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IP Update: Recent RNAi- and microRNA-Related Patents and Patent Applications: Aug 11, 2011

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Title: Methods for Producing microRNAs

Patent Number: 7,993,925

Filed: June 5, 2008

Lead Inventor: Ross Dickins, Cold Spring Harbor Laboratory

“The invention relates to recombinant vectors for inducible and/or tissue specific expression of double-stranded RNA molecules that interfere with the expression of a target gene,” the patent's abstract states. “In certain embodiments, the invention relates to the use of Tet-responsive RNA polymerase II promoters to direct inducible knockdown in certain cells of an integrated or an endogenous gene, such as p53. The invention also relates to a method for producing transgenic animals expressing inducible, reversible, and/or tissue-specific double-stranded RNA molecules that interfere with the expression of a target gene.”


Title: Method for Treatment of Huntington's Disease Through Intracranial Delivery of siRNA

Patent Number: 7,994,149

Filed: Feb. 9, 2009

Lead Inventor: William Kaemmerer, Medtronic

The invention, the patent's abstract states, claims “devices, small interfering RNAs, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The … invention also provides valuable small interfering RNA vectors, systems, and methods for treating Huntington's disease in vivo without impairment of cell endoplasmic reticulum, spontaneous motor activity, or locomotor activity of a patient.”


Title: Compositions and Methods for siRNA Inhibition of Angiopoietin 1 and 2 and Their Receptor Tie-2

Patent Number: 7,994,305

Filed: April 19, 2004

Lead Inventor: Samuel Reich, University of Pennsylvania (Opko Health)

The patent, its abstract states, claims “RNA interference using small interfering RNAs … specific for mRNA produced from the Ang1, Ang2, or Tie2 genes inhibits expression of these genes. Diseases which involve Ang1-, Ang2-, or Tie2-mediated angiogenesis, such as inflammatory and autoimmune diseases, diabetic retinopathy, age-related macular degeneration, and many types of cancer, can be treated by administering the small interfering RNAs.”


Title: Methods of Treating Cancer Using siRNA Molecules Directed Against CD24

Patent Number: 7,994,306

Filed: Nov. 13, 2007

Lead Inventor: Nadir Arber, Tel Aviv Medical Center

The invention, the patent's abstract states, comprises “a method of treating a CD24-related medical condition [involving] … administering to a subject in need thereof at least one siRNA molecule selected from [a pre-defined] group.”


Title: RNAi Modulation of the BCR-ABL Fusion Gene and Uses Thereof

Patent Number: 7,994,307

Filed: July 27, 2009

Lead Inventor: Philipp Hadwiger, Alnylam Pharmaceuticals

“The invention relates to compositions and methods for modulating the expression of Bcr-Abl and, more particularly, to the down-regulation of Bcr-Abl mRNA and Bcr-Abl protein levels by oligonucleotides via RNA interference,” the patent's abstract states.


Title: Compositions and Methods for Inhibiting Expression of a Mutant Gene

Patent Number: 7,994,309

Filed: June 16, 2010

Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals

The invention, the patent's abstract states, “relates to a double-stranded ribonucleic acid for inhibiting the expression of a mutant gene, comprising a complementary RNA strand having a complementary region that is substantially complementary to a portion of the mutant gene, and which is partially complementary to the corresponding wild-type gene. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.”


Title: Group of Nucleic Acid Fragments for Prevention of HIV Infection or AIDS and the Usage Thereof

Application Number: 20110189264

Filed: Jan. 10, 2011

Lead Inventor: Zhiwen Zhou, Beijing Solobio Genetechnology

“The invention provides a group of nucleic acid fragments ... for prevention of HIV infection or AIDS and the usage thereof,” the patent application's abstract states. “The double-stranded RNA derived from these fragments can effectively inhibit the expression of the HIV genes. The RNA transcribed by plasmid also can suppress the expression of the HIV in the cell. After the adenovirus or associated virus … infect the cell, the transcription dsRNA can inhibit the expression of the HIV genes.”


