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IP Update: Recent Patents, Patent Applications Awarded to Alnylam; Merck; Sylentis; and More

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Title: Nanoparticle-Amphipol Complexes for Nucleic Acid Intracellular Delivery and Imaging

Patent Number: 8,063,131

Filed: June 18, 2009

Inventor: Xiaohu Gao, University of Washington

The invention, the patent's abstract states, comprises “nanoparticle-amphiphilic polymer complexes for nucleic acid delivery and real-time imaging.”


Title: Techniques for Linking Non-Coding and Gene-Coding Deoxyribonucleic Acid Sequences and Applications Thereof

Patent Number: 8,065,091

Filed: Oct. 30, 2007

Lead Inventor: Isidore Rigoutsos, International Business Machines

The invention comprises “techniques for linking non-coding and gene coding regions of a genome,” the patent's abstract states. “In one aspect, a method of determining associations between non-coding sequences and gene coding sequences in a genome of an organism comprises … [identifying] at least one conserved region ... from one or more non-coding sequences. Additional instances of the conserved region are located in the untranslated or amino acid coding regions of one or more genes in the organism under consideration, and the conserved region is associated with the one or more biological processes in which these one or more genes participate.”


Title: Synthetic Genes and Genetic Constructs Comprising the Same

Patent Number: 8,067,383

Filed: Jan. 17, 2003

Lead Inventor: Michael Wayne Graham, Commonwealth Scientific and Industrial Research Organization (Benitec)

The invention relates “generally to synthetic genes for modifying endogenous gene expression in a cell, tissue, or organ of a transgenic organism, in particular a transgenic animal or plant,” the patent's abstract states. “More particularly, the … invention provides novel synthetic genes and genetic constructs which are capable of repressing delaying, or otherwise reducing the expression of an endogenous gene or a target gene in an organism when introduced thereto.”


Title: Silencing of TGF-Beta Type II Receptor Expression by siRNA

Patent Number: 8,067,389

Filed: July 20, 2009

Lead Inventor: Nalin Kumar, University of Illinois

The patent application, its abstract states, claims “siRNA-based silencing of the type II receptor of TGF-beta. [The] siRNAs that target this receptor abrogate the receptor protein and transcript. TGF-beta-mediated processes such as fibronectin assembly and cell migration also are inhibited and the molecules of the invention are efficacious in reducing the inflammatory response and matrix deposition. … Methods and compositions exploiting the findings of the ... invention have a wide-ranging application, extending from treatment of disorders of the eye to other organs and tissues throughout the body.”


Title: Therapeutic Targeting of Interleukins Using siRNA in Neutral Liposomes

Patent Number: 8,067,390

Filed: Feb. 29, 2008

Lead Inventor: William Merritt, University of Texas

The invention, the patent's abstract states, comprises “compositions that include an siRNA targeted to an interleukin and a neutral lipid, and methods of treating a human subject with cancer involving administering to the subject a pharmaceutically effective amount of an interleukin-8 antagonist or a composition.”


Title: Therapeutic Uses of Inhibitors of RTP801

Patent Number: 8,067,570

Filed: Jan. 18, 2007

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals (Silence Therapeutics)

The invention relates to “novel molecules, compositions, methods, and uses for treating microvascular disorders, eye diseases, respiratory conditions, and hearing disorders based upon inhibition of the RTP801 gene and/or protein,” the patent's abstract states.


Title: Hybrid Interfering RNA

Patent Number: 8,067,572

Filed: May 24, 2006

Lead Inventor: Jo Milner, University of York

“The invention relates to a hybrid interfering RNA molecule comprising a duplex RNA and a single-stranded DNA molecule, and its use in the ablation of mRNA and in polymerase chain reactions,” according to the patent's abstract.


Titles: Nucleic Acid Agents for Down-Regulating H19 and Methods of Using Same

Patent Numbers: 8,067,574; 8,067,573

Filed: July 6, 2006

Lead Inventor: Abraham Hochberg, Hebrew University of Jerusalem

The inventions, the patents' abstracts state, comprise “isolated oligonucleotides capable of down-regulating ... H19 mRNA in cancer cells,” as well as treating cancer by combining the oligos with an additional treatment.


