Speakers

Jonathan Appleby, Ph.D. is the chief scientific officer at Gene and Cell Therapy Catapult. Previously he was the chief scientific officer for Cell and Gene Therapy in GSK’s Rare Diseases Unit, where he led the team that developed and launched Strimvelis, the multiaward-winning and first autologous stem cell gene therapy to be approved for commercial use, and was the director and portfolio manager at the GSK Centre of Excellence for External Drug Development. In this role he discovered, in-licensed and developed new technologies and assets with SME companies across a broad range of therapeutic areas. Dr Appleby has worked in all phases of medicines development from discovery to launch and brings decades of industry experience in both conventional medicines and advanced therapy medicinal products (ATMP) development. He received his honors degree in genetics and his Ph.D. in biochemistry and molecular biology from the University of Leeds.

Christopher P. Austin, M.D., is the director of the National Center for Advancing Translational Sciences (NCATS) at the U.S. National Institutes of Health (NIH). NCATS’ mission is to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. Before becoming NCATS director in September 2012, he was the director of the NCATS Division of Preclinical Innovation, which focuses on translating basic science discoveries into new treatments, particularly for rare and neglected diseases, and developing new technologies and paradigms to improve the efficiency of therapeutic and diagnostic development. In this role, Dr. Austin founded and directed numerous initiatives including the NIH Chemical Genomics Center (NCGC), the Therapeutics for Rare and Neglected Diseases (TRND) Program and the Toxicology in the 21st Century (Tox21) Program. From 2016 to 2018, he was the elected chair of the International Rare Disease Research Consortium (IRDiRC). Before joining NIH in 2002, Dr. Austin directed research programs on genomics-based target discovery, pharmacogenomics and neuropsychiatric drug development, with a particular focus on schizophrenia, at Merck. He received his A.B. in biology from Princeton University and his M.D. from Harvard Medical School and completed clinical training in internal medicine and neurology at Massachusetts General Hospital and a research fellowship in genetics at Harvard.

Robert A. Baffi, Ph.D., is the president of Global Manufacturing and Technical Operations at BioMarin, where he is responsible for overseeing the company’s manufacturing, process development, quality and analytical chemistry departments. He also serves on the board for the National Institute for Bioprocessing Research and Training. He has contributed to more than 20 regulatory submissions for product approval in the United States and Europe and to more than 50 regulatory submissions for investigational new drug testing. Previously Dr. Baffi served in a number of increasingly responsible positions at Genentech, Inc., primarily in the functional area of quality control. Prior to Genentech, Dr. Baffi worked for Cooper BioMedical as a research scientist and at Becton Dickinson Research Center as a postdoctoral fellow. He received his Ph.D. in biochemistry, M. Phil. and B.S. in biochemistry from the City University of New York and his M.B.A. from Regis University.  

Barry Byrne, M.D., Ph.D., is the associate chair of pediatrics, the director of the University of Florida Powell Center and Child Health Research Institute and the Earl and Christy Powell University Chair in Genetics. He also serves as the chief medical advisor of the Muscular Dystrophy Association, a member of TREAT-NMD’s Advisory Committee for Therapeutics (TACT) and an advisor to the Pfizer Rare Disease Therapeutic Area Advisory Committee. He is a clinician scientist who studies a variety of rare diseases with the specific goal of developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness and abnormalities in heart and respiratory function. Dr. Byrne’s group has made significant contributions to the understanding and treatment of Pompe disease, a type of neuromuscular disease due to glycogen storage in motor units. The research team has been developing new therapies using adeno-associated virus (AAV)-mediated gene therapy to restore cardiac and skeletal muscle function in Duchenne muscular dystrophy (DMD), Friedreich’s ataxia, and Pompe and other inherited neuromuscular diseases. His group at the Powell Center also has established a series of new methods for large-scale clinical manufacturing of AAV. The work is supported by several National Institutes of Health (NIH) and foundation awards. He received his B.S. from Denison University and his M.D. and Ph.D. from the University of Illinois. He completed his pediatrics residency, cardiology fellowship and postdoctoral training in biological chemistry at the Johns Hopkins Hospital.  

