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: