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Wednesday, June 22 • 9:00am - 10:00am
#303: Grappling with Uncertainty: Pediatric Gene Therapy Trials and Ethics

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Component Type: Workshop
Level: Basic
CE: ACPE 1.00 Application UAN: 0286-0000-22-595-L04-P ; CME 1.00; RN 1.00

Gene therapy research in pediatric patients faces a number of ethical concerns. Trial sponsors must ensure that clinical trials are conducted ethically and balance stakeholder needs and priorities. This workshop will include facilitators from a multidisciplinary working group on ethical issues related to pediatric GT research and distribute realistic cases to groups.

Learning Objectives

Examine common ethical issues currently facing pediatric gene therapy research; Identify how different stakeholder communities may differ in their stances on these issues; Evaluate proposed responses on the part of sponsors.


Lesha D. Shah, MD, FAPA


Emma James, PhD

Cara Hunt, MA


Cara Hunt

Research Associate, NYU Grossman School of Medicine, United States
Ms. Hunt a staff an associate researcher in NYU Grossman School of Medicine’s Division of Medical Ethics. She has a demonstrated interest in translating topics of ethical concern into content of relevance to patient populations and increasing transparency of gene therapy clinical... Read More →
avatar for Emma James

Emma James

Vice President of Medical and Patient Affairs, Encoded Therapeutics, United States
Emma James is VP, Medical & Patient Affairs at Encoded Therapeutics, a biotech company developing gene therapies for CNS disorders. She obtained her PhD from the University of Oxford, after completing research at its Nuffield Department of Medicine and Wellcome Trust Centre for Human... 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 →

Wednesday June 22, 2022 9:00am - 10:00am CDT
Room 470 A McCormick Place 2301 South Indiana Avenue Gate 40 Chicago, IL 60616
  02: ClinTrials -ClinOps, Workshop |   06: PreClinical Dev-EarlyPhaseCR, Workshop