Press Information 04 December 2012 Adeno-associated virus now easily available for research and preclinical studies. Munich, Bielefeld, Heidelberg (Germany), SIRION Biotech and PlasmidFactory agreed to actively promote adeno-associated virus (AAV) vectors for advanced preclinical research. Dr. Oliver Mueller, University of Heidelberg, will serve as scientific advisor. AAV vectors have been identified as most promising gene delivery candidates for curative therapy of gene defects that need long-term treatment. It is estimated that over 4000 human diseases are caused by single gene defects. Besides acting as highly versatile gene delivery agents they also serve for functional genomic studies. To date 44 clinical trials are being conducted with AAV for diseases like Parkinson, Alzheimer or Stage IV Gastric cancer. The first AAV vector-based gene therapy for lipoprotein lipase deficiency (LPLD) has been approved in Europe for Human Use just a few weeks ago. Generating AAV, especially for use in preclinical and clinical applications requires a mix of skills: plasmid DNA in reproducible and certified quality both in research and clinical grades, solid experience in generating AAV vectors for preclinical use and the ability to generate entire cell models according to desired specifications. This week’s agreement between PlasmidFactory and SIRION Biotech allows for commercial supplies of highly innovative AAV vectors for preclinical research and up to clinical phase III studies. Within just 8 weeks SIRION Biotech is able to supply research quantities for in vivo studies in small animals. Working with AAV vectors for many more clinical trials is now made a realistic option for researchers worldwide. About SIRION BIOTECH, PlasmidFactory and PD Dr. Oliver Müller SIRION Biotech was founded in 2006 in Munich and has sites in Tokyo and New Hampshire. It stands for sophisticated cell modelling and viral vector platforms. These enable much improved target identification and compound screening in the drug, the food & cosmetic industries. Its technology has over the years been validated in more than 250 commercial projects with more than 70 academic and industrial organizations worldwide. As a result, primary cell immortalizations are being technically advanced, so is the recombinant virus vector construction serving for gene therapy applications and novel vaccines. PlasmidFactory is a biopharmaceutical company, founded in Bielefeld in 2000. Today the company is formed by a young team of biologists, chemists, engineers and technicians and is Europe's leading contract manufacturer of plasmid DNA. The company produces clients’ plasmids according to their requirements (incl. GMP use) in modern laboratories with high quality standards. PlasmidFactory holds the worldwide exclusive license from DKFZ for manufacturing, distribution and use of the Helper 1 & Packaging Plasmids from the pDG and pDP family, respectively . Dr. Oliver Mueller leads a team at the Dept. of Internal Medicine III at the University of Heidelberg whose aim is to develop gene therapies for hereditary heart diseases. The department’s research is addressing chronic diseases by close interaction of medical and biology basic research. Contact: SIRION BIOTECH GmbH Dr. Christian Thirion Am Klopferspitz 19 D-82152 Martinsried Tel.: +49-89-700 961 99-15 eMail: [email protected] www.Sirion-Biotech.com PlasmidFactory GmbH & Co. KG Dr. Martin Schleef Meisenstr. 96 D-33607 Bielefeld Tel.: +49-521-299 7350 eMail: [email protected] www.PlasmidFactory.com 1 References: [1] Grimm et al. (1998), Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors, Human Gene Therapy, Vol. 9, 2745-2760. [2] Raake PWJ, Schlegel P, Ksienzyk J, Reinkober J, Barthelmes J, Schinkel S, Pleger S, Mier W, Haberkorn U, Katus HA, Koch WJ, Most P, Müller OJ. AAV6.βARKct cardiac gene therapy ameliorates cardiac function and normalizes the catecholaminergic axis in a clinically relevant large animal heart failure model. Europ Heart J. Jan 12. (2012) [Epub ahead of print] [3] Grimm et al. (2003), Helper Virus-Free, Optically Controllable, and Two-Plasmid-Based Production of Adeno-associated Virus Vectors of Serotypes 1 to 6, Molecular Therapy, Vol. 7, 839-850. [4] Kronenberg et al. (2005), A Conformational Change in the Adeno-Associated Virus Type 2 Capsid Leads to the Exposure of Hidden VP1 N Termini, Journal of Virology, Vol. 79, 5296-5303. [5] Moullier, P. and Snyder, R.O. (2008), International efforts for recombinant adenoassociated viral vector reference standards, Molecular Therapy, Vol. 16, 1185-1188 [6] AAV-DNA helper sequences EP 0 934 423