Title: Methods and Compositions for Silencing Genes without Inducing Toxicity
Patent Number: 8,299,042
Filed: Sept. 24, 2008
Lead Inventor: Catherine Pachuk, Nucleonics (Alnylam Pharmaceuticals)
The ... invention, the patent's abstract states, “provides methods of post-transcriptional gene silencing [that] involve the use of a ... dsRNA having substantial sequence identity to a target nucleic acid and a short, second dsRNA [that] inhibits dsRNA-mediated toxicity. These methods can be used to prevent or treat a disease or infection by silencing a gene associated with the disease or infection. The invention also provides methods for identifying nucleic acid sequences that modulate a detectable phenotype, including the function of a cell, the expression of a gene, or the biological activity of a target polypeptide.”
Title: Adenoviral VA1 Pol III Expression System for RNAi Expression
Patent Number: 8,299,045
Filed: Aug. 25, 2011
Lead Inventor: John Rossi, City of Hope
The invention comprises “an adenoviral VA1 Pol III expression system for RNAi expression,” the patent's abstract states.
Title: siRNA Hat Inhibits WT1 Gene Expression and Uses Thereof
Patent Number: 8,299,234
Filed: March 29, 2005
Lead Inventor: Haruo Sugiyama, International Institute of Cancer Immunology
According to the patent's abstract, “siRNAs targeting the 17AA site of the WT1 gene not only suppress the expression of the WT1 gene, but also demonstrate remarkable cell growth-suppressing effects and cell death-inducing effects in cancer cell lines.”
Title: RNA Molecules and Vectors for Gene Silencing
Patent Number: 8,299,235
Filed: Dec. 17, 2004
Lead Inventor: David Baulcombe, Plant Bioscience Limited
The invention, the patent's abstract states, comprises “compositions for gene silencing by providing short RNA molecules to cells.”
Title: Compositions and Methods for Enhancing Delivery of Nucleic Acids into Cells and for Modifying Expression of Target Genes in Cells
Patent Number: 8,299,236
Filed: Sept. 8, 2008
Lead Inventor: Lishan Chen, Marina Biotech
“Polynucleotide delivery-enhancing polypeptides are admixed or complexed with, or conjugated to, nucleic acids for enhancing delivery the nucleic acids into cells,” the patent's abstract states. “The transported nucleic acids are active in target cells as small inhibitory nucleic acids that modulate expression of target genes, mediated at least in part by RNA interference. The siNA/polypeptide compositions and methods of the invention provide effective tools to modulate gene expression and alter phenotype in mammalian cells, including by altering phenotype in a manner that eliminates disease symptoms or alters disease potential in targeted cells or subject individuals to which the siNA/polypeptide compositions are administered.”
Title: siRNA Targeting Fructose-1, 6-Biphosphatase
Patent Number: 8,304,528
Filed: Dec. 3, 2009
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)'
“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent's abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for FBP1.”
Title: Methods and Compositions for Enhancing the Efficacy and Specificity of RNA Silencing
Patent Number: 8,304,530
Filed: March 23, 2010
Lead Inventor: Phillip Zamore, University of Massachusetts
The invention, the patent's abstract states, “provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.”
Title: Method of Inducing an Immune Response
Application Number: 20120269850
Filed: June 21, 2012
Lead Inventor: Nigel McMillan, University of Queensland
The invention, the patent application's abstract states, comprises “a method ... for inducing or enhancing an immune response in a mammal to a target polypeptide expressed in a plurality of cells of the mammal.” The method involves “administering to the mammal an inhibitory nucleic acid which targets a region of a ribonucleic acid [that] encodes [the] polypeptide. Also provided is a pharmaceutical composition comprising an inhibitory nucleic acid which targets a region of an RNA [that] encodes a target polypeptide expressed in a plurality of cells of a mammal.”
Title: microRNA-140-5P as a Tumor Suppressor and Sensitizing Agent for Chemotherapy
Application Number: 20120269883
Filed: April 23, 2012
Lead Inventor: Preethi Gunaratne, University of Houston
The invention relates to “a method of improving a therapeutic response to a cancer treatment … [by] administering an effective amount of an agent that enhances the expression of microRNA-140-5p or an agent that mimics the effects of microRNA-140-5p,” according to the patent application's abstract. “Further provided is a method of treating a cancer … [by] administering an effective amount of a microRNA-140-5p or an agent that enhances the expression of microRNA-140-5p.”
