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The Basics of Gene Therapy: Preclinical Development

(2022 ACMG Summer Gene Therapy Education Series: Advancements in Gene Therapy Options for Rare Diseases – This course is supported by an independent medical education grant from Pfizer, Inc.)

ACMG Genetics Academy – Live Webinar

Session Description:

This session will review the basics of the preclinical development of a new gene therapy for a rare disorder. The presentation will span the bench to bedside spectrum with a focus on proof-of-concept and preclinical studies needed to initiate a clinical trial.

Target Audience:

This summer webinar series is targeted to genetics professionals in research, lab, and clinical settings, as well nongenetic healthcare professionals caring for patients with rare disease.

 Learning Objectives:

At the conclusion of this session, participants should be able to:

• Describe key aspects of preclinical studies prior to Investigational New Drug (IND) application
• Review the key steps in the interaction with the FDA prior to submitting an IND application
• Explain the relevance of an IND number

Moderators:

Lindsay C Burrage, MD, PhD, FACMG

Assistant Professor, Molecular & Human Genetics

Baylor College of Medicine

Dwight Koeberl, MD, PhD, FACMG

Professor, Department of Pediatrics/Division of Medical Genetics, and Department of Molecular Genetics and Metabolism, Medical Director, Biochemical Genetics Laboratory

Duke University Medical Center

Presenters: