MANUFACTURING OF PLASMID AND VECTOR AND GLP DOSE-ESCALATION TOXICOLOGY STUDIES

Battelle Centers/Pub Hlth Res & Evaluatn, Columbus OH

Investigators

Abstract

Currently, the National Institute of Neurological Disorders and Stroke (NINDS), via its URGenT network, is funding a cooperative agreement for the manufacturing of an AAV9 vector, plasmids, and GLP dose-escalating studies as a potential therapy the severe and progressive genetic neurological disorder, Aspartylglucosaminuria (AGU). The goals of the URGenT cooperative agreement are to manufacture the vector, plasmid, and initial GMP batch.