In an update issued November 27, 2019, Audentes Therapeutics, a biotech company that is focused on developing innovative gene therapy products, provided an update that they are on track to file an Investigational New Drug (IND) application in 2019 for their clinical development program in Pompe disease.
Audentes is currently testing candidate vectors in mouse-model studies (non-human) using an approach that aims to express GAA directly in the tissues affected by Pompe disease. The data generated in these studies will be used as part of a package of preclinical safety and efficacy information to support an Investigational New Drug (IND) filing.
Tagged: gene therapy, preclinical