Andrew P. 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 (CBER). He has 19 years of experience at FDA in review of 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. 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 and his Ph.D. from the University of Oxford and then conducted postdoctoral research at Johns Hopkins University.

Nathalie Clément, Ph.D., is the associate director and an associate professor at the Powell Gene Therapy Center, where she supervises adeno-associated virus (AAV) production and testing at research, preclinical and clinical scales. She also is the director of the Process and Development Lab and Quality Control Lab, which is responsible for the production and release of all AAV pre-clinical and clinical lots, at the University of Florida. She has more than 20 years of experience in the field of gene therapy with a strong expertise in viral vectors, specifically adeno-associated vectors. During her thesis work, she developed new recombinant viruses derived from the parvovirus minute virus of mice (MVM) for cancer-selective gene therapy treatments. Previously Dr. Clément was part of Dr. Michael Linden’s laboratory at Mount Sinai School of Medicine, New York, where she developed novel recombinant AAV vectors and directed the AAV Vector Core. Her research is largely focused on optimizing processes supporting large-scale production of high-quality rAAV stocks and their implementation into the good manufacturing practice (GMP) settings. She received her B.S. in molecular biology and her Ph.D. in virology, molecular biology, and gene therapy from the Universite Libre de Brussels, Belgium.

Scott Dorfman is president and CEO of Odylia Therapeutics, a nonprofit biotech company whose mission is to facilitate the translation of rare disease with proof-of-concept from the lab into clinical trials. He also serves as an operations partner for Fulcrum Equity Partners and sits on the Board of Nacuity Pharmaceuticals Inc., Dropoff Inc., Complemar Corporation, Fulcrum Equity, Odylia Therapeutics and the Usher 2020 Foundation. Prior to Odylia, Mr. Dorfman founded and was chairman and CEO of Innotrac Corporation, a provider of eCommerce services. Founded in 1984, Innotrac employed more than 2,500 employees with $250 million in revenues. The company merged with eBay Enterprises (division of eBay) and was sold to Belgium Post. 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), Radial, Chatham Capital, Return.com and Market Velocity, Inc. He received his B.B.A. from the University of Georgia.  

Erich Ehlert, Ph.D., is the director of process development at uniQure. He leads a team of talented professionals in generating the biological starting materials and developing the production process for new products in uniQure’s product pipeline. Prior to joining uniQure, Dr. Ehlert worked at the Netherlands Institute for Neuroscience, where he studied the mechanisms that underlie regeneration and plasticity of the central nervous system using lentiviral and adeno-associated viral vectors. He has a background in molecular biology and gene therapy. He received his B.A.Sc. in biotechnology from Rotterdam University of Applied Sciences and his Ph.D. in neuroscience from Vrije Universiteit Amsterdam.

Nicole Faust, Ph.D. is the chief executive officer at CEVEC Pharmaceuticals GmbH. She has extensive management experience in the biotechnology sector. Prior to CEVEC, she held scientific management positions with Artemis/Taconic Biosciences, Amaxa and Lonza, engaging in the fields of pharmaceutical models, gene transfer, protein production and gene therapy. She received her Ph.D. in biology from the University of Freiburg.  

Simon Frost is the chief executive officer of Tiber Capital Group, a real estate investment firm. He also serves as director of both Cure AHC and Hope for Annabel, charities dedicated to finding viable genetic therapies for alternating hemiplegia of childhood (AHC). His daughter Annabel has a de novo mutation in ATP1A3, the gene most commonly associated with AHC. Mr. Frost has previously served as the chief investment officer of Greencourt Capital Group, co-founder and chief financial officer of The American Home, president and chief operating officer of Key Properties and chief financial officer of Goal Financial. He received his B.A. and M.A. in economics from Cambridge University in England and his bachelor’s degree in finance from the University of South Africa. 

