Patents Examined by Sean McGarry
  • 5?-phosphorothiolate mRNA 5?-end (cap) analogs, mRNA comprising the same, method of obtaining and uses thereof
    Patent number: 11066436
    Abstract: The present invention relates to nucleotides, analogs of mRNA 5?-end (cap) containing sulfur atom at the position 5? of 7-methylguanosine nucleoside. The disclosed compounds are recognized (bound and non-hydrolyzed) by DcpS enzyme (Decapping Scavenger), and thus may find therapeutic use as inhibitors thereof. DcpS is cap-specific enzyme with pyrophosphatase activity, which was identified as a therapeutic target in the treatment of spinal muscular atrophy (SMA). Some of the compounds disclosed have additional modifications in the phosphate chain, which modulate their affinity for DcpS enzyme.
    Type: Grant
    Filed: January 27, 2017
    Date of Patent: July 20, 2021
    Assignee: UNIWERSYTET WARSZAWSKI
    Inventors: Jacek Jemielity, Kaja Fac-Da̧browska, Blażej Wojtczak, Marek Baranowski, Anna Nowicka, Joanna Kowalska, Paweł Sikorski, Marcin Warmiński
  • Hepatitis B virus (HBV) iRNA compositions and methods of use thereof
    Patent number: 11060091
    Abstract: The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the hepatitis B virus (HBV) genome, and methods of using such RNAi agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection and/or HBV-associated disorder, e.g., chronic hepatitis B infection.
    Type: Grant
    Filed: November 4, 2019
    Date of Patent: July 13, 2021
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Gregory Hinkle, Laura Sepp-Lorenzino, Vasant Jadhav, Martin Maier, Stuart Milstein, Muthiah Manoharan, Kallanthottathil G. Rajeev
  • Modulation of prekallikrein (PKK) expression
    Patent number: 11053500
    Abstract: Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.
    Type: Grant
    Filed: September 7, 2018
    Date of Patent: July 6, 2021
    Assignee: lonis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Huynh-Hoa Bui
  • Oligonucleotide sequences targeting transcription factor TSC22D4 for the treatment of insulin resistance
    Patent number: 11053499
    Abstract: The present invention relates to oligonucleotide inhibitors of the TSC22D4 activity or expression and their uses for the prevention, treatment, and/or regulation of insulin resistance, metabolic syndrome and/or diabetes and/or for improving insulin sensitivity in a mammal.
    Type: Grant
    Filed: April 30, 2020
    Date of Patent: July 6, 2021
    Assignee: DEUTSCHES KREBSFORSCHUNGSZENTRUM STIFTUNG DES ÖFFENTLICHEN RECHTS
    Inventors: Stephan Herzig, Mauricio Berriel Diaz, Tobias Schafmeier
  • Patatin-like phospholipase domain containing 3 (PNPLA3) iRNA compositions and methods of use thereof
    Patent number: 11052103
    Abstract: The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such RNAi agents to inhibit expression of a PNPLA3 gene and methods of treating subjects having Nonalcoholic Fatty Liver Disease (NAFLD) and/or a PNPLA3-associated disorder.
    Type: Grant
    Filed: February 5, 2019
    Date of Patent: July 6, 2021
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Kevin Fitzgerald, Gregory Hinkle
  • Modified AAV constructs and uses thereof
    Patent number: 11046955
    Abstract: The present disclosure relates to the field of rAAV delivery of transgenes. In some aspects, the disclosure relates to RNAi. Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising modified ITRs. In some embodiments, the modified ITRs comprise a sequence encoding a shRNA, miRNA, or AmiRNA.
    Type: Grant
    Filed: April 15, 2016
    Date of Patent: June 29, 2021
    Assignee: University of Massachusetts
    Inventors: Guangping Gao, Jun Xie, Phillip D. Zamore
  • Pharmaceutical compositions and methods for potentiating gene silencing
    Patent number: 11040057
    Abstract: In various aspects and embodiments, the invention provides compounds or agents that potentiate siRNA cellular entry or activity, and provides methods for identifying such compounds or agents. Exemplary agents that act as L-type calcium channel blockers are described herein, and are shown to potentiate gene silencing with cp-asiRNAs.
    Type: Grant
    Filed: June 29, 2017
    Date of Patent: June 22, 2021
    Assignee: OliX Pharmaceuticals, Inc.
