The emergence of commercial gene therapy products requires a transition from bench-scale production of gene therapy vectors to manufacturing-scale operations. This transition necessitates training – of operators, scientists, engineers, and regulators – in manufacturing methods for gene therapy vectors.
A model system/process (i.e., platform) for production of gene therapy vectors would create a resource to (1) enable development and testing of new process and analytical technologies and (2) enable development of hands-on courses in the area of gene therapy vector production.
Advancement of gene therapy manufacturing through platform process and skills training for adeno-associated virus vectors
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 (1) development of hands-on courses in the area of gene therapy vector production and (2) development and testing of new process and analytical technologies for vector production.
Suspension cell line for teaching and research that has been optimized for vector – adeno-associated virus (AAV) – production and is accessible to NIIMBL members. [A HEK293 cell line adapted to growth in suspension, without serum, is available. Optimization for AAV2 production ongoing.]
Protocols for all process steps and analytical assays made available to NIIMBL members.
Report detailing use of Sudhin Biopharma Co.’s BioSettler 150 in the AAV2 process.
Short course titled “Hands-on GMP Manufacturing of Vectors for Gene Therapy.” [First offering held May 13–16, 2019 at the Biomanufacturing Training and Education Center (BTEC) at NC State University]
Preparing for the Future: A Gene Therapy Vector Production Platform (SP1.0-002), NIIMBL Member Forum, Virtual, October 25, 2018.
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North Carolina State University
Sudhin Biopharma Co
University of North Carolina, Chapel Hill
