Who We Are
Who We Are 2017-11-02T10:56:03+00:00

Our mission is to develop and commercialize innovative gene therapy products to dramatically and positively transform the lives of patients living with serious, life-threatening rare diseases with limited or no treatment options.

We currently have four programs in development

A world-class management team with over 100 years of combined experience in gene therapy, rare disease drug development and commercialization, and biologics manufacturing.

Matthew R. Patterson
Matthew R. PattersonPresident and Chief Executive Officer
Mr. Patterson is the co-founder of Audentes Therapeutics and has served as president and CEO since the Company’s inception in November 2012. He has more than 20 years of experience in the research, development, and commercialization of innovative treatments for rare diseases and has held positions of senior management in both private and public biotechnology companies. Previously Mr. Patterson worked for Genzyme Corporation, BioMarin Pharmaceutical, and Amicus Therapeutics. Prior to Audentes he was an Entrepreneur-In-Residence with OrbiMed, the world’s largest health-care dedicated investment firm.

Mr. Patterson is a member of the Board of Directors of Gilda’s Club of New York City, which provides social and emotional support for people living with cancer. He also currently serves as Vice Chairman of the Alliance for Regenerative Medicine (ARM). Mr. Patterson received his B.A. in Biochemistry from Bowdoin College.

Natalie Holles
Natalie HollesSenior Vice President, Chief Operating Officer
Ms. Holles has served as Senior Vice President and Chief Operating Officer since August 2015. Ms. Holles brings to this role over fifteen years of corporate development, strategic planning, and commercial experience gained in a range of therapeutic areas, with a focus in orphan diseases. Most recently, Ms. Holles served as Senior Vice President, Corporate Development at Hyperion Therapeutics, Inc. from 2013 through its acquisition by Horizon Pharma, plc in May 2015. From 2010-2013, Ms. Holles provided executive-level strategy and business development advisory services to a number of privately-held biopharmaceutical companies, most recently acting as Executive Vice President, Corporate Development at Immune Design, Inc. Earlier in her career, Ms. Holles served as Vice President, Business Development at KAI Pharmaceuticals, Inc. (acquired by Amgen in 2012) and previously held corporate development and commercial roles at InterMune, Inc. (acquired by Roche in 2014) and Genentech, Inc.

Ms. Holles holds an M.A. in molecular, cellular and developmental biology from the University of Colorado, Boulder, where she was a Howard Hughes Medical Institute Predoctoral Fellow, and an A.B. in Human Biology from Stanford University.

Tom Soloway
Tom SolowaySenior Vice President, Chief Financial Officer
Mr. Soloway has served as Senior Vice President, Chief Financial Officer since October 2015, bringing over 20 years of experience in operations, corporate finance and venture capital in the life sciences industry. Prior to joining Audentes, Mr. Soloway served as the Senior Vice President, Chief Financial Officer of Ascendis Pharma A/S, a Danish biopharmaceutical company, where he helped lead a successful initial public offering on NASDAQ. Prior to Ascendis, Mr. Soloway co-founded Transcept Pharmaceuticals, Inc. At Transcept, Mr. Soloway held positions of increasing responsibility, serving initially as Senior Vice President, Operations and Chief Financial Officer and over time as Executive Vice President, Chief Operating Officer. In this role he oversaw project planning, manufacturing, pharmaceutical sciences, legal, human resources, regulatory and corporate communications. Prior to joining Transcept, Mr. Soloway financed and advised early stage healthcare and life sciences companies as a Principal at Montreux Equity Partners, a venture capital firm.

Mr. Soloway earned a B.S. in Entrepreneurial Studies from the University of Southern California and an M.B.A. from Georgetown University.

Suyash Prasad, M.D.
Suyash Prasad, M.D.Senior Vice President, Chief Medical Officer

Dr. Prasad has served as Senior Vice President and Chief Medical Officer since February 2014. He has a wide range of experience and achievement in international drug development across Phase I to IV, with a specific focus in the clinical development of therapies to treat rare pediatric disorders. Dr. Prasad has worked in drug development for the past 14 years in positions of increasing responsibility at BioMarin Pharmaceutical, Inc., Genzyme Corporation, and Eli Lilly and Company. He has broad therapeutic expertise in neuromuscular disease, metabolic medicine, bone, neuroscience, endocrinology, immunology, and genetics. Dr. Prasad has significant experience with the development and commercialization of enzyme replacement therapies to treat lysosomal storage disorders, including Cerezyme®, Aldurazyme®, Fabrazyme®, and Myozyme®. For Pompe Disease, he led the global medical planning activities for Lumizyme® for the treatment of adult Pompe Disease and for Myozyme® to treat infantile Pompe. Most recently, he was responsible for the clinical development of novel treatments for phenylketonuria (PKU) and achondroplasia.