    Inventors: Dong Ki Lee, Da Seul Son, Yang Hee Kim
  • Treatment using truncated Trk B and Trk C antagonists
    Patent number: 11034961
    Abstract: Disclosed herein are methods, compositions, vectors, and kits comprising an antagonist of a truncated TrkC or a truncated TrkB. Also described herein are methods of treating and/or preventing an otic disease or condition associated with an elevated expression level of a truncated TrkC or truncated TrkB isoform.
    Type: Grant
    Filed: July 20, 2018
    Date of Patent: June 15, 2021
    Assignee: OTONOMY, INC.
    Inventors: Horacio Uri Saragovi, Fabrice Piu
  • Modulation of Lnc05 expression
    Patent number: 11033570
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of Inc05 in a cell or individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a cancer in an individual.
    Type: Grant
    Filed: December 1, 2017
    Date of Patent: June 15, 2021
    Assignees: Cold Spring Harbor Laboratory, Ionis Pharmaceuticals, Inc.
    Inventors: Allen Tingjin Yu, David L. Spector, Frank Rigo, Susan M. Freier, Jan Bergmann, Carmen Berasain
  • Targeting PAX2 for the treatment of breast cancer
    Patent number: 11033628
    Abstract: The present application provides methods of prevention and/or treatment of breast cancer in a subject by inhibiting expression of PAX2. In the cancer treatment methods disclosed, the method of inhibiting expression of PAX2 can be by administration of a nucleic acid encoding an siRNA for PAX2. A method of treating cancer in a subject by administering DEFB1 is also provided. Similarly, provided is a method of treating cancer in a subject by increasing expression of DEFB1 in the subject.
    Type: Grant
    Filed: August 20, 2014
    Date of Patent: June 15, 2021
    Assignee: PHIGENIX, INC.
    Inventor: Carlton D. Donald
  • Antisense nucleic acids
    Patent number: 11028122
    Abstract: The present invention provides an oligomer which efficiently enables to cause skipping of the 53rd exon in the human dystrophin gene. Also provided is a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency.
    Type: Grant
    Filed: February 12, 2021
    Date of Patent: June 8, 2021
    Assignees: NIPPON SHINYAKU CO., LTD., NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY
    Inventors: Naoki Watanabe, Youhei Satou, Shin'ichi Takeda, Tetsuya Nagata
  • Exonic splicing enhancers and exonic splicing silencers
    Patent number: 11015222
    Abstract: Compounds and methods for regulation of exonic splicing enhancers and exonic splicing silencers. Compounds include polynucleotides targeted to aberrant exonic splicing enhancers and exonic splicing silencers. Compounds and methods for the diagnosis of diseases and conditions associated with aberrant exonic splicing enhancers and exonic splicing silencers. Methods for identifying splicing-sensitive disease mutations, and functional RNA elements as targets for amelioration of aberrant pre-mRNA splicing.
    Type: Grant
    Filed: May 29, 2019
    Date of Patent: May 25, 2021
    Assignee: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
    Inventors: Jeremy Sanford, Timothy Steme-Weiler
  • Methods and compositions for inhibiting detoxification response
    Patent number: 10988765
    Abstract: Disclosed herein are compositions and methods for attenuating detoxification response and related symptoms thereof induced by translation defect. The compositions and methods herein are useful for attenuating detoxification response and/or treat related symptoms thereof in subjects comprising translation defect. The composition and methods herein are also useful for improving pharmacokinetics of a pharmaceutical compound.
    Type: Grant
    Filed: August 29, 2016
    Date of Patent: April 27, 2021
    Assignee: THE GENERAL HOSPITAL CORPORATION
    Inventors: Gary Ruvkun, J. Amaranath Govindan
  • Single-stranded RNA-editing oligonucleotides
    Patent number: 10988763
    Abstract: The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA in a eukaryotic cell, wherein said oligonucleotide does not, in itself, form an intramolecular hairpin or stem-loop structure, and wherein said oligonucleotide comprises a cytidine (a non-complementary nucleotide) or a uridine in position opposite to the target adenosine to be edited in the target RNA region.
    Type: Grant
    Filed: June 22, 2017
    Date of Patent: April 27, 2021
    Assignee: PROQR THERAPEUTICS II B.V.
