Speaker Biographies

Eric Auger is a Partner and Chair of the Partnership Committee at Putnam Associates, LLC. He advises life sciences clients in the areas of new product planning, competitive strategy, pricing, and market access. His clients include leading and emerging manufacturers of innovative biopharmaceuticals, vaccines, medical devices, and clinical diagnostics. His specialty is advising on product investments and go to market strategy, helping clients to evaluate and identify the critical path to success in new and developing markets as well as in established and highly competitive markets. His work cuts across internal functions, building strategic alignment within organizations around the business case for strategic and operational recommendations and developing the confidence for client stakeholders to move forward with conviction. Mr. Auger has led or co-led numerous internal thought-leadership and capability-building initiatives around timely industry topics, including commercial planning for the currently emerging cell, gene, and tissue therapy landscape. He has worked directly with several cell and gene therapy clients regarding the complex challenge of pricing, reimbursement, and patient access to these important therapeutic innovations.

Wilson Bryan, M.D., is the Director of the Office of Tissues and Advanced Therapies (OTAT) in the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research (CBER). Dr. Bryan is board certified in neurology and neuromuscular medicine. He served on the neurology faculty of the University of Texas Southwestern Medical School for 13 years. He has been an investigator on clinical trials in cerebrovascular disease and neuromuscular disorders, particularly amyotrophic lateral sclerosis. He received his M.D. from the University of Chicago Pritzker School of Medicine.

Andrew Byrnes, Ph.D., is Chief of the Gene Transfer and Immunogenicity Branch in the Division of Cellular and Gene Therapies at the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research. He has 18 years of experience at the FDA in review of the manufacturing of gene therapies, cell therapies, and other products for clinical use. Dr. Byrnes has a background in virology and gene therapy, and his laboratory research focuses on adenovirus gene therapy in rodent models. His research interests include vector biodistribution, clearance of vector by the liver, interactions of virions with natural antibodies and complement, and innate immune reactions to viral vectors. He received his undergraduate degree from Yale University, earned his Ph.D. from the University of Oxford, and then conducted postdoctoral research at Johns Hopkins University.

Theresa Chen, Ph.D., is a Pharmacology/Toxicology reviewer in the Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT) in the Office of Tissues and Advanced Therapies (OTAT) at the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research (CBER). She is responsible for reviewing preclinical toxicological and pharmacological data that are submitted in the pre-pre–investigational new drug applications (INDs), pre-INDs, INDs, and/or biologics license applications (BLAs). Before joining FDA in 2003, she was a senior scientist at Genetic Therapy, Inc./Novartis. She received her Ph.D. in biochemistry from the University of North Texas.

John (Jay) Chiorini, Ph.D., is Chief of the Adeno-Associated Virus Biology Section at the National Institute of Dental and Craniofacial Research. Adeno-associated virus (AAV) vectors have several characteristics that make them attractive vectors for gene therapy, including stability, ease of manipulation, low immunogenicity, and ability to direct long-term expression of transgenes. The overall research goal of the AAV Biology Section is to define the interactions of AAV with its target cell. The underlying hypothesis is that by understanding these interactions and the biology of the virus, vectors with enhanced activity can be developed and existing vectors can be better targeted to specific applications. Ongoing projects look at the application of these vectors for diseases with unmet clinical needs. Although vectors are being developed to many targets, of special interest are diseases of the salivary gland including radiation-induced xerostomia and Sjogren’s syndrome. Findings from the group’s research are in clinical development and early stage clinical trials at the NIH and around the world. Dr. Chiorini received his B.A. in biochemistry and molecular biology from the University of California, Santa Cruz, and his Ph.D. in genetics from The George Washington University. He completed postdoctoral training fellowships at the National Institute of General Medical Science and the National Heart, Lung, and Blood Institute. As a postdoctoral fellow his work focused on the role of the Rep proteins in AAV replication and the virus lifecycle. He cloned some of the first non-AAV2 serotypes including Bovine AAV, Avian AAV, AAV4, AAV5, and AAV12.

Kenneth Cornetta, M.D., is a Clinical Professor of Medical and Molecular Genetics at Indiana University School of Medicine. He directs the Indiana University Vector Production Facility that was established in 1995 with the goal of generating clinical retroviral and lentiviral vector products for phase I trials. Dr. Cornetta also has an interest in the development of release testing to assist investigators in meeting Food and Drug Administration (FDA) requirements, an effort assisted by the National Heart, Lung, and Blood Institute/National Institutes of Health (NHLBI/NIH) National Gene Vector Biorepository, which is hosted in his laboratory. He received his M.D. from Albany Medical College.

