Regulatory Aspects of Gene Therapy and Cell Therapy 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 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.

• 1. Regulatory Oversight of Gene Therapy and Cell Therapy Products in the US - FDA/CBER.- 2. US Oversight of Gene Therapy Products – NIH RAC.- 3. Regulatory Oversight of Gene Therapy and Cell Therapy Products in Canada - Health Canada.- 4. Overview of the Regulatory Oversight Implemented by the French Regulatory Authorities for the Clinical Investigation of Gene Therapy and Cell Therapy Products.- 5. Regulation of Clinical Trials with Advanced Therapy Medicinal Products (ATMP) in Germany.- 6. EU Clinical Trials Regulatory Oversight of Advanced Therapy Medicinal Products (ATMP): Perspectives from Various Member States.- 7. Regulatory Oversight of Gene Therapy and Cell Therapy Products in Switzerland - Swissmedic.- 8. Regulatory Oversight of Gene Therapy and Cell Therapy Products in Japan - Japan Ministry of Health, Labour and Welfare (MHLW) and Pharmaceutical and Medical Device Agency (PMDA).- 9. Regulatory Oversight of Gene Therapy and Cell Therapy Products in Korea - Korean Ministryof Food and Drug Safety (MFDS).- 10. Regulatory Oversight of Gene Therapy and Cell Therapy Products in Taiwan - Taiwan Food and Drug Administration (TFDA).- 11. Regulatory Oversight of Gene Therapy and Cell Therapy Products in Singapore - Singapore Health Science Authorities (HSA).- 12. Regulatory Oversight of Gene Therapy and Cell Therapy Products in Brazil - National Health Surveillance Agency (ANVISA).