Dr. Prasad graduated in Medicine at the University of Newcastle-upon-Tyne, UK, where he received commendations for Pediatrics, Obstetrics and Gynecology, and Medical Ethics. He is a United Kingdom board certified physician with a sub-specialty interest in Pediatric Critical Care, and is a member of the Royal College of Physicians (MRCP) and the Royal College of Pediatrics and Child Health (MRCPCH). He received his Diploma in Pharmaceutical Medicine from the Royal College of Physicians of the United Kingdom, and has recently become an elected Fellow to the Faculty of Pharmaceutical Medicine (FFPM) and is a past recipient of the Outstanding Contribution Award from the Faculty of Pharmaceutical Medicine of the UK Royal College of Physicians. Dr. Prasad also currently sits on the board of the National PKU Association.

John Gray, Ph.D.
John Gray, Ph.D. Senior Vice President, Chief Scientific Officer
John Gray has served as Senior Vice President, Chief Scientific Officer since May 2017 and prior to that was Senior Vice President, Research and Development. Dr. Gray has over 20 years of experience designing genetic therapies and vaccines, and developing manufacturing processes for those products. For 11 years prior to joining Audentes, Dr. Gray was the Director of Vector Production and Development at St. Jude Children’s Research Hospital where he led a team devoted to advancing the gene therapy vector science. In the area of lentiviral vector production, his team derived the GPRG stable cell line, the first such line to be used to successfully produce an HIV-based vector tested in a human clinical trial (for treatment of X-linked Severe Combined Immunodeficiency). He also contributed significantly to the Hemophilia B gene therapy project, for which he designed the self-complementary AAV Factor IX vector expression cassette and developed the production process used to manufacture the first two batches of clinical vector. During his tenure at St. Jude, Dr. Gray also worked on Chimeric Antigen Receptor modified cell therapy, lysosomal storage disorder gene therapy, and multiple hematopoietic stem/progenitor cell gene therapy projects.

Prior to joining St. Jude Children’s Research Hospital in 2003, Dr. Gray was a researcher in the laboratory of Dr. Richard Mulligan, where for 5 years he served as the Assistant Director of the Harvard Gene Therapy Initiative. Prior to this he worked for 5 years at Pfizer Animal Health designing bacterial and viral vectors for vaccine applications. Dr. Gray has a B.A. degree in Biochemistry from the University of California, Berkeley, and a Ph.D. degree in Biochemistry from the University of Colorado, Boulder.

Mary Newman
Mary NewmanSenior Vice President, Regulatory Affairs

Ms. Newman joins Audentes with over 20 years of experience in regulatory affairs, and research and development, within the biotechnology industry. She joined Audentes after serving 7 years at SARcode Biotherapeutics Inc. (acquired by Shire Ltd.), most recently as Senior Vice President, Regulatory Affairs and Quality Assurance. Ms. Newman’s responsibilities included oversight of global regulatory strategic development, all primary regulatory agency interactions, and quality assurance for SARcode products. She previously held various management positions, with increasing responsibilities, in Regulatory Affairs at BioMarin Pharmaceutical, Inc., Berlex Inc. (now Bayer HealthCare Pharmaceuticals Inc.), and Sequus Pharmaceuticals, Inc. (now Johnson and Johnson). While at BioMarin, Ms. Newman oversaw the development and approval of Kuvan (sapropterin HCl) for the treatment of phenylketonuria (PKU), Naglazyme (galsulfase) for mucopolysaccharidosis (MPS) VI, and supported the final approval of Aldurazyme (laronidase) for MPS I. She has also held various leadership roles in research and development in oncology, neurology, and antifungal therapeutic areas.

Ms. Newman holds a B.S. in physiology from Oregon State University.

David Nagler
David NaglerSenior Vice President, Human Resources & Corporate Affairs

Mr. Nagler has served as Senior Vice President, Human Resources and Corporate Affairs since February 2015. He has been in the life sciences industry for over 25 years, first as a leader in government affairs and later serving as a senior executive in human resources, along with corporate communications, legal affairs, and operations. Mr. Nagler joined Audentes having served the company as a human resources consultant. Prior to his consulting practice, Mr. Nagler spent nearly 10 years as Vice President Corporate Affairs and Secretary at ARYx Therapeutics, Inc. There he was responsible for human resources, public company compliance, legal, information technology, investor relations, and corporate communications. Prior to ARYx, Mr. Nagler served as Vice President Human Resources at Genentech, Inc., where he was responsible for a department with an annual $20 million budget that served an employee population of 7,600. During his tenure at Genentech, Mr. Nagler also served as Senior Director Government Affairs. Prior to joining Genentech, Mr. Nagler co-founded JLA Associates in Sacramento, a legislative consulting and association management company that he later sold to Nossaman, Guthner, Knox and Elliott, where he spent 10 years as a legislative advocate.

