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IP Update: Oct 22, 2009

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Title: siRNA Targeting Kinase Insert Domain Receptor

Number: 7,605,252

Filed: Oct. 26, 2007

Lead Inventor: Anstasia Khvorova, Dharmacon (Thermo Fisher Scientific)

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent's abstract states. "By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for KDR."


Title: Compositions and Methods for Inhibiting Expression of the PCSK9 Gene

Number: 7,605,251

Filed: May 10, 2007

Lead Inventor: Pamela Tan, Alnylam Pharmaceuticals

"The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the proprotein convertase subtilisin kexin 9 gene, comprising an antisense strand having a nucleotide sequence which is less than 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene," according to the patent's abstract. "The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier and methods for treating diseases caused by PCSK9 gene expression by using the pharmaceutical composition."


Title: siRNA Targeting cAMP-Specific Phosphodiesterase 4D

Number: 7,605,250

Filed: Nov. 9, 2006

Lead Inventor: Anstasia Khvorova, Dharmacon (Thermo Fisher Scientific)

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent's abstract states. "By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed."


Title: Treatment of Neurodegenerative Disease Through Intracranial Delivery of siRNA

Number: 7,605,249

Filed: Nov. 25, 2003

Inventor: William Kaemmerer, Medtronic

The invention "provides devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein," the patent's abstract states. The invention also provides "valuable small interfering RNA vectors and methods for treating neurodegenerative disorders such as Alzheimer's disease, Parkinson's disease, Huntington's disease, spinocerebellar ataxia type 1, type 2, type 3, and/or dentatorubral-pallidoluysian atrophy."


Title: Methods and Compositions for Inhibiting HIV Infection

Number: 7,604,977

Filed: April 21, 2006

Lead Inventor: Kelli Kuhen, IRM

The invention "provides novel methods for identifying agents that inhibit HIV infection," the patent's abstract states. "The anti-HIV agents are identified by screening test compounds for ability to modulate a biological activity of isopeptidase T, e.g., its isopeptidase activity or its binding to another molecule such as viral protein R. Such IsoT modulators can be further examined for their activity in inhibiting an activity indicative of HIV infection or HIV replication. These novel anti-HIV agents are useful in the prevention or treatment of HIV infection and conditions associated with or caused by HIV infection."

The patent specifically claims an siRNA that inhibits human IsoT.


Title: pCryptoRNAi

Number: 20090258366

Filed: March 25, 2009

Lead Inventor: Jon Ander Ochoa de Eribe Casas

"A vector developed to transform fungi can be used to study the expression of a gene of interest," the patent application's abstract states. "The vector can provide for the expression of signal proteins in fungi that can be observed and/or monitored. The vector can be used to investigate the effects of RNA interference on a gene of interest in pathogenic fungi. Systems and methods of using the vector are provided."


Title: Cellular Delivery of siRNA

Number: 20090258424

Filed: March 1, 2007 PCT Filed: March 1, 2007

Inventor: Yingqun Huang, Yale University

"The invention provides a method for delivering a nucleic acid to a cell using a targeting molecule that is bound non-covalently to the nucleic acid," according to the patent application's abstract. "Compositions and kits are also provided."


Title: Gene Knockdown by Intracellular Expression of Aptamers

Number: 20090258425

Filed: June 17, 2009

Lead Inventor: David Epstein, Archemix

The invention, the patent application's abstract states, comprises "materials and methods … for target validation by gene knockdown with intracellularly expressed aptamers and siRNAs. The aptamers produced by the materials and methods of the invention are useful in target validation for therapeutics development."


Title: Carrier Composition for Nucleic Acid Transport

Number: 20090258923

Filed: Oct. 17, 2006 PCT Filed: Oct. 17, 2006

Lead Inventor: Hidekazu Toyobuku, Otsuka Pharmaceutical

The invention, the patent application's abstract states, involves "a nucleic acid-delivery carrier composition of low toxicity and high safety.

"The carrier composition, when used to administer a nucleic acid such as an siRNA into an animal-derived cell or organism, [is] capable of delivering efficiently the nucleic acids into the cell while protecting it from being degraded," the abstract notes. The invention also relates to a "nucleic acid-delivery composition containing the carrier and a nucleic acid … [which] is prepared by mixing a cationic lipid having a steroid skeleton with a tertiary ammonium salt-type cationic lipid."


Title: Methods, Compositions, and Drug-Delivery Systems for Intraocular Delivery of siRNA Molecules

Number: 20090258924

Filed: April 15, 2008

Lead Inventor: Robert Lyons, Allergan

The patent application, its abstract states, claims "biocompatible intraocular drug-delivery systems in the form of an implant for intraocular administration of siRNA molecules. The drug-delivery systems may be placed in an eye to treat or reduce the occurrence of one or more ocular conditions, such as retinal damage, including glaucoma and proliferative vitreoretinopathy among others."


Title: Natural Antisense and Non-Coding RNA Transcripts as Drug Targets

Number: 20090258925

Filed: Dec. 28, 2006 PCT Filed: Dec. 28, 2006

Inventor: Claes Wahlestedt, Scripps Research Institute (Curna)

"Small interfering RNA knock down antisense transcripts, and regulate the expression of their sense partners. This regulation can either be discordant — antisense knockdown results in sense transcript elevation — or concordant — antisense knockdown results in concomitant sense transcript reduction."


Title: Methods and Compositions for Delivering siRNA into Mammalian Cells

Number: 20090258926

Filed: Feb. 23, 2009

Lead Inventor: Gilles Divita, Centre National De La Recherche Scientifique

The invention, the patent application's abstract states, comprises a complex comprising a peptide carrier of a defined sequence and an "appropriate siRNA."


Title: Double-Stranded RNA Structures and Constructs, and Methods for Generating and Using the Same

Number: 20090258930

Filed: Oct. 6, 2008

Lead Inventor: Catherine Pachuk, Nucleonics (Alnylam Pharmaceuticals)

The invention, the patent application's abstract states, "relates to novel double-stranded RNA structures and dsRNA-expression constructs, methods for generating them, and methods of utilizing them for silencing genes. Desirably, these methods specifically inhibit the expression of one or more target genes in a cell or animal without inducing toxicity. These methods can be used to prevent or treat a disease or infection by silencing a gene associated with the disease or infection.

"The invention also provides methods for identifying nucleic acid sequences that modulate a detectable phenotype, such as the function of a cell, the expression of a gene, or the biological activity of a target polypeptide," the abstract adds.


Title: Compositions and Methods for Inhibiting Expression of Nav1.8 Gene

Number: 20090258934

Filed: June 18, 2009

Lead Inventor: Dinah Sah, Alnylam Pharmaceuticals

"The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the Nav1.8 gene, comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Nav1.8 gene," according to the patent application's abstract. "The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, methods for treating diseases caused by the expression of the Nav1.8 gene using the pharmaceutical composition, and methods for inhibiting the expression of the Nav1.8 gene in a cell."

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