Building a blueprint for rare disease gene therapies

Join us to learn about a father’s journey to find a cure for AP-4, a rare genetic disorder that causes a range of neural and musculoskeletal disabilities. Starting with a search for more information in scientific literature, over the course of several years, a team of scientists, physicians, and CDMOs was assembled to develop a plan for manufacturing and testing a clinical-grade AAV vector.

In this webinar we’ll discuss the successful early drug discovery strategy for a little-studied, rare genetic disorder. We’ll cover an overview of a pathway from “concept to clinic” for a gene therapy product using an AAV for AP-4 as a model system, as well as:

  • How AAV vectors using plasmids as a critical starting reagent are developed and manufactured
  • Gene therapy drug development for AP-4
  • Optimizing plasmids and viral vectors for accelerated gene therapy development

About the Presenters

Chris Edwards
Parent, Co-founder & President, Cure AP-4 Inc.

James Cody, PhD
Associate Director Technical Sales and Evaluations, Charles River