Mr. Nagler currently sits on the Planning Commission in the City of Pleasanton (California), having previously served as Chair of the Pleasanton Human Services Commission. He is a past member of the board of directors of U.C. Davis CONNECT, as well as the boards of the Northern California Chapter of the American Liver Foundation and the John Vasconcellos Legacy Project.

Mr. Nagler studied public policy and philosophy at the University of California Berkeley.

Fulvio Mavilio, Ph.D.
Fulvio Mavilio, Ph.D.Vice President Scientific Affairs, Europe

Dr. Mavilio has served as our Vice President Scientific Affairs, Europe since July 2017. Prior to joining Audentes, he served as Scientific Director of Genethon, Evry, France (2012-2017) and prior to that he was co-Director of the Center for Regenerative Medicine of the University of Modena, Italy (2006-2011), Director of Discovery of Molmed SpA (2002-2005), founder and Chief Scientific Officer of Genera SpA (1999-2002), and co-Director of the San Raffaele-Telethon Institute of Gene Therapy in Milan, Italy (1995-2002). Dr. Mavilio is a member of the European Molecular Biology Association (EMBO), Member of the Board of the American Society of Gene and Cell Therapy (ASGCT), and a member of the Editorial Board of many international journals in the fields of genetics, molecular biology and gene therapy. He graduated in Biology at the University of Rome in 1976, obtained a Ph.D. in Medical Genetics at the School of Medicine of the same University in 1979, and trained as a visiting scientist at the Wistar Institute in Philadelphia, PA from 1985 to 1989. An expert and a pioneer in the fields of gene therapy and stem cell research, Dr. Mavilio has published over 170 articles in major international journals. He also serves as Professor of Molecular Biology at the University of Modena and Reggio Emilia (Modena, Italy).

Matthew R. PattersonPresident and Chief Executive Officer
Mr. Patterson is the co-founder of Audentes Therapeutics and has served as president and CEO since the Company’s inception in November 2012. He has more than 20 years of experience in the research, development, and commercialization of innovative treatments for rare diseases and has held positions of senior management in both private and public biotechnology companies.
Previously Mr. Patterson worked for Genzyme Corporation, BioMarin Pharmaceutical, and Amicus Therapeutics. Prior to Audentes he was an Entrepreneur-In-Residence with OrbiMed, the world’s largest health-care dedicated investment firm.

Mr. Patterson is a member of the Board of Directors of Gilda’s Club of New York City, which provides social and emotional support for people living with cancer. He also currently serves as Vice Chairman of the Alliance for Regenerative Medicine (ARM). Mr. Patterson received his B.A. in Biochemistry from Bowdoin College.

Jennifer Jarrett
Ms. Jarrett serves as the Chief Financial and Business Officer of Arcus Biosciences, Inc. She served as Chief Financial Officer of Medivation, Inc. until the company’s purchase by Pfizer, Inc. in 2016. Previously, Ms. Jarrett served as Managing Director at Citigroup, where she was responsible for building and managing Citigroup’s West Coast life sciences investment banking practice.
Prior to Citigroup, Ms. Jarrett was a Managing Director in Credit Suisse’s Health Care Group focused on the biotechnology sector. She earned a bachelor’s degree in economics, Cum Laude, from Dartmouth College and holds a masters of business administration from Stanford Graduate School of Business.
Louis G. Lange, M.D., Ph.D.
Dr. Lange began his career with 22 years of experience in academic medicine at Harvard and Washington University, where he served as Chief of Cardiology and Professor of Medicine at Jewish Hospital from 1985-1992 and was one of the first academicians in molecular cardiology. He then founded CV Therapeutics, and as Chairman, CEO and Chief Scientific Officer, led the company through an IPO in 1996, the approval of two first in class cardiovascular drugs, RANEXA® and LEXISCAN®, and the sale of the company to Gilead in 2009 for $1.4 billion dollars.
Since, 1998, Dr. Lange has served as a member of the Board of Trustees at the University of Rochester and also served as Chair of the Health Affairs committee that oversaw the strategic re-invigoration of the medical center. As a member of the BIO Board of Directors from 1999 to 2009, Dr. Lange led the largest committee of member companies for two years and was picked as one of two biotech executives to attend the ceremonies at the White House for the signing of the Bioterrorism bill in 2004. Dr. Lange has been a General Partner at Asset Management since 2009, remains a senior advisor to Gilead and serves on numerous other public and private Boards in both the non-profit and for-profit arena.

