Qualification of cellular starting materials for cell-based therapies

Published: 28 March 2019
Expert Insight
Elizabeth Read
Elizabeth Read
Principal Consultant at EJ Read Consulting LLC
Elizabeth Read, MD is an independent consultant focusing on CMC development and CMC regulatory issues for cell- and tissue-based therapies. Dr. Read received her M.D. from the State University of New York (Buffalo, NY). After clinical training in Internal Medicine, Hematology, Oncology, and Blood Banking/Transfusion Medicine, Dr. Read worked at the National Cancer Institute and later in the Clinical Center’s Department of Transfusion Medicine at the National Institutes of Health (NIH; Bethesda, MD), where she served as Section Chief and Medical Director of the Cell Therapy Core Facility from 1995-2006. Initially engaged with novel cellular therapies in the context of hematopoietic transplantation, she later worked on more complex cell, tissue, and gene therapies for a range of clinical indications. From 2007-2010, she headed the Cell Therapy Program at Blood Systems Research Institute (San Francisco, CA), collaborating with UCSF investigators on grant-funded stem cell projects. She previously served as Medical Director at the American Red Cross Blood & Tissue Services (Los Angeles, CA). Over the past 10 years, she held leadership positions at small biotech companies, including Fate Therapeutics (San Diego, CA), StemCyte (Baldwin Park, CA), Medeor Therapeutics (San Mateo, CA), and Adicet Bio (Menlo Park, CA). Dr. Read has authored over 100 scientific publications, and has served as a faculty lecturer at UCSF and for the American Course on Drug Development and Regulatory Science. She has served on advisory committees focused on quality, safety, and efficacy of blood products and cell, tissue, and gene therapies, including the American Association of Blood Banks, the US DHHS/HRSA Advisory Council for Blood Stem Cell Transplantation, and the US Pharmacopeia. She currently serves as an advisor on clinical-stage cell therapy projects funded by the California Institute of Regenerative Medicine.

Cellular starting material is unique because of the living, dynamic nature of cells that must be collected from a human donor. The process for donor screening and testing to reduce risk of infectious disease transmission is complex and not harmonized globally. Biologic, donor-to-donor variability is difficult to control and has major impacts on development of the manufacturing process and final product specifications. Maintaining stability of the material requires attention to the complex supply chain for a given manufacturing model. This article presents the key elements, regulatory expectations and special challenges for qualification of cellular starting material.

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