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IP Update: Feb 24, 2011

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Title: Allele-Specific RNA Interference

Patent Number: 7,892,793

Filed: Sept. 30, 2005

Inventor: Zuoshang Xu, University of Massachusetts

“Human diseases caused by dominant, gain-of-function mutations develop in heterozygotes bearing one mutant and one wild-type copy of a gene,” the patent's abstract states. “Because the wild-type gene often performs important functions, whereas the mutant gene is toxic, any therapeutic strategy must selectively inhibit the mutant while retaining wild-type gene expression. The ... invention includes methods of specifically inhibiting the expression of a mutant allele, while preserving the expression of a co-expressed wild-type allele using RNAi. ... The invention also includes small interfering RNAs and small hairpin RNAs that selectively suppress mutant, but not wild-type, expression of copper zinc superoxide dismutase, which causes inherited amyotrophic lateral sclerosis,” it adds. The invention further provides “asysmmetric siRNAs and shRNAs with enhanced efficacy and specificity and mediating RNAi.”


Title: Regulation of Oncogenes by microRNAs

Patent Number: 7,893,034

Filed: Sept. 2, 2005

Lead Inventor: Frank Slack, Yale University

The invention, the patent's abstract states, relates to “naturally occurring miRNAs that regulate human oncogenes and methods of use thereof. ... Suitable nucleic acids for use in the methods and compositions described herein include, but are not limited to, pri-miRNA, pre-miRNA, mature miRNA, or fragments of variants thereof that retain the biological activity of the mature miRNA and DNA encoding a pri-miRNA, pre-miRNA, mature miRNA, fragments or variants thereof, or regulatory elements of the miRNA. The compositions containing nucleic acids are administered to a patient in need of treatment or prophylaxis of at least one symptom or manifestation of cancer. In one embodiment, the compositions are administered in an effective amount to inhibit gene expression of one or more oncogenes. Methods for treatment or prevention of at least one symptom or manifestation of cancer are also described.”


Title: Multi-Cistronic Constructs with siRNA to Inhibit Tumors

Patent Number: 7,893,035

Filed: May 15, 2007

Lead Inventor: Jasti Rao, University of Illinois

“Multi-cistronic short interfering RNA constructs targeting in various combinations a human urokinase-type plasminogen activator receptor, human urokinase-type plasminogen activator, human matrix metalloprotease 9, and cathepsin B inhibit tumors,” the patent's abstract states.


Title: In Vivo Production of Small Interfering RNAs that Mediate Gene Silencing

Patent Number: 7,893,036

Filed: March 26, 2008

Lead Inventor: Phillip Zamore, University of Massachusetts

“The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs that selectively silence targeted genes using the cell's own RNA interference pathway,” the patent's abstract states. “By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.”


Title: Oligonucleotides Comprising a Ligand Tethered to a Modified or Non-Natural Nucleobase

Patent Number: 7,893,224

Filed: July 31, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention “relates to a double-stranded oligonucleotide comprising at least one ligand tethered to an altered or non-natural nucleobase,” the patent's abstract states. “In certain embodiments, the non-natural nucleobase is difluorotolyl, nitropyrrolyl, or nitroimidazolyl. In certain embodiments, the ligand is a steroid or aromatic compound … [and in others] only one of the two oligonucleotide strands comprising the double-stranded oligonucleotide contains a ligand tethered to an altered or non-natural nucleobase.”

In other embodiments, “both of the oligonucleotide strands comprising the double-stranded oligonucleotide independently contain a ligand tethered to an altered or non-natural nucleobase … [or] the oligonucleotide strands comprise at least one modified sugar moiety,” it adds. “Another aspect of the ... invention relates to a single-stranded oligonucleotide comprising at least one ligand tethered to an altered or non-natural nucleobase. In certain embodiments, the non-natural nucleobase is difluorotolyl, nitropyrrolyl, or nitroimidazolyl. In certain embodiments, the ligand is a steroid or aromatic compound … [and in others] the ribose sugar moiety that occurs naturally in nucleosides is replaced with a hexose sugar, polycyclic heteroalkyl ring, or cyclohexenyl group. In certain embodiments, at least one phosphate linkage in the oligonucleotide has been replaced with a phosphorothioate linkage.”