Mark Galbraith is the head of Quality Control & Analytical Sciences at Spark Therapeutics Inc. Spark is a leader in the field of gene therapy, seeking to transform the lives of patients by developing potential one-time, life-altering treatments for debilitating genetic diseases. Mr. Galbraith has worked in the biopharmaceutical industry for 20 years and has gained diverse experience and knowledge in all phases of product development from preclinical to commercial. He began his career at Merck, where he held positions of increasing responsibility in several functional areas related to vaccine clinical development and vaccine commercial manufacturing including analytical research and development, regulatory sciences, commercial analytical support and quality control operations. He continued to develop his career in quality control management and analytical development by directing the quality control activities at Celldex Therapeutics, Bristol Myers-Squibb, and Gilead Sciences. He has gained a broad knowledge of vaccines, biotechnology products, sterile pharmaceuticals and, now, gene therapy.  

Guangping Gao, Ph.D., is the co-director of the Li Weibo Institute for Rare Diseases Research, the director of the Horae Gene Therapy Center and Viral Vector Core and professor of microbiology and physiological systems and Penelope Booth Rockwell Professor in Biomedical Research at the University of Massachusetts Medical School. He also is an elected fellow of the National Academy of Inventors (NAI) and the American Academy of Microbiology and president of the American Society of Gene and Cell Therapy. Dr. Gao is an internationally recognized gene therapy researcher, instrumental in the discovery and characterization of a new family of adeno-associated virus (AAV) serotypes that revitalized the gene therapy field, hugely impacting many currently untreatable human diseases. For nearly 30 years of his scientific research career, Dr. Gao primarily focused on molecular genetics and viral vector gene therapy of rare genetic diseases, encompassing disease gene cloning, causative mutation identification, pathomechanism investigation, animal modeling, novel viral vector discovery and engineering for in vivo gene delivery, vector biology, preclinical and clinical gene therapy product development and manufacturing and technology platforms development as novel approaches for human gene therapy. He has published 267 research papers, 6 book chapters and 5 edited books and holds 135 patents with 239 more patent applications pending. He was ranked #4 in the World Top 20 Translational Researchers in 2018 by Nature Biotechnology. He received his Ph.D. in biological sciences from Florida International University and his postdoctoral training at the University of Pennsylvania School of Medicine.

Denise Gavin, Ph.D., is the chief of the Gene Therapy Branch (GTB) in the Office of Tissues and Advanced Therapies (OTAT) in the United States Food and Drug Administration’s (FDA’s) Center for Biologic Evaluation and Research (CBER). 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 Ph.D. 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. 

Steven Gray, Ph.D., is an associate professor in the Department of Pediatrics at the University of Texas Southwestern Medical Center (UTSW). He also 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. His 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 gene delivery, Dr. Gray’s research focus has also 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); Tay-Sachs, Sandhoff, Krabbe, aspartylglycosaminuria (AGU), Charcot-Marie-Tooth, Batten, and Austin diseases and have expanded into human clinical studies to test a gene therapy approach for GAN. He received his B.S. with honors from Auburn University and 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 the University of North Carolina, Chapel Hill.  

Josh Grieger, Ph.D., is the chief technology officer at Asklepios BioPharmaceutical, Inc. (AskBio) and leads its manufacturing, process and analytical development initiatives. Previously, he was a co-founder and vice president of process development and manufacturing for Bamboo Therapeutics, Inc., where he led the good manufacturing practice (GMP) manufacturing and quality release of the recombinant adeno-associated virus (rAAV) drug product for the GAN Phase I clinical trial and supported the pre- investigational new drug application (IND) for its Duchenne muscular dystrophy AAV gene therapy program. Pfizer acquired Bamboo Therapeutics in 2016, and Dr. Grieger served as the senior director of gene therapy and focused his efforts on process development and optimization of scalable rAAV vector manufacturing for early- and late-phase gene therapy clinical trials. Prior to this, he was employed by the University of North Carolina (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 scalable transfection-based manufacturing process for rAAV vectors. The AskBio 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. Dr. Grieger also consulted for AskBio during that time and was critical in the Pro10 rAAV vector manufacturing process technology transfer to Baxter to support the hemophilia gene therapy preclinical and clinical programs. He received his Ph.D. in molecular biology and genetics from the University of North Carolina at Chapel Hill, where he carried out his dissertation work in the laboratory of R. Jude Samulski, Ph.D.