Title: RNA Interference-Mediated Inhibition of Cyclin D1 Gene Expression Using Short Interfering Nucleic Acid

Patent Number: 8,067,575

Filed: Aug. 28, 2008

Lead Inventor: James McSwiggen, Merck

The invention, the patent's abstract states, “relates to compounds, compositions, and methods useful for modulating cyclin and/or cyclin dependent kinase gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of cyclin and/or cyclin dependent kinase gene expression and/or activity by RNA interference using small nucleic acid molecules.”


Title: siRNA Targeting Serine/Threonine Kinase 12

Patent Number: 8,067,576

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'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: Methods and Compositions for the Treatment of Intestinal Conditions

Patent Number: 8,067,577

Filed: Aug. 30, 2010

Lead Inventor: Ana Jimenez, Sylentis

The patent claims “methods and compositions for the treatment of intestinal disorders, such as [irritable bowel disease] and Crohn's disease,” its abstract states. “Also disclosed is a method of specifically targeting [short interfering nucleic acids] to treat intestinal disorders by intrarectal administration of [such] compounds.”


Title: siRNA of NF-KB P105 for Inhibiting Cell Proliferation and Migration and a Compound Comprising the Same

Application Number: 20110280929

Filed: Aug. 22, 2008

Lead Inventor: Myung-Ok Park, BiopolyMed

The invention, the patent application's abstract states, “relates to a siRNA which targets mRNA of the NF-kappa B p105 gene to cause its degradation by RNAi induction. In addition, the ... invention provides a pharmaceutical composition for the treatment of diseases associated with aberrant cell proliferation and migration, comprising the siRNA and delivery vehicle capable of intracellular delivery of the same.”


Title: Oligoribonucleotide or Peptide Nucleic Acid Capable of Inhibiting Activity of Hepatitis C Virus

Application Number: 20110281271

Filed: Nov. 26, 2009

Lead Inventor: Michinori Kohara, Tokyo Metropolitan Institute of Medical Science

The invention comprises “siRNA sequences that exhibit … effective RNAi activity against hepatitis C virus RNAs, the patent application's abstract states.


Title: Compositions and Methods for microRNA Expression Profiling of Colorectal Cancer

Application Number: 20110281756

Filed: Nov. 13, 2009

Lead Inventor: Ying Wu, Shanghai Medical College

The invention, the patent application's abstract states, relates to “compositions and methods for microRNA expression profiling of colorectal cancer. In particular, the invention relates to a diagnostic kit of molecular markers for identifying one or more mammalian target cells exhibiting or having a predisposition to develop colorectal cancer.”

The invention also relates to “a pharmaceutical composition for the prevention and/or treatment of colorectal cancer.”


Title: siRNA Targeting Beta Secretase

Application Number: 20110281769

Filed: July 1, 2011

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 BACE.”


Titles: RNA Sequence-Specific Mediators of RNA Interference

Application Numbers: 20110281931; 20110289611

Filed: Oct. 4, 2010

Lead Inventor: Thomas Tuschl, Rockefeller University (Max Planck Institute)

The invention relates to a “Drosophila in vitro system [that] was used to demonstrate that dsRNA is processed to RNA segments 21 [to] 23 nucleotides in length,” according to the patent application's abstract. “Furthermore, when these ... fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21 [to] 23 [nucleotide long] fragments are the sequence-specific mediators of RNA degradation.” The invention specifically comprises the fragments and “their use for specifically inactivating gene function.”


Title: Methods and Compositions for the Management of Cardiovascular Disease with Oligonucleotides

Application Number: 20110281933

Filed: May 13, 2011

Lead Inventor: Angel Baldan, Saint Louis University

The invention comprises “compositions and methods for treating cardiovascular disease and reducing the adverse effects induced by the administration of statins,” the patent application's abstract states. More specifically, the invention comprises “antisense compounds to augment the expression of miR-33 and associated genetic elements. In particular, methods of [treating] cardiovascular disease and the modulation of miR-33 levels [are] disclosed, as [are] treatment of the secondary effects including cholestasis, induced by the administration of statins.

