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IP Update: May 5, 2011

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Title: RNA Interference-Mediated Inhibition of Hepatitis C Virus Expression Using Short Interfering Nucleic Acid

Patent Number: 7,935,812

Filed: April 3, 2009

Lead Inventor: James McSwiggen, Merck

The invention, the patent's abstract states, “relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of hepatitis C virus gene expression and/or activity. The ... invention is also directed to compounds, compositions, and methods relating to traits, diseases, and conditions that respond to the modulation of expression and/or activity of genes involved in hepatitis C virus gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases, and conditions. Specifically, the invention relates to double stranded nucleic acid molecules capable of mediating or that mediate RNA interference against hepatitis C virus gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules.”


Title: siRNA Target Hypoxia-Inducible Factor 1

Patent Number: 7,935,813

Filed: April 12, 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.”


Title: Methods and Compositions for Increasing the Activity of Inhibitory RNA

Application Number: 20110097328

Filed: June 23, 2010

Lead Inventor: Paul Chang, Massachusetts Institute of Technology

“The invention provides methods for increasing the activity of an inhibitory RNA in a subject requiring administering one or more poly-ADP-ribose polymerase inhibitors and/or one or more PARG activators to the subject,” the patent application's abstract states. “The invention also provides methods for increasing the activity of an inhibitory RNA in a cell or cell population requiring contacting a cell or cell population with one or more PARP inhibitors and/or one or more PARG activators. The invention further provides compositions and kits containing one or more PARP inhibitors and/or one or more PARG activators.”


Title: Ultra-Small RNAs as Toll-Like Receptor-3 Antagonists

Application Number: 20110097390

Filed: Feb. 20, 2009

Inventor: Jayakrishna Ambati, University of Kentucky

The invention, the patent application's abstract states, comprises “methods and compositions for the treatment or prevention of macular degeneration or other diseases or disorders associated with activation of TLR3. Administration of double-stranded RNAs having a length of 22 nucleotides or less treats or prevents macular degeneration or other diseases or disorders associated with activation of TLR3 due to the ability of the RNAs to bind to but not activate TLR3. Furthermore, all double-stranded RNAs — both targeted and non-targeted — of 22 nucleotides or less in length can bind to but not activate TLR3 and thereby treat or prevent such conditions.

“Also provided is a method for increasing the specificity of a desire siRNA target knockdown [involving] administering an amount of a target siRNA sufficient to knockdown a target gene and an amount of a double-stranded RNA of 22 nucleotides or less which prevents the target siRNA from activating TLR3,” the abstract adds.


Title: RNAi Agents Comprising Universal Nucleobases

Application Number: 20110097707

Filed: Oct. 29, 2010

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“One aspect of the ... invention relates to an oligonucleotide agent comprising at least one universal nucleobase,” the patent application's abstract states. “In certain embodiments, the universal nucleobase is difluorotolyl, nitroindolyl, nitropyrrolyl, or nitroimidazolyl … [while] in a preferred embodiment, the universal nucleobase is difluorotolyl. In certain embodiments, the oligonucleotide is double-stranded … [and in others it] is single-stranded.

“Another aspect of the ... invention relates to a method of altering the expression level of a target in the presence of [a] target sequence polymorphism,” it states. “In a preferred embodiment, the oligonucleotide agent alters the expression of different alleles of a gene. In another preferred embodiment, the oligonucleotide agent alters the expression level of two or more genes. In another embodiment, the oligonucleotide agent alters the expression level of a viral gene from different strains of the virus. In another embodiment, the oligonucleotide agent alters the expression level of genes from different species.”


Title: Methods for Detecting Oligonucleotides

Application Number: 20110097716

Filed: Aug. 29, 2009

Lead Inventor: Francois Jean-Charles Natt, Novartis

“The invention provides methods and compositions for detecting and/or quantifying nucleic acid oligonucleotides,” the patent application's abstract states. “These methods and compositions are useful for detecting and quantifying diagnostic and/or therapeutic synthetic target oligonucleotides, such as aptamers, RNAi, siRNA, antisense oligonucleotides, or ribozymes in a biological sample.”


Title: Screening Methods for Selected Amino Lipid-Containing Compositions

Application Number: 20110097720

Filed: Dec. 31, 2008

Lead Inventor: Marco Ciufolini, Alnylam Pharmaceuticals

“The invention features a method of identifying therapeutically relevant compositions which include a therapeutic agent and 2,2-Dilinoley 1-4-dimethylaminomethyl-[1,3]-dioxolane by screening for an effect of the agent on the liver of a model subject,” the patent application's abstract states.


Title: Inhibitors of RTP801 and Their Use in Disease Treatment

Application Number: 20110098337

Filed: Sept. 10, 2010

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention comprises “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,” according to the patent application's abstract.


Title: Methods Using dsDNA to Mediate RNA Interference

Application Number: 20110098200

Filed: Sept. 4, 2003

Lead Inventor: Gregory Martin Arndt, Johnson & Johnson Research

The invention provides “methods of producing dsDNA molecules that can be used to mediate RNA interference,” the patent application's abstract states. “These methods include the production of hairpin DNAs including random sequences, and the use of convergent promoters to co-express sense and antisense RNAs. As such, the invention allows the production of random short hairpin RNA and a small interfering RNA expression libraries for forward genetic screening.”


Title: RNA Interference for the Treatment of Heart Failure

Application Number: 20110098338

Filed: Dec. 23, 2008

Lead Inventor: Roger Hajjar, Mount Sinai Medical Center

The invention, the patent application's abstract states, “relates to targeted RNAi for the treatment of heart failure by modulating defective cardiac Ca2+ homeostasis via decreasing expression or activity of phospholamban using adeno-associated virus transfection of cardiomyocytes. Methods for decreasing ventricular arrhythmias, as well as methods for overall improvement of survival from heart failure in subjects, are also disclosed. Further, the ... invention provides methods which can be used to diagnose susceptibility to treatment by RNAi, and includes pharmaceutical compositions, kits and vectors including an RNAi sequence.”


Title: Novel RNA Interference Methods Using DNA-RNA Duplex Constructs

Application Number: 20110098461

Filed: Oct. 25, 2010

Lead Inventor: Shao-Yao Ying, University of Southern California

The invention provides “novel compositions and methods for suppressing the function or activity of a targeted gene through a novel intracellular piRNA-mediated RNAi mechanism using RNA-DNA duplex constructs,” according to the patent application's abstract. “The invention further provides novel methods and compositions for generating or producing RNA-DNA duplex agents, whose quantity is high enough to be used for the invention's gene silencing transfection and possibly in therapeutics applications. This improved RNA-polymerase chain reaction method utilizes thermocycling steps of promoter-linked DNA or RNA template synthesis, in vitro transcription, and then reverse transcription to bring the amount of RNA-DNA duplexes up to two thousand folds within one round of the above procedure for using in D-RNAi-directed gene silencing.”


Title: Nucleic Acid Molecule and Method of Targeting Gene Expression to Gliomas

Application Number: 20110098346

Filed: Nov. 4, 2008

Lead Inventor: Shu Wang, Agency for Science, Technology, and Research

The invention, the patent application's abstract states, comprises a nucleic acid molecule with “a glial-specific promoter, a coding sequence for a transgene, and a plurality of miRNA target sites. Each miRNA target site binds an miRNA that is down-regulated in a glioma cell compared to a normal glial cell, and the glial-specific promoter and the plurality of miRNA target sites are both operably linked to the coding sequence for the transgene.”

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