Regulatory Aspects of Gene Therapy and Cell Therapy Products ― A Global Perspective

?This book will include chapters authored by experts from various regulatory bodies throughout the international community. Each chapter will describe and discuss the basic background, regulatory policies, and requirements applied in different global regions (e.g., North America, Europe, Asia) for the development of gene therapy (GT) and cell therapy (CT) medicinal products. Each chapter will walk the reader through the applications of nonclinical research to translational clinical research to licensure for product types that are considered to be at the forefront of biomedical research. This book will be unique in that it will offer its readers the perspective of regulators across the globe with varying levels of hands-on experience in the regulation of GT and CT products. Examples of some of the contents that each chapter will offer include: 1) an understanding of the overall regulatory framework of the organization for CT and for GT products; 2) insight into the applicable regulatory pathway(s) for CT and for GT products; 3) an overview of specific considerations/requirements in the areas of product manufacturing, pharmacology/toxicology, clinical trial designs for CT and for GT products; and 4) access to relevant guidelines and regulations for CT and for GT products.

Maria Cristina Galli holds a University degree in Biological Sciences and a PhD in Molecular Medicine; currently she is in-staff senior researcher, Cell Biology and Neurosciences Department, Istituto Superiore di Sanita, Roma, Italy. Her main expertise is in regulatory sciences for translational medicine supported by scientific education and research experience in experimental oncology, cellular biology, and molecular immunology. Dr. Galli spent more than twenty years as a basic researcher in experimental oncology, cellular biology, and molecular immunology, and wrote or co-wrote sixty publications in international journals. Over the past two decades, she has been active in the field of translational medicine as quality assessor for gene therapy and biotechnology medicines in national as well as European procedures; she has also been active for most of this time as GMP and GLP inspector. Dr. Galli was a member of CAT-EMA for three years and for four years served as vice-chair/chair of CAT-EMA Gene Therapy Working Party, in which she has participated since its first meeting. She is currently co-chair of the ATMP platform in the European infrastructure for translational medicine EATRIS-ERIC.

Mercedes Serabian holds an MS degree in Toxicology from American University and is a Diplomat of the American Board of Toxicology (DABT). She currently serves as Chief of the Pharmacology/Toxicology Branch in the Office of Cellular, Tissue, and Gene Therapies (OCTGT) in the Center for Biologics Evaluation and Research (CBER) at the USFDA. She is responsible for overseeing the pharmacology/toxicology review, regulation, and policy development for cellular and gene therapy products submitted to FDA. She provided expert pharmacology/toxicology advice on FDA guidance documents such as cancer vaccines, cellular therapies for cardiac disease, cartilage repair/replacement products, long-term follow-up of subjects administered gene therapy products, and viral shedding. Ms. Serabian championed the Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products, outlining FDA recommendations on preclinical data to support clinical studies of cellular and gene therapy products. She has participated in several expert working groups under the International Conference for Harmonisation and has presented to outside parties on preclinical regulatory considerations for cellular and gene therapy products intended for administration in clinical trials.