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IP Update: Sep 9, 2010


Title: Double-Stranded Compositions Comprising a 3'-Endo-Modified Strand for Use in Gene Modulation

Patent Number: 7,790,691

Filed: June 3, 2004 PCT Filed: June 3, 2004

Lead Inventor: Bryan Kraynack, Isis Pharmaceuticals

The invention "provides double stranded compositions that have a region that is complementary to a target nucleic acid," the patent's abstract states. "One of the targeting strand or the second strand comprises linked ribofuranosyl nucleosides and the other strand comprises linked modified nucleosides that have 3'-endo conformational geometry. The strands can be linked together or separate and may contain additional groups. The … invention also provides methods of using the compositions for modulating gene expression."

Title: Vaccinia Virus-Related Nucleic Acids and microRNA

Patent Number: 7,790,867

Filed: Aug. 28, 2003

Inventor: Isaac Bentwich, Rosetta Genomics

The invention, the patent's abstract states, "relates to a group of viral RNA regulatory genes, here identified as viral genomic address messenger genes or VGAM genes, and as genomic record or GR genes. VGAM genes selectively inhibit translation of known host target genes, and are believed to represent a pervasive viral attack mechanism. GR genes encode an operon-like cluster of VGAM genes.

"VGAM and viral GR genes may therefore be useful in diagnosing, preventing, and treating viral disease," the abstract adds. "Several nucleic acid molecules are provided respectively encoding several VGAM genes, as are vectors and probes, both comprising the nucleic acid molecules, and methods and systems for detecting VGAM genes, and for counteracting their activity."

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

Patent Number: 7,790,878

Filed: June 25, 2009

Inventor: Sailen Barik, Alnylam Pharmaceuticals

The invention, according to the patent's abstract, "is based on the in vivo demonstration that RSV and PIV 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: Methods to Regulate miRNA Processing by Targeting Lin-28

Application Number: 20100221266

Filed: Oct. 8, 2008 PCT Filed: Oct. 8, 2008

Lead Inventor: Richard Gregory, Children's Medical Center

The invention relates to "methods to regulate microRNA biogenesis, in particular the regulation of the processing of pri-miRNA to mature miRNA by Lin-28 and/or variants such as Lin28B," the patent application's abstract states. "In particular, the … invention relates to methods and compositions comprising at least one agent which inhibits Lin-28 function or activity and/or expression to increase the processing of pri-mRNA to mature miRNA. More specifically, one aspect of the invention is directed to treating and/or preventing cancer in a subject by administering an agent that inhibits Lin-28 activity or expression to a subject, preferably a human subject."

Title: Drug-Delivery Product and Methods

Application Number: 20100221357

Filed: May 17, 2010

Inventor: Gary Ostroff, University of Massachusetts

The invention "provides a particulate delivery system comprising an extracted yeast cell wall comprising beta-glucan, a payload molecule, and a payload trapping molecule," the patent application's abstract states. "The invention further provides methods of making and methods of using the particulate delivery system."

That payload, the application notes, can be an siRNA.

Title: High Potency siRNAs for Reducing the Expression of Target Genes

Application Number: 20100221789

Filed: Aug. 31, 2009

Lead Inventor: David Brown, Life Technologies

The invention, the patent application's abstract states, "provides improved methods of attenuating gene expression through the phenomenon of RNA interference. The invention provides methods of synthesis of double stranded RNAs of increased potency for use as small interfering RNA. Surprisingly and unexpectedly, siRNAs made by the methods of the invention are significantly more potent than previously available siRNAs."

Title: Compositions and Methods for Inhibiting Expression of Pro-Apoptotic Genes

Application Number: 20100222409

Filed: June 26, 2008 PCT Filed: June 26, 2008

Lead Inventor: Hagar Kalinski, Quark Pharmaceuticals

"The invention relates to one or more inhibitors, in particular siRNA compounds, which down-regulate the expression of a pro-apoptotic gene selected from the group consisting of TP53, HTRA2, KEAP1, SHC1-SHC, ZNHIT1, LGALS3, and HI95," the patent application's abstract states. "The invention also relates to a pharmaceutical composition comprising the compound and a pharmaceutically acceptable carrier. The … invention further provides methods of treating a subject afflicted with a disease or a condition associated with those genes, comprising administering to the subject a pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject."

