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IP Update: May 26, 2011

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Title: RNA Interference for the Treatment of Gain-of-Function Disorders

Patent Number: 7,947,658

Filed: Sept. 13, 2004

Lead Inventor: Neil Aronin, University of Massachusetts

The invention, the patent's abstract states, “relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease. The … invention utilizes RNA interference technology against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.”


Title: iRNA Agents Targeting VEGF

Patent Number: 7,947,659

Filed: March 11, 2005

Lead Inventor: Antonin de Fougerolles, Alnylam Pharmaceuticals

According to the patent's abstract, the invention relates to “compounds, compositions and methods useful for modulating the expression of vascular endothelial growth factor, such as by the mechanism of RNA interference. The compounds and compositions include iRNA agents that can be unmodified or chemically modified.”


Title: RNAi-Mediated Inhibition of Frizzled-Related Protein-1 for Treatment of Glaucoma

Patent Number: 7,947,660

Filed: March 10, 2006

Lead Inventor: Abbot Clark, Alcon

The invention, the patent's abstract states, claims RNA interference for the “inhibition of frizzled-related protein-1 mRNA expression, in particular, for treating patients having glaucoma or at risk of developing glaucoma.”


Title: siRNA-Expression System

Patent Number: 7,947,823

Filed: July 9, 2004

Lead Inventor: Irene Bozzoni, Universita degli Studi di Roma 

The invention, the patent's abstract states, is “an expression system for the correct, stable, and effective expression in mammalian cells of a siRNA comprising a polymerase II RNA-dependent promoter derived from the U1 RNA gene.

Downstream from the sequence are “suitable restriction sites for the cloning of the sequence transcribing the pre-siRNA,” and downstream from these sites are “sequences derived from the sequences at 3' of the gene for U1 snRNA that are necessary and sufficient for the correct formation of 3' of the pre-siRNA,” it adds.


Title: Therapeutic Uses of Inhibitors of RTP801

Application Number: 20110117102

Filed: June 17, 2010

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

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


Title: microRNA-Based Diagnostic Testing and Therapies for Inflammatory Bowel Disease and Related Diseases

Application Number: 20110117111

Filed: March 26, 2009

Lead Inventor: John Kwon, Johns Hopkins University

The invention, the patent application's abstract states, “is based, at least in part, on the novel discovery that certain microRNAs are associated with inflammatory bowel diseases and other related diseases. Accordingly, the invention relates to microRNA-based compositions, kits, and methods for detecting, characterizing, modulating, preventing, and treating inflammatory bowel diseases and other related diseases.”


Title: Compositions and Methods for the Delivery of Nucleic Acids

Application Number: 20110117125

Filed: March 26, 2009

Lead Inventor: Michael Hope, Tekmira Pharmaceuticals

The invention provides “compositions and methods for the delivery of therapeutic agents to cells,” the patent application's abstract states. “In particular, these include novel lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the ... invention are highly potent, thereby allowing effective knockdown of specific target protein at relatively low doses. In addition, the compositions and methods of the ... invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.”


Title: Treating Hepatitis C Virus Infection with Over-Expression of microRNA-196

Application Number: 20110117181

Filed: Oct. 8, 2009

Lead Inventor: Herbert Bonkovsky, Carolinas Medical Center

The invention is “directed to methods of treating cells infected with HCV and mammals suffering from HCV infection by transfecting the infected cells with [a] miRNA-196 mimic,” the patent application's abstract states. The “miRNA-196 mimic significantly down-regulates Bach1 protein and HCV gene expression, while also up-regulating HMOX1 gene expression.”

The miRNA “binds with the 3'-UTR of Bach1 mRNA to reduce the expression of Bach1 … [and therefore] can play an important role in the regulation of HCV replication and HMOX1/Bach1 expression in hepatocytes,” the abstract adds. The invention also provides “a formulation for the treatment of cells expressing HCV comprising a therapeutically effective amount of miRNA-196 such that Bach1 and HCV gene expression are down-regulated while HMOX1 expression is increased. The formulations are adapted to enable the transfection of miRNA-196 mimic into hepatocytes expressing HCV proteins.”


Title: Novel Therapeutic RNA Interference Technology Targeted to the PDX-1 Oncogene in PDX-1-Expressing Neuroendocrine Tumors

Application Number: 20110117183

Filed: Oct. 27, 2010

Lead Inventor: John Nemunaitis, Gradalis

The patent application, its abstract states, claims “a bifunctional shRNA-based composition and methods for knocking down the expression of the PDX-1 oncogene in target cells. ... The invention also provides methods to deliver the shRNA-containing expression vectors to target tissues over-expressing the PDX-1 oncogene.”


Title: microRNA Associated with Rheumatoid Arthritis

Application Number: 20110117567

Filed: Feb. 23, 2009

Lead Inventor: Seiji Kawano, Kobe University

The patent application, its abstract states, claims a “novel marker for rheumatoid arthritis, and more specifically … a marker whose expression may be specifically increased or decreased in RA. … The marker includes [miRNAs] whose expression is specifically increased or decreased in RA synovial cells based on a small RNA expression profile in the RA synovial cells. In addition, the therapeutic agent for RA includes” miRNAs as active ingredients.


