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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Tekmira, Alcon, and More

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Title: SnoRNAi-Small Nucleolar RNA Degradation by RNA Interference in Trypanosomatids

Patent Number: 8,486,910

Filed: March 12, 2012

Inventor: Shulamit Michaeli, Bar-Ilan University

The patent, its abstract states, claims “polynucleotides and a method suitable for downregulation of small nuclear RNA… to treat diseases associated with activity of small nuclear RNA. … Specifically, the … invention can be used to downregulate snoRNA molecules or box H/ACA-containing RNA molecules, which are involved in diseases such as cancer.”


Title: MicroRNA Fingerprints During Human Megakaryocytopoiesis

Patent Number: 8,486,912

Filed: Aug. 27, 2012

Lead Inventor: Carlo Croce, Ohio State University

The patent, its abstract states, claims “a method of decreasing expression of MAFB in a subject having a cancer and/or myeloproliferative disorder associated with overexpression of a MAFB gene product where an effective amount of at least one miR-130a gene product or an isolated variant or biologically-active fragment thereof is administered to the subject sufficient to decrease expression of the MAFB gene product in the subject.”


Title: MicroRNA Fingerprints During Human Megakaryocytopoiesis

Patent Number: 8,486,913

Filed: Aug. 27, 2012

Lead Inventor: Carlo Croce, Ohio State University

The patent, its abstract states, claims “a method of decreasing expression of HOXA1 in a subject having a cancer and/or myeloproliferative disorder associated with overexpression of a HOXA1 gene product where an effective amount of at least one miR-10a gene product or an isolated variant or biologically-active fragment thereof is administered to the subject sufficient to decrease expression of the HOXA1 gene product in the subject.”


Title: RNA Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof

Patent Number: 8,487,088

Filed: Dec. 23, 2010

Lead Inventor: Beverly Davidson, University of Iowa

The invention, the patent’s abstract states, is “directed to RNA interference molecules targeted against a nucleic acid sequence that encodes poly-glutamine repeat diseases, and methods of using these RNAi molecules.”


Title: Method and Medicament for Inhibiting the Expression of a Given Gene

Application Number: 20130177631

Filed: Jan. 29, 2013

Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals

“The invention relates to an oligoribonucleotide of [a] double-stranded structure for inhibiting the expression of a given target gene in mammalian cells and a method of mediating RNA interference of an mRNA of a gene in mammalian cells using the dsRNA,” the patent application’s abstract states.


Title: Transferrin/Transferrin Receptor-mediated siRNA Delivery

Application Number: 20130177580

Filed: Feb. 12, 2013

Lead Inventor: Jon Chatterton, Alcon

“The invention provides interfering RNA molecule-ligand conjugates useful as a delivery system for delivering interfering RNA molecules to a cell in vitro or in vivo,” according to the patent application’s abstract. “The conjugates comprise a ligand that can bind to a transferrin receptor. Therapeutic uses for the conjugates are also provided.”


Title: Nucleic Acid Molecules and Collections Thereof, Their Application, and Modification

Application Number: 20130178387

Filed: Dec. 20, 2012

Lead Inventor: Ronald Plasterk, Royal Netherlands Academy of Arts and Sciences

The invention, the patent application’s abstract states, relates to a “method for characterizing a sample … nucleic acid derived from a cell. The method [involves] determining whether a sample comprises at least a minimal sequence of at least one new microRNA … or a mammalian ortholog thereof, and characterizing the sample on the basis of the presence or absence of the miRNA. The invention further provides nucleic acid molecules and collections thereof and their use in therapeutic and diagnostic applications. The invention furthermore provides a method for identifying a miRNA molecule or a precursor molecule thereof.”


Title: Interfering RNA Delivery System and Uses Thereof

Application Number: 20130178421

Filed: Dec. 6, 2012

Lead Inventor: Ying Yu, Alcon

“The invention provides a delivery system comprising a cell penetrating peptide, 10 histidines, and an interfering RNA molecule,” the patent application’s abstract states. “The system can be used for delivering interfering RNA molecules into a cell in vivo or in vitro. Therapeutic uses for the delivery system are also provided.”


Title: Silencing of CSN5 Gene Expression Using Interfering RNA

Application Number: 20130178511

Filed: July 17, 2012

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

The invention, the patent application’s abstract states, “provides compositions comprising nucleic acids that target CSN5 gene expression and methods of using such compositions to silence CSN5 gene expression. More particularly, the … invention provides unmodified and chemically modified interfering RNA molecules which silence CSN5 gene expression and methods of use thereof, e.g., for treating cell proliferative disorders such as cancer. The … invention also provides nucleic acid-lipid particles that target CSN5 gene expression comprising an interfering RNA molecule, a cationic lipid, a non-cationic lipid, and optionally a conjugated lipid that inhibits aggregation of particles.”


Title: Novel Structurally Designed shRNAs

Application Number: 20130179999

Filed: April 22, 2011

Lead Inventor: Greg Hannon, Cold Spring Harbor Laboratory

The invention comprises “an improved design of shRNA based on structural mimics of miR-451 precursors,” according to the patent application’s abstract. “These miR-451 shRNA mimics are channeled through a novel small RNA biogenesis pathway, require AGO2 catalysis and are processed by Drosha but are independent of DICER processing. This miRNA pathway feeds active elements only into Ago2 because of its unique catalytic activity. These data demonstrate that this newly identified small RNA biogenesis pathway can be exploited in vivo to produce active molecules.”

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