Scott Dorfman, BBA, is President and Chief Executive Officer (CEO) of Odylia Therapeutics, a nonprofit biotech company whose mission is to facilitate the translation of rare disease therapies with proof-of-concept from the lab into clinical trials. Prior to Odylia, Mr. Dorfman founded and was Chairman and CEO of Innotrac Corporation, which was a full-service provider of eCommerce technology, fulfillment, and call center services. Founded in 1984, Innotrac employed more than 2,500 employees with $250 million in revenues. The company was taken private by Mr. Dorfman along with Sterling Capital and Longview Capital in 2014 and combined with eBay Enterprises (division of eBay) to form Radial Corporation. Radial Corporation is the one of the world’s leading eCommerce service companies with revenues of more than $1 billion and with 30 locations in North America, Europe, and Asia. Mr. Dorfman served on the Board of Visitors for Emory University and is a past member of the Board of Directors of the Partnership against Domestic Violence (PADV). In addition to serving on the Radial Board, he has also sat on the Board of Directors for Chatham Capital, Return.com, and Market Velocity, Inc. Mr. Dorfman currently sits on the Board of Dropoff Inc., Complemar Corporation, Fulcrum Ventures, Odylia Therapeutics, and the Usher 2020 Foundation. He received his BBA from the University of Georgia.

Florian Eichler, M.D., is an Associate Professor of Neurology at Massachusetts General Hospital (MGH) and Harvard Medical School and the Director of the Center for Rare Neurological Diseases at MGH. The Center aims to eradicate rare disorders of the nervous system by leveraging the power of biological insights toward design and implementation of clinical trials. He also is the principal investigator of several NIH-funded studies on neurogenetic disorders as well as a gene therapy trial of adrenoleukodystrophy that recently reported first successful outcomes in the New England Journal of Medicine. For this work, he received the Martin Research Prize from MGH and the Herbert Pardes Clinical Excellence Award from the Clinical Research Forum. He also serves as chair of the Rare Disease Think Tank at MGH and is Founder and President of the international consortium ALD Connect, a patient-powered research network that is dedicated to curing adrenoleukodystrophy. Dr. Eichler’s career has been dedicated to advancing the care and treatment for devastating neurogenetic conditions. His laboratory at MGH explores the relationship of mutant genes to specific biochemical defects and their contribution to neurodegeneration. To develop novel treatments, his laboratory assesses the consequences of gain or loss of function due to disease-causing genes. In 2009 the laboratory identified two neurotoxic desoxysphingoid bases that accumulate in mutant transgenic mice and humans with HSAN1. For this work Dr. Eichler received the Wolfe Neuropathy Research Prize from the American Neurological Association. This further led to a first clinical trial of substrate supplementation therapy for patients with HSAN1. He received his M.D. from the University of Vienna Medical School and neurogenetics training at Johns Hopkins with the late Dr. Hugo Moser and performed a residency in pediatric neurology at MGH.

Jaysson Eicholtz, M.S., is the Director of Good Manufacturing Practice (GMP) Operations at Nationwide Children’s Hospital in Columbus, Ohio. He has 19 years of related industry experience in operations leadership roles including vaccines, cell-based therapies, over-the-counter drugs, and gene therapy at organizations that include Wyeth, Sanofi Pasteur, Dendreon, and Unither. He received his B.S. in animal science from the University of Maryland and his M.S. in biotechnology from the University of Maryland University College. He has been a member of the Food and Drug Administration’s (FDA’s) Devices Good Manufacturing Practice Advisory Committee since 2017 and has expertise in GMP facility design and process development with a focus on operational efficiency.

Denise Gavin, Ph.D., is the Chief of the Gene Therapy Branch (GTB) in the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologic Evaluation and Research (CBER) at the United States Food and Drug Administration (FDA). In this role, she oversees the manufacturing, testing, and controls evaluations for gene therapy products under Investigational New Drug and Biologics License Applications. The GTB oversees a wide variety of products including viral and non-viral vectors, ex vivo genetically modified cells, and products that involve genome editing. The GTB also is responsible for developing policy related to regulation of cell and gene therapy products and performing outreach to gene therapy stakeholders. Dr. Gavin received her PhD. in molecular virology from Rush University School of Medicine in Chicago, Illinois, and performed fellowships in the Laboratory of Molecular Biology (LMB) in the National Institute of Neurological Disease and Stroke at the National Institutes of Health in Bethesda, Maryland, and at the University of North Carolina-Chapel Hill Gene Therapy Center in North Carolina.

