Preparing for the Future: A Gene Therapy Vector Production Platform

Performance Period: 4/15/2018 to 6/30/2019

The emergence of commercial gene therapy products will require a transition from bench-scale production of gene therapy vectors to manufacturing-scale operations. This project will develop a scalable platform process for production, purification, and analysis of adeno-associated virus (AAV) vectors used in gene therapy. This resource will be available for use throughout the NIIMBL network, and enable:

• development of hands-on courses in the area of gene therapy vector production; and,
• development and testing of new process and analytical technologies for vector production. 

In addition to these broad project goals, the following specific objectives will be met:

• Develop and offer a hands-on short course in gene therapy vector manufacturing that would be offered to NIIMBL members and the broader biopharmaceutical community.
• Test Sudhin Biopharma’s inclined settler technology for separation of cells and cell debris from liquid streams in the vector production process.