Gina Hann is a marketing manager at Texas Instruments. She also serves as the president of both Batten Hope and RARE Dallas, two foundations dedicated to serving rare disease families in pursuit of treatment through applied therapies. Ms. Hann founded Batten Hope in 2017, when her son Joseph was diagnosed with Batten disease (CLN7) and she has worked closely with UT Southwestern Medical Center and Children’s Health based in Dallas, Texas, in the development of an adeno-associated virus (AAV) vector for future use in gene therapy for CLN7. She received her B.S.E.E. in electrical and computer engineering and her M.B.A. from the University of Arizona.

Andrew Harmon, Ph.D., is a chemistry, manufacturing and control (CMC) reviewer in the Division of Cellular and Gene Therapies at the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research (CBER) and is responsible for regulatory review of the Initial Targeted Engagement for Regulatory Advice on CBER Products (INTERACT) program, pre–investigational new drug (IND) and IND submissions for viral vectors and genetically modified cell therapy products. Dr. Harmon started at the FDA as a postdoctoral researcher studying immune responses to viral gene therapy vectors. He transitioned to regulatory review activities and is an active participant in intra-agency working groups, meetings and conferences focused on advancing cell and gene therapy products. Dr. Harmon received his Ph.D. in molecular, cell and developmental biology from the University of California, Los Angeles (UCLA).

Stephen Kaminsky, Ph.D., is a professor of research in genetic medicine and an associate director of the Belfer Gene Therapy Core Facility at Weill Cornell Medical College. He directs multiple projects ranging from cancer therapeutics, metabolic disorders such as alpha-1 antitrypsin and gene therapies for the eye and heart to vaccines for addictive drugs. He contributes expertise to preclinical development, safety and toxicology study design, good manufacturing practice (GMP) manufacture, facility design and lab management. He has prior experience designing and developing several candidate prophylactic and therapeutic HIV vaccines as well as a candidate prostate cancer therapeutic vaccine, each of which went to clinical trial. Dr. Kaminsky participated in the design of the Weill Cornell Gene Therapy Core Facility, created the GMP production laboratory and established all of the underlying processes and controls and is a member of the project team that developed gene therapies to the central nervous system (CNS) for Batten disease and Alzheimer’s disease, both from idea through clinic trial. He has authored more than 14 Food and Drug Administration (FDA) regulatory submissions with application to diagnostics, vaccines and therapeutics; he has sat on the National Institutes of Health (NIH) grant review panels for HIV vaccine program projects (HIVRAD), Partnerships for Development of Vaccine Technologies; and he has co-chaired an FDA panel on peptide-based vaccines products. He received his B.S. in engineering physics from Cornell University and his Ph.D. in molecular biophysics and biochemistry from Yale University. 

Andrew Knudten, M.S., M.B.A., is the founder and principal consultant at Gene Therapy Consulting, LLC. His biotechnology career started at Amgen, Inc., in Thousand Oaks, California, where he served in several leadership roles within protein chemistry, product development, finance and operations. Then at Novartis Vaccines he served as the global head of contract manufacturing and strategy and focused on viral and cell-based vaccine production.  Following his tenure at Novartis, he became the site general manager for sterile injectables in McPherson, Kansas, and eventual head of Hospira API Operations prior to the acquisition of the company by Pfizer. Mr. Knudten then joined AveXis as the senior vice president of manufacturing and supply chain and led the development and successful commercialization of Zolgensma® from a manufacturing, process development and supply chain perspective. Since his retirement from AveXis, he has stayed connected in the gene therapy arena by consulting for a number of startup gene and cell therapy companies. He received his B.S. in biology and health from Concordia University, his M.S. in cell biology and genetics from the University of Nebraska, Lincoln, and his M.B.A. focusing on finance and accounting from the University of California, Los Angeles, Anderson School of Management.

