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Preparing for the Future: A Gene Therapy Vector Production Platform

This project will develop a scalable platform process for production, purification, and analysis of adeno-associated virus (AAV) vectors used in gene therapy.
Categories
Cell and Gene therapies
Drug substance
Assays
Incumbent worker training
Project status
100% Completed

Industry Need

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.

Solution

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.

Outputs/Deliverables

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]

Impacts

Advancement of gene therapy manufacturing through platform process and skills training for adeno-associated virus vectors

Publications

Additional Project Information (Members Only)

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Project Lead

North Carolina State University

North Carolina State University

Participating Organizations

Sudhin Biopharma Co

Sudhin Biopharma Co

University of North Carolina, Chapel Hill

University of North Carolina, Chapel Hill