Alliance Webinar with FDA Director of CBER, Dr. Peter Marks

Gene therapy, including genome editing, has the potential to be transformational in the treatment of disease, particularly for rare conditions that may have no current treatments. Despite the potential, the field has evolved slowly and often with a lot of frustration expressed by FDA, companies, and patient groups about the challenges of developing a gene therapy and bringing it to market. There are known limitations in manufacturing, clinical development, and global regulatory alignment.

As the Director of the Center for Biologics Evaluation and Research (CBER), Dr. Marks’ leadership and vision are central to the development of gene therapies. For this webinar, Dr. Marks is going to present an overview of some of the current challenges and provide his perspective on how the field can move toward greater success and increased patient benefit.

Bio:

Peter Marks, M.D., Ph.D. is the director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration. The center is responsible for assuring the safety and effectiveness of biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies.

Dr. Marks and center staff are committed to facilitating the development of biological products and providing oversight throughout the product life cycle. Examples of these activities include:

• reviewing and providing advice during product development
• evaluating applications and making approval decisions based on safety and effectiveness data
• monitoring the safety of biological products
• conducting research that supports product development and characterization

"The center regulates and does research on complex biologic products that touch people's lives on a daily basis," says Dr. Marks. "Many of the products that we regulate are vital for promoting and protecting the public health, including vaccines, blood products, and tissues for transplantation. I'm very proud to lead a team of highly committed individuals whose efforts help to ensure the timely development of safe and effective products to meet important medical needs."

Dr. Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University. Following this, he completed an Internal Medicine residency and Hematology/Medical Oncology fellowship at Brigham and Women's Hospital in Boston, where he subsequently joined the attending staff as a clinician-scientist and eventually served as Clinical Director of Hematology.

He then moved on to work for several years in the pharmaceutical industry on the clinical development of hematology and oncology products prior to returning to academic medicine at Yale University where he led the Adult Leukemia Service and served as Chief Clinical Officer of Smilow Cancer Hospital. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in 2016. Dr. Marks is board certified in internal medicine, hematology and medical oncology, and is a Fellow of the American College of Physicians. In 2022, he became a Member of the National Academy of Medicine, one of the highest honors in the fields of health, science and medicine.