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Tuesday, June 21 • 11:15am - 12:15pm
#228: Individualized Therapeutics: Walking the Line Between Research and Treatment

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Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-22-559-L04-P ; CME 1.00; RN 1.00

This multidisciplinary panel introduces the most pressing novel ethical considerations facing individualized therapeutics (antisense olgionucleotide) for those with ultra-rare genetic diseases, and suggests ways to address these challenges. Join the DIA Patient Engagement Community for a follow up round table discussion (session #237 RT) on Tuesday, June 21, 12:30pm - 1:30pm.

Learning Objectives

Describe the ethical challenges facing the emerging field of individualized anti-sense oglionucleotide (ASO) therapeutics; Discuss the overlapping roles of treatment and research in individualized therapeutics; Identify ways in which individualized therapies require unique regulatory and development considerations.


Alison Bateman-House, PhD, MA, MPH


James P. Smith, MD, MS

Sarah Glass, PhD

Timothy W. Yu, MD, PhD

Edward Neilan, MD, PhD

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 →

Sarah Glass

Chief Development Officer, N-Lorem Foundation, United States
Sarah Glass, PhD is the Chief Development Officer at n-Lorem Foundation. She has over twenty years of experience as a strategic leader in rare diseases across academic, pharmaceutical, and clinical research organizations. Sarah marries her professional experience with her passion... Read More →
avatar for Edward Neilan

Edward Neilan

Chief Medical and Scientific Officer, National Organization for Rare Disorders (NORD), United States
Dr. Neilan is a physician-scientist and rare disease expert with experience in both academic medicine and the pharmaceutical industry. His expertise spans laboratory research, medical practice, as well as clinical trial design and execution across all phases of development. After... Read More →
avatar for James Smith

James Smith

Deputy Director, Office of New Drug Policy, OND, CDER, FDA, United States
Dr. Smith is deputy director of the Office of New Drug Policy in the Office of New Drugs (OND), with his primary focus being clinical and scientific policy priorities. He was formerly deputy division director of the Division of Metabolism and Endocrinology Products, overseeing development... Read More →
avatar for Timothy Yu

Timothy Yu

Associate Professor, Harvard Medical School; Division of Genetics and Genomics, Boston Children’s Hospital, United States
Dr. Tim Yu, MD, PhD is a neurologist in the Division of Genetics and Genomics at Boston Children’s Hospital, Associate Professor at Harvard Medical School, and an Associate Member of the Broad Institute. His research group applies genetics, neurobiology, and bioinformatics to study... Read More →

Tuesday June 21, 2022 11:15am - 12:15pm CDT
Room 183 B McCormick Place 2301 South Indiana Avenue Gate 40 Chicago, IL 60616
  06: PreClinical Dev-EarlyPhaseCR, Forum