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Review
. 2016 Apr 15;25(R1):R42-52.
doi: 10.1093/hmg/ddv451. Epub 2015 Oct 30.

Progress and challenges in viral vector manufacturing

Johannes C M van der Loo  1 , J Fraser Wright  2
Affiliations
Review

Progress and challenges in viral vector manufacturing

Johannes C M van der Loo et al. Hum Mol Genet. .

Abstract

Promising results in several clinical studies have emphasized the potential of gene therapy to address important medical needs and initiated a surge of investments in drug development and commercialization. This enthusiasm is driven by positive data in clinical trials including gene replacement for Hemophilia B, X-linked Severe Combined Immunodeficiency, Leber's Congenital Amaurosis Type 2 and in cancer immunotherapy trials for hematological malignancies using chimeric antigen receptor T cells. These results build on the recent licensure of the European gene therapy product Glybera for the treatment of lipoprotein lipase deficiency. The progress from clinical development towards product licensure of several programs presents challenges to gene therapy product manufacturing. These include challenges in viral vector-manufacturing capacity, where an estimated 1-2 orders of magnitude increase will likely be needed to support eventual commercial supply requirements for many of the promising disease indications. In addition, the expanding potential commercial product pipeline and the continuously advancing development of recombinant viral vectors for gene therapy require that products are well characterized and consistently manufactured to rigorous tolerances of purity, potency and safety. Finally, there is an increase in regulatory scrutiny that affects manufacturers of investigational drugs for early-phase clinical trials engaged in industry partnerships. Along with the recent increase in biopharmaceutical funding in gene therapy, industry partners are requiring their academic counterparts to meet higher levels of GMP compliance at earlier stages of clinical development. This chapter provides a brief overview of current progress in the field and discusses challenges in vector manufacturing.

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Figures

Figure 1.
Figure 1.
Progression of cGMP compliance at different stages of product development: illustration of the increase in the level of compliance with cGMP as products progress from a pre-clinical stage to the Biological License Application and Commercial Manufacturing.
See this image and copyright information in PMC

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