Dr. Lange holds a Ph.D. degree in Biochemistry from Harvard University and an M.D. degree from Harvard Medical School. Dr. Lange has a Bachelor’s degree from the University of Rochester.

Scott Morrison
Mr. Morrison was a Partner with Ernst & Young LLP from 1996 to December 2015, serving as its U.S. Life Sciences Leader from 2002 to December 2015. Mr. Morrison retired in December 2015.
He serves on the boards and chairs the audit committees of Global Blood Therapeutics and Corvus Pharmaceuticals. Mr. Morrison previously served on the boards of directors of the Life Sciences Foundation, the Bay Area Biosciences Association, the Biotechnology Institute and the Emerging Companies Section of the Biotechnology Innovation Organization. Mr. Morrison received a B.S. in business administration from the University of California, Berkeley.
Kush M. Parmar, M.D., Ph.D.
Dr. Parmar, M.D., Ph.D. is a Partner at 5AM Ventures. Previously he was an NIH-sponsored M.D./Ph.D. Physician Scientist Fellow at Harvard Medical School. Dr. Parmar currently serves as Director or Observer on the Boards of Achaogen, Arvinas, Audentes, Cleave, Novira and Pulmatrix.
He previously served as Board Observer for Envoy (acquired by Takeda). He is a Fellow of the Society of Kauffman Fellows. Before joining 5AM, Dr. Parmar completed clinical clerkships at the Massachusetts General & Brigham and Women’s Hospitals and consulted for an oncology startup. Dr. Parmar also founded a non-profit international development organization, the Cruz Blanca Initiative. He holds an A.B. in Molecular Biology and Medieval Studies from Princeton University, a Ph.D. in Experimental Pathology from Harvard University, and an M.D. from Harvard Medical School.
Thomas J. Schuetz, M.D., Ph.D.
Dr. Schuetz, M.D., Ph.D. is the co-founder of Audentes Therapeutics and has served as a member of the board of directors since July 2013. He is currently the Chief Executive Officer of Compass Therapeutics, LLC.
Dr. Schuetz is currently the Chief Executive Officer of Compass Therapeutics, LLC. Previously, Dr. Schuetz was a Venture Partner at OrbiMed. Dr. Schuetz served as the Chief Medical Officer of Therion Biologics Corporation and the Vice President of Clinical Affairs at Transkaryotic Therapies, Inc. (now Shire Pharmaceuticals, Inc.). Dr. Schuetz has also served as the Chief Medical Resident at Massachusetts General Hospital and completed a medical oncology fellowship at the Dana-Farber Cancer Institute. Dr. Schuetz also serves on the board of directors of Compass Therapeutics, Inc. and Glypharma, Inc. Dr. Schuetz holds a B.S. in chemistry from Xavier University, an M.D. from Harvard Medical School and a Ph.D. in genetics from Harvard University. Dr. Schuetz is Board Certified in Medical Oncology.
Julie Anne Smith
Ms. Smith is a Director and the Chief Executive Officer of Nuredis, Inc., a biotech company developing treatments that target the cause of neurological and neuromuscular nucleotide repeat diseases. Prior to her current role, Ms. Smith served as President and Chief Executive Officer of Raptor Pharmaceuticals, Inc. from January 2015 until the company’s acquisition by Horizon Pharma plc in October 2016.
Previously, Ms. Smith served as Raptor’s Executive Vice President and Chief Operating Officer after joining the company in September 2012 and serving as President and Chief Executive Officer Designee from July 2014 to December 2014. From 2008 to 2012, Ms. Smith served as Chief Commercial Officer of Enobia Pharma Corporation, prior to the company’s acquisition by Alexion. Additionally, Ms. Smith served in positions of increasing responsibility at Jazz Pharmaceuticals plc (2006-2008), Genzyme, Inc. (2001-2006) and Novazyme Pharmaceuticals, Inc. (2000-2001). Ms. Smith began her industry career at Bristol-Myers Squibb, first as a Product Manager and later as Director of channel strategy and design. Ms. Smith serves as an Independent Director at Exelixis, Inc. and previously as Director on the Health and Emerging Companies Sections of the Biotechnology Industry Organization (BIO) board. She received her Bachelor of Science in biological and nutritional sciences from Cornell University.
Stephen Squinto, Ph.D.
Dr. Squinto, Ph.D. has over 25 years of experience in the biotechnology industry, as both a scientist and senior executive. He is currently a Venture Partner with OrbiMed. Dr. Squinto was a co-founder of Alexion Pharmaceuticals Inc. and recently served as its Executive Vice President and Chief Global Operations Officer.
Prior to 2013, he was Alexion’s Global Head of Research and Development. Alexion is dedicated to developing therapies for certain rare and ultra-rare diseases. From 1988 to 1992, Dr. Squinto held various positions at Regeneron Pharmaceuticals, Inc. Prior to Regeneron, he held a joint academic position at both the Tulane University and LSU Medical Schools. He is a recipient of numerous honors and awards from academic and professional organizations for his scientific work.

