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European Biopharmaceutical Review

Proactive Progress

Although gene therapy is a promising tool within disease treatment, progress has been slow in developing effective clinical approaches. The issue lies in the difficulty to develop safe and efficient gene-delivery systems (1). An ideal vector system should deliver a certain amount of genetic material into the target cells. The transfer should be stable and cell-type-specific, allowing a high and controllable expression of the gene product without causing an immune response or other toxicity (2).

The revolutionary potential of adeno-associated viruses (AAV) is undisputed and underlined by the growing number of clinical trials using them as a central gene delivery tool. Recent groundbreaking success stories from clinical AAV applications have been reported in the media. The publication of one therapy successfully treating haemophilia B via liver-specific AAV vectors demonstrated their long-term potency, aside from a low number of temporary side effects (3). The first AAV FDA approval for an AAV-based therapy in history happened just before Christmas 2017, giving patients who suffer from bi-allelic RPE65 mutationassociated retinal dystrophy new hope that they may retain their eyesight (4). Impressive in their own right, these publications also help to uncover the secret to success when it comes to engaging in the development of any new AAV-based therapy: viral engineering – customising existing virus models to fit the specific goals of the therapeutic approach. More precisely, both the genetic strategy itself – in terms of plasmid manufacturing – and the viral capsid carrying it to the designated cell type or tissue need to be optimised to fit every aspect of their intended application, including the determination of the correct titre of the gene shuttle (see Figure 1).

A Solid Start

Among other applications, plasmid DNA is often used as starting material in the Good Manufacturing Practicecompliant (GMP) production of recombinant viruses, antibodies, and RNA, where these are the API used in clinical trials. In many cases, producing the plasmid DNA under GMP conditions is not necessary. An alternative is the highquality grade plasmid DNA, which is both highly purified and well-characterised, thus meeting the requirements of most regulating agencies (5). High-quality grade plasmid DNA is produced based on a research cell bank and the patented ccc-grade DNA technology. A number of quality controls, both to the cell bank and to the plasmid DNA product, ensure that the final result is a product designed especially for the intended application that complies with the appropriate regulatory standards (5).

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Tatjana Buchholz holds degrees in molecular biotechnology (BSc) and genome-based systems biology (MSc) from the Bielefeld University, Germany, and is an examined medical technical assistant. Her bachelor’s thesis was occupied with signal studies of the promoter activity of the nuclear LHCII translational repressor NAB1 in the microalgae Chlamydomonas reinhardtii. Tatjana's master’s thesis was concerned with MC technology optimisation. She joined PlasmidFactory in Bielefeld, Germany, as Marketing Manager in 2016. 

Dr Carl J Christel has a strong background in molecular physiology that has helped him advise, organise, and accompany gene engineering projects for commercial and academic clients. In 2009, he gained his PhD from the Department for Pharmacology and Toxicology at the Technical University, Munich, and completed four years of post-doctorate work at the Department of Molecular Physiology and Biophysics at the University of Iowa, US. Since 2013, Carl works in the commercial biotech sector as Senior Manager for Sales and Marketing at Sirion Biotech in Munich, Germany.

Dr Hüseyin Besir studied chemistry at the Ludwig Maximilian University in Munich, Germany. His dissertation at the Max Planck Institute of Biochemistry in Martinsried focused on lipid induced crystallisation of halophilic archaeal rhodopsins. After his post-doctoral work at Roche Diagnostics in Penzberg, Hüseyin became sub-project leader of the EU-funded project Interaction Proteome at the Max Planck Institute in Munich and later Head of the Protein Expression and Purification Core Facility at EMBL in Heidelberg. In 2016, he joined PROGEN Biotechnik in Heidelberg as Head of R&D.
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Tatjana Buchholz
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Dr Carl J Christel
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Dr Hüseyin Besir
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