Current Gene Therapy - Volume 10, Issue 6, 2010

As the successes of Gene Therapy trials become ever more frequent so does the need to develop production processes allowing faster transfer into the clinical development phases. The Gene Therapy field is significantly pulverised, with many different vectors being currently available. Furthermore, new developments on vector design and safety as well as in vector production and purification technologies raise new chal Read More

In the past 5 years, European investigators have played a major role in the development of clinical gene therapy. The provision of substantial funds by some individual member states to construct GMP facilities makes it an opportune time to network available gene therapy GMP facilities at an EU level. The integrated coordination of GMP production facilities and human skills for advanced gene and genetically-modified (GM) cell th Read More

The use of recombinant adeno-associated viral vectors (rAAVs) as gene transfer tools has increased dramatically during the past several years, establishing AAV as the vector of choice for many therapeutic applications. With the steady advance of the field toward clinical studies, and the isolation and engineering of several novel AAV serotypes, efficient, scalable, and versatile production and purification methods are conti Read More

Replication deficient adenovirus vectors are frequently used tools for the delivery of transgenes in vitro and in vivo. In addition, several therapeutic products based on adenovirus are under clinical development. This review outlines adenovirus vector production discussing different vector types, available production cell lines and state of the art of production process development and purification.

Retroviral vectors are presently amongst the most widely used vectors in gene therapy clinical trials to target pathologies of different origins, such as cancers, genetic diseases or neurological disorders. This review provides an overview on the evolution of retroviral vector design and production for gene therapy applications, including state of the art developments in flexible producer cells and safe vectors. In addition, production Read More

Lentiviral vectors become more and more famous for the use as gene vector for gene therapy purposes for the treatment of acquired or inherited diseases. In this review, the present state of the art of the production of lentiviral vectors is presented with particular emphasis on the large scale production of these vectors for preclinical and clinical purposes. In contrast to oncoretroviral vectors which are produced using stable pr Read More

After some decades of research, development and first clinical approaches to use DNA vectors in gene therapy, cell therapy and DNA vaccination, the requirements for the pharmaceutical manufacturing of gene vectors has improved significantly step by step. Even the expression level and specificity of non viral DNA vectors were significantly modified and followed the success of viral vectors. The strict separation of “viral” a Read More

To be able to produce advanced therapy medicinal products, compliance with regulatory standards while maintaining flexibility is mandatory. For this purpose, careful planning is vital in the design or upgrade of a facility. Similarly, extensive foresight is elemental to anticipate upcoming needs and requirements. Failing this may lead to the facility's inability to meet the demands. In this chapter we aim to outline the current issue Read More