Lunch + Learn: Diving into Preclinical Studies

July 28, 2022

Researchers break down the preclinical models for gene therapies and a patient advocate shares direct examples of how communities can gather this data to further expand the knowledge around their disease and possible treatments.

Agenda

Introduction 
Rachel Bailey, PhD, Assistant Professor at UT Southwestern Medical Center 

The Preclinical Process 
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  
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