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

Patent Numbers: 7,893,243 & 7,893,244

Filed: June 29, 2007 & Feb. 8, 2008

Lead Inventor: Yijia Liu, Intradigm

“Compositions and methods are provided for treatment of diseases involving unwanted neovascularization,” the patents' abstracts state. “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 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: Interfering RNA Molecules

Patent Number: 7,893,245

Filed: Aug. 28, 2008

Lead Inventor: Klaus Giese, Silence Therapeutics

The invention is related to a “ribonucleic acid comprising a double-stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid; and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended,” the patent's abstract states.


Title: siRNA Capable of Inhibiting the Expression of an Oncogene Involved in Cervical Cancer

Patent Number: 7,893,246

Filed: Sept. 9, 2008

Lead Inventor: Akira Saito, NEC

The invention, the patent's abstract states, “provides a novel oncogene polynucleotide from [a] human involving development of cervical cancer, comprising a nucleotide sequence encoding [a predefined] amino acid sequence.”


Title: siRNA Targeting Spleen Tyrosine Kinase

Patent Number: 7,893,247

Filed: Nov. 23, 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.”


Title: RNA Interference-Mediated Inhibition of Myc and/or Mycb Gene Expression Using Short Interfering Nucleic Acid

Patent Number: 7,893,248

Filed: Dec. 10, 2009

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

“This invention relates to compounds, compositions, and methods useful for modulating Myc and/or Myb gene expression using short interfering nucleic acid molecules,” the patent'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 Myc and/or Myb gene expression and/or activity by RNA interference using small nucleic acid molecules. ... The small nucleic acid molecules are useful in the treatment of cancer and other diseases and disorders.”


Title: Deprotection and Purification of Oligonucleotides and Their Derivatives

Patent Number: 7,893,249

Filed: Dec. 7, 2009

Lead Inventor: Keith Bowman, Sirna Therapeutics (Merck)

The invention comprises a “method for synthesis, deprotection, and/or purification of nucleic acid molecules, such as oligonucleotides comprising one or more ribonucleotides,” the patent's abstract states. “Such nucleic acid molecules include siRNA, dsRNA, ribozymes, antisense, and aptamers.”


Title: Inhibitory Polynucleotide Compositions and Methods for Treating Cancer

Application Number: 20110038849

Filed: Dec. 21, 2006

Lead Inventor: Frank Xie, Intradigm

The invention comprises “compositions and methods for treating diseases, such as cancers,” the patent application's abstract states. “The compositions are effective to silence, down-regulate, or suppress the expression of a validated target gene by stimulating the process of RNA interference of gene expression, thus inhibiting tumor growth. The invention also provides methods for treating diseases, such as cancers, by inactivation of a validated target gene product, using neutralizing antibody or small-molecule drug, to inhibit tumor growth.

“More particularly, the compositions and methods are directed toward a cancer or a precancerous growth in a mammal, associated with pathological expression of a certain target genes identified herein,” the abstract adds. “The compositions inhibit expression of the target gene when introduced into a tissue of the mammal. The methods include administering the compositions of the invention to a subject in need thereof in an amount effective to inhibit expression of a target gene in a cancerous tissue or organ.”


Title: Methods for Delivering siRNA Via Ionthophoresis

Application Number: 20110038937

Filed: Dec. 5, 2008

Lead Inventor: William Schubert, EyeGate Pharma

The invention, the patent application's abstract states, comprises “formulations of siRNA suitable for delivery by ocular iontophoresis, devices for iontophoretic delivery of siRNA ,and methods of use thereof.”


