USPTO issues two patents to VIRxSYS SMaRT RNA platform technology

Feb 18 2011

VIRxSYS Corporation, a privately-held biotechnology company with proprietary RNA payload and lentiviral vector delivery platform technologies, today announced that the United States Patent and Trademark Office has issued the Company two patents for its SMaRT™ (Spliceosome-Mediated RNA Trans-splicing) RNA platform technology, capable of reprogramming or repairing genes to create, increase or change protein expression. The patents cover the use of the SMaRT™ RNA platform for a broad array of possible therapeutic and diagnostic applications, including treatment of cardiovascular disease, hemophilia A, and antibody gene transfer leading to antibody production.

"The issuance of these patents is another demonstration of our leadership in the development of RNA-based therapies for the treatment of serious human diseases," said Dr. Riku Rautsola, VIRxSYS President & CEO. "With over 30 issued patents in our robust SMaRT^TM-based intellectual property portfolio, as well as numerous patent applications in the U.S. and worldwide, VIRxSYS is positioned to deliver on the promises of genetic medicine. VIRxSYS will soon be initiating a non-human primate study to test the ability of our lead clinical candidate to raise serum apoA-I levels, leading to an increase in HDL."

By targeting gene transcripts in a cell, the SMaRT™ RNA platform technology is capable of repairing defective genes and reprogramming specific gene transcripts at the RNA level. SMaRT™ can efficiently delete any defective or otherwise unwanted part of a gene transcript, and insert, or "splice-in," new genetic information to create a healthy, functional protein or a totally different protein of therapeutic interest, such as diverting plasma albumin production in the liver towards production of apolipoprotein AI (apoA-I), factor VIII, or even small antibody fragments.

The first of the two patents, U.S. Patent No. 7,871,795, covers the use of the SMaRT™ platform for trans-splicing into highly abundant gene transcripts to produce a range of therapeutic products, including apoA-I or apoA-I milano variant polypeptides for the treatment of cardiovascular disease, single chain antibody polypeptides, and Factor VIII protein for the treatment of hemophilia A. The second, U.S. Patent No. 7,879,321 issued on February 1, 2011, covers the use of its SMaRT™ platform for trans-splicing into highly abundant gene transcripts for antibody gene transfer and antibody production.