Title: Method for Analyzing Secretome, Biomarker for Lung Cancer Metastasis, and siRNA Compound for Inhibiting Lung Cancer Metastasis
Application Number: 20120270254
Filed: Dec. 27, 2011
Lead Inventor: Pao-Chi Liao, National Cheng Kung University
The invention comprises “a method for analyzing [the] secretome, a biomarker for lung cancer metastasis, and a siRNA compound for inhibiting lung cancer metastasis,” according to the patent application's abstract.
Title: Alternative Export Pathways for Vector-Expressed RNA Interference
Application Number: 20120270317
Filed: June 21, 2012
Lead Inventor: Scott Harper, University of Iowa
The invention is “directed to nucleic acid molecules containing a loop sequence designed to circumvent exportin-5 mediated export, and methods using these novel molecules,” according to the patent application's abstract.
Title: siRNA Targeting Diacylglycerol O-Acyltransferase Homolog 2
Application Number: 20120270751
Filed: July 2, 2012
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent application's abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for DGAT2.”
Title: Lipid-Formulated Compositions and Methods for Inhibiting Expression of a Gene from the Ebola Virus
Application Number: 20120270921
Filed: Aug. 13, 2010
Lead Inventor: Antonin de Fougerolles, Alnylam Pharmaceuticals
“The invention relates to lipid formulated double-stranded ribonucleic acid for inhibiting the expression of a gene from the Ebola virus,” the patent application's abstract states.
Title: siRNA Targeting Apolipoprotein
Application Number: 20120270926
Filed: June 28, 2012
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent application's abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for ApoB.”
Title: Polyamides for Nucleic Acid Delivery
Application Number: 20120270927
Filed: July 3, 2012
Inventor: Theresa Reineke, University of Minnesota
The invention, the patent application's abstract states, comprises “a new class of non-viral transduction vectors that can be used for both in vivo and in vitro applications, including, a gene transfer vector that has comparable efficiency to a viral vector without the potential for a life-threatening immune response. … Complexes including a cellular delivery molecule or agent that can facilitate the translocation of the complex or portion thereof into cells is also provided. The cellular delivery molecules may include one or more polymers.”
Title: Identification of RNAi Targets and Use of RNAi for Rational Therapy of Chemotherapy-Resistant Leukemia and Other Cancers
Application Number: 20120272346
Filed: March 29, 2010
Lead Inventor: Bruce Stillman, Cold Spring Harbor Laboratory
The patent application, its abstract states, claims “a mosaic mouse model for use in determining the potency of an shRNA in vivo for reducing survival of cancer cells of chemotherapy-resistant leukemia. The syngeneic mouse recipient is transplanted with tet-on competent leukemia cells carrying a bi-cistronic nucleic acid construct comprising a promoter operably linked to a fusion gene associated with chemotherapy-resistant leukemia, and a sequence encoding a reverse tet-transactivator protein, such that both coding sequences are co-expressed from the promoter. Also provided are methods of treating soft tissue cancers.”
Title: Nucleic Acid-Containing Lipid Particles and Related Methods
Application Number: 20120276209
Filed: May 4, 2012
Lead Inventor: Pieter Cullis, University of British Columbia
The patent application, its abstract states, claims “lipid particles containing a nucleic acid, devices, and methods for making the lipid particles, and methods for using the lipid particles.”
Title: Means and Methods for the Specific Modulation of Target Genes in the CNS and the Eye and Methods for Their Identification
Application Number: 20120277288
Filed: June 11, 2012
Lead Inventor: Karina Drumm, Acuity Pharmaceuticals (Opko Health)
The patent application, its abstract states, claims “methods for the treatment of disorders of the central nervous system and the eye. In particular, use of compositions comprising a compound capable of modulating a target gene or gene product is described for the preparation of a pharmaceutical composition for the treatment of disorders of the CNS and/or the eye, wherein the composition is designed to be administered outside the blood-CNS and the blood-retina barriers. Furthermore, methods are provided for identifying and obtaining nucleic acid molecules encoding polypeptides involved in CNS disorders or of the eye, methods for diagnosing said disorders, as well as transgenic animal deficient in the expression of target genes. ... In addition, methods of identifying and isolating drugs that are particularly useful for the treatment of disorders related to the CNS and/or the eye are disclosed.”
Title: Activity-Generating Delivery Molecules
Application Number: 20120277289
Filed: Nov. 4, 2010
Lead Inventor: Renata Fam, Marina Biotech
The invention, the patent application's abstract states, comprises “activity-generating delivery molecules ... compositions and methods of use thereof. The activity-generating delivery compounds and compositions are useful for generating activity of an active agent in a cell, tissue, or subject.”