The FDA's Role in Gene Therapy

Описание: The potential for gene therapy products to change the lives of patients with debilitating or terminal conditions provides hope for the future. Gene therapy research is exploding at a rapid pace and a number of products are advancing in clinical development. The Center for Biologics and Evaluation Research (CBER) at the Food and Drug Administration (FDA) supports progress in gene therapy and helps expedite new product development for unmet medical needs using review pathways designed to advance innovative, safe and effective treatment options.

Dr. Peter Marks, Director of CBER, and Julie Tierney, Senior Policy Advisor for Strategic Planning and Legislation at CBER, discuss regulatory pathways for gene therapies, how gene therapies are deemed safe, specific indications for gene therapies and more.

Webinar Speakers
Peter Marks, MD received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in January 2016.

Julie Tierney, JD advises the CBER Center Director and senior staff on a wide range of issues related to regulatory policy development, implementation of statutory mandates, and legislative strategy. She joined FDA in 2008 as an Associate Chief Counsel for Drugs in FDA’s Office of the Chief Counsel and, immediately prior to her current role, served as FDA’s detailee to the Senate Health, Education, Labor & Pensions Committee during negotiation and enactment of the 21st Century Cures Act. Before working at FDA, Ms. Tierney practiced food and drug law at private law firms. She received her J.D. from Georgetown University Law Center and her undergraduate degree from Johns Hopkins University.

Katie Kowalski, MPH is the Senior Manager for NORD’s Educational Initiatives. In that role, she works to develop and implement educational programs for medical professionals, patients and their families. She serves as a Program Manager for key programs within NORD’s Education Department including continuing medical education, the educational webinar series and student programs.