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:10/11/2017)... ... October 11, 2017 , ... ComplianceOnline’s Medical Device Summit is ... and 8th June 2018 in San Francisco, CA. The Summit brings together current and ... distinguished CEOs, board directors and government officials from around the world to address key ...
(Date:10/11/2017)... ... , ... Disappearing forests and increased emissions are the main causes of the ... Especially those living in larger cities are affected by air pollution related diseases. , ... pollution-affected countries globally - decided to take action. , “I knew I had to ...
(Date:10/10/2017)... ... October 10, 2017 , ... ... advancing targeted antibody-drug conjugate (ADC) therapeutics, today confirmed licensing rights that give ... Liposomal Nanoparticle), a technology developed in collaboration with Children’s Hospital Los Angeles ...
(Date:10/10/2017)... Calif. , Oct. 10, 2017 SomaGenics ... from the NIH to develop RealSeq®-SC (Single Cell), expected ... for profiling small RNAs (including microRNAs) from single cells ... Program highlights the need to accelerate development of approaches ... "New techniques for measuring levels ...
Breaking Biology Technology:
(Date:4/11/2017)... Research and Markets has announced the addition of the "Global ... ... at a CAGR of 30.37% during the period 2017-2021. ... based on an in-depth market analysis with inputs from industry experts. ... the coming years. The report also includes a discussion of the ...
(Date:4/5/2017)... 5, 2017 Today HYPR Corp. , ... server component of the HYPR platform is officially ... end-to-end security architecture that empowers biometric authentication across Fortune ... already secured over 15 million users across the financial ... connected home product suites and physical access represent a ...
(Date:3/30/2017)... -- The research team of The Hong Kong Polytechnic ... by adopting ground breaking 3D fingerprint minutiae recovery and matching technology, ... accuracy for use in identification, crime investigation, immigration control, security of ... ... A research team led by Dr ...
Breaking Biology News(10 mins):