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Hear from leaders around the globe as they share insights about their experiences and lessons learned throughout their certification journey.
The curative potential of cell and gene therapies (CGTs) has led to an explosion of interests and investment for this novel therapeutic modality, and there are currently over 2,400 active INDs in various stages of clinical development. While parallels can be drawn with small molecule drugs and protein therapeutics, there are a number of unique and significant challenges for CGT manufacturing: variability in source materials, limited material for testing, absence of reliable reference standards, maintaining the chain of identity and custody, and the human cost of manufacturing failures, just to name a few. Furthermore, many CGTs are on an expedited development path, leading to compressed CMC development timelines and possible misalignment with clinical development. As a result, establishing proper manufacturing control can become bottlenecks for product registration. This unusual situation was best captured by former FDA Commissioner Scott Gottlieb in his speech at the Alliance for Regenerative Medicine's Annual Board Meeting: “In contrast to traditional drug review, where 80 percent of the review is focused on the clinical portion of that process, and maybe 20 percent is focused on the product issues, I’d say that this general principle is almost completely inverted when it comes to cell and gene therapy.”
Although each product has its own unique challenges, there are common themes in successful CMC development for diverse CGT products. In this symposium, key opinion leaders from academia, industry, and a regulatory agency will discuss these strategies in addressing CGT manufacturing challenges to bring transformative medicines with consistent quality to patients. Hot topics include potency assays, comparability studies, and common quality issues for CGT manufacturing.
1 June 2022 – 3 July 2022: Early Bird $195 member | $230 nonmember
4 July 2022 – 3 August 2022: Regular $230 member | $270 nonmember
After the event, the attendees should be able to
Intermediate: Content is designed based upon the assumption that individuals have basic knowledge of the topic(s) and/or demonstrated competence related to the topic(s). Higher-level concepts are introduced during lectures; exercises requiring synthesis and/or application of concepts are incorporated into the activity.
Regulatory professionals working or interested in Cell and Gene therapy investigational products.
Bruce Levine, Ph.D.
Barbara and Edward Netter Professor in Cancer Gene Therapy, University of Pennsylvania Perelman School of Medicine
Dr. Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, is the Founding Director of the Clinical Cell and Vaccine Production Facility (CVPF) in the Department of Pathology and Laboratory Medicine and the Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania. He received a B.A. (Biology) from Penn and a Ph.D. in Immunology and Infectious Diseases from Johns Hopkins. First-in-human adoptive immunotherapy trials include the first use of a lentiviral vector, the first infusions of gene edited cells, and the first use of lentivirally-modified cells to treat cancer. Dr. Levine is co-inventor of the first FDA approved gene therapy (Kymriah), chimeric antigen receptor T cells for leukemia and lymphoma, licensed to Novartis. Dr. Levine is co-inventor on 30 issued US patents and co-author of >200 manuscripts and book chapters with a Google Scholar citation h-index of 100. He is a Co-Founder of Tmunity Therapeutics, and of Capstan Therapeutics both spinouts of the University of Pennsylvania. Dr. Levine is a recipient of the William Osler Patient Oriented Research Award, the Wallace H. Coulter Award for Healthcare Innovation, the National Marrow Donor Program/Be The Match ONE Forum 2020 Dennis Confer Innovate Award, serves as Immediate Past-President of the International Society for Cell and Gene Therapy, and serves on the Board of Directors of the Alliance for Regenerative Medicine. He has written for Scientific American and Wired and has been interviewed by the NY Times, Wall Street Journal, Washington Post, NPR, Time Magazine, National Geographic, Bloomberg, Forbes, BBC, and other international media outlets.
Deputy Director, Division of Cellular and Gene Therapies, CBER/FDA
Dr. Steven Oh provides leadership in reaching regulatory decisions on advanced therapy regenerative medicine products such as cellular and gene therapy products, tissue-engineered products, combination products, and point-of-care devices submitted to CBER, FDA. Dr. Oh is actively engaged in developing CBER policies addressing regulatory challenges that involve cross-cutting science and emerging technologies in regenerative medicine therapy. He also participates in efforts to develop international standards for manufacturing and testing cellular, tissue-engineered, and gene therapy products as well as in various activities promoting global regulatory convergence for these products. Before joining FDA in 2007, Dr. Oh served on the faculty of Tufts University School of Medicine in Boston. He was trained as a cell biologist and biochemist at Massachusetts Institute of Technology, Johns Hopkins University School of Medicine, and the University of Michigan.
Don Fink, Ph.D.
Regulatory Master Practice Expert, Dark Horse Consulting (DHC)
Dr. Don Fink provides strategic support across several DHC service areas pertaining to cell and gene therapies including manufacturing, process development and comparability, analytical development, and quality systems. Since joining DHC in 2021, Don has become engaged with over 50 clients in the cell and gene therapy space from early-stage product development (INTERACT and pre-IND meetings) through IND and eventual biologics license application (BLA). Don recently led a DHC initiative resulting in submission of a proposed draft guidance to FDA on the topic of testing AAV-based gene therapy products for empty capsid composition during product manufacture.
Prior to joining DHC in 2021, Don was an expert CMC reviewer in the Division of Cellular and Gene Therapies, Office of Tissues and Advanced Therapies (OTAT), Center for Biologics Evaluation and Research (CBER), FDA accumulating over 20-years of regulatory experience primarily in the cell therapy space. Don was responsible for review of an extensive portfolio of applications that included hematopoietic, mesenchymal, cord blood, placenta-derived, and pluripotent stem cell-derived cellular products with the latter being an area of focused expertise. While at FDA, Don organized an FDA advisory committee meeting on the topic of cellular replacement therapies for neurological disorders focused on stem cell-based treatments. He served as FDA liaison to the NIH Stem Task Force as well as the International Society for Stem Cell Research Task Force on Clinical Translation of Stem Cells. Don was a co-founder and co-chair of an FDA-NIH interagency working group involving the extramural program office of the National Institute of Neurological Disorders and Stroke which served to promote cross-agency dialogue to facilitate clinical translation of cellular and gene transfer-based treatments. Dr. Fink has given numerous presentations and authored or co-authored several book chapters describing FDA’s approach for evaluation of stem cell-based therapies.
Jiwen Chen, PhD., RAC
Associate Director, Nurix Therapeutics
Jiwen Chen, Ph D, RAC, is associate director of cell therapy at Nurix Therapeutics. He has also worked at Bristol Myers Squibb, contributing to the development of several medicines that are now widely prescribed to patients.
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