SteveGray_20150417Steven Gray, Ph.D., is an Associate Professor in the Department of Pediatrics at the University of Texas Southwestern (UTSW) Medical Center. He is the Director of the UTSW Viral Vector Facility and maintains affiliations with the Department of Molecular Biology, the Department of Neurology and Neurotherapeutics, the Eugene McDermott Center for Human Growth and Development, and the Hamon Center for Regenerative Science and Medicine at UTSW. Dr. Gray’s core expertise is in adeno-associated virus (AAV) gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system. As AAV-based platform gene transfer technologies have been developed to achieve global, efficient, and in some cases cell-type specific central nervous system (CNS) gene delivery, his research focus also has included preclinical studies to apply these reagents toward the development of treatments for neurological diseases. Currently these include preclinical studies for Rett syndrome; giant axonal neuropathy (GAN); and Tay-Sachs, Sandhoff, Krabbe, AGU, Charcot-Marie-Tooth, and Batten diseases and have expanded into human clinical studies to test a gene therapy approach for GAN. He received his Ph.D. in molecular biology from Vanderbilt University and performed a postdoctoral fellowship focusing on gene therapy in the laboratory of Jude Samulski at UNC Chapel Hill.

Josh Grieger, Ph.D., is Chief Technology Officer at Asklepios Biopharmaceuticals, where he was key in the Pro10 recombinant adeno-associated virus (rAAV) vector manufacturing process technology transfer to Baxter to support the hemophilia gene therapy pre-clinical and clinical programs. Previously he was the co-founder and Vice President of Process Development and Manufacturing at Bamboo Therapeutics, Inc., where he led and managed the good manufacturing practice (GMP) manufacturing and quality release of the rAAV drug product for the GAN phase I clinical trial and supported the preinvestigational new drug (IND) for the Duchenne Muscular Dystrophy AAV Gene Therapy Program. Bamboo Therapeutics, Inc., was acquired by Pfizer in August 2016. As part of Pfizer, Dr. Grieger served as a Senior Director of Gene Therapy focused on process development and optimization of scalable rAAV vector manufacturing processes for early- and late-phase gene therapy clinical trials. From 2008 through 2015, he was employed by the UNC-Chapel Hill Gene Therapy Center as a postdoctoral research scientist, Research Associate Professor, and Director of the UNC Vector Core Facility. His research focused on the development of a scalable transfection-based manufacturing process for rAAV vectors. The Pro10 cell line was established through this work and led to the transition from adherent cell-based manufacturing of rAAV vectors to animal-derived component-free suspension cell manufacturing in WAVE and Stir tank bioreactors with associated scalable purification technology. He received his Ph.D. in molecular biology and genetics from UNC-Chapel Hill and carried out his dissertation work, focused on various aspects of the AAV life cycle, in Dr. R. Jude Samulski’s laboratory.

Marlene Haffner, M.D., M.P.H., is the Chief Executive Officer of Haffner Associates, LLC, a firm dedicated to the strategy, development, and policy of drug development with a special emphasis on rare diseases and the products that treat them. She also is an Adjunct Professor in the Department of Preventive Medicine and Biometrics and a Clinical Professor in the Department of Medicine at the F. Edward Hébert School of Medicine at the Uniformed Services University of the Health Sciences (USUHS) in Bethesda, Maryland. Prior to establishing her own company in March 2009, she was Executive Director of Global Regulatory Policy and Intelligence at Amgen, Inc., for 2 years. Previously, Dr. Haffner served as Director of the Office of Orphan Products Development (OOPD) of the Food and Drug Administration (FDA). As OOPD Director she was responsible for the leadership and management of the FDA orphan products development program, the first orphan products program in the world. She is well known as an expert in orphan drug development and is a sought-after speaker and consultant in that area of regulatory science. For 36 years she served in the United States Public Health Service (USPHS), beginning her career with the Indian Health Service in Gallup, New Mexico. During her public health career, she rose to the rank of Rear Admiral in the USPHS. A sought after speaker and consultant, Dr. Haffner has received many awards for her work in drug development including The Outstanding Contributions to Pharmaceutical Medicine Award from the American Academy of Pharmaceutical Physicians and the Woodrow Wilson Award for Outstanding Government Service from the Johns Hopkins University. She is the author of multiple articles in peer-reviewed literature concerning issues of orphan product development. She received her M.D. from the George Washington University School of Medicine where she then interned in internal medicine. She received further training in internal medicine, dermatology, and hematology at the Presbyterian Hospital, New York, and the Albert Einstein College of Medicine, New York. She received her M.P.H. from the Johns Hopkins University Bloomberg School of Public Health.

Brian Halak, Ph.D. is the President and Chief Executive Officer (CEO) of WindMIL Therapeutics. In 2016, he assisted scientific founders Kim Noonan and Ivan Borrello in creating WindMIL and has served as President and CEO since its inception. He remains a partner at Domain Associates, a leading healthcare venture capital fund focused exclusively on the life sciences sector, where he has been involved with new company creation and investments in all sectors in which Domain invests. He also established and directed Domain’s prior initiatives in China, including its collaboration with Beijing Elite to create new medical device companies in China based on technology licensed from U.S. and European markets. One such company, Elite Neurovascular, was successfully sold to Terumo Corporation in 2017. Since forming WindMIL, Brian’s duties as a Domain partner have focused on his continuing service on the board of directors for two companies, Alimera Sciences and Dicerna Pharmaceuticals. He previously has served on the boards of Eddingpharm, Esprit Pharma, GI Dynamics, Tobira Therapeutics, and Vanda Pharmaceuticals. He received his B.S.E. in bioengineering from the University of Pennsylvania and his Ph.D. in immunology from Thomas Jefferson University.