Dr. Squinto received his B.A. in Chemistry and Ph.D. in Biochemistry and Biophysics from Loyola University of Chicago.

Alan H. Beggs, Ph.D.
Alan H. Beggs, Ph.D.

Director of The Manton Center for Orphan Disease Research,
Boston Children’s Hospital

Sir Edwin and Lady Manton Professor of Pediatrics,
Harvard Medical School

Dr. Alan Beggs is Director of The Manton Center for Orphan Disease Research at Boston Children’s Hospital and the Sir Edward and Lady Manton Professor of Pediatrics at Harvard Medical School. His current research is focused on utilizing genetic approaches in human patients and animal models to understand the pathophysiology and to develop targeted therapies to treat these devastating disorders of childhood.

Dr. Beggs received his AB in biology at Cornell University and his Ph.D. in human genetics at Johns Hopkins University. He completed postdoctoral fellowships in medical genetics at Johns Hopkins University and in clinical molecular genetics at Harvard Medical School, and has directed an independent research laboratory in the Genetics Division at Boston Children’s Hospital since 1992. Dr. Beggs has led studies to identify the genetic basis for a wide variety of rare conditions with a particular focus on skeletal muscle and its defects in congenital myopathies and related neuromuscular diseases. Over the past two decades, he has published over 150 articles on neuromuscular and other genetic diseases and his laboratory is at the forefront of research to understand the pathophysiology of X-linked myotubular myopathy (XLMTM), and to develop therapies for this disease.

Deborah Bilder, M.D.
Deborah Bilder, M.D.

Associate Professor in the Department of Psychiatry, Division of Child and Adolescent Psychiatry, University of Utah

Dr. Deborah Bilder is Medical Director of the University of Utah’s Autism Spectrum Disorder Clinic and the Neurobehavior HOME Program. The Neurobehavior HOME Program is a novel Medicaid medical home setting that provides primary and behavioral health care to over 1000 children and adults with neurodevelopmental disabilities. She is also on the faculty of the Utah Leadership Education in Neurodevelopmental Disabilities (ULEND) training program.

Dr. Bilder earned her medical degree from Vanderbilt University School of Medicine in Nashville, Tennessee and completed the triple board residency program (pediatrics, general psychiatry, child and adolescent psychiatry) at the University of Utah. Following residency, she participated in the Utah Leadership Education in Neurodevelopmental Disabilities (ULEND) program, of which she is now on the faculty. As a clinical investigator, Dr. Bilder leads a research team that investigates autism spectrum disorder (ASD) etiology and epidemiology. Her other areas of clinical and research expertise are ASD adult outcomes and psychiatric comorbidity associated with phenylketonuria. Her work on excess mortality in ASD was listed by the US Department of Health and Human Services’ Interagency Autism Coordinating Committee as among the most significant autism research advances in 2012. She currently serves on a Phase 3 Clinical Trials Steering Committee and works with the pharmaceutical industry to design clinical trials that capture neuropsychiatric outcomes of treatment for genetic disorders. A key component of Dr. Bilder’s role on Audentes’ Board of Scientific and Clinical Advisors is to ensure that the emotional and psychological well being of patients and families, across Audentes’ pipeline, is appropriately represented and considered.

Alan Boyd, B.Sc., M.B., Ch.B., F.F.P.M.
Alan Boyd, B.Sc., M.B., Ch.B., F.F.P.M.

