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IP Update: Jun 17, 2010

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Title: Method for Expression of Small Antiviral RNA Molecules with Reduced Cytotoxicity Within a Cell

Patent Number: 7,737,124

Filed: March 8, 2007

Lead Inventor: Carlo Lois-Caballe, California Institute of Technology

"In one aspect, the invention provides methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector," the patent's abstract states. "Small interfering RNA can be expressed using the methods of the invention within a cell. In a further aspect, the invention provides methods for producing siRNA encoding lentivirus where the siRNA activity may interfere with the lentiviral life cycle. In yet a further aspect, the invention provides methods for expression of a small RNA molecule within a cell, such as an siRNA capable of down-regulating CCR5, wherein expression of the small RNA molecule is relatively non-cytotoxic to the cell."


Title: RNAi Modulation of HIF-1 and Therapeutic Uses Thereof

Patent Number: 7,737,265

Filed: June 27, 2006

Lead Inventor: Akin Akinc, Alnylam Pharmaceuticals

The invention, the patent's abstract states, relates to "compounds, compositions, and methods useful for modulating the expression of HIF-1 alpha, such as by the mechanism of RNA interference. The compounds and compositions include iRNA agents that can be unmodified or chemically modified."


Title: RNAi Modulation of SCAP and Therapeutic Uses Thereof

Patent Number: 7,737,266

Filed: Sept. 18, 2007

Lead Inventor: Juergen Soutschek, Alnylam Pharmaceuticals

"The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of a SCAP 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 a SCAP gene," the patent's abstract states. "The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, methods for treating diseases caused by human SCAP expression and the expression of a SCAP gene using the pharmaceutical composition, and methods for inhibiting the expression of a SCAP gene in a cell."


Title: siRNA Targeting Hypoxia-Inducible Factor 1

Patent Number: 7,737,267

Filed: Aug. 4, 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: Development of Asthma Therapy with rAAV-Mediated AMCase shRNA

Application Number: 20100143450

Filed: Dec. 4, 2008

Lead Inventor: Chao-Lin Liu, Ming Chi University of Technology

"The invention provides small interfering RNA and their carriers that could treat asthma in [mammals] through inhibiting acidic mammalian chitinase gene expression," according to the patent application's abstract. "The invention further provides a composition comprising siRNA of the … invention and a carrier. The invention also provides a method for treating asthma of a subject."


Title: siRNA-Mediated Gene Silencing with Viral Vectors

Application Number: 20100144026

Filed: May 27, 2009

Lead Inventor: Beverly Davidson, University of Iowa

The invention, the patent application's abstract states, "is directed to viral vectors encoding small interfering RNA molecules targeted against a gene of interest, and methods of using these viral vectors."


Title: siRNA Targeting Serine/Threonine Kinase 12

Application Number: 20100144552

Filed: Jan. 21, 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. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to STK12."


Title: Cancer Cell Identification Marker and Cancer Cell Proliferation Inhibitor

Application Number: 20100144830

Filed: Sept. 19, 2007 PCT Filed: Sept. 19, 2007

Inventor: Masahiko Kuroda, Tokyo Medical University

The patent application, its abstract states, claims an "identification marker [that] can be utilized for detection of various human cancer cells and whose expression closely relates to malignant alteration of cells; and compositions for human cancer treatment [that] are based on suppression of cancer cell proliferation through inhibition of expression of the identification marker. The marker is human heterochromatin protein 1 gamma, and the compositions for cancer treatment comprises one or more agents [that suppress] the expression of human HP1 gamma gene, such as siRNAs to human HP1 gamma."


Title: Blocking of Gene Expression in Eukaryotic Cells

Application Number: 20100144831

Filed: Sept. 27, 2007 PCT Filed: Sept. 27, 2007

Lead Inventor: Habib Fakhral, NovaRx

The invention, the patent application's abstract states, "provides methods for designing a sequence for efficient short interference RNA molecules. In particular, the … invention defines a universal target for siRNA derived from a poly A sequence, optionally in conjunction with unique sequences for gene silencing and inhibition of viral replication in a eukaryotic host cell."

The invention "further provides methods for the treatment and prevention of diseases and disorders by silencing a gene of a virus, an oncogene, genes encoding transcription factors, and many other disease-related genes," it adds. "The … invention describes antisense nucleic acids compositions comprising sequences complementary to a target nucleic acid. The antisense sequences are designed to hybridize to complementary nucleic acid target regions in a target RNA, and inhibit translation, processing, transport, or binding by proteins or riboproteins. Target regions include, and are limited to, a poly-A tail, and exclude AUG, 5' non-translated sequences, translation initiation factor binding sites, ribosome subunit binding sites, Shine Dalgarno sequence, 3' nontranslated sequences, poly-addition site, 3' cleavage site, coding region, intron, intron branch site, intron/exon junction, and splice sequence."


Title: RNAi Modulation of RSV, PIV, and Other Respiratory Viruses and Uses Thereof

Application Number: 20100144833

Filed: June 25, 2009

Inventor: Sailen Barik, University of South Alabama (Alnylam Pharmaceuticals)

The invention, the patent application's abstract states, "is based on the in vivo demonstration that [respiratory syncytial virus and parainfluenza virus] can be inhibited through intranasal administration of RNAi agents, as well as by parenteral administration of such agents. Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently. Based on these findings, the … invention provides general and specific compositions and methods that are useful in reducing RSV or PIV mRNA levels, RSV or PIV protein levels, and viral titers in a subject, e.g., a mammal, such as a human. These findings can be applied to other respiratory viruses."


