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IP Update: Jan 6, 2011

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Title: shRNA Library

Patent Number: 7,863,222

Filed: Aug. 1, 2008

Lead Inventor: Donald Payan, Rigel Pharmaceuticals

The invention, the patent's abstract states, relates to "a library of nucleic acid vectors, as well as a method for making the same."


Title: Interfering RNAs Against the Promoter Region of p53

Patent Number: 7,858,592

Filed: Feb. 22, 2008

Lead Inventor: David Shames, University of Texas

The invention, the patent's abstract states, "relates to the inhibition of p53 transcription by interfering with the activity of a p53 promoter using inhibitory double-stranded RNAs. Use of these inhibitory RNAs in the treatment of cancers also is disclosed."


Title: Conjugates and Compositions for Cellular Delivery

Patent Number: 7,858,625

Filed: June 24, 2005

Lead Inventor: Jasenka Matulic-Adamic, Sirna Therapeutics (Merck)

"This invention features conjugates, compositions, methods of synthesis, and applications thereof, including folate-derived conjugates of nucleosides, nucleotides, non-nucleosides, and nucleic acids including enzymatic nucleic acids and antisense nucleic acid molecules," the patent's abstract states.


Title: RNA Interference-Mediated Inhibition of Gene Expression Using Multifunctional Short Interfering Nucleic Acid

Patent Number: 7,858,769

Filed: Feb. 9, 2005

Lead Inventor: Vasant Jadhav, Sirna Therapeutics (Merck)

The invention concerns "methods and nucleic acid-based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target-validation, and genomic-discovery applications," the patent's abstract states.
"Specifically, the invention relates to multifunctional short interfering nucleic acid molecules that modulate the expression of one or more genes in a biologic system, such as a cell, tissue, or organism, via RNA interference. The bi-functional short interfering nucleic acid molecules of the invention can target more than one regions of nucleic acid sequence in a single target nucleic acid molecule or can target regions of nucleic acid sequence in differing target nucleic acid molecules. The self multifunctional siNA molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism."


Title: siRNA Having Antiviral Activity Against Non-Polio Enterovirus

Patent Number: 7,858,770

Filed: May 30, 2006

Lead Inventor: Heuiran Lee, University of Ulsan

The invention, the patent's abstract states, "relates to an siRNA having antiviral activity against non-polio enteroviruses, and a pharmaceutical composition comprising same as an active ingredient for preventing and treating diseases caused by non-polio enterovirus infection."


Title: RNA Interference-Mediated Inhibition of Muscarinic Cholinergic Receptor Gene Expression

Patent Number: 7,858,771

Filed: Dec. 10, 2009

Lead Inventor: James McSwiggen, Merck

The invention "relates to compounds, compositions, and methods useful for modulating the expression of genes associated with respiratory and pulmonary disease, such as cholinergic muscarinic receptor genes, using short interfering nucleic acid molecules," according to the patent's abstract. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of cholinergic muscarinic receptor genes, or other genes involved in pathways of cholinergic muscarinic receptor gene expression and/or activity by RNA interference using small nucleic acid molecules."


Title: siRNA Targeting Survivin

Application Number: 20100331214

Filed: Aug. 13, 2010

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent application'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. Through these methodologies, one can select siRNA that target genes, including survivin."


Title: Compositions and Methods for the Specific Inhibition of Gene Expression by dsRNA Containing Modified Nucleotides

Application Number: 20100331389

Filed: Sept. 17, 2009

Inventor: Bob Brown, Dicerna Pharmaceuticals

"The invention features compositions and methods that are useful for reducing the expression or activity of a specified gene in a eukaryotic cell," the patent application's abstract states.


Title: Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure

Application Number: 20100331394

Filed: Sept. 2, 2010

Lead Inventor: Ana Jimenez, Sylentis

The invention, the patent application's abstract states, "relates to methods and compositions that decrease intraocular pressure of the eye. The compositions of the invention comprise short interfering nucleic acid molecules including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of an eye conditions displaying increased [intraocular pressure] such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention."


Title: In Vivo Delivery of Double-Stranded RNA to a Target Cell

Application Number: 20100323001

Filed: Nov. 6, 2007

Inventor: Catherine Pachuk, Veritas

"The invention encompasses methods of delivering nucleic acids, including dsRNA, to mammalian target cells in vivo via intercellular transfer, wherein the dsRNA is delivered to or expressed in a first cell different from the target cell, wherein the first cell facilitates delivery of the dsRNA to the target cell," the patent application's abstract states.


Title: microRNA Expression Profiling and Targeting in Peripheral Blood in Lung Cancer

Application Number: 20100323357

Filed: Nov. 26, 2008

Lead Inventor: Serge Nana-Sinkam, Ohio State University

The invention, the patent application's abstract states, comprises "a method for the diagnosis, prognosis, and treatment of lung cancer by detecting at least one microRNA in peripheral blood."


Title: siRNA Targeting TATA Box-Binding Protein-Associated Factor

Application Number: 20100323922

Filed: Aug. 10, 2010

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent application'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 TAF1."


Title: Delivery Method

Application Number: 20100324113

Filed: June 1, 2007

Lead Inventor: Bruce Sullenger, Duke University

The invention relates to "a method of effecting targeted delivery of siRNAs, and to compounds suitable for use in such a method," the patent application's abstract states.


