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.
Deliverables:
1) 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.]
2) Protocols for all process steps and analytical assays made available
to NIIMBL members. [Completed]
3) Report detailing use of Sudhin Biopharma Co.’s BioSettler 150 in the AAV2 process. [Completed]
4) 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]
Advancement of gene therapy manufacturing through platform process and skills training for adeno-associated virus vectors
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North Carolina State University
Sudhin Biopharma Co
University of North Carolina, Chapel Hill
