Description
Overview of Gene Therapy with a focus on AAV based therapies. We will look at major differences in preclinical safety evaluations, such as small molecule vs gene therapy. Some of the key study components will be reviewed with the audience for knowledge of FDA requirements to get to an IND approved study design. Gene editing, biodistribution assay development, along with updates and program designs in the history of preclinical development in 15 years of gene therapy at Lovelace Biomedical.
Learning Objectives:
- How are preclinical programs run for gene therapy?
- What does FDA require to approve your study?
- What is the history of gene therapy and updates on running studies in the field?
- How does it all come together to get to the IND stage?
Contributors
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Edward (Ted) Barrett, Ph.D.
Ted Barrett, Ph.D., is Senior Director, Pharmacology at Lovelace Biomedical. Dr. Barrett has spent most of his career investigating the effects of small molecule, biologic, nucleic acid and medical device technologies in various animal models of disease. His programs aim to translate nonclinical efficacy, pharmacokinetic, and pharmacodynamic data obtained by from species ranging from rodents to nonhuman primates to the human clinical paradigm. Dr. Barrett received his doctorate in toxicology from the University of Rochester and joined Lovelace in 1999.
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Janet Benson, Ph.D.
Dr. Benson has been a toxicologist at Lovelace for 42 years. She has investigated the toxicity, PK, or biodistribution of metals, solvents, polymers, natural products, select agents, vesicants and gene therapeutics. As a member of the NHLBI Gene Therapy Resources Program, Pharmacology/Toxicology core for over 11 years, she has assisted investigators in the development of their pre-clinical safety and biodistribution studies to support IND applications. She has also participated in preparation and/or review of INTERACTor pre-IND packages and participated in multiple, associated meetings with investigators and the FDA.
Dr. Benson holds a BS degree in Chemistry from the University of California, Berkeley and a PhD in Comparative Pharmacology and Toxicology from UC, Davis. She was a Diplomate of the American Board of Toxicology from 1981 - 2017. She has over 100 publications in the scientific literature. She currently serves on the US Transuranium Uranium Registry Scientific Advisory Committee and has served on scientific advisory Committees (National Academy of Sciences, the EPA IRIS review Committee, and American Conference of Industrial Hygienists Threshold Limit Value Committee).