Marianne Hamilton Lopez, Ph.D., M.P.A. is Research Director of the Value-Based Payment Reform portfolio at the Duke-Margolis Center for Health Policy. In this role, she manages the Center’s activities aimed at identifying barriers and facilitating implementation of new value-based payment models for pharmaceuticals, including gene therapies, and medical devices. She oversees the Value-Based Payment for Medical Products Consortium and partners with Duke University faculty, scholars, and external health experts to advance this work. Prior to joining Duke-Margolis, Dr. Hamilton Lopez was a Senior Program Officer with the National Academy of Medicine’s Leadership Consortium for a Value and Science-Driven Health System and provided strategic direction and oversight of the Consortium’s Science and Technology Portfolio, Clinical Effectiveness Research Innovation, and the Digital Learning Collaboratives. She was a Senior Manager at AcademyHealth, a Public Health Community Advisor for the United States Cochrane Center, and the Federal Women’s Program Manager and American Indian/Alaska Native Employment Program Manager for the National Institutes of Health. She received her Ph.D. in public policy from the University of Maryland Baltimore County and her M.P.A. from The George Washington University.

Katherine A. High, M.D., is President of Spark Therapeutics, where she has led the development and regulatory approval of the first gene therapy for a genetic disease in the United States. Spark has shown human proof-of-concept of its leading gene therapy platform in both the retina and the liver and has received breakthrough therapy designations for three different therapeutics across two different tissue targets, the eye and the liver. She is an accomplished hematologist with a longstanding interest in gene therapy for genetic disease and began her career studying the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders. Dr. High was a long-time member of the faculty at the University of Pennsylvania and of the medical staff at the Children’s Hospital of Philadelphia (CHOP), where she was also an Investigator at the Howard Hughes Medical Institute. Her pioneering bench-to-bedside studies of gene therapy for hemophilia led to a series of studies that characterized the human immune response to adeno-associated virus (AAV) vectors in a variety of target tissues. Her work has evolved to encompass clinical translation of potential gene therapies for multiple inherited disorders. As the director of the Center for Cellular and Molecular Therapeutics at CHOP, she assembled a multidisciplinary team of scientists and researchers working to discover new gene and cell therapies for genetic diseases and to facilitate rapid translation of preclinical discoveries into clinical application. Dr. High served a 5-year term on the Food and Drug Administration (FDA) Advisory Committee on Cell, Tissue and Gene Therapies and is a past president of the American Society of Gene & Cell Therapy (ASGCT). She received her A.B. in chemistry from Harvard University, her M.D. from the University of North Carolina School of Medicine, her business certification from the University of North Carolina Business School Management Institute for Hospital Administrators, and her M.A. from the University of Pennsylvania.

Ilan Irony, M.D., is the Acting Director of the Food and Drug Administration’s (FDA’s) Office of Orphan Products Development (OOPD). Previously he was the Deputy Director in the Division of Clinical Evaluation and Pharmacology/Toxicology in the Office of Tissues and Advanced Therapies. He also worked in the Endocrine Division in the Center for Drug Evaluation and Research (CDER) as a reviewer and team leader. Over the course of his career at FDA, he has reviewed many applications intended for treatment of rare disorders; has presented on development of orphan products, in particular regarding cellular and gene therapies; and participated in activities with rare disease organizations. After he received his M.D. from Faculdade de Ciencias Medicas da Santa Casa de Sao Paolo, he received training at the University of California, San Francisco (UCSF) and the National Institutes of Health (NIH) and had years of practice in internal medicine and endocrinology.

Stephen G. Kaler, M.D., is a Senior Investigator at the Eunice Kennedy Shriver National Institute of Child Health and Human Development. He identified the molecular basis for the copper transport disorder occipital horn syndrome and, with international collaborators, delineated several other conditions affecting copper homeostasis. These include ATP7A-related isolated distal motor neuropathy and unique syndromes caused by mutations in SLC33A1, an acetylCoA transporter, and CCS, a copper chaperone. His laboratory illuminated the mechanisms underlying ATP7A-related motor neuron degeneration, establishing a link between genetically distinct forms of motor neuron disease and highlighting the role of ATP7A in the peripheral nervous system. He pioneered early identification of infants at risk for Menkes disease using plasma neurochemical measurements and his laboratory utilized a yeast complementation assay to develop a predictive test for responsiveness to copper treatment. Dr. Kaler leads a clinical service for patients with this illness and related disorders of copper metabolism at the National Institutes of Health (NIH) Clinical Center. The Kaler laboratory recently rescued a lethal mouse model of Menkes disease by a brain-directed adeno-associated virus (AAV)-mediated gene. Extension of these latter proof-of-concept investigations, in tandem with preclinical toxicology studies, will pave the way for a first-in-human gene therapy trial for Menkes patients with complete loss of function ATP7A gene defects. He received his M.D. from the University of Rochester; received clinical training in internal medicine and pediatrics in Boston before coming to the National Institutes of Health (NIH) for a fellowship in medical genetics; and became board certified in clinical genetics, biochemical genetics, and molecular genetics.

