Manufacturing and testing a clinical-grade AAV vector: from concept to clinic

AP-4 is a hereditary spastic paraplegia which is caused by one of four specific genetic mutations. Collectively known as AP-4 deficiency syndrome, this rare genetic disorder causes a range of neural and muscular developmental disabilities. Given the rarity of AP-4 HSP, it is not well-studied, and there are no established treatments. However, due to the nature of the genetic mutations that cause AP-4, this disease may be amenable to a gene therapy-based approach.

In this webinar, the father of an AP-4 patient will describe his journey to find a cure for this disease. Starting with a search for more information in the 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.

This webinar will provide an overview of a pathway from “concept to clinic” for a gene therapy product, using an AAV for AP-4 as a model system. It will include a description of how AAV vectors, using plasmids as a critical starting reagent, are developed and manufactured.

View this webinar for insights into:

• A parent's journey to find a cure for AP-4
• A successful early drug discovery strategy for a little-studied, rare genetic disorder
• Gene therapy drug development for AP-4
• Optimizing plasmids and viral vectors for accelerated gene therapy development

Chris Edwards

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

Chris is a Serial Entrepreneur and founded or co-founded 8 companies so far and one of them among is Cure AP-4 Inc., Alternative Therapies Group, Inc. and Canna Drinks. Chris is serving as a Board member for Novel Beverage Co. as well.

James Cody

Associate Director Technical Sales and Evaluations, Charles River Laboratories

Since 2018, James has worked in the viral vector CDMO space for Vigene Biosciences, which was acquired by Charles River in 2021, and is a member of the business development team based at Rockville’s (MD) Viral Vector manufacturing site. In his current role, James provides technical support to the business development team, helping to onboard new projects by working in close collaboration with subject matter experts across various functional groups.

James received his Ph.D. in cancer gene therapy from the University of Alabama (UAB), Birmingham, and completed his postdoctoral training, studying two viral vector systems (adenovirus and HSV). Afterwards, he worked as a scientist conducting research in virology, cell biology, cancer, and parasitology, for four years.

Watch now

SPEAKERS

Chris Edwards

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

James Cody

Associate Director Technical Sales and Evaluations, Charles River Laboratories

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