Regulatory Aspects of Gene Therapy and Cell Therapy Products

Advances in Experimental Medicine and Biology

Book 871
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This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations.

Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

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About the author

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 Sanità, 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.

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Additional Information

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Published on
Sep 15, 2015
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Medical / Clinical Medicine
Medical / Genetics
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The circulatory system is usually considered to be composed of tubes of various diameters, characterized by collateral and terminal branches. There is also a tendency to treat blood vessels merely as conducting tubes in which the various structures of the wall act as mechanical pumps wlrich modify their diameter. This is, of course, not so. In fact, we know that blood vessels, and in particular arteries, are organs with personalities of their own and a particular susceptibility to several diseases. In addition, blood vessels differ in structure, according to their localization, and age at differing rates. The experimental work car ried out so far clearly confirms the data that have come from spontaneous human pathology; experimentally induced arterial lesions have a definite tendency to appear in certain arteries and not in others, depending on the experimental procedures used, and in each specific artery the lesions appear to have a specific location. We now know that the arterial wall is a metabo licallyactive structure, in which a number of enzyme activities have been clearly demonstrated. It possesses a sensitive vasa vasorum apparatus and a specific reactivity to various lesion-inducing stimuli. We must also remember that the arterial wall is in continuous contact with the blood circulating through the endothelial cells lining the vascular bed. It is obvious, therefore, that any variation in the circulating blood mass can modify the morphology as well as the function of the vessel wall.
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