Walter Kowtoniuk, Ph.D., is a company creator with nearly a decade of experience working at the intersection of strategy and science. At Third Rock Ventures, he focuses on discovering, launching, and building companies aimed at treating rare genetic diseases and transforming the lives of patients. He is the architect of Fulcrum Therapeutics, joining the company full time to lead strategy, business development, and operations. As part of the leadership team, he was involved in advancing the pipeline to its first clinical candidate and growing the company to 40-plus employees. Dr. Kowtoniuk also has worked in strategy consulting with Clarion Healthcare developing new business strategies and providing commercial insight into early product development in the rare disease space. He also is a member of   the Corporate Advisory Council for National Tay-Sachs and Allied Diseases (NTSAD). He received his B.S. in biochemistry, molecular biology, and philosophy from Gettysburg College and his Ph.D. in chemical biology from Harvard University.

Harry L. Malech, M.D., is Chief of the Genetic Immunotherapy Section and Deputy Chief of the Laboratory of Clinical Immunology and Microbiology at the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH). He cares for and studies patients who have a variety of inherited immune deficiencies, with long-term focus on children and young adults with chronic granulomatous disease (CGD) or X-linked severe combined immune deficiency (XSCID). His clinical service conducts clinical trials of allogeneic hematopoietic stem cell transplant and autologous stem cell ex vivo lentivector transduction gene therapy for CGD, SCID, and other immune deficiencies. Laboratory research is focused on achieving efficient genetic correction of patient hematopoietic stem cells. Related work includes studies of the generation of induced pluripotent stem cells from patients with CGD and XSCID and the use of gene editing methods to genetically correct iPSC or patient hematopoietic stem cells. Dr. Malech is author of 342 publications. He currently serves on the Medical Advisory Board of the Immune Deficiency Foundation; as a clinical reviewer for the California Institute for Regenerative Medicine; as a member of the NIH Institutional Biosafety Committee, which reviews all gene therapy protocols conducted at the NIH Clinical Center; as a member of the Scientific Advisory Board of Orchard Therapeutics; and as an elected member of the Association of American Physicians and the American Society for Clinical Investigation. He was President of the American Society of Gene & Cell Therapy for 2014–2015. He received his M.D. from Yale University and performed his residency at the University of Pennsylvania and postdoctoral training at the National Cancer Institute and at Yale University.

Douglas R. Martin, Ph.D., is a Professor of Anatomy, Physiology, and Pharmacology and a scientist at the Scott-Ritchey Research Center at Auburn University’s College of Veterinary Medicine. He has 25 years of experience with feline models of gangliosidosis, neuropathic lysosomal storage disorders that include Tay-Sachs disease and GM1 gangliosidosis. His lab focuses on developing gene therapy for translation from animal models to human application. In this pursuit, affected cats treated by intracranial or intravascular adeno-associated virus (AAV) gene therapy have shown dramatic improvements in quality of life and greater than fivefold increases in life span. In 2007, Dr. Martin co-founded the Tay-Sachs Gene Therapy Consortium, a team of international scientists dedicated to finding an effective treatment for neuropathic storage disorders. Preclinical proof-of-concept and toxicity/biodistribution studies are complete for both Tay-Sachs disease and GM1 gangliosidosis. Early-phase clinical trials are expected to begin in 2019. He received his Ph.D. in biomedical sciences from Auburn University.

 

Lynne F. McGrath, M.P.H., Ph.D., is currently an independent regulatory consultant specializing in gene therapy, rare disease  and oncology product development. Most recently she was the Vice President of Regulatory Affairs at REGENXBIO, a leading AAV gene therapy focused clinical-stage biotechnology company. In addition, she serves on the Scientific Advisory Committee for BioPontis Alliance for Rare Diseases.  Prior to joining REGENXBIO in 2015, Dr. McGrath spent 3 years as an R&D global leader for Novartis Consumer Health and 8 years at Novartis Oncology managing global strategy for multiple development teams, accelerating approval of multiple drugs including several for rare diseases. As a member of the executive team for Novartis Oncology US organization, she participated in multiple business-critical endeavors, e.g., compliance initiatives, pharmacovigilance, due diligence and oversight of promotional material review and approval. Prior to Novartis, Dr. McGrath spent over 15 years leading regulatory teams developing global products for primary care, oncology and rare genetic diseases.  Dr. McGrath holds a PhD and an MPH in public health from the University of Medicine and Dentistry of NJ - Robert Wood Johnson Medical School, where her research focused on the use of innovative scientific discoveries to inform regulatory decision making through effective risk analysis. In addition, she received a B.S. in biology from the University of Connecticut.

