processes using antisense oligonucleotides.
  • Charles R. Allerson, Ph.D., is VP, Head of Chemistry; he has extensive expertise in oligonucleotide therapeutics and previously held senior positions at Regulus Therapeutics and Ionis Pharmaceuticals.

In addition to Dr. Kaye, the company’s Board of Directors includes:

  • Adrian R. Krainer, Ph.D., is co-founder of Stoke and is the St Giles Professor of Molecular Genetics and Program Chair of Cancer & Molecular Biology at Cold Spring Harbor Laboratory.
  • Arthur A. Levin, Ph.D., is currently Executive Vice President, R&D at Avidity Biosciences; previously he held that position at miRagen Therapeutics, and prior to that he held senior drug development roles at Ionis Pharmaceuticals and Santaris Pharma.
  • Seth L. Harrison, M.D., is the founder and Managing Partner of Apple Tree Partners and is Chairman of the Board; he is currently Chairman of Braeburn Pharmaceuticals, Elstar Therapeutics, Limelight Bio, and Syntimmune, and is a Director of Corvidia Therapeutics.
  • Sam Hall, Ph.D., is a Principal at Apple Tree Partners; he was previously a member of the investment team at Symphony Capital and prior to that a member of the healthcare investment banking team at Citigroup.

About Stoke Therapeutics

Stoke Therapeutics is a biotechnology company working to increase gene expression to treat a wide array of severe genetic diseases, including genetic conditions affecting the central nervous system, eye, and liver. Stoke was launched in 2018 with a $40 million Series A investment funded by Apple Tree Partners. For more information visit www.stoketherapeutics.com.

About Apple Tree Partners

Apple Tree Partners (ATP) is a New York-based venture capital firm dedicated to building transformative life sciences businesses. The firm is actively investing its fourth fund, with $1.5 billion in capital commitments. ATP considers therapeutics and medical device investments at all stages, from discovery research through to commercialization and takes a long-term view to create sustainable value. For more information visit www.appletreepartners.com.

About Cold Spring Harbor Laboratory

Founded in 1890, Cold Spring Harbor Laboratory has shaped contemporary biomedical research and education with programs in cancer, neuroscience, plant biology and quantitative biology. Home to eight Nobel Prize winners, the private, not-for-profit Laboratory employs 1,100 people including 600 scientists, students and technicians. For more information, visit www.cshl.edu.

Media Contact

Dan Quinn, Ten Bridge Communications
Dan@Tenbridgecommunications.com
781-475-7974

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

Senior Research Associate/Scientist, Bioanalytical
STOKE-008
Senior Research Associate/Scientist, Bioanalytical
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 bioanalytical scientist to join the bioanalytical and biomarker group. The successful candidate will be responsible for:

  • Characterization of oligonucleotides and development of LC-MS and HELISA based bioanalytical methods, sample testing as well as data interpretation and reporting to support research and non-clinical studies.
  • Transfer methods to CROs and manage their work.
  • Support initiatives including immunohistochemistry and biomarkers methods (LC-MS/MS, ELISA, MSD etc.) development.
  • Help maintain the bioanalytical lab and keep the instruments running properly.
  • Help manage internal compound inventory.

Qualifications & Experience:

  • B.S/M.S. with 3-5 years or Ph.D. with 0-3 years of academic or industry experience in relevant field
  • In depth knowledge of bioanalytical chemistry and techniques including HPLC, mass spectrometry (MS/MS) and ligand binding assays.
  • Strong hands-on LC-MS instrumentation skills and the ability to independently develop, implement, and execute bioanalytical LC-MS based assays.
  • Excellent problem-solving skills and ability to work as a team player in a dynamic environment.
  • Drug development experience particularly in nucleic acid therapeutics is highly desirable.
  • Prior experience in quantitation of oligonucleotides in biological matrices 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-008.

