Ottawa Hospital Research Institute

Purpose

The Project will accelerate the rate at which the NRC’s Lipoprotein lipase deficiency (LPLD) AAV-based gene therapy technology is GMP manufactured and readied for testing in the clinic, by formally combining the efforts, experience and expertise of the National Research Council and the Biotherapeutics Manufacturing Centre- Virus Manufacturing Facility (BMC-VMF) at the Ottawa Hospital Research Institute (OHRI). The project will result in the finalization of upstream and downstream process parameters leading to the execution of a non-GMP run of the complete, finalized process. Importantly, this non-GMP run will generate material that is suitable and required for a pivotal GLP toxicology study while serving to validate the feasibility of the overall process (at reduced scale) at the BMC