Submitted by Z. Alex Xu, PhD, DABT, AACT 2017–2018 President
The American Association of Chinese in Toxicology (AACT) is pleased to invite you to a webinar presentation to be held on November 14, 2017, from 1:00 pm to 2:00 pm, Eastern. Register to participate.
Title: Preclinical Considerations for Gene Therapy Products: An FDA Perspective
Presenter: Ying Huang, PhD, Office of Tissues and Advanced Therapies (OTAT), Center for Biologics Evaluation and Research (CBER), US Food and Drug Administration (US FDA)
Description: The US FDA/CBER has a long history of regulating gene therapy (GT) products. The regulatory approach for assessing the safety and effectiveness of individual GT products can vary based on the biological properties of each respective product. There is no standard set of preclinical studies and testing parameters that are uniformly applicable to all GT products. The regulatory review of GT products employs a science-based approach that considers the benefits and risks of each investigational product in the framework of the respective clinical trial. The introduction of gene editing (GE) technologies in this generation of GT products raises unique concerns regarding how the GE process may impact the product safety profile. This presentation will include an overview of CBER preclinical considerations for GT products, with a focus on specific safety concerns for GT products that incorporate GE technologies and the types of preclinical evaluations conducted for these products to support administration in human clinical trials.
Speaker Biography: Dr. Huang has been a Pharm/Tox master reviewer at US FDA/CBER/OTAT/DCEPT since 2004. She is responsible for the review of the preclinical evaluations for proof-of-concept and safety related to cell and gene therapies, and gene editing derived gene therapy products on oncology and non-oncology diseases. Her review activities have been involved in submissions of Investigational New Drug (IND) applications, including pre-preIND and pre-IND, and Biologics License Application. In addition to her review work, she is one of the representatives for the US FDA in the International Pharmaceutical Regulators Forum/ Gene Therapy Working Group. She was the co-representative for US FDA/CBER in the Interagency Coordinating Committee on the Validation of Alternative Methods during 2006– April 2017. Prior to the US FDA, Dr. Huang received her PhD degree in Pharmacology and Toxicology at the University of Toronto, Canada, and subsequently an National Institutes of Health (NIH) Intermural Research Training Award fellowship at NIH/National Institute of Diabetes and Digestive and Kidney Diseases before becoming a senior scientist at the former Genetic Therapy Inc., a Novartis Company.
Moderator: Z. Alex Xu, PhD, DABT, Chief Scientist in Pharmacology and Toxicology, US FDA/CDER
We look forward to your participation. This webinar is scheduled for scheduled for ~60 minutes, including 10–15 minutes for questions. Questions from the audience participating online are encouraged.