Navigation Links
VIRxSYS Publishes New Study on RNA Therapy

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.


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

    Contact: Russell LaMontagne
    Phone:   212-255-5340

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:
(Date:6/27/2016)... - BIOREM Inc. (TSX-V: BRM) ("Biorem" or "the Company") announces ... Clean Technology Fund I, LP and Clean Technology Fund ... venture capital funds which together hold approximately 59% of ... as converted basis), that they have entered into an ... in Biorem to TUS Holdings Co. Ltd. ("TUS") ( ...
(Date:6/27/2016)... ... June 27, 2016 , ... Rolf K. ... the faculty of the University of North Carolina Kenan-Flagler Business School ... entrepreneurship at UNC Kenan-Flagler, with a focus on the school’s international efforts, leading ...
(Date:6/24/2016)... ... 2016 , ... While the majority of commercial spectrophotometers and fluorometers use the ... models are higher end machines that use the more unconventional z-dimension of 20mm. ... the bottom of the cuvette holder. , FireflySci has developed several Agilent flow ...
(Date:6/23/2016)... , June 23, 2016 /PRNewswire/ - FACIT ... Ontario biotechnology company, Propellon Therapeutics ... development and commercialization of a portfolio of first-in-class ... Epigenetic targets such as WDR5 represent an exciting ... significantly in precision medicine for cancer patients. Substantial ...
Breaking Biology Technology:
(Date:6/2/2016)... , June 2, 2016   The Weather Company , ... Watson Ads, an industry-first capability in which consumers will be ... able to ask questions via voice or text and receive ... Marketers have long sought an advertising ... that can be personal, relevant and valuable; and can scale ...
(Date:5/16/2016)... YORK , May 16, 2016   EyeLock ... solutions, today announced the opening of an IoT Center ... to strengthen and expand the development of embedded iris ... an unprecedented level of convenience and security with unmatched ... authenticate one,s identity aside from DNA. EyeLock,s platform uses ...
(Date:4/28/2016)... First quarter 2016:   , Revenues amounted to ... of 2015 The gross margin was 49% (27) ... operating margin was 40% (-13) Earnings per share rose ... was SEK 249.9 M (21.2) , Outlook   ... The operating margin for 2016 is estimated to exceed ...
Breaking Biology News(10 mins):