Robert Kotin, M.D., is a professor in the Department of Microbiology and Physiological Systems at the University of Massachusetts Medical School. He is one of the world’s top experts on the viral transport of gene therapies and has been a leader in adeno-associated virus (AAV) research for more than 30 years. He spent most of his career in the Intramural Research Program at the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) as a tenure track investigator, a tenured senior investigator and head of the Laboratory of Molecular Biology and Gene Therapy. While at the NIH, Dr. Kotin’s laboratory invented and developed a scalable recombinant adeno-associated virus (rAAV) production process in Sf9 cells, which was licensed by several companies and was used to produce Glybera™, the first rAAV product granted regulatory agency approval for sale. The research at the NIH resulted in the discovery of an AAV replicative product that has been described as closed-ended linear duplex DNA. Previous positions include vice president and founder of Generation Bio, vice president of Voyager Therapeutics and adjunct associate professor at the University of Maryland. He received his B.A. in biology from the University of California, Santa Cruz, and his Ph.D. in microbiology from Rutgers University. 

Kelvin H. Lee, Ph.D., is Gore Professor of Chemical and Biomolecular Engineering at the University of Delaware and director of the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), one of 14 Manufacturing USA institutes. He is a Fellow of the American Institute for Medical and Biological Engineering and a Fellow of the American Association for the Advancement of Science. He previously served as director of the Delaware Biotechnology Institute and spent several years at the Biotechnology Institute at the ETH in Zurich, Switzerland. Dr. Lee also was on the faculty of Cornell University, where he held the titles of Samuel C. and Nancy M. Fleming Chair and Professor, professor in the School of Chemical and Biomolecular Engineering, director of the Cornell Institute for Biotechnology and director of the New York State Center for Life Science Enterprise. He received his B.S.E. in chemical engineering from Princeton and his Ph.D. in chemical engineering from Caltech and completed a postdoctoral fellowship in Caltech’s Biology Division.

Michael Lehmicke is the director of Science and Industry Affairs at the Alliance for Regenerative Medicine (ARM). He is responsible for shaping and leading science and manufacturing initiatives at ARM as well as leading ARM’s science and technology-related member committees and task forces. Mr. Lehmicke has more than 20 years of research and development experience in biomaterials, medical devices and regenerative medicine. He has led product development teams for class II devices, human cell and tissue-based products and drug/device combination products. He is a creator and an inventor with multiple U.S. patents to his name. He received his M.Sc. in biomedical engineering, with a focus on tissue engineering, from Drexel University.

Peter Marks, M.D., Ph.D., is the director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). The center is responsible for assuring the safety and effectiveness of biological products, including vaccines; allergenic products; blood and blood products; and cellular, tissue and gene therapies. Previously he was the deputy center director for CBER. Dr. Marks is board certified in internal medicine, hematology and medical oncology and is a Fellow of the American College of Physicians. Prior to the FDA, he spent several years in the pharmaceutical industry working on the clinical development of hematology and oncology products, leading the Adult Leukemia Service and serving as chief clinical officer of Smilow Cancer Hospital at Yale University and as a member of the attending staff as a clinician-scientist and clinical director of hematology at Brigham and Women’s Hospital in Boston. He received his graduate degree in cell and molecular biology and his M.D. from New York University. Following this, he completed an internal medicine residency and hematology/medical oncology fellowship at Brigham and Women's Hospital in Boston.

Otto-Wilhelm Merten, Ph.D., is a specialist in gene therapy/vector manufacturing at Miltenyi Biotec and a visiting professor at Instituto de Tecnologia Quimica e Biologica Antonio Xavier (ITQB)/Universidade NOVA de Lisboa (UNL). Previous positions include head of the Laboratory of Cell Technology at Sandoz Forschungsinstitut in Vienna, investigator and manager of the Laboratory of Cell Technology at the Institut Pasteur in Paris and head of the Applied Vectorology and Innovation Group at Généthon in Evry. As a biotechnologist he is involved in cell culture development and optimization of production, purification and analytical methods of recombinant proteins (to a large extent monoclonal antibodies), viruses including rabies and influenza virus and viral vectors such as murine leukemia virus (MLV), lentiviruses (LV) and adeno-associated virus (AAV). Dr. Merten was involved in the development of large-scale production methods for the routine manufacturing of LV and AAV vectors. In the context of his expertise in animal cell culture technology he was a long-term member of the executive committee of the European Society of Animal Cell Technology (ESACT) and its chairman from 2001 to 2005. Today he is an honorary member of ESACT as well as a member of the European Society of Gene and Cell Therapy (ESGCT) and the American Society of Gene & Cell Therapy (ASGCT). He received his Ph.D. in biotechnology from the University of Life Sciences in Vienna and an HDR at the University of Evry/Val-d’Essonne.