Research Associate/Sr. Research Associate, Biology
STOKE-007
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 a dynamic and flexible laboratory-based technician to support our lead programs. The ideal candidate will have a strong in vivo background and will have experience in cell biology and molecular biology techniques.  Duties will include performing in vivo studies and processing tissues for RNA and protein-based assays.  This role may also involve doing tissue culture and performing cell-based assays.  Qualified candidates will be expected to analyze and report data as well as effectivity communicate results at meetings. Careful record keeping, laboratory organization and strong communication skills are additional key attributes. Previous experience in neuroscience is highly desirable.

Qualifications & Experience:

  • Master’s or Bachelor’s degree in bio-medical related field is required
  • Mouse handling, test article administration, surgery and tissue dissection skills are required
  • Excellence in record keeping, laboratory organization and communication skills is required
  • Flexibility and willingness to learn new techniques is required
  • Experience in neuroscience or RNA research is highly desired
  • Experience performing RNA extraction, protein extraction, RT-PCR, qPCR, Western blotting, and/or immunohistochemistry from in vivo samples is highly desired
  • Experience in cell culture 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-007.

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 several research associates with a strong record of success in molecular biology research.  Candidates must hold a Bachelor’s or Master’s degree in molecular or cell biology, a record of peer-reviewed publications, and at least four years’ post-graduate experience, ideally in industry.  Experience in RNA techniques, RNA metabolism, and cell-based assays is required.  Research experience in human genetic diseases and/or molecular therapeutics development, and mouse handling is also highly desirable.  The successful candidate will join Stoke’s scientific team and will be directly responsible for target validation and hit identification and optimization activities at Stoke.

Qualifications & Experience:

  • Bachelor’s or Master’s degree in molecular or cell biology
  • At least 4 years’ post-graduate experience, ideally in industry
  • Expertise in RNA techniques, cell-based assays & RNA metabolism
  • Expertise in human genetic diseases and/or molecular therapeutics development, and mouse handling is ideal but not essential
  • Expertise in antisense-based target validation 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.

Scientists/Senior Scientists, Biology
STOKE-003
Scientists/Senior Scientists, 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 several scientists/senior scientists with a strong record of success in pre-clinical research.  Candidates must hold a Ph.D. degree in molecular or cell biology, a record of peer-reviewed publications, and at least two years’ post-graduate experience in neurologic, metabolic or ocular disease.  Research experience in RNA metabolism, in vivo animal models, human genetic diseases and/or molecular therapeutics development is highly desirable.  The successful candidate will join Stoke’s scientific team and will be directly responsible for target validation, hit & lead identification and optimization activities at Stoke.

Qualifications & Experience:

  • Ph.D. degree in molecular or cell biology
  • At least 2 years’ post-graduate experience, ideally in industry
  • Expertise in RNA techniques, cell-based assays, and in vivo animal models
  • Expertise in RNA metabolism, human genetic diseases and/or molecular therapeutics development is highly desirable
  • Expertise in antisense-based therapeutics or target validation 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-003.

Principal Scientist / Director Biology
STOKE-001
Principal Scientist / Director 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 one or more Principal Scientists / Directors with a strong record of success in pre-clinical and early clinical translational research focused on neuroscience or ophthalmology. The scientist should possess deep academic training and at least five years’ industrial experience in neurological or ocular diseases. The successful candidate will take a key role in identifying & prioritizing Stoke’s neuroscience or ophthalmology programs, will be responsible for the generation and application of animal models to achieve in vivo proof-of-mechanism & efficacy, and will serve as a project lead for Stoke’s growing pipeline of drug candidates through to early clinical proof-of-concept.

Qualifications & Experience:

  • PhD or MD/PhD with specialization in neurological or ocular diseases
  • At least 5 years’ experience in pharma/biopharma directly responsible for pre-clinical and early clinical translational research (lead ID to clinical POC)
  • Expertise in drug target identification, validation & prioritization
  • Expertise in oligonucleotide or nucleic acid-based therapeutic drug development is ideal but not essential
  • Expertise in pharmacology & safety/toxicology 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-001.