Title: Chemically Modified Oligonucleotides for Use in Modulating microRNA and Uses Thereof

Application Number: 20100222413

Filed: March 1, 2010

Lead Inventor: Markus Stoffel, Rockefeller University (Alnylam Pharmaceuticals)

"This invention relates generally to chemically modified oligonucleotides useful for modulating expression of microRNAs and pre-microRNAs," the patent application's abstract states. "More particularly, the invention relates to single-stranded, chemically modified oligonucleotides for inhibiting microRNA and pre-microRNA expression and to methods of making and using the modified oligonucleotides. Also included in the invention are compositions and methods for silencing microRNAs in the central nervous system."

Title: siRNA Sequence-Independent Modification Formats for Reducing Off-Target Phenotypic Effects in RNAi, and Stabilized Forms Thereof

Application Number: 20100222414

Filed: March 19, 2009

Lead Inventor: Nitin Puri, Applied Biosystems

The patent application, its abstract states, claims "modification formats having modified nucleotides … for siRNA. Short interfering RNA having modification formats and modified nucleotides provided herein reduce off-target effects in RNA interference of endogenous genes. Further, modification-formatted siRNAs are demonstrated to be stabilized to nuclease-rich environments. Unexpectedly, increasing or maintaining strand bias, while necessary to maintain potency for endogenous RNA interference, is not sufficient for reducing off-target effects in cell biology assays."

Title: Compositions and Methods for Enhancing Oligonucleotide Delivery Across and Into Epithelial Tissues

Application Number: 20100222417

Filed: Nov. 25, 2009

Lead Inventor: Tracy Zimmerman, Alnylam Pharmaceuticals

The invention, the patent application's abstract states, "relates to the delivery of oligonucleotide across and into epithelial tissues by conjugating the oligonucleotide with a lipophile and/or coadministering with a penetration enhancer. In particular, the … invention provides a composition comprising a conjugated siRNA, which are advantageous for the in vivo delivery of nucleic acids across and into epithelial tissue. Additionally, the … invention provides methods of improving delivery of oligonucleotides across the epithelial tissues with the aid of a mechanical enhancer."

Title: Methods and Compositions for Isolating Small RNA Molecules

Application Number: 20100222564

Filed: Nov. 2, 2009

Inventor: Richard Conrad, Life Technologies

The invention "concerns the use of methods and compositions for the isolation of small RNA molecules, such as microRNA and siRNA molecules," the patent application's abstract states. "Such molecules are routinely lost in commonly used isolation procedures, and therefore the … invention allows for a much higher level of enrichment or isolation of these small RNA molecules."

The Scan

Study Finds Sorghum Genetic Loci Influencing Composition, Function of Human Gut Microbes

Focusing on microbes found in the human gut microbiome, researchers in Nature Communications identified 10 sorghum loci that appear to influence the microbial taxa or microbial metabolite features.

Treatment Costs May Not Coincide With R&D Investment, Study Suggests

Researchers in JAMA Network Open did not find an association between ultimate treatment costs and investments in a drug when they analyzed available data on 60 approved drugs.

Sleep-Related Variants Show Low Penetrance in Large Population Analysis

A limited number of variants had documented sleep effects in an investigation in PLOS Genetics of 10 genes with reported sleep ties in nearly 192,000 participants in four population studies.

Researchers Develop Polygenic Risk Scores for Dozens of Disease-Related Exposures

With genetic data from two large population cohorts and summary statistics from prior genome-wide association studies, researchers came up with 27 exposure polygenic risk scores in the American Journal of Human Genetics.