Anne Pariser, M.D., is the director of the Office of Rare Diseases Research (ORDR) at the National Institute of Health’s (NIH’s) National Center for Advancing Translational Sciences (NCATS). The ORDR is dedicated to accelerating rare diseases research to benefit patients through rare diseases programs such as the Rare Diseases Clinical Research Network, Genetic and Rare Diseases (GARD) Information Center and the NCATS Toolkit for Patient-Focused Therapy Development. Important translational science research initiatives for rare diseases at ORDR include establishing best practices and tools for good quality natural history studies, data standards and sharing initiatives, the development of diagnostic support tools, rare diseases therapeutics development and translational and basic science research grants and collaborative programs. Dr. Pariser came to NCATS in 2017; before this, she worked for 16 years at the Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER), where she founded the Rare Diseases Program in CDER’s Office of New Drugs in 2010 and served as a medical officer and team leader for rare diseases drug and biologics product development, review and regulation. She has 20 years of experience in rare diseases research, and her current research interests include “many diseases at a time” research approaches such as platforms for gene therapies and other rare disease product development and informatics approaches for diagnosis. She received her M.D. from Georgetown University School of Medicine.

Nicole Paulk, Ph.D., is an assistant adjunct professor of biochemistry and biophysics and a K01 fellow in viral gene therapy at the University of California, San Francisco (UCSF). She is a pioneer in the development of next-generation adeno-associated virus (AAV) gene delivery platforms and has engineered payloads for gene repair and gene transfer for numerous rare diseases, utilized directed evolution to evolve capsid serotypes with novel tropisms and applied comparative proteomic approaches to interrogate challenges in vector manufacturing. Dr. Paulk’s translational research lab at UCSF now develops solutions for the biggest problems in gene therapy: cost, delivery and efficacy, with a focus on treatments for rare diseases and cancer. She received her B.S. in medical microbiology from Central Washington University and her Ph.D. in viral gene therapy and regenerative hepatology from Oregon Health and Science University with Dr. Markus Grompe and completed her postdoctoral fellowship in human gene therapy with Dr. Mark Kay at Stanford University.

Herbert Runnels, Ph.D., is a senior research fellow and gene therapy global analytical lead at the Analytical R&D facility in Pfizer’s Biotherapeutics Pharmaceutical Science (BioTx PS) division. In BioTx PS, he focuses on candidates in clinical development and licensure stages and has worked on complex bioassays for many modality types and led global analytical teams in the areas of vaccines and gene therapy. This work involves the development of the analytical strategy and implementation for both early- and late-phase projects. His current focus is advancing viral therapeutics for critical unmet needs with accelerated timelines. Dr. Runnels trained as a molecular and cellular immunologist. Previously he helped design effective antigen constructs for dendritic cancer vaccines at Pharmacia (legacy GD Searle/Monsanto) Discovery Research. This led to work on the discovery and preclinical proof-of-concept studies of therapeutic antibodies for oncology. When Pharmacia merged with Pfizer, he moved into the inflammation therapeutic area where he led projects on chemokine receptor antagonism (small and large molecule) and shuttling therapeutic antibodies, against various soluble and cell-bound targets, into first-in-human/patient clinical trials.

R. Jude Samulsky, Ph.D., is chief scientific officer of Asklepios BioPharmacuetical (AskBio), a company he co-founded. The foundational discoveries and advancements of Dr. Samulski and other longtime associates have set the groundwork for gene therapy today. His vision has led to the development of advanced gene therapy and adeno-associated virus (AAV) technology that has created a future in which the goal to erase genetic disease is now within sight. He also is a professor and the director of the University of North Carolina Gene Therapy Center. Dr. Samulski was the first person to receive the Outstanding Achievement Award from the American Society of Gene and Cell Therapy, which recognized his lifetime of significant scientific contributions to the field of gene therapy. He received his B.S. in microbiology from Clemson University and his Ph.D. in molecular biology from the University of Florida.

