VIRxSYS Publishes New Study on RNA Therapy
New RNA therapy reversed hemophilia in mice
“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,
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
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
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SOURCE VIRxSYS Corporation