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

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.
Specifics of the process will come during the tech transfer described in the base task. The final product
qualification and characterization testing will include assays of sterility, endotoxin, purity, identity,
activity/potency, vector genome titer, capsid particle titer, appearance, and genomic structure. Upon
establishment of the vectors, and optimal characterization, this project will progress to IND-enabling toxicology studies to support the IND.

Grant Number: 75N95022D00021-P00001-759502300003-1
NIH Institute/Center: NIH
Principal Investigator: Amanda Burnaugh