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IP Update: Mar 31, 2011


Title: Modified siRNA Molecules and Uses Thereof

Patent Number: 7,915,399

Filed: June 8, 2007

Lead Inventor: Ian MacLachlan, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention, the patent's abstract states, “provides chemically modified siRNA molecules and methods of using such siRNA molecules to silence target gene expression. Advantageously, the modified siRNA … is less immunostimulatory than its corresponding unmodified siRNA sequence and retains full RNAi activity against the target sequence.”

The invention also provides “nucleic acid-lipid particles comprising a modified siRNA, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. Methods for identifying and/or modifying an siRNA having immunostimulatory properties are also provided.”

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

Patent Number: 7,915,400

Filed: June 11, 2008

Lead Inventor: James McSwiggen, Merck

The invention comprises “methods and reagents useful in modulating hepatitis C virus gene expression in a variety of applications, including use in therapeutic, diagnostic, target-validation, and genomic-discovery applications,” the patent's abstract states. “Specifically, the invention relates to small nucleic acid molecules ... capable of mediating RNA interference against hepatitis C virus gene expression and/or activity. The small nucleic acid molecules are useful in the treatment and diagnosis of HCV infection, liver failure, hepatocellular carcinoma, cirrhosis, and any other disease or condition that responds to modulation of HCV expression or activity.”

Title: IMP-1 Oncogene as a Therapeutic Target and Prognostic Indicator for Lung Cancer

Application Number: 20110070245

Filed: Aug. 16, 2007

Lead Inventor: Yusuke Nakamura, Oncotherapy Science

“IMP-1 was abundantly expressed in the majority of lung cancers examined,” the patent application's abstract states. “Suppression of IMP-1 expression with siRNA effectively suppressed growth of [non-small cell lung cancer] cells. IMP-1 was able to bind to mRNAs encoding a variety of proteins involved in signal transduction, cell-cycle progression, cell adhesion, cytoskeleton, and various types of enzymatic activities. These results suggest that IMP-1 expression is likely to play important roles in lung cancer development and progression, and that IMP-1 is a prognostic marker and a promising therapeutic target for treatment of lung cancer.”

Title: Lipid-Encapsulated Dicer-Substrate Interfering RNA

Application Number: 20110071208

Filed: June 4, 2010

Lead Inventor: Ian MacLachlan, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention comprises “novel, stable nucleic acid-lipid particles comprising one or more Dicer-substrate dsRNAs and/or small hairpin RNAs, methods of making the particles, and methods of delivering and/or administering the particles,” the patent application's abstract states. “In some embodiments, the nucleic acid-lipid particles of the invention comprise Dicer-substrate dsRNAs and/or shRNAs. In other embodiments, the nucleic acid-lipid particles of the invention comprise Dicer-substrate dsRNAs and/or shRNAs in combination with one or more additional interfering RNAs. In further embodiments, the Dicer-substrate dsRNAs and/or shRNAs present in the nucleic acid-lipid particles of the invention are chemically synthesized.”

Title: RNAi Modulation of the RHO-1 Gene and Uses Thereof

Application Number: 20110071209

Filed: July 30, 2010

Lead Inventor: Juergen Soutschek, Alnylam Pharmaceuticals

“The invention relates to compositions and methods for modulating the expression of the RhoA gene, and more particularly to the down-regulation of RhoA by chemically modified oligonucleotides,” the patent application's abstract states.

Title: microRNA and Downstream Targets for Diagnostic and Therapeutic Purposes

Application Number: 20110071211

Filed: Feb. 26, 2009

Lead Inventor: Thomas Thum, University of Wurzberg

The invention, the patent application's abstract states, “relates to a promoter region of a microRNA, the use of a microRNA, in particular miR-21, and related elements for the diagnosis and for the manufacture of a medicament for the treatment and/or prevention of fibrosis and/or fibrosis related diseases. Additionally, the invention concerns antisense oligonucleotides against targets of miR-21.

“A cell deficient for miR-21, the promoter region and targets of miR-21, and a knock-out organism thereof are also encompassed,” it adds. “Finally, the invention is directed to a method for diagnosing fibrosis and/or fibrosis related diseases and to a method for screening a pharmaceutically active compound for the treatment of fibrosis and/or fibrosis related diseases.”

The invention “further relates to compositions for use in the treatment, amelioration, and/or prevention of fibrosis,” the abstract states. “In certain embodiments, the compositions modulate the activity of a miRNA for the treatment, amelioration, and/or prevention of fibrosis. In certain embodiments, the compositions inhibit the activity of miR-21 for the treatment, amelioration, and/or prevention of fibrosis.”

The Scan

Review of Approval Process

Stat News reports the Department for Health and Human Services' Office of the Inspector General is to investigate FDA's approval of Biogen's Alzheimer's disease drug.

Not Quite Right

A new analysis has found hundreds of studies with incorrect nucleotide sequences reported in their methods, according to Nature News.

CRISPR and mRNA Together

Time magazine reports on the use of mRNA to deliver CRISPR machinery.

Nature Papers Present Smartphone Platform for DNA Diagnosis of Malaria, Mouse Lines for Epigenomic Editing

In Nature this week: a low-cost tool to detect infectious diseases like malaria, and more.