Title: siRNA Silencing of Apolipoprotein B

Application Number: 20110189300

Filed: May 20, 2010

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

The invention, the patent application's abstract states, “provides nucleic acid-lipid particles comprising siRNA molecules that silence ApoB expression and methods of using such nucleic acid-lipid particles to silence ApoB expression.”


Title: microRNAs and Uses Thereof

Application Number: 20110189766

Filed: Feb. 8, 2010

Lead Inventor: Isaac Bentwich, Rosetta Genomics

The patent application, its abstract states, claims “novel polynucleotides associated with prostate and lung cancer. The polynucleotides are miRNAs and miRNA precursors. Related methods and compositions that can be used for diagnosis, prognosis, and treatment of those medical conditions are disclosed. Also described herein are methods that can be used to identify modulators of prostate and lung cancer.”


Title: microRNA-Based Methods and Compositions for the Diagnosis, Prognosis, and Treatment of Lung Cancer Using miR-21

Application Number: 20110190160

Filed: March 30, 2011

Lead Inventor: Carlo Croce, Ohio State University

The invention provides “novel methods and compositions for the diagnosis, prognosis, and treatment of lung cancer,” the patent application's abstract states. “The invention also provides methods of identifying anti-lung cancer agents.”


Title: Compositions Comprising C-Myc siRNA and Methods of Uses Thereof

Application Number: 20110190375

Filed: June 11, 2009

Inventor: Frank Xie, Intradigm (Silence Therapeutics)

The invention provides “nucleic acid molecules that inhibit c-Myc expression,” the patent application's abstract states. “Methods of using the nucleic acid molecules are also provided.”


Title: RNAi-Related Inhibition of TNFa Signaling Pathway for Treatment of Macular Edema

Application Number: 20110190376

Filed: Feb. 21, 2011

Lead Inventor: Jon Chatterton, Alcon

“RNA interference is provided for inhibition of tumor necrosis factor-alpha by silencing TNF-alpha cell surface receptor TNF receptor-1 mRNA expression, or by silencing TNF-alpha converting enzyme mRNA expression,” according to the patent application's abstract. “Silencing such TNF-alpha targets, in particular, is useful for treating patients having or at risk of developing macular edema.”


Title: Methods for Delivery of siRNA to Bone Marrow Cells and Uses Thereof

Application Number: 20110190380

Filed: Oct. 23, 2008

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention relates to a “method for the delivery of therapeutic oligonucleotides to bone marrow and, in particular, delivery of siRNA to a subset of bone marrow cells,” according to the patent application's abstract. “The method comprises systemically administering siRNA to a subject in need thereof to reduce or inhibit expression of a gene associated with a disease or disorder or to symptoms associated with a disease or disorder associated with the cells. The invention further relates to chemically modified siRNA compounds, to pharmaceutical compositions comprising such compounds, and to methods of using such compounds and compositions in the treatment of disease.”


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

Application Number: 20110190381

Filed: April 12, 2011

Lead Inventor: Abbot Clark, Alcon

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


Title: Diagnostic, Prognostic, and Therapeutic Uses of miRs in Adaptive Pathways and/or Disease Pathways

Application Number: 20110190383

Filed: Sept. 18, 2009

Lead Inventor: Clay Marsh, Ohio State University

The invention, the patent application's abstract states, comprises “methods and compositions for the diagnosis, prognosis, and treatment of various adaptive and/or of disease pathways by examining samples containing one or more miRs therein, and by formulating therapeutic agents therefrom.”


Title: siRNA Specific to WT1 17AA- Isoform and Use Thereof

Application Number: 20110190384

Filed: March 28, 2007

Lead Inventor: Haruo Sugiyama

The invention comprises a specific siRNA sequence that can be used as a “cancer cell-specific molecular targeted therapy,” according to the patent application's abstract.

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