Title: Lipid Nanoparticle Compositions and Methods of Making and Using the Same

Application Number: 20110038941

Filed: Dec. 23, 2008

Lead Inventor: Robert Lee, Ohio State University

The patent application, its abstract states, claims “oligonucleotide-lipid nanoparticles made of at least one oligonucleotide, at least one lipid and at least one complexation agent for the oligonucleotide, methods of making and using, and devices for making the same.”


Title: siRNA Targeting Connective Tissue Growth Factor

Application Number: 20110039734

Filed: Oct. 1, 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 CTGF.”


Title: Polypeptide-Nucleic Acid Conjugates and Uses Thereof

Application Number: 20110039785

Filed: Dec. 19, 2008

Lead Inventor: Richard Beliveau, Angiochem

The invention “is directed to polypeptide-nucleic acid conjugates,” the patent application's abstract states. “These conjugates can allow for targeted application of a therapeutic RNAi agent across the blood-brain barrier to treat, for example, a cancer, neurodegenerative disease, or lysosomal storage disorder.”


Title: Methods and Materials for Reducing GLI2 Expression

Application Number: 20110039909

Filed: Oct. 13, 2009

Lead Inventor: Vladimir Spiegelman, University of Wisconsin, Madison

The invention, the patent application's abstract states, comprises “methods and materials for reducing expression of GLI2 … including nucleic acid molecules such as short hairpin RNAs that direct cleavage of GLI2 encoding transcripts and the use of such molecules for reducing prostate cancer cell growth.”


Title: Modified RNAi Polynucleotides and Uses Thereof

Application Number: 20110039914

Filed: Feb. 11, 2009

Lead Inventor: Pamela Pavco, RXi Pharmaceuticals

“The invention relates to improved RNAi constructs and uses thereof,” the patent application's abstract states. “The construct has a double-stranded region of 19 [to] 49 nucleotides, preferably 25, 26, or 27 nucleotides, and preferably blunt-ended. The construct has selective minimal modifications to confer an optimal balance of biological activity, toxicity, stability, and target gene specificity. … The constructs of the invention largely avoids the interferon response and sequence-independent apoptosis in mammalian cells, exhibits better serum stability, and enhanced target specificity.”


Title: Immunostimulatory siRNA Molecules

Application Number: 20110039915

Filed: Feb. 16, 2009

Lead Inventor: Michael Paul Marie Gantier, Monash University

The invention relates to “a double-stranded siRNA molecule that is capable of silencing gene expression, as well as inducing an immune response,” the patent application's abstract states. “The molecule comprises a sense strand and an antisense strand, wherein the antisense strand comprises a first nucleotide sequence that is specifically complementary to mRNA transcribed from a target gene, and the sense strand comprises a second nucleotide sequence that is substantially or perfectly complementary to the antisense strand with the exception that the second nucleotide sequence comprises at least one immunostimulatory motif comprising two or more non-complementary nucleotides. Such a molecule may be utilized in a method of treating or preventing a disease or condition in a subject.”


Title: miRNA-Regulated Differentiation-Dependent Self-Deleting Cassette

Application Number: 20110041196

Filed: Aug. 13, 2010

Lead Inventor: David Frendewey, Regeneron Pharmaceuticals

The patent application, its abstract states, claims “targeting constructs and methods of using them ... for differentiation-dependent modification of nucleic acid sequences in cells and in non-human animals. Targeting constructs comprising a promoter operably linked to a recombinase are provided, wherein the promoter drives transcription of the recombinase in a differentiated cell but not an undifferentiated cell. Promoters include Blimp1, Prm1, Gata6, Gata4, Igf2, Lhx2, Lhx5, and Pax3. Targeting constructs with a cassette flanked on both sides by recombinase sites can be removed using a recombinase gene operably linked to a 3'-UTR that comprises a recognition site for [a microRNA] that is transcribed in undifferentiated cells but not in differentiated cells. The constructs may be included in targeting vectors, and can be used to automatically modify or excise a selection cassette from an ES cell, a non-human embryo, or a non-human animal.”

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