The Antibody Question: How Does it Impact Trial Enrollment?

Antibodies play a major role at several stages of GT trials and can be cause for concern among many rare disease families especially given the current inability to re-dose. Panelists will briefly discuss where the field is at scientifically with re-dosing and why pre-existing antibodies are so important from a clinical perspective. They will also discuss ethical issues related to antibodies such as the utility of antibody testing outside of clinical trials and how decisions around viral vectors affects equity.
Moderator:
Lesha Shah, MD; Assistant Professor, Department of Child & Adolescent Psychiatry, NYU Grossman School of Medicine
Panelists:
Barry J Byrne, MD, PhD; Associate Chair of Pediatrics; Director, Powell Gene Therapy Center, University of Florida
Gregory de Hart, PhD; Director, BioMarin Pharmaceutical, Inc
Michelle Rengarajan, PhD, MD; Assistant in Medicine, Mass General Hospital; Instructor in Medicine, Harvard Medical School