“Also disclosed is the treatment of benign recurrent intrahepatic cholestasis and reverse cholesterol transport,” the abstract adds.


Title: Process for Synthesizing Oligonucleotide Phosphate Derivatives

Application Number: 20110282044

Filed: July 30, 2010

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention comprises a “simple, efficient, and enzyme-free method of making oligonucleotide phosphate derivatives,” the patent application's abstract states. “This invention presents [a] novel process using [an] automated synthesizer for synthesizing oligonucleotide phosphate derivatives using a diaryl phosphonate as reagent.”


Title: Compositions and Methods for Inhibiting Expression of a Mutant Gene

Application Number: 20110287090

Filed: July 6, 2011

Lead Inventor: Roland Kreutzer, Roche

The invention relates to “a double-stranded ribonucleic acid for inhibiting the expression of a mutant gene, comprising a first strand that has a ... region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand,” the patent application's abstract states. “The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.”


Title: Nucleic Acid-Delivery Compositions and Methods

Application Number: 20110287547

Filed: May 25, 2011

Inventor: Cory Berkland, University of Kansas

The invention, the patent application's abstract states, comprises “complexes [made of] a cationic polymer, a nucleic acid, and a metal ion. In some embodiments, a complex may be used as a means for delivering nucleic acid to a cell. … Methods of making a complex comprising a cationic polymer, a nucleic acid, and a metal ion are also described. Methods of condensing a polyplex comprising a cationic polymer and a nucleic acid are also provided.”


Title: microRNA Fingerprints During Human Megakaryocytopoiesis

Application Number: 20110287970

Filed: June 27, 2011

Lead Inventor: Carlo Croce, Ohio State University

The invention comprises “novel methods and compositions for the diagnosis, prognosis, and treatment of cancer and myeloproliferative disorders,” the patent application's abstract states. “The invention also provides methods of identifying anti-cancer agents.”


Title: Compositions and Methods for the Specific Inhibition of Gene Expression by dsRNA Containing a Tetraloop

Application Number: 20110288147

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: RNA Interference-Mediated Inhibition of Epithelial Sodium Channel Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20110288154

Filed: Nov. 18, 2009

Lead Inventor: Walter Strapps, Merck

The invention relates to “compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of ENaC gene expression and/or activity, and/or modulate [an] ENaC gene expression pathway,” the patent application's abstract states. “Specifically, the invention relates to double-stranded nucleic acid molecules … capable of mediating or that mediate RNA interference against ENaC gene expression.”


Title: siRNA Compounds and Methods of Use Thereof

Application Number: 20110288155

Filed: Dec. 17, 2009

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The patent application, its abstract states, “relates to double stranded oligonucleotide inhibitors of target genes, pharmaceutical compositions comprising same, and the use of such molecules to treat ... neurodegenerative disorders including Alzheimer's disease and amyotrophic lateral sclerosis, eye diseases … acute renal failure, hearing loss, acute respiratory distress syndrome, and in preventing or treating ischemia-reperfusion injury in organ transplant patients.”


Title: RNAi-Mediated Inhibition of Spleen Tyrosine Kinase-Related Inflammatory Conditions

Application Number: 20110288157

Filed: June 30, 2011

Lead Inventor: John Yanni, Alcon

“RNA interference is provided for inhibition of spleen tyrosine kinase mRNA expression, in particular, for treating patients having a Syk-related inflammatory condition or at risk of developing a Syk-related inflammatory condition such as allergic conjunctivitis, ocular inflammation, dermatitis, rhinitis, asthma, allergy, or mast-cell disease,” the patent application's abstract states.


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

Application Number: 20110288158

Filed: July 6, 2011

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

“The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene,” according to the patent application's abstract. “More particularly, the invention is directed to compositions that contain double stranded RNA, and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells.”

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Sandwich Sampling

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In Nature this week: catalog of DNA viruses of the human gut microbiome, new dense depth data dump format to store quantitative genomic data, and more.