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IP Update: Sep 17, 2009

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Title: Inhibition of the Expression of Huntingtin Gene

Number: 7,589,189

Filed: April 30, 2004 PCT Filed: April 30, 2004

Lead Inventor: Kanazawa Ichiro, Japan Science and Technology Agency

The invention, the patent's abstract states, comprises "methods for suppressing … huntingtin gene expression by using a double-stranded RNA, huntingtin gene expression inhibitors to suppress … huntingtin gene expression, and preventives and/or remedies of Huntington's disease. Targeting against a specific sequence of mRNA at immediately upstream of CAG repeats in HD genes of Huntington's disease … the huntingtin gene expression is suppressed by using a dsRNA homologous to the sequence.

"In this invention, [an] siRNA … as short as around 21-23 [base pairs] can be effectively used as the dsRNA homologous to a specific RNA sequence in a region at immediately upstream of CAG repeats," the abstract notes. "The dsRNA of this … invention can be used as a huntingtin gene expression inhibitor, or a preventive and/or a remedy of Huntington's disease."


Title: siRNA Targeting Hypoxia-Inducible Factor 1

Number: 7,589,191

Filed: Feb. 2, 2009

Lead Inventor: Anastasia 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: Encapsulated Nanoparticles for Nucleic Acid Delivery

Number: 20090226528

Filed: Oct. 29, 2008

Lead Inventor: Michael Czech, University of Massachusetts (RXi Pharmaceuticals)

The patent application, its abstract states, claims "methods and compositions for delivering agents … [such as] gene-silencing agents, and molecules to cells using yeast cell wall particles. … Embodiments of the invention are particularly useful for the delivery of nucleic acids [including] siRNAs … to cells."


Title: Methods and Composition for Intraocular Delivery of Therapeutic siRNA

Number: 20090226531

Filed: March 7, 2008

Lead Inventor: Robert Lyons, Allergan

"Biocompatible intraocular drug-delivery systems include nanoparticles that encapsulate siRNA molecules," the patent application's abstract states. "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: Modulation of HIV Replication by RNA Interference

Number: 20090227509

Filed: April 3, 2009

Lead Inventor: Mario Stevenson, University of Massachusetts

The patent application, its abstract states, claims "small interfering RNAs and vectors encoding one or more siRNAs that are sufficiently homologous to a portion of the HIV genome to mediate RNA interference in vivo. Also disclosed are methods wherein siRNAs, or vectors encoding siRNAs, are administered to prevent or inhibit HIV infection in a subject, cell, or tissue. Knockout and/or knockdown cells or organisms are also disclosed that utilize the siRNAs or vectors of the … invention."


Title: miR-34-Regulated Genes and Pathways as Targets for Therapeutic Intervention

Number: 20090227533

Filed: June 6, 2008

Lead Inventor: Andreas Bader, Asuragen

The invention "concerns methods and compositions for identifying genes or genetic pathways modulated by miR-34, using miR-34 to modulate a gene or gene pathway, using this profile in assessing the condition of a patient, and/or treating the patient with an appropriate miRNA," according to the patent application's abstract.


Title: Composition and Methods of RNAi Therapeutics for Treatment of Cancer and Other Neovascularization Diseases

Number: 20090227657

Filed: April 9, 2009

Lead Inventor: Yijia Liu, Intradigm

The patent application, its abstract states, claims "compositions and methods … for treatment of diseases involving unwanted neovascularization. The invention provides treatments that control [neovascularization] through selective inhibition of pro-angiogenic biochemical pathways, including inhibition of the VEGF pathway gene expression and inhibition localized at pathological [neovascularization] tissues.

"Tissue-targeted nanoparticle compositions comprising polymer conjugates and nucleic acid molecules that induce RNA interference are provided," the abstract adds. "The nanoparticle compositions of the invention can be used alone or in combination with other therapeutic agents such as VEGF pathway antagonists. The compositions and methods can be used for the treatment of [neovascularization] diseases such as cancer, ocular disease, arthritis, and inflammatory diseases."


Title: siRNA Targeting Connexin 43

Number: 20090227780

Filed: May 14, 2009

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," according to the patent application's abstract. "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 connexin 43."

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