One of the most exciting promises of cell and gene therapies is their potential to tailor treatment to individuals and thus improve the lives of those with rare diseases. However, developing these therapeutics blur the traditional line between treatment and research and therefore invite a number of important ethical questions. This panel will ask: How should we think about the development of these novel medical interventions—are they research or treatment? How should the development of such individual therapeutics be regulated? How can we work towards equitable access to these exciting new interventions?
Featuring Panelists:
Alison Bateman-House, MPH, PhD (Assistant Professor of Population Health, NYU Grossman School of Medicine)
Alison Bateman-House is an assistant professor in the department of population health at NYU Grossman School of Medicine. Dr. Bateman-House’s research is primarily focused on ethics and policy issues concerning access to investigational medical products, both inside and outside of clinical trials. One particular area of interest is in the equity of access: who gets access to what, how? Dr. Bateman-House also serves as the co-chair of the Working Group on Compassionate Use of PreApproval Access (CUPA).
Allison Bradbury, MS, PhD (Assistant Professor of Pediatrics, OSU & PI in the Center for Gene Therapy, Abigail Wexner Research Institute)
Allison M. Bradbury, MS, PhD, is an assistant professor in the Department of Pediatrics at The Ohio State University and a principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute. Dr. Bradbury is the PI of the Bradbury Laboratory, which is dedicated to understanding disease mechanisms and developing therapeutic approaches for rare, pediatric neurologic disorders. A primary focus of the Bradbury lab is to improve AAV targeting of myelinating cells in order to advance therapeutic outcomes for leukodystrophies and other white matter disorders.