CEO and Managing Director at Boyds

Fellow and President of the Faculty of Pharmaceutical Medicine at the Royal College of Physicians

Chair of the Specialist Advisory Committee in Pharmaceutical Medicine at The Royal College of Physicians

Honorary Professor in the College of Medical and Dental Sciences at the University of Birmingham Medical School

Professor Alan Boyd is a Fellow and President of the Faculty of Pharmaceutical Medicine at the Royal College of Physicians, Chair of the Specialist Advisory Committee in Pharmaceutical Medicine at The Royal College of Physicians, Honorary Professor in the College of Medical and Dental Sciences at the University of Birmingham Medical School, and the Founder, CEO and Managing Director at Boyds consulting firm. The focus of Boyds is to aid and support early stage life science-based companies. His clients now include many companies in Europe, North America and Japan who are devoted to the development of cell and gene based medicines.

Dr. Boyd studied biochemistry and medicine at the University of Birmingham, UK. His 30 years pharmaceutical career began at Glaxo Group Research Ltd. From 1988 he led the cardiovascular medical research team at ICI Pharma, Canada, and later assumed a role as their Director of Clinical and Medical Affairs. In 1999, after four years as Head of Medical Research for Zeneca Pharmaceuticals, Dr. Boyd became Director of Research and Development for Ark Therapeutics Ltd in the UK, where he was responsible for delivering the majority of key development milestones that have shaped the company. In particular, he led the development of their gene based medicines portfolio.

Barry J. Byrne, M.D., Ph.D.
Barry J. Byrne, M.D., Ph.D.

Director of the University of Florida Powell Center for Rare Disease Research

Earl and Christy Powell University Chair in Genetics and Associate Chair of Pediatrics, University of Florida

Member of the American Society of Gene and Cell Therapy (ASGCT) Board of Directors

Founding Editor of Human Gene Therapy, Clinical Development

Member of the Editorial Board for Plos ONE

Dr. Barry Byrne is the Director of the University of Florida Powell Center for Rare Disease Research at the University of Florida and Associate Chair of Pediatrics. He joined the University of Florida in 1997 and is now the Earl and Christy Powell University Chair in Genetics. Dr. Byrne was recently elected to the American Society of Gene & Cell Therapy (ASGCT) Board of Directors. He is a member of the Editorial Board of Plos ONE and the founding editor of Human Gene Therapy, Clinical Development.

Dr. Byrne obtained his B.S. degree from Denison University, his M.D. and Ph.D. from the University of Illinois and completed his pediatrics residency, cardiology fellowship training and post-doctoral training in biological chemistry at the Johns Hopkins Hospital. As a clinician scientist, he is currently studying a variety of rare diseases with specific attention to developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness and problems in heart and respiratory function. His group has made significant contributions to the understanding and treatment of Pompe disease, a type of muscular dystrophy due to abnormal glycogen storage in muscle. His research team has been developing new therapies using the missing cellular protein or the corrective gene to restore muscle function in Pompe and other inherited myopathies.

Martin K. (Casey) Childers, DO, Ph.D.
Martin K. (Casey) Childers, DO, Ph.D.

Professor in the Department of Rehabilitation Medicine and Investigator in the Institute for Stem Cell & Regenerative Medicine, University of Washington

Dr. Martin (Casey) Childers is Professor in the Department of Rehabilitation Medicine and an Investigator at the Institute for Stem Cell and Regenerative Medicine at the University of Washington.

Dr. Childers received his B.A. from Seattle Pacific University, his D.O. from Western University, and his Ph.D. in physiology and pharmacology from the University of Missouri. Professor Childers’ laboratory is currently pursuing two areas of investigation: Systemic gene replacement delivery for patients with X-linked myotubular myopathy (XLMTM) and induced pluripotent stem (iPS) cell technology to study heart disease in patients with Duchenne muscular dystrophy (DMD). Dr. Childers’ rehabilitation medicine practice at the University of Washington Medical Center (UWMC) is dedicated to serve patients with neuromuscular diseases. The UWMC neuromuscular outpatient clinic is co-sponsored by the Muscular Dystrophy Association (MDA) and staffed by medical specialists focused on neuromuscular medicine.

David Dimmock, M.D.
David Dimmock, M.D.

Associate Professor of Pediatrics, Human and Molecular Genetics Center, Medical College of Wisconsin

Dr. David Dimmock is Associate Professor of Pediatrics at the Human and Molecular Genetics Center at the Medical College of Wisconsin. He is board certified by the ABP in Pediatrics and by the ACMG in Clinical Metabolic Genetics and Clinical Genetics. Dr. Dimmock provides consult to the State of Wisconsin Newborn Screening Umbrella Committee and Metabolic Subcommittee, and has been acting Chair of the Metabolic Subcommittee since 2010.

