Cell & gene therapy (CGT) holds significant promise to advance patient treatments. As shown by recent trends, such as the growing interest in CAR T-cell therapy and mRNA gene editing, CGT is the future. Despite its potential, CGT often does not progress from the lab to commercial markets. This is in part due to the unique requirements of working with these technologies, and the new challenges they present to clinical trials.
This roundtable reviewed the necessary considerations and logistics of conducting CGT trials, and how sponsors and service providers can optimize their clinical research to maximize the prospect of success.
Other discussion points included:
- The need for and management of study stopping rules to mitigate risks - protocol amendments - for patients and sites
- The importance of transportation, including the complexity of handling living materials
- How sequencing on site can reduce both the costs and risk associated with CGT clinical trials ?
As head of the Custom Intelligence team for the Americas and lead for cell and gene therapy thought leadership at Informa, Ly partners with clients to develop personalized solutions using Informa’s suite of pharma data and insights. She has 20+ years of experience in the healthcare and pharmaceutical industry and extensive experience in market research and analysis.
Tamie has over 25 years of experience in commercializing specialty biologics and has worked in cell and gene therapies for the last 8 years in global cryologistics and operations. As a former senior executive at one of the largest drug distributors, she specialised in commercialisation, patient hub services and reimbursement support for plasma derived drugs and therapies for oncology and rare diseases. At ICON, she is a member of the Cell and Gene Therapy Centre of Excellence and supports ongoing strategy and innovation across therapeutic areas. She is very active in industry groups focused on developing standards for the regenerative medicine sector.
Dr. Jakob Dupont is a renowned expert in the fields of cell therapy and oncology, with long-standing and deep experience in developing therapies and programs dedicated to addressing high unmet medical needs. Dr. Dupont serves as Global Head of Research & Development including Medical and Regulatory Affairs. Prior to joining Atara, he served as the Chief Medical Officer at Gossamer Bio, overseeing global development, regulatory, and quality activities for the company, and advancing therapeutics in the disease areas of immunology, inflammation, and oncology. Dr. Dupont is committed to bringing transformative therapies to patients.
Dr. Dupont has been involved in tumor immunology research and clinical investigations for more than 25 years, ranging from cellular therapy to tumor vaccine therapy and immune checkpoints. Dr. Dupont has received numerous grants and awards, and has co-authored 47 peer-reviewed publications, has 30 patents, and has served as a faculty member and laboratory researcher at Memorial Sloan Kettering Cancer Center (MSK) and adjunct clinical faculty in medical oncology at Stanford University.
Dr. Pauza received his PhD in Molecular Biology from the University of California, Berkeley and was a postdoctoral fellow in the laboratory of Sydney Brenner, MD, Director, Laboratory of Molecular Biology in Cambridge, England, Noble Laureate (2002). Dr. Pauza held faculty positions at the Salk Institute for Biological Studies, La Jolla, CA, where he founded Salk’s HIV research program, University of Wisconsin-Madison, University of Maryland Biotechnology Institute, and University of Maryland School of Medicine where he served as Associate Director for the Institute of Human Virology and Co-Leader for the Viral Oncology Program in the Greenebaum Cancer Center.
Since 2016 Dr. Pauza has been Chief Science Officer for American Gene Technologies International in Rockville, Maryland. He is an inventor on 14 issued US patents and numerous foreign filings representing efforts to treat and cure HIV, develop gene therapies for phenylketonuria (PKU) and apply genetic medicines to cancer. He led a successful effort to obtain an IND and initiate a Phase 1 clinical trial using an innovative gene therapy strategy for HIV disease. He is a frequent speaker at academic and industry conferences.
Dr. Gallia Levy is the Chief Medical Officer at Spark Therapeutics and leads strategic and operational teams throughout the product development lifecycle with responsibility for setting the global development strategy for current and pipeline programs. She serves as the principal physician and resident medical expert at Spark.
Prior to joining Spark, Gallia was Vice President and Global Head of the Rare Blood Disorders Franchise in Product Development at Genentech, another member of the Roche Group, where she was responsible for the clinical development of treatments for hemophilia A and other rare blood disorders such as paroxysmal nocturnal hemoglobinuria (PNH), and atypical hemolytic uremic syndrome (aHUS). Gallia first joined Genentech in 2009, where she worked in both early and late-stage clinical development. She later moved to Portola Pharmaceuticals, where she led the clinical development program for hematology and oncology indications spanning Phases 1 to registrational trials. She eventually returned to Genentech, taking responsibility for the global clinical development program for HEMLIBRA® during which time she led the transition from Phase 1 through to global registration for adults and children with hemophilia A.
Gallia is board-certified in hematology and earned an M.D. and Ph.D. in Molecular and Cellular Biology from the University of Michigan, during which time discovered ADAMTS13, responsible for Thrombotic Thrombocytopenic Purpura (TTP). She completed her residency in internal medicine at Stanford University and a fellowship in hematology at the University of California, San Francisco. She also spent time at the Pasteur Institute during which she received an M.S. in Molecular and Cellular Biology from the University of Paris, VI and she holds a B.A. from the University of California, Berkeley. Gallia was also appointed as a College Fellow to the College of Physicians of Philadelphia in 2021.