    Inventors: Janne Juha Turunen, Petra Geziena De Bruijn, Bart Klein, Roxana Simona Redis, Lenka Van Sint Fiet
  • Read through of truncated proteins in premature termination codon diseases by suppressor tRNAs
    Patent number: 10982209
    Abstract: Provided are a method for constructing a suppressor tRNA, and 19 suppressor tRNAs corresponding to three termination codons, a plasmid, a vector or a kit comprising the above-mentioned tRNA. Also provided are use of the above-mentioned tRNA, plasmid, vector or kit in the manufacture of a medicament for treating a hereditary disease or a cancer caused by a nonsense mutation of a gene. Also provided are a method for evaluating the efficiency of a suppressor tRNA for reading through a nonsense mutation, and a method for restoring the expression of a truncated protein of a nonsense mutant of a pathogenic gene in a monogenic hereditary disease and a tumor suppressor gene in a tumor cell.
    Type: Grant
    Filed: March 3, 2017
    Date of Patent: April 20, 2021
    Assignee: PEKING UNIVERSITY
    Inventors: Qing Xia, Tianchang Wang, Qi Yang
  • Micro-RNAS and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone-associated medical conditions
    Patent number: 10975373
    Abstract: A method of treating a bipolar disorder in a subject in need thereof is disclosed. The method comprising administering to the subject sa therapeutically effective amount of a miR-135, a precursor thereof or a nucleic acid molecule encoding said miR-135 or said precursor thereof, thereby treating the bipolar disorder. Methods of diagnosing a mood disorder in a human subject and of monitoring treatment of an anti-depressant drug or a medicament for the treatment of a mood disorder are also disclosed.
    Type: Grant
    Filed: November 5, 2018
    Date of Patent: April 13, 2021
    Assignee: Yeda Research and Development Co. Ltd.
    Inventors: Alon Chen, Orna Issler
  • Combination vectors and methods for treating cancer
    Patent number: 10975374
    Abstract: A composition for treating cancer is disclosed. The composition includes a lentiviral particle and an aminobisphosphonate drug. The lentiviral particle is capable of infecting a target cell, such as a cancer cell, and includes an envelope protein optimized for targeting such target cell and a viral vector. The viral vector includes a small RNA optimized to target an FDPS mRNA sequence. The aminobisphosphonate drug includes zoledronic acid.
    Type: Grant
    Filed: July 30, 2020
    Date of Patent: April 13, 2021
    Assignee: American Gene Technologies International Inc.
    Inventors: Tyler Lahusen, Mei-Ling Liou, Lingzhi Xiao, Haishan Li, Charles David Pauza
  • HER2-regulated RNA as a diagnostic and therapeutic targets in HER2+ breast cancer
    Patent number: 10961589
    Abstract: A method for treating HER2 positive breast cancer in a subject in need thereof includes administering to cancer cells of the subject an agent effective to modulate the level of HER2-associated RNA in the breast cancer cells of the subject.
    Type: Grant
    Filed: March 7, 2016
    Date of Patent: March 30, 2021
    Assignee: CASE WESTERN RESERVE UNIVERSITY
    Inventors: Ahmad Khalil, Lyndsay Harris
  • Methods and pharmaceutical compositions for the treatment of pulmonary bacterial infections
    Patent number: 10954299
    Abstract: The present invention relates to methods and pharmaceutical compositions for the treatment of pulmonary bacterial infections. The increasing burden of antimicrobial resistance coupled with the decreasing number of antibiotics in development has urged for strategies to elaborate new therapies. The inventors showed a therapeutic effect of IL-20 receptor 10 antagonists in pulmonary bacterial infection mouse model. Indeed, they demonstrated that treatment with IL-20 receptor antagonists reduces bacterial burden, cellular infiltration and inflammation in bronchoalveolar lavage fluid (BALF) and lung. In particular, the present invention relates to an antagonist of IL-20 cytokines, or/and an antagonist of IL-20RB receptor for use in a method for the treatment of pulmonary bacterial infections in a subject in 15 need thereof.
    Type: Grant
    Filed: May 24, 2017
    Date of Patent: March 23, 2021
    Assignees: INSERM (Institut National de la Santé et de la Recherche Médicale), Centre National de la Recherche Scientifique (CNRS), Institut Paseur de Lille, Universitéde Lille
    Inventors: Philippe Gosset, Fahima Madouri, Muriel Pichavant
  • Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, HAO1) and methods of use thereof
    Patent number: 10946107
    Abstract: The invention relates to polynucleotide agents targeting an hydroxyacid oxidase (HAO1) gene, and methods of using such polynucleotide agents to inhibit expression of HAO1 and to treat subjects having an HAO1-associated disease, e.g., hyperoxaluria.
    Type: Grant
    Filed: December 14, 2017
    Date of Patent: March 16, 2021
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventor: Gregory Hinkle