Title: Double-Stranded Nucleic Acid

Application Number: 20110117608

Filed: Oct. 28, 2010

Lead Inventor: Michael Graham, Benitec

“The invention is directed towards constructs for RNAi techniques … [and] provides a ribonucleic acid for use as interfering RNA in gene silencing techniques to silence a target gene comprising in a 5' to 3' direction at least a first effector sequence, a second effector sequence, a sequence substantially complementary to the second effector sequence and a sequence substantially complementary to the first effector sequence, wherein the complementary sequences are capable of forming double stranded regions with their respective effector sequences and wherein at least one of these sequences is substantially identical to the predicted transcript of a region of the target gene, and a nucleic acid construct encoding such an RNA,” according to the patent application's abstract.


Title: Modified Dicer Polypeptide and Methods of Use Thereof

Application Number: 20110117610

Filed: March 18, 2009

Lead Inventor: Jennifer Doudna, University of California, Berkeley

The invention, according to the patent application's abstract, comprises “a modified Dicer polypeptide … [that] exhibits enhanced catalytic activity. Also provided is a method for producing small regulatory RNAs from a dsRNA, involving contacting a dsRNA with a subject modified Dicer. Small regulatory RNAs produced by a subject method find use in a variety of applications, including research and therapeutic applications.”


Title: Cationic siRNAs, Synthesis, and Use for RNA Interference

Application Number: 20110118331

Filed: Jan. 30, 2009

Lead Inventor: France Jean-Paul Behr, Centre National de la Recherche Scientifique

“The invention relates to cationic siRNAs, characterized in that they are double-stranded RNA fragments, grafted to the ends of which are oligocations, the number of cationic charges grafted being comparable to or greater than that of the anionic charges carried by the internucleoside phosphates of the RNA strands,” according to the patent application's abstract.


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

Application Number: 20110118335

Filed: Nov. 16, 2010

Lead Inventor: Vasant Jadhav, Merck

The invention comprises “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 application'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 bifunctional short interfering nucleic acid (multifunctional siNA) molecules of the invention can target more than one region 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: Method of Using Compositions Comprising miR-192 and/or miR-215 for the Treatment of Cancer

Application Number: 20110118337

Filed: July 9, 2009

Lead Inventor: Nelson Chau, Merck

“The invention provides methods and compositions for inhibiting the proliferation of mammalian cells,” the patent application's abstract states. “In some embodiments, the methods comprise contacting mammalian cells with an effective amount of at least one small interfering nucleic acid agent that inhibits the level of expression of at least two miR-192 family responsive genes.”


Title: Chemically Modified Oligonucleotides and Uses Thereof

Application Number: 20110118339

Filed: Jan. 16, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“This invention relates generally to chemically modified oligonucleotides useful for augmenting activity of microRNAs and pre-microRNAs,” the patent application's abstract states. It also relates to “single-stranded chemically modified oligonucleotides for augmenting microRNA and pre-microRNA expression and to methods of making and using the modified oligonucleotides.”


Title: Delivery of RNAi Constructs to Oligodendrocytes

Application Number: 20110118340

Filed: Feb. 6, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“The invention provides methods for delivering a double-stranded ribonucleic acid to the central nervous system of a subject, and particularly, to oligodendrocytes of a subject by localized delivery to the brain,” including to the corpus callosum, the patent application's abstract states.


Title: Interfering RNA Molecules

Application Number: 20110118456

Filed: Jan. 7, 2001

Lead Inventor: Klaus Giese, Silence Therapeutics

The invention, the patent application's abstract states, “is related to a ribonucleic acid comprising a double-stranded structure... 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.”


Title: Compositions and Methods for Inhibiting Expression of TGF-Beta Receptor Genes

Application Number: 20110119781

Filed: July 8, 2009

Lead Inventor: Birgit Bramlage, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of a TGF-beta receptor type I gene comprising an antisense strand having a nucleotide sequence which is less than 30 nucleotides in length and which is substantially complementary to at least a part of a TGF-beta receptor type I gene,” the patent application's abstract states. “The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a TGF-beta receptor type I gene using said pharmaceutical composition; and methods for inhibiting the expression of a TGF-beta receptor type I gene in a cell.”

The Scan

Driving Malaria-Carrying Mosquitoes Down

Researchers from the UK and Italy have tested a gene drive for mosquitoes to limit the spread of malaria, NPR reports.

Office Space to Lab Space

The New York Times writes that some empty office spaces are transforming into lab spaces.

Prion Pause to Investigate

Science reports that a moratorium on prion research has been imposed at French public research institutions.

Genome Research Papers on Gut Microbe Antibiotic Response, Single-Cell RNA-Seq Clues to Metabolism, More

In Genome Research this week: gut microbial response to antibiotic treatment, approach to gauge metabolic features from single-cell RNA sequencing, and more.