R. Scott McIvor, Ph.D., is a Professor in the Department of Genetics, Cell Biology and Development and the Center for Genome Engineering at the University of Minnesota. His primary areas of interest have been (i) the application of integrating vectors (retroviral and lentiviral vectors) into hematopoietic stem cells for protection against the toxicity of antifolate chemotherapeutics and for treatment of primary immune deficiencies, currently in clinical testing for Artemis-deficient severe combined immunodeficiency at the University of California, San Francisco; (ii) development of the non-viral integrating Sleeping Beauty transposon system for genetic therapies; and (iii) genetic therapies for metabolic disease, with emphasis on the mucopolysaccharidoses using several different approaches and in collaboration with different commercial partners. In addition to his academic position, Dr. McIvor managed Discovery Genomics, Inc., as Chief Executive Officer and Chief Science Officer from 2006 until 2016 when it was acquired by Immusoft Corporation, where he is currently Chief Development Officer. Immusoft uses the Sleeping Beauty transposon system for genetic engineering of B cells to express therapeutic proteins as a novel cellular therapy for a variety of conditions, including metabolic disease. Dr. McIvor received his Ph.D. in microbiology from the University of Minnesota and performed postdoctoral work at the University of California, San Francisco, and at Genentech, Inc.

Jerry R. Mendell, M.D., is the Founder of the Clinical Translational Gene Therapy Program at the Research Institute at Nationwide Children’s Hospital and holds professorships in Neurology, Pediatrics, Pathology, and Physiology and Cell Biology at The Ohio State University. He currently holds the Curran-Peters Chair of Pediatric Research. He is a clinician-scientist directing laboratory projects and carrying these to the bedside while at the same time caring for neuromuscular patients in the clinic at Nationwide Children’s Hospital. He has published more than 350 articles with a focus on neuromuscular disease and authored books on muscle and nerve disease. He was a founding member of the study group known as “The Clinical Investigation of Duchenne Dystrophy (CIDD),” which defined testing methods for clinical trials in boys with Duchenne muscular dystrophy (DMD), delineated the natural history of the disease, and tested many pharmacologic agents including corticosteroids. He was the corresponding author on the randomized clinical trial that demonstrated the efficacy of prednisone for DMD. Dr. Mendell was the first to perform gene therapy for LGMD2D, showing gene expression in the EDB. For DMD, gene expression in a region of the patient’s deletion showed an immune response with implications for later trials. He also started a gene therapy study in limb-girdle muscular dystrophy type 2D, demonstrating sustained alpha-sarcoglycan gene expression for the first time. Dr. Mendell led the clinical trial on exon skipping—the first therapeutic agent to show increased dystrophin expression in DMD—and a follow-up, long-term exon skipping trial demonstrating slowing in DMD progression. He also was instrumental in the development of the two-tier system for detection of DMD in newborns, setting the stage for the implementation of newborn screening for DMD. In Becker muscular dystrophy, he showed that follistatin, a myostatin inhibitor, could build muscle and improve the 6-min-walk time. He was the principal investigator on the recently published article on spinal muscular atrophy (SMA) gene therapy that showed unequivocal efficacy, and this work received the 2017 People’s Choice Award as the Science Breakthrough of the Year. Today the Mendell Lab focuses on understanding and correcting gene defects in the muscular dystrophies. He received his M.D. from the University of Texas Southwestern Medical Center.

Raj Puri, M.D., Ph.D. is the Director of the Division of Cellular and Gene Therapies (DCGT) in the Office of Tissues and Advanced Therapies (OTAT) at the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research (CBER). He also is a Chief of Tumor Vaccines and the Biotechnology Branch in the same division. Dr. Puri oversees evaluation and regulation of cancer vaccines, immunotherapy, cellular and gene therapy, tissue engineering, and xenotransplantation products and development of policies and guidance documents in these 21st century cutting edge areas of medical research. In addition, Dr. Puri oversees and manages mission-critical regulatory science-related research performed by 11 principal investigators in the DCGT to support cutting-edge medical product development and directs a translational research program in the field of cancer vaccines, cancer targeting, and cellular immunotherapy of cancer. He has contributed more than 300 peer-reviewed regulatory and research articles in numerous scientific and medical journals. Prior to joining the FDA, Dr. Puri was trained in immunotherapy approaches for cancer at the National Cancer Institute’s Surgery Branch and in targeting hormone receptors at the Mayo Clinic in Rochester, Minnesota