Caroline Smith-Moore, Ph.D., is the assistant director, analytical, of the Golden LEAF Biomanufacturing Training and Education Center (BTEC) at North Carolina State University, where she has a leadership role in BTEC’s gene therapy initiatives, specifically the development of assays for characterizing adeno-associated virus (AAV) and the creation and delivery of course content for gene therapy courses. She is currently developing an AAV analytical characterization short course and is principal investigator (PI) on a National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) project to develop new methods for AAV analysis. She leads a group developing assays, performing testing and conducting research for grant-funded projects as well as external client service projects. She also directs work in cell line development, where previously she was a senior scientist. She has extensive experience in biochemical characterization, assay development, molecular biology and recombinant expression in prokaryotic and eukaryotic systems. She received her P.S.M. in microbial biotechnology, her M.B.A. in biosciences management and her Ph.D. in functional genomics from North Carolina State University.

Richard O. Snyder, Ph.D., is the vice president for Science and Technology, Pharma Services, Viral Vector Services at Thermo Fisher Scientific. He has been investigating virus biology, vector development, current good manufacturing practice (cGMP) vector manufacturing and analytical technologies and viral vector-mediated gene transfer for more than 33 years and was a member of teams that developed novel viral vector-based human gene therapies. Previously Dr. Snyder was an associate professor of molecular genetics and microbiology and the director of Biotherapeutic Programs at the University of Florida and an assistant professor of pediatrics at Harvard Medical School and was previously employed by Cell Genesys, Somatix, Merlin, and Avigen where he was engaged in the development of gene transfer vector and vaccine technology along with therapeutic applications. He received his B.A. in biology from Washington University in St. Louis and his Ph.D. in microbiology from The State University of New York at Stony Brook. He completed a postdoctoral fellowship at Johns Hopkins University School of Medicine. 

Zenobia F. Taraporewala, Ph.D., is a chemistry, manufacturing and control (CMC) reviewer and team lead in the Gene Therapies Branch at the Office of Tissues and Advanced Therapies (OTAT) in the Food and Drug Association’s (FDA’s) Center for Biologics Evaluation and Research (CBER). She is trained as a molecular virologist with expertise in viral replication, genomics, vaccinology, reverse genetics and structure-function analysis of viral proteins. She is responsible for regulatory review and oversight of pre–investigational new drug (IND), IND and biologics license application (BLA) submissions for gene therapy products. She also participates in several inter- and intra-agency working groups, meetings and conferences focused on the development of cell and gene therapy products and has contributed to the development of several gene therapy–related guidance documents. She was a member of the BLA review team for the first adeno-associated virus (AAV)-vectored gene therapy product (Luxturna). Previously she was a staff scientist at the National Institute of Health’s (NIH’s) National Institute of Allergy and Infectious Diseases (NIAID). She received her B.Sc. in microbiology from the University of Bombay in Mumbai, India; her M.Sc. in microbiology from M.S. University in Baroda, India; and her Ph.D. in molecular and cell biology from the University of Maryland. She received her postdoctoral training in the Rotavirus Molecular Biology Unit of the Laboratory of Infectious Disease at the NIAID/NIH.

Rajesh Thangapazham, Ph.D. is the head of regulatory strategy in the Cell and Gene Technologies unit of Lonza Pharma Biotech & Nutrition. He is a regulatory affairs professional with more than 10 years of experience and specialization in regenerative medicine and cell and gene therapies. He joined Lonza with a background in biotechnology commercialization and intellectual property creation and considerable experience in orphan drug development. At Lonza, he supports sponsor’s chemistry, manufacturing and controls (CMC) sections of regulatory filing, regulatory briefing documents, registration/licenses for Lonza’s establishments and lifecycle management of master files for facilities, cell banks and donor programs. He also provides continued regulatory support and scientific guidance to Lonza’s innovative technologies such as pluripotent stem cell manufacturing, viral vector manufacturing platforms, automated and closed cell processing systems for personalized medicines and qualification of single-use disposable systems. He received his M.E. in biotechnology from the Birla Institute of Technology and Science and his Ph.D. in biotechnology from the Uniformed Services University of Health Sciences. His training includes a postdoctoral fellowship, staff scientist tenure and research assistant professorship at the U.S. Department of Defense, where he advanced regenerative medicine and was actively involved in the preclinical development of cell and gene therapies.

