Navigation Links
VIRxSYS Publishes New Study on RNA Therapy
Date:4/6/2009

New RNA therapy reversed hemophilia in mice

GAITHERSBURG, Md., April 6 /PRNewswire/ -- VIRxSYS Corporation announced today the publication of a new research article, "Trans-splicing into Highly Abundant Albumin Transcripts for Production of Therapeutic Proteins In Vivo," in the journal Molecular Therapy. The study describes a novel RNA therapy technique in which an RNA molecule that is inserted into the albumin gene "hijacks" the albumin, causing it to produce the newly added gene's proteins instead of its own. Using this strategy, the researchers were able to produce three different therapeutic proteins in animals. With one of these, Factor VIII, they showed they could correct hemophilia A in mice. The researchers believe their technology can be used to produce therapeutic proteins in humans.

"Two of the RNA molecules that were spliced into albumin are normally produced in the liver. These are the apoA-I, which produces the principal protein in good cholesterol, and Factor VIII, which makes the protein missing in patients with hemophilia A. We instructed the liver to make more apoA-I protein than it normally does, and in separate experiments we instructed the liver to make the correct form of Factor VIII instead of a mutated form. We also added an RNA molecule that makes a monoclonal antibody that is not normally produced in liver," said VIRxSYS Executive Vice President of Scientific and Clinical Affairs, Gerard J. McGarrity, Ph.D. "The work is at an early stage, but we are definitely teaching old genes some new tricks."

Albumin is the most abundantly expressed gene in liver. Results showed that each of the three gene sequences spliced into the albumin gene sequence was expressed in high concentrations by liver cells in laboratory mice. The specific approach that is used ensures that added RNA was only expressed in liver cells, and not in other cells, which could have triggered an immune response. The technology used to perform the albumin splicing process is owned by VIRxSYS and is known as spliceosomal mediated RNA trans-splicing, or SMaRT(TM). VIRxSYS now has 39 publications in top tier journals showing the effectiveness of SMaRT(TM) in a broad variety of applications. The company expects to begin testing this technology in human trials in the second half of 2010.

The study was headed by Madaiah Puttaraju, Ph.D., of VIRxSYS. The full citation for the study is: Wang J, Mansfield SG, Cote CA, Jiang PD, Weng K, Amar MJ, Brewer BH Jr, Remaley AT, McGarrity GJ, Garcia-Blanco MA, Puttaraju M. Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo. Mol Ther. 2009; 17(2):343-51; doi:10.1038/mt.2008.260. Drs. Amar, Brewer and Remaley were collaborators at the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health. Part of these studies was performed under a cooperative research and development agreement (CRADA) between VIRxSYS and the NHLBI.

About VIRxSYS

Founded in 1998, VIRxSYS is a private biotechnology company that focuses on the development of a novel lentiviral gene delivery platform technology for the treatment of serious diseases. The Company has exclusively licensed its patented, proprietary lentiviral technology platform from The Johns Hopkins University (JHU) in Baltimore, Maryland where the original research was conducted. The Company obtained its SMaRT(TM) technology from Intronn Inc. More information regarding VIRxSYS can be found at www.virxsys.com.

    Contact: Russell LaMontagne
    Phone:   212-255-5340
    E-mail:  russell@corinthgroup.com


'/>"/>
SOURCE VIRxSYS Corporation
Copyright©2009 PR Newswire.
All rights reserved

Related biology technology :

1. VIRxSYS and Lentigen Announce Litigation Settlement
2. VIRxSYS Announces Interim Results from VRX496 Gene Therapy Phase II Trial
3. Circulation Publishes MERLIN TIMI-36 Data Showing Safety and Anti-Arrhythmic Effects of Ranexa(R)
4. Thomson Scientific Publishes Who Is Making The Biggest Splash? - A Quarterly Review of Scientific Literature on Drugs and Therapies From April - June 2007
5. Consortium publishes Phase II map of human genetic variation
6. The Lancet Publishes Vasogens ACCLAIM Results
7. Dendreon Publishes Manuscript on CD54 as a Surrogate Marker of Antigen Presenting Cell Activation
8. The Lancet Publishes Results From Worlds First Clinical Trial of a Fully Bioabsorbable Drug Eluting Coronary Stent
9. Springer publishes anthology with the Nanoethics Group
10. The Scientific Business of Thomson Reuters Publishes Its 2008 Liquent Regulatory Affairs Trends Survey Results
11. Waters Publishes Rapid and Specific Method of Detection for Melamine in Infant Formula and Liquid Milk, in Support of Chinas Ministry of Science and Technology
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/2/2016)... , ... December 01, 2016 , ... ... long-term client Nanowear on their recent FDA Class II 510(k) clearance for their ... significant hurdle in commercializing remote cardiac monitoring devices that rely on cloth-based nanosensors. ...
(Date:12/2/2016)... a world leader in rapid infectious disease tests, introduced the Company,s newest product, the ... (Photo: http://photos.prnewswire.com/prnh/20161201/444905 ) Continue Reading ... ... , , bioLytical was invited by the ... INSTI HIV Self Test to 350 pharmacy representatives in Nairobi ...
(Date:11/30/2016)... Biotest Pharmaceuticals Corporation (BPC), a leading manufacturer ... addition of its newest plasma collection center located at ... The 15,200 square foot state-of-the-art facility officially opened its ... the total number of BPC,s plasma collection centers to ... Chief Executive Officer said "We are pleased to become ...
(Date:11/30/2016)... November 2016   Merck , ein ... Unterzeichnung einer Reihe von Vereinbarungen mit Evotec ... AG Screeningleistungen für Mercks Palette genetischer Reagenzien ... auf diese Bibliotheken in Kombination mit Evotecs ... Weg zur Ermittlung und Erforschung neuer Arzneimitteltargets.    ...
Breaking Biology Technology:
(Date:11/29/2016)... Nearly one billion matches per second with DERMALOG,s high-speed AFIS    ... ... DERMALOG is Germany's largest Multi-Biometric supplier: The ... Identification Systems) ... Germany's largest Multi-Biometric supplier: The company's Fingerprint Identification System is part of ...
(Date:11/22/2016)... According to the new market research report "Biometric ... Signature, Voice), Multi-Factor), Component (Hardware and Software), Function (Contact and Non-contact), Application, ... is expected to grow from USD 10.74 Billion in 2015 to reach ... and 2022. Continue Reading ... ...
(Date:11/19/2016)... , Nov. 18, 2016 Securus Technologies, ... solutions for public safety, investigation, corrections and monitoring, announced ... smaller competitor, ICSolutions, to have an independent technology judge ... the most modern high tech/sophisticated telephone calling platform, and ... customers that they do most of what we do ...
Breaking Biology News(10 mins):