Tejashri Purohit-Sheth, M.D., is the Director of the Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT) in the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). She provides supervisory oversight for the clinical and pharmacology/toxicology review of submissions to OTAT. She previously served as the Clinical Deputy Director in the Division of Anesthesiology, General Hospital, Respiratory, Infection Control, and Dental Devices (DAGRID); the Acting Division Director and Branch Chief in the Office of Scientific Investigation; and as a Medical Officer in the Division of Pulmonary and Allergy Products, all at the FDA. She completed an internal medicine residency at Naval Medical Center Portsmouth followed by a fellowship in allergy/immunology at Walter Reed Army Medical Center. Following the fellowship, she became Service Chief of the Allergy/Immunology Clinic at the National Naval Medical Center in Bethesda, Maryland. Following the end of her obligated service as an active duty Naval Officer, she transferred her commission to the U.S. Public Health Service and began her FDA career.

Dawn Rotellini is the Senior Vice President of Program Development at the National Hemophilia Foundation. She is the parent of a 20-year-old son with hemophilia and founded the Rocky Mountain Hemophilia Association in Montana when he was an infant. After moving to Pittsburgh, Pennsylvania, she served as a board member for the Hemophilia Center of Western Pennsylvania. Eventually she became the Executive Director of the Western Pennsylvania Chapter of the National Hemophilia Foundation. Ms. Rotellini has been with the National Hemophilia Foundation for 10 years and oversees three departments: Chapter Services, Education, and Conference and Travel Services. 

Kristin Smedley is the President of the Curing Retinal Blindness Foundation. She is an award winning nonprofit leader, TEDx speaker, and author, but she never planned on any of that. She did plan to be a great third grade teacher; however, her personal path to greatness took an unexpected turn when two of her three children were diagnosed as blind. She had to learn the tools of blindness and build a team of experts who would help her navigate this path that she had not been trained for. Her two blind sons are now thriving as gifted high school students, elected student council officials, baseball championship winners, International Braille competition finalists, and social butterflies. In 2011 Ms. Smedley founded the Curing Retinal Blindness Foundation, the only patient organization in the world for her sons’ blindness, CRB1 LCA/RP. Kristin has led the Curing Retinal Blindness Foundation to raise more than $1 million, introduced the first-ever legislation in the United States in Braille, and has moved rare eye diseases from rarely talked about to being discussed in key circles worldwide. She delivered a TEDx talk in New York City regarding how her blind sons completely changed her perception of blindness and testified at the Food and Drug Administration (FDA) on behalf of the first-ever gene therapy to treat blindness (LUXTURNA). In 2018 she and three colleagues launched a podcast on iTunes, 4 Chicks Chatting, and she will publish her first book, Thriving Blind: Stories of Success Without Sight.

Charles Venditti, M.D., Ph.D., is a Senior Investigator in the Genetics and Molecular Biology Branch at the National Human Genome Research Institute. He is board certified in pediatrics, clinical genetics, and biochemical genetics and is an attending physician at the Mark O. Hatfield Clinical Center at the National Institutes of Health (NIH), where he has initiated a translational research program to study the natural history and clinical phenotype(s) of the hereditary methylmalonic acidemias (MMA) and cobalamin metabolic disorders. The clinical research studies are paralleled by laboratory investigations that have focused on the development of experimental systems to study the genetics, genomics, and biochemistry of organic acid metabolism in model organisms, including roundworms, mice, and zebrafish. Using a translational research approach, Dr. Venditti and his colleagues have published a number of papers that connect disease pathophysiology in MMA to mitochondrial dysfunction and prove the efficacy of gene therapy as a treatment for both MMA and propionic acidemia. In 2009, he was the recipient of a Presidential Early Career Award for Scientists and Engineers (PECASE), the U.S. government's highest honor for early-career scientists. Other awards include selection as an Outstanding New Investigator from the American Society of Gene and Cell Therapy in 2010 and election into the American Society of Clinical Investigation (ASCI) in 2012. He is a member of numerous professional organizations in the fields of inborn errors of metabolism, genetics, and gene therapy and serves on the medical advisory board of the Organic Acid Association (OAA). He was an M.D., Ph.D. scholarship recipient at Penn State University. He completed a pediatrics residency at Massachusetts General Hospital/Harvard Medical School and a combined clinical and biochemical genetics fellowship at the Children's Hospital of Philadelphia/University of Pennsylvania School of Medicine. 

