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Five RNAi-Related Patent Applications Published by the US Patent Office: Jul 23, 2004

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Title: RNA Interference-Mediated Inhibition of Vascular Endothelial Growth Factor and Vascular Endothelial Growth Factor Receptor Gene Expression Using Short Interfering Nucleic Acid. Number: 20040138163. Filed: Sept. 18, 2003. Lead Inventor: James McSwiggen, Sirna Therapeutics.

“The present invention concerns methods and reagents useful in modulating vascular endothelial growth factor (VEGF, VEGF-A, VEGF-B, VEGF-C, VEGF-D) and/or vascular endothelial growth factor receptor (e.g., VEGFr1, VEGFr2 and/or VEGFr3) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications,” the patent application’s abstract states.

“Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid … short interfering RNA … double-stranded RNA … microRNA … and short hairpin RNA … molecules capable of mediating RNA interference … against VEGF and/or VEGFr gene expression and/or activity,” it adds. “The small nucleic acid molecules are useful in the diagnosis and treatment of cancer, proliferative diseases, and any other disease or condition that responds to modulation of VEGF and/or VEGFr expression or activity.”


Title: Methods and Compositions for Inhibiting the Function of Polynucleotide Sequences. Number: 20040138168. Filed: Feb. 6, 2004. Lead Inventor: Chandrasekhar Satishchandran, Wyeth (Nucleonics).

The patent application’s abstract states that the invention comprises “a therapeutic composition for inhibiting the function of a target polynucleotide sequence in a mammalian cell [including] an agent that provides to a mammalian cell an at least partially double-stranded RNA molecule comprising a polynucleotide sequence of at least about 200 nucleotides in length, said polynucleotide sequence being substantially homologous to a target polynucleotide sequence.”

The abstract adds that “the agents useful in the composition can be RNA molecules made by enzymatic synthetic methods or chemical synthetic methods in vitro; or made in recombinant cultures of microorganisms and isolated therefrom, or alternatively, can be capable of generating the desired RNA molecule in vivo after delivery to the mammalian cell.

“In methods of treatment of prophylaxis of virus infections, other pathogenic infections, or certain cancers, these compositions are administered in amounts effective to reduce or inhibit the function of the target polynucleotide sequence, which can be of pathogenic origin or produced in response to a tumor or other cancer, among other sources,” states the abstract.


Title: Compositions and Processes Using siRNA, Amphipathic Compounds, and Polycations. Number: 20040137064. Filed: Jan. 15, 2003. Lead Inventor: David Lewis, Mirus.

“Described is a composition with low toxicity comprising an amphipathic compound and a polycation, useful for delivering siRNA to a cell,” the patent application’s abstract states. “The composition may be used in the process of delivering an siRNA to an animal cell ... in a multi-well format.”


Title: Devices and Processes for Distribution of Genetic Material to Mammalian Limb. Number: 20040136960. Filed: Jan. 10, 2003. Lead Inventor: Jon Wolff, Mirus.

According to the patent application’s abstract, the invention comprises “a process for functionally distributing genetic material to mammalian limb muscle cells [by] inserting genetic material into a limb vessel. Delivery efficiency and distribution is enhanced by combining injection of a solution containing the genetic material with the use of an externally applied cuff to increase vascular permeability,” it adds.


Title: Methods for Making Polynucleotide Libraries, Polynucleotide Arrays, and Cell Libraries for High-Throughput Genomics Analysis. Number: 20040137490. Filed: Dec. 20, 2003. Lead Inventor: Robert Finney, PanGenex.

The patent application, its abstract states, covers “a method for high-throughput genomics analysis to identify the therapeutic or diagnostic utility of genes [involving] the use of a construct to disrupt a gene or alleles of a gene in cells of interest. Arrays of such cells can be used to monitor such disrupted cells phenotypically in the context, for example, of testing drug candidates,” it notes.

“Polynucleotides that comprise part of the disrupted genes can be recovered from such knockout cells by virtue of an origin of replication or a host cell selection marker sequence that is part of the construct,” the abstract adds. “The recovered polynucleotides can be used to identify the disrupted genes or to make homologous recombination vectors, which in turn can be employed to make multi-allele knockout cells.”

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