Sirion Biotech GmbH: Adeno-associated virus now easily available for research and preclinical studies

(DGAP-Media / 04.12.2012 / 10:15) 
 
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 & Packaging Plasmids 
from the pDG and pDP family, respectively1. 
 
 
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.: [phone] 
eMail: [e-mail] 
www.Sirion-Biotech.com 
 
PlasmidFactory GmbH & Co. KG 
Dr. Martin Schleef 
Meisenstr. 96 
D-33607 Bielefeld 
Tel.: [phone] 
eMail: [e-mail] 
www.PlasmidFactory.com 
 
 
 
1References: 
[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, Muller OJ. AAV6.ßARKct 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 
 
 
End of Media Release 
 
=-------------------------------------------------------------------- 
 
Issuer: Sirion Biotech GmbH 
Key word(s): Health 
 
04.12.2012 Dissemination of a Press Release, transmitted by DGAP - a 
company of EquityStory AG. 
The issuer is solely responsible for the content of this announcement. 
 
DGAP's Distribution Services include Regulatory Announcements, 
Financial/Corporate News and Press Releases. 
Media archive at www.dgap-medientreff.de and www.dgap.de 
 
=-------------------------------------------------------------------- 
 
 
195744 04.12.2012 
 

(END) Dow Jones Newswires

December 04, 2012 04:15 ET (09:15 GMT)