Dr. Dimmock graduated from St George’s Hospital Medical School (University of London) in 1998, then undertook internships in general adult medical training (Internal Medicine and Surgery) before training in pediatrics in the UK. With a focus on adult and pediatric metabolic diseases, he began a pediatric residency program in Phoenix, Arizona. He then undertook Genetics and Clinical Metabolic Genetics fellowship training at Baylor College of Medicine in Houston, Texas. As a clinical researcher, Dr. Dimmock has developed an international reputation for bringing innovative diagnostic testing strategies into the clinic. He is passionate about improving the standard of care for children and adults with inborn errors of metabolism. His research laboratory focuses on improving the diagnosis of mitochondrial and single gene disorders that affect the liver. He has published scientific articles on improved diagnostic testing for these disorders as well as small molecule and gene therapy approaches. Dr. Dimmock has been an invited advisor to the FDA and the Institute of Medicine, and has been involved in more than 15 trials for novel therapeutics in rare disorders, serving as the site primary investigator for 13 trials. He is excited at the opportunities that gene therapy through Audentes can afford his patients with rare disorders.

Kevin M. Flanigan, M.D.
Kevin M. Flanigan, M.D.

Professor of Pediatrics and Neurology, Ohio State University

Co-Director, Nationwide Children’s Muscular Dystrophy Association Clinic

Investigator, Research Institute of Nationwide Children’s Hospital Center for Gene Therapy

Dr. Kevin Flanigan is an investigator at the Center for Gene Therapy at the Research Institute of Nationwide Children’s Hospital, Co-Director of the Nationwide Children’s Muscular Dystrophy Association Clinic, and a Professor of Pediatrics and Neurology at the Ohio State University in Columbus, Ohio. His current research is directed toward the molecular characterization and therapy of neuromuscular diseases, and the identification of genetic modifiers of disease.

Dr. Flanigan completed residency training in Neurology and fellowship training in Neuromuscular Disease at the Johns Hopkins Hospital in Baltimore. He then completed a postdoctoral research fellowship in the Human Molecular Biology and Genetics program at the University of Utah, prior to joining their faculty in 1997. He has been a member of the Executive Board of the World Muscle Society since 2001, and is a member of the Executive Committee of TREAT-NMD, the international alliance directed toward establishing the infrastructure to ensure that promising new therapies reach patients as quickly as possible. He has conducted multiple clinical trials in Duchenne muscular dystrophy, including trials of gene modifying therapies such as nonsense suppression and exon skipping, and holds INDs for gene transfer trials of vectors directed toward Duchenne muscular dystrophy and mucopolysaccharidosis type 3A.

Mike Lawlor, M.D., Ph.D.
Mike Lawlor, M.D., Ph.D.

Director of the Pediatric Pathology Clinical Neuromuscular Laboratory and Congenital Muscle Disease Tissue Repository, Medical College of Wisconsin

Dr. Michael Lawlor is Director of the Pediatric Pathology Clinical Neuromuscular Laboratory at the Medical College of Wisconsin. He is also Director of the Congenital Muscle Disease Tissue Repository, which processes and diagnoses muscle and nerve biopsy specimens for Children’s Hospital of Wisconsin.

Dr. Lawlor completed his clinical training in pathology/neuropathology at Massachusetts General Hospital and postdoctoral research training in the laboratory of Dr. Alan Beggs at Boston Children’s Hospital. His postdoctoral research work focused on disease pathogenesis and experimental treatments for X-linked myotubular myopathy (XLMTM) and nemaline myopathy. This work has continued and expanded at the Medical College of Wisconsin, including on-site treatment trials of animal models as well as pathological analyses for preclinical trial studies (including anti-myostatin therapy, gene therapy, and protein replacement therapy) currently being performed worldwide. Additional research efforts in the Lawlor laboratory are focused on nemaline myopathy, congenital contractures, and mitochondrial myopathy. Dr. Lawlor is currently working with Audentes Therapeutics to plan muscle biopsy and pathology procedures for a human gene therapy clinical trial for XLMTM based on one of these projects.

Suyash Prasad, M.D.
Suyash Prasad, M.D.

Senior Vice President and Chief Medical Officer, Audentes Therapeutics

Member of the Royal College of Physicians (MRCP)

Member of the Royal College of Pediatrics and Child Health (MRCPCH)

Fellow of the Faculty of Pharmaceutical Medicine (FFPM)

Dr. Suyash Prasad has served as Senior Vice President and Chief Medical Officer at Audentes Therapeutics since February 2014. He is a United Kingdom board certified physician, having trained at Pediatric Centers of excellence in the UK and Australia; and is a member of the Royal College of Physicians (MRCP) and the Royal College of Pediatrics and Child Health (MRCPCH). He is also an elected Fellow to the Faculty of Pharmaceutical Medicine (FFPM).

