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Friday, June 18 • 12:00pm - 1:00pm
#522 OD: Gene Therapy Research in Pediatric Populations: Ethical Issues

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Component Type: Forum
Level: Basic

Many gene therapy trials enroll pediatric patients. Clinical research in children raises discrete ethical challenges, as does clinical research involving gene therapy; this forum identifies these challenges and identifies relevant guidance documents.

Learning Objectives

Identify the primary ethical challenges facing pediatric gene therapy trials; Assess which ethical challenges are common to all clinical research in children versus which are unique to gene therapy trials in this population; Recognize existing ethical guidance on these issues.


Alison Bateman-House, PhD, MA, MPH


Rafael Escandon, DrPH, PhD, MPH, MS

Alexandra Hall, MA

Lesha D. Shah, MD, FAPA

avatar for Alison Bateman-House

Alison Bateman-House

Assistant Professor, Division of Medical Ethics, Dept of Population Health, NYU Grossman School of Medicine, United States
Alison Bateman-House, PhD, MPH, MA, is an assistant professor in the Division of Medical Ethics at NYU Grossman School of Medicine and a Professor of Practice in Law at University of Arizona James E. Rogers College of Law. She is co-chair of the Working Group on Compassionate Use... Read More →
avatar for Lesha D. Shah

Lesha D. Shah

Assistant Professor; Medical Director, Icahn School of Medicine at Mount Sinai, United States
Dr. Shah is Assistant Professor of psychiatry and the Medical Director of Child, Adolescent & Family Services at the Icahn School of Medicine at Mount Sinai, with dual board certification in psychiatry and child and adolescent psychiatry. She co-chairs the Pediatric Gene Therapy... Read More →
avatar for Rafael Escandon

Rafael Escandon

Senior Vice President Medical Affairs, Policy and Patient Engagement, Bridgebio Gene Therapy, United States
Rafael has been involved in clinical research and development for the past 25 years and developed interests in public health ethics, justice and human rights while conducting clinical trials in the developing world. He believes that sponsors and investigators alike owe great debts... Read More →

Alexandra Hall

Managing Director: Policy, Patient Support, and Industry, The Isaac Foundation, Canada
Alix is the Managing Director of Policy, Patient Support and Industry Liaison at The Isaac Foundation and Equal Access for Rare Disorders, where her work focuses on advocating for patients’ safe and ethical access to treatment, and managing patient education and rare disease awareness... Read More →

Friday June 18, 2021 12:00pm - 1:00pm EDT
TBD Virtual Event Horsham, PA 19044
  06: PreClin Dev-EarlyPhaseCR, Forum |   20: Early Access Content, Forum