Title: Inhibition of GPR4

Application Number: 20100144835

Filed: Dec. 13, 2007 PCT Filed: Dec. 13, 2007

Lead Inventor: Klaus Seuwen, Novartis

The invention "relates to the use of a GPR4 inhibitor for the manufacture of a medicament for the inhibition of angiogenesis, for instance for the inhibition of tumor growth in the treatment of cancer," the patent application's abstract states. "In a preferred embodiment, said inhibitor is a siRNA, preferably double-stranded. In addition, the … invention further encompasses non-human animals wherein the GPR4 has been inactivated, for instance a knock-out mouse lacking GPR4, and the use of said animals as an experimental model for angiogenesis and for screening for compounds modulating angiogenesis."


Title: Methods for Modulating the Efficacy of Nucleic Acid-Based Therapies

Application Number: 20100144840

Filed: Dec. 11, 2009

Lead Inventor: Dayton Reardan, Eleos

The patent application, its abstract states, claims "covalently reactive antigen analogs … [which] may be used to stimulate production of catalytic antibodies specific for predetermined antigens associated with particular medical disorders. The antigen analogs may also be used to permanently inactivate endogenously produced catalytic antibodies produced in certain autoimmune diseases as well as in certain lymphoproliferative disorders. Also provided are methods for modulating the efficacy of nucleic acid based therapeutics."


Title: RNA Interference-Mediated Inhibition of NOGO and NOGO Receptor Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20100144842

Filed: Jan. 26, 2010

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

"This invention relates to compounds, compositions, and methods useful for modulating NOGO and/or NOGO receptor gene expression using short interfering nucleic acid molecules," the patent application's abstract states. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of NOGO and/or NOGO receptor gene expression and/or activity by RNA interference using small nucleic acid molecules … methods used to modulate the expression of NOGO and/or NOGO receptor genes, such as NOGO-A, NOGO-B, NOGO-C, NOGO-66 receptor, NI-35, NI-220, NI-250, myelin-associated glycoprotein, tenascin-R, and NG-2."


Title: RNAi Therapeutic for Respiratory Virus Infection

Application Number: 20100144843

Filed: Feb. 12, 2010

Lead Inventor: James McSwiggen, MDRNA

The invention, the patent application's abstract states, claims "double-stranded siRNA molecules for combating a respiratory virus, wherein the strands of an siRNA molecule may be from about 15 to about 60 nucleotides, and uses thereof. One strand of an siRNA molecule can be a nucleic acid sequence identical to a conserved site, or a variant thereof, within the nucleic acid sequence of the respiratory virus."


Title: RNAi-Mediated Inhibition of H1F1A for Treatment of Ocular Angiogenesis

Application Number: 20100144844

Filed: Feb. 16, 2010

Lead Inventor: Jon Chatterton, Alcon

The patent application, its abstract states, claims "RNA interference … for inhibition of HIF1A mRNA expression for treating patients with ocular angiogenesis, particularly for treating retinal edema, diabetic retinopathy, sequela associated with retinal ischemia, posterior segment neovascularization, and neovascular glaucoma, and for treating patients at risk of developing such conditions."


Title: Oligonucleotide Systems for Targeted Intracellular Delivery

Application Number: 20100144845

Filed: Aug. 6, 2007 PCT Filed: Aug. 6, 2007

Lead Inventor: Omid Farokhzad, Massachusetts Institute of Technology

The invention, the patent application's abstract states, "provides methods for deriving oligonucleotides for specific internal delivery to one or more target cell types. The method generally includes selecting at least once with a target cell type to provide a plurality of internalizing oligonucleotides for the target cell type, and in some embodiments, counter-selecting at least once with a non-target cell type to provide a plurality of oligonucleotides that do not bind to features present in the non-target cell type. Therapeutic and diagnostic compositions including the oligonucleotides and methods of treatment are also provided."


Title: RNA Interference-Mediated Inhibition of Platelet-Derived Endothelial Cell Growth Factor Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20100144851

Filed: Jan. 19, 2010

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

"This invention relates to compounds, compositions, and methods useful for modulating platelet-derived endothelial cell growth factor and/or receptor gene expression using short interfering nucleic acid molecules," according to the patent application's abstract. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of ECGF1 and/or ECGF1r gene expression and/or activity by RNA interference using small nucleic acid molecules … and methods used to modulate the expression of ECGF1 and/or ECGF1r genes."


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

Application Number: 20100145038

Filed: Nov. 9, 2009

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

"This invention relates to compounds, compositions, and methods useful for modulating gene expression using short interfering nucleic acid molecules," the patent application's abstract states. "In particular, the … invention features small nucleic acid molecules … and methods used to modulate the expression of genes, such as expressed pseudogenes associated with the maintenance or development of diseases, disorders, traits, and conditions in a subject or organism."


Title: siRNA Targeting Nucleoprotein 62kDa

Application Number: 20100145039

Filed: Jan. 26, 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. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for Nup62."

The Scan

Possibly as Transmissible

Officials in the UK say the B.1.617.2 variant of SARS-CoV-2 may be as transmitted as easily as the B.1.1.7 variant that was identified in the UK, New Scientist reports.

Gene Therapy for SCID 'Encouraging'

The Associated Press reports that a gene therapy appears to be effective in treating severe combined immunodeficiency syndrome.

To Watch the Variants

Scientists told US lawmakers that SARS-CoV-2 variants need to be better monitored, the New York Times reports.

Nature Papers Present Nautilus Genome, Tool to Analyze Single-Cell Data, More

In Nature this week: nautilus genome gives peek into its evolution, computational tool to analyze single-cell ATAC-seq data, and more.