Emerging Platform Bioprocesses for Viral Vectors and Gene Therapies.

BioProcess International 14(4)s.

Emerging Platform Bioprocesses for Viral Vectors and Gene Therapies.

Pettitt DA, Smith J, Fuzerstenau-Sharp M, Holländer G, Predki P, Slade A, Jones P, Weed L, Bure K, Brindley DA.



Recent advances in molecular biology are expediting genomic sequencing to underpin precision medicine. Such progress is positioning gene and gene-modified cell therapy on the cusp of an extraordinary revolution in patient care for presently unmet medical needs — and a new therapeutic class that could rival monoclonal antibodies (MAbs) in importance. However, despite substantial strides made in clinical trials, the bioprocessing community is struggling to fulfill growing demands for biomanufacturing capacity to make gene and gene-modified cell therapies — including current good manufacturing practice (CGMP) viral vectors.

Here we review the basic science of gene therapy bioproduction and evaluate critical emerging bioprocess opportunities and challenges. An interrelationship exists between viral-vector upstream and downstream bioprocessing strategies and those for other therapeutic platforms such as chimeric antigen receptor (CAR) T cells and induced pluripotent stem cells (iPSCs).