Johannes van der Loo, Ph.D., is the director of the Clinical Vector Core, which manufactures viral vector products for Phase 1–3 clinical trials in the United States and abroad, at Children’s Hospital of Philadelphia. He has expertise in the manufacturing of adeno-associated virus (AAV), gamma-retroviral and lentiviral vectors, is a member of the Editorial Board of Molecular Therapy – Methods & Clinical Development and has acted as consultant on viral vector manufacturing and good manufacturing practice (GMP) facility design. Previous positions include chair of the Institutional Biosafety Committee and director of the Aseptic Processing Laboratories at Cincinnati Children’s Hospital Medical Center and associate professor in the Department of Pediatrics at the University of Cincinnati. He received his M.S. in immunology from the University of Utrecht and his Ph.D. in hematology from Erasmus Universiteit Rotterdam.

Wei Wang, Ph.D., is a microbiologist in the Division of Manufacturing and Product Quality (DMPQ) at the Office of Compliance and Biologics Quality (OCBQ) at the Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research (CBER). Dr. Wang has more than 10 years of experience at FDA in review of chemistry, manufacturing and control (CMC) and a facility for manufacturing of vaccines, gene and cell therapy products. She received her undergraduate degree in industrial microbiology from the South China Institute of Technology and her Ph.D. in molecular biology from New York University.

James M. Wilson, M.D., Ph.D., is the Rose H. Weiss Professor and Director of the Orphan Disease Center, professor of medicine and pediatrics and director of the Gene Therapy Program at the University of Pennsylvania’s Perelman School of Medicine, where he has led an effort to develop the field of gene therapy. He has been at the nexus of this emerging therapeutic area from its birth. His laboratory discovered a family of viruses from primates called adeno-associated viruses (AAV) that could be engineered to be very effective gene transfer vehicles. These so-called vectors have become the technology platform of choice and have set the stage for the recent resurgence of the field of gene therapy. Dr. Wilson has also been active in facilitating the commercial development of these new gene therapy platforms through the establishment of several biotechnology companies. He is currently leading a national dialogue on the challenges of commercializing these potentially lifesaving treatments due to the disruptive nature they will have on traditional business models. Throughout his career, the focus of Dr. Wilson’s research has been rare inherited diseases, ranging from cystic fibrosis to dyslipidemias to a variety of neurologic disorders. He has published more than 580 papers, reviews, commentaries and editorials in the peer-reviewed literature and is an inventor on more than 153 U.S. patents and patent applications and more than 200 total issued patents worldwide. He was noted by the journal Nature Biotechnology to be the “second most productive bio-entrepreneur in life sciences.” He received his M.D. and his Ph.D. in biological chemistry from the University of Michigan Medical School. 

J. Fraser Wright, Ph.D., is a professor of pediatrics at the Center for Definitive and Curative Medicine and a member of the Maternal & Child Health Research Institute (MCHRI) at Stanford University School of Medicine. He is an expert in the field of gene therapy, viral vectors and monoclonal antibodies and a pioneer and innovator in viral vector design, process development and good manufacturing practice (GMP) manufacture, analytics methods development and quality systems/quality control for DNA-based biologics. He established viral vector manufacturing and quality systems and led vector chemistry, manufacturing and controls (CMC) functions supporting 12 investigational drugs administered to more than 150 subjects in US and EU clinical studies. Dr. Wright is author or coauthor of more than 200 publications, including 15 patents issued or pending, with more than 11,000 literature citations. He is a scientist dedicated to innovation and effective team-based advancement of new medicines to address unmet medical needs. Previous positions include principal at Wright Biologics Consulting; chief technology officer at Axovant Gene Therapies, Ltd; cofounder and chief technology officer at Spark Therapeutics, Inc. and professor of pathology and laboratory medicine at the University of Pennsylvania School of Medicine. He received his Ph.D. in biochemistry and molecular immunology from the University of Toronto. 

 

U.S. Department of Health and Human Services National Center for Advancing Translational Sciences Food and Drug Administration