Dr. Prasad studied Medicine at the University of Newcastle-upon-Tyne, UK, where he received commendations for Pediatrics, Obstetrics and Gynecology, and Medical Ethics. He received his Diploma in Pharmaceutical Medicine from the Royal College of Physicians of the United Kingdom, and is a past recipient of the Outstanding Contribution Award from the Faculty of Pharmaceutical Medicine of the UK Royal College of Physicians.

Dr. Prasad has worked in drug development for 15 years in positions of increasing responsibility at BioMarin Pharmaceutical, Inc., Genzyme Corporation, and Eli Lilly and Company. Dr. Prasad has a wide range of experience and achievement in international drug development from Phase I to IV, with a specific focus in the clinical development of therapies to treat rare pediatric disorders. His therapeutic expertise includes neuromuscular disease, metabolic medicine, osteology, neuroscience, endocrinology, immunology, cardiology, hepatic disease, genetics and translational science.

Dr. Prasad holds considerable experience in bringing highly innovative therapies to families and children afflicted with severe, chronic disease, and is a dedicated advocate of such children and families.

Benedikt Schoser, M.D.
Benedikt Schoser, M.D.

Professor, Senior consultant neurologist
Friedrich-Baur-Institut, Department of Neurology
Klinikum München, Ludwig-Maximilians University

Dr. Benedikt Schoser is Senior Consultant Neurologist at the Friedrich-Baur-Institute, Department of Neurology and head of the interdisciplinary neuromuscular unit at the Klinikum München, Ludwig-Maximilians-University of Munich, Germany. He is member of the German reference center for neuromuscular disease and co-head of the diagnostic working group of the German Muscular Dystrophy Network (MD-Net). He is assigned to co-chair the neuromuscular special interest board of the European Academy of Neurology in 2016.

Dr. Schoser graduated with a Doctorate in Medicine from the Johannes-Gutenberg-University of Mainz, Germany, in 1993, before completing further residencies in the Departments of Neurology and Psychiatry at the University Medical Center Hamburg-Eppendorf in Hamburg, Germany. Later, he worked as a postdoctoral research fellow in the channelopathy laboratory of Thomas Jentsch at the Center of Molecular Neuroscience (ZMNH) in Hamburg. In 2001, he moved to his current position at the Friedrich-Baur-Institute, Munich, Germany, the German reference center for neuromuscular disorders. Here, he completed his habilitation on the caveolinopathy rippling muscle disease as Professor of Neurology in 2004. As a trained adult neurologist, intensive care neurologist, and myopathologist, Dr. Schoser’s present research is focused on metabolic myopathies, triadopathies, and repeat expansion disorders exploiting methods in human patient cells to understand the pathophysiology and to develop targeted therapies to treat these disorders of all ages. He has conducted several clinical phase 1–3 studies in neuromuscular disorders during the past decade and has a special interest in translational therapy. Dr. Schoser has led studies to identify the pathology and genetic basis for a variety of rare neuromuscular diseases, and has published more than 150 peer-reviewed manuscripts and several book chapters.

Caroline Sewry, B.Sc., Ph.D., F.R.C.Path.
Caroline Sewry, B.Sc., Ph.D., F.R.C.Path.

Founder of the Muscle Pathology Service at the
Robert Jones & Agnes Hunt Orthopaedic Hospitals
Muscle Pathologist, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital

Co-founder of the Wolfson Centre for Inherited Neuromuscular Diseases

Dr. Caroline Sewry has worked in the field of muscle pathology for over 45 years, initially with the histochemist Professor A.G.E. Pearse, then with Professor Victor Dubowitz at Hammersmith Hospital, London, and currently with Professor Francesco Muntoni at Great Ormond Street Hospital for Children, London. In 1998 she established a muscle pathology service at the Robert Jones & Agnes Orthopaedic Hospital, Oswestry, and with Professor Glenn Morris and Dr. Ros Quinlivan helped to found the Wolfson Centre for Inherited Neuromuscular Diseases, a multidisciplinary clinical and research centre for all neuromuscular disorders.

Dr. Sewry’s initial work was in the application of histochemistry and electron microscopy to diseased muscle, and with advent of the molecular era she developed the application of immunohistochemistry in her department. She is co-author of the 3rd and 4th edition of ‘Muscle Biopsy: A Practical Approach’ and co-editor of ‘Muscle Disease: Pathology & Genetics’. She has contributed chapters to several textbooks on muscle disease, and has over 300 peer-reviewed publications.