Chester (Chet) B. Whitley, Ph.D., M.D. is a Professor of Pediatrics and Experimental and Clinical Pharmacology and Director of the Advanced Therapies Program at the University of Minnesota. He also directs the Clinical Laboratory Improvement Amendments (CLIA)-certified Gene Therapy and Diagnostics Lab, the first laboratory in North America to offer complete gene sequencing as a clinical diagnostic test. In 1983, he led the first U.S. trials of bone marrow transplantation for Hurler syndrome (mucopolysaccharidosis type I, [MPS I]) and other lysosomal diseases. During these early studies, he coined the term “metabolic cross-correction” and, with the Food and Drug Administration (FDA), coined the term “ultra-orphan disease.” In the late 1990s, Dr. Whitley conducted the first clinical trial of gene therapy for an MPS condition, Hunter syndrome. At that time, he invented the instant DMB dye-binding test to quantify urine glycosaminoglycans (GAG), the most common method for quantifying GAG in diagnostic laboratories globally. Other firsts include (1) discovery of a pseudo-deficiency allele for MPS I; (2) showing that combining enzyme replacement therapy (ERT) after bone marrow transplantation enhances metabolic correction; (3) demonstrating that small amounts of intravenous laronidase enzyme cross the blood-brain barrier, reduce brain pathology, and improve learning of murine Hurler syndrome; and (4) demonstrating that intravenous gene therapy prevents brain disease in mice with Hurler syndrome. He has been principal investigator (PI) on the NIH program project grant Gene Therapy for Metabolic Disease that evaluated gene therapy for metabolic brain disorders over a period of 20 years. He is PI of the NIH-funded Lysosomal Disease Network, coordinating 21 clinical studies at 18 institutions. Dr. Whitley is the founding organizer of the annual scientific meeting WORLDSymposium, an international forum fusing basic, translational, and clinical research for lysosomal diseases. Beginning in 2015, he and his colleagues evaluated Sangamo Therapeutics’ zinc-finger nuclease technology in murine models of MPS I and II, leading to the first clinical trial of in vivo gene editing for which the first subject was treated in a phase I clinical trial. He received his Ph.D. in human biochemical genetics and his M.D. from the University of Minnesota.

Rachel Witten M.D., FAAP, is a Senior Medical Officer in the Division of Clinical Evaluation and Pharmacology/Toxicology in the Office of Tissues and Advanced Therapies at the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research. She is responsible for conducting primary clinical reviews of clinical protocols submitted in the pre–Investigational New Drug Applications (pre-IND), INDs, and/or Biologics License Applications (BLAs). Dr. Witten’s reviews cover a wide range of gene and cell therapy products across different therapeutic areas that, among others, include rare and orphan diseases. She actively participates in scientific and regulatory working groups within and outside the FDA, teaches courses for new investigators, participates in writing guidances on rare diseases and gene therapy, and organizes relevant workshops at the FDA.

image1.jpegKeith M. Wonnacott, Ph.D., is an Executive Director of Regulatory Affairs in the Rare Diseases Unit at Pfizer, where he works on regulatory policy and outreach related to cell and gene therapy. He is a member of the regulatory affairs committee for the American Society of Gene & Cell Therapy (ASGCT) and ARM and participates in other trade organizations that work on cell and gene therapy policy. He also contributes to regulatory strategy on gene therapies to treat rare diseases. Prior to working at Pfizer, Dr. Wonnacott was a Director of Regulatory Affairs in the Cell and Gene Therapy Group at Novartis Pharmaceuticals. In that role he advised on regulatory strategy and led the team responsible for developing the chemistry, manufacturing, and control (CMC) module for the Biologics License Application (BLA) leading to Food and Drug Administration (FDA) approval for Kymriah (tisagenlecleucel). Prior to working at Novartis, Dr. Wonnacott was the Chief of the Cellular Therapies Branch at the Center for Biologics Evaluation and Research (CBER) at the FDA. His branch was responsible for the CMC review of all cellular therapies including stem cells, allogeneic pancreatic islets, immunotherapies, cancer vaccines, xenotransplantation products, and tissue engineered products.  His branch was also responsible for the review of medical devices used in the processing and storage of cellular products. Dr. Wonnacott has published several articles and book chapters on the regulation of cellular therapies. He received his bachelor's degree in microbiology from Brigham Young University in Provo, Utah, and his Ph.D. in microbiology and immunology from The Pennsylvania State University College of Medicine in Hershey, Pennsylvania. 

Iwen Wu, Ph.D., is the Branch Chief of the Pharmacology/Toxicology Branch 2 in the Office of Tissues and Advanced Therapies at the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research (CBER). She has previously held positions as a Pharmacology/Toxicology Team Lead and Reviewer. Prior to joining CBER, she was a Lead Reviewer in the Renal Devices Branch at the Center for Devices and Radiological Health and a Commissioner’s Fellow at FDA. She received her B.S. in bioengineering-biotechnology from the University of California, San Diego, and her Ph.D. in biomedical engineering from Johns Hopkins University.

 

NCATS U.S. Food and Drug Administration