Title: Hepatitis C dsRNA Effector Molecules, Expression Constructs, Compositions, and Methods of Use

Application Number: 20100324117

Filed: June 23, 2008

Lead Inventor: Daniel McCallus, Alnylam Pharmaceuticals

The patent application, its abstract states, claims "agents, compositions, constructs, and methods for silencing HCV polynucleotides, as well as methods and compositions for treating or preventing HCV infection in a mammalian cell.

"In one aspect, the … invention provides an agent or composition comprising at least one double-stranded RNA effector molecule or complex … [with] a sequence of at least 19 nucleotides having at least 90 percent identity with a nucleotide sequence within" a conserved portion of the HCV genome, and its complementary sequence, it adds. "In another aspect, the present invention provides a construct suitable for replication in a host cell, and/or suitable for expression of an RNA molecule or complex of the invention in vitro or in vivo.

"In a third aspect, the … invention provides a method for silencing HCV RNA in a mammalian cell [involving] administering to the mammalian cell an agent, composition, or construct of the invention in a manner and amount effective to silence HCV RNA in the cell," the abstract states. "In a related aspect, the invention provides a method for treating or preventing HCV infection in a patient, comprising administering to the patient an effective amount of an agent, composition, or construct of the invention as described herein."


Title: Method for the Promotion of Angiogenesis, Vascularization, or Vascular Repair or for the Inhibition of Tumor Angiogenesis

Application Number: 20100324118

Filed: Oct. 30, 2008

Lead Inventor: Stefanie Dimmeler, Goethe University

"The invention relates to a method for influencing the miR-92 expression in a cell," the patent application's abstract states. It further claims a method of "reducing the miR-92 expression in the cell in order to promote the vascularization or vessel repair by introducing an antisense molecule against miR-92 into the cell, or increasing the miR-92 expression in the cell for an inhibition of the tumor angiogenesis by introducing a construct into the cell, wherein said construct includes an expressible miR-92 sequence. Furthermore, the invention relates to a pharmaceutical composition comprising an agent for reducing the miR-92 activity or expression in a cell in the form of an antisense molecule against miR-92, or an agent for increasing the miR-92 expression in a cell in the form of a construct for expressing miR-92."


Title: Lipid Formulation

Application Number: 20100324120

Filed: June 10, 2010

Lead Inventor: Jianxin Chen, Alnylam Pharmaceuticals

"The invention features a cationic lipid … an improved lipid formulation comprising a cationic lipid of [a specified formula], and corresponding methods of use," the patent application's abstract states. "Also disclosed are targeting lipids and specific lipid formulations comprising such targeting lipids."


Title: Methods and Compositions for the Specific Inhibition of Gene Expression by Double-Stranded RNA

Application Number: 20100324121

Filed: Aug. 12, 2010

Lead Inventor: John Rossi, City of Hope (Integrated DNA Technologies)

"The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene," the patent application's abstract states. "The method involves introducing into the environment of a cell an amount of a double-stranded RNA such that a sufficient portion of the dsRNA can enter the cytoplasm of the cell to cause a reduction in the expression of the target gene. The dsRNA has a first oligonucleotide sequence that is between 26 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of from about 19 to about 23 nucleotides is complementary to a nucleotide sequence of the RNA produced from the target gene."


Title: Oligomeric Compounds Comprising 4'-Thionucleosides for Use in Gene Modulation

Application Number: 20100324277

Filed: Aug. 31, 2010

Lead Inventor: Bhat Balkrishen, Isis Pharmaceuticals

The invention provides "modified oligomeric compounds and compositions of oligomeric compounds for use in the RNA interference pathway of gene modulation," the patent application's abstract states. "The modified oligomeric compounds include siRNA and asRNA having at least one affinity modification."


Title: Methods and Sequences to Suppress Primate Huntington Gene Expression In Vivo

Application Number: 20100325746

Filed: Aug. 9, 2006

Lead Inventor: William Kaemmerer, Medtronic

The invention, the patent application's abstract states, comprises "sequences, molecules, and methods used to suppress the expression of [Huntington's disease] genes encoding for huntingtin protein in primates including Macaca mulatta and Homo sapiens. These sequences, molecules, and methods aid in the study of the pathogenesis of [Huntington's disease] and can also provide a treatment for this disease by reducing [Huntington's disease] mRNA without causing death, locomotor impairment, or cellular alterations of the Macaca mulatta and Homo sapiens."

The Scan

Just Breathing

A new analysis suggests that most Mycobacterium tuberculosis is spread by aerosols from breathing, rather than by coughing, the New York Times reports.

Just Like This One

NPR reports that the World Health Organization has hired a South African biotech company to recreate mRNA vaccine for SARS-CoV-2 that is similar to the one developed by Moderna.

Slow Start

The Wall Street Journal reports that Biogen's Alzheimer's disease treatment had revenues for July through September that totaled $300,000.

Genome Research Papers on Cancer Chromatin, Splicing in the Thymus, Circular RNAs in Cancer

In Genome Research this week: analysis of bivalent chromatin sites, RBFOX splicing factors' role in thymic epithelial cells, and more.