
Thursday, June 9, 2022 | 4:30 pm – 5:30 pm ET
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