Agenda
Introduction
12-12:05 p.m.
Rachel Bailey, PhD, Assistant Professor at UT Southwestern Medical Center
The Preclinical Process
12:05-12:25 p.m.
Allison Bradbury, PhD, Principal Investigator and Assistant Professor, Nationwide Children’s Hospital
Dr. Allison Bradbury will give an overview of the various models for preclinical studies that are conducted to develop a gene therapy, and how route of delivery changes based on the model used. Attendees will learn about the process behind generating sufficient efficacy and safety data to enable an IND submission and get to a clinical trial.
The Patient Advocate Role
12 :25-12 :45 p.m.
Kim Nye, Founder, President, and Executive Director of the TESS Research Foundation
Kim Nye will share how foundations and advocate communities can enable and help the preclinical process. Attendees will learn specific examples from Kim’s experience with the TESS Research Foundation on how they can affect and help the preclinical process for studies on rare disease.
Moderated Q&A
12:45 p.m.- 1:00 p.m.
Thank You to Our Sponsors
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