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Five New RNAi-Related Patent Applications Published by the USPTO

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Title: Compositions and Methods for Treatment of Hepatitis C Virus-Associated Diseases. Number: 20040033978. Filed: June 9, 2003. Lead Inventor: Kevin Anderson, Isis Pharmaceuticals.

The patent application, its abstract states, covers “antisense oligonucleotides … which are complementary to and hybridizable with at least a portion of HCV RNA, and which are capable of inhibiting the function of the HCV RNA.”

The oligos of the invention, the abstract adds, can be administered in vivo or in vitro, and can be used either therapeutically or prophylactically to “reduce the severity of diseases associated with hepatitis C virus.” Diagnostic applications are also covered.

Specifically, the application covers “an RNA compound between eight and 80 nucleobases in length targeted to HCV genomic or messenger RNA, wherein said compound specifically hybridizes with said HCV genomics or MRNA and inhibits expression of HCV.”


Title: Oligonucleotide Modulation of Cell Adhesion. Number: 20040033977. Filed: June 4, 2003. Lead Inventor: Frank Bennett, Isis Pharmaceuticals.

According to the patent application’s abstract, the invention involves “compositions and methods for the treatment of ophthalmic disorders, particularly preservation of corneal explants and prevention of corneal allograft rejection. These compositions comprise oligonucleotides which are specifically hybridizable with nucleic acids encoding the cellular adhesion molecules: intercellular adhesion molecule-1 (ICAM-1), vascular adhesion molecule-1 (VCAM-1), and endothelial leukocyte adhesion molecule-1 (ELAM-1).”

The application specifically claims “an RNA compound between about eight and 80 nucleobases in length targeted to” the aforementioned cellular adhesion molecules.


Title: Use of Genes Identified to be Involved in Tumor Development for the Development of Anti-Cancer Drugs. Number: 20040033974. Filed: Aug. 19, 2002. Lead Inventor: Anders Henrik Lund, Kylix.

The patent application’s abstract states the invention “relates to the use of inhibitors of the expressed proteins of the murine genes and/or their human homologs listed [elsewhere in the patent application] for the preparation of a therapeutical composition for the treatment of cancer,” in particular solid tumors of the lung, colon, breast, prostate, ovaries, and pancreas, as well as leukemia.

The application notes that inhibitors covered include double-stranded RNA molecules used to inhibit mRNA transcripts.


Title: Methods and Compositions Relating to Polypeptides with RNase III Domains that Mediate RNA Interference. Number: 20040033602. Filed: June 12, 2003. Lead Inventor: Lance Ford, Ambion.

“The present invention concerns methods and compositions involving RNase III and polypeptides containing RNase III domains to generate RNA capable of triggering RNA-mediated interference in a cell,” the patent application’s abstract states.

“In some embodiments, the RNase III is from a prokaryote. RNase III activity will cleave a double-stranded RNA molecule into short RNA molecules that trigger or mediate RNAi,” it adds.

Compositions of the invention, the abstract notes, are “kits that include an RNase III domain-containing polypeptide,” among others. “The present invention further concerns methods using polypeptides with RNase III activity for generating RNA molecules that effect RNAi, including the generation of a number of RNA molecules to the same target.”


Title: Transgenic Organism. Number: 20040040052. Filed: April 24, 2003. Lead Inventor: Philippa Radcliffe, Oxford BioMedica.

According to the patent application’s abstract, the invention is a method of producing a transgenic cell by “introducing into a cell a non-primate lentiviral expression vector comprising a nucleotide of interest.”

The abstract adds that the application also covers “a method of producing a transgenic cell [by] introducing into a cell a lentiviral expression vector comprising a [nucleotide of interest] capable of generating an antisense oligonucleotide, a ribozyme, an siRNA, a short hairpin RNA, a microRNA or a group 1 intron.”

Also described by the patent application is “a viral vector comprising a first nucleotide sequence, wherein said first nucleotide sequence comprises: a second nucleotide sequence comprising an aptazyme; and a third nucleotide sequence capable of generating a polynucleotide; wherein [the first and second sequences] are operably linked and wherein the aptazyme is activatable to cleave a transcript of the first nucleotide sequence such that said polynucleotide is generated,” the abstract states.