Patents Examined by Brian Whiteman
  • Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
    Patent number: 11198872
    Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
    Type: Grant
    Filed: December 12, 2019
    Date of Patent: December 14, 2021
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: William Querbes, Kevin Fitzgerald, James Butler, Stephanie Williams, Gregory Hinkle, Martin A. Maier
  • Modulatory polynucleotides
    Patent number: 11193129
    Abstract: The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of modulatory polynucleotides.
    Type: Grant
    Filed: January 22, 2020
    Date of Patent: December 7, 2021
    Assignee: VOYAGER THERAPEUTICS, INC.
    Inventors: Dinah Wen-Yee Sah, Jinzhao Hou, Mathieu E. Nonnenmacher
  • Compositions and methods for inhibition of the lncRNA SAF to drive apoptotic cell death in human immunodeficiency virus (HIV) infected human macrophages
    Patent number: 11186841
    Abstract: Provided are compositions and methods for prophylaxis or therapy for human immunodeficiency virus (HIV) infection. The compositions and methods involve use of RNAi agents targeted to an anti-apoptotic long non-coding RNA (lncRNA) that is IncRNA SAF (FAS-AS1) or HOXA-AS2. The RNAi agents preferentially induce apoptosis of HIV infected macrophages. RNAi agents, and macrophages containing the RNAi agents, are also provided.
    Type: Grant
    Filed: December 9, 2019
    Date of Patent: November 30, 2021
    Assignee: Cornell University
    Inventor: David G. Russell
  • Methods of treating intestinal fibrosis using SMAD7 inhibition
    Patent number: 11162097
    Abstract: The present invention relates to methods of treating, preventing or managing intestinal fibrosis by inhibiting SMAD7. The invention is also directed to methods of monitoring effectiveness of treatment or management of intestinal fibrosis using a SMAD7 antisense oligonucleotide, as well as methods of regulating SMAD7 antisense oligonucleotide treatment, based on analysis of Transforming Growth Factor-? (TGF-?) levels, ?-Smooth Muscle Actin (a-SMA) levels, and/or phosphorylated Mothers Against Decapentaplegic Homolog 3 (p-SMAD3) levels.
    Type: Grant
    Filed: February 23, 2017
    Date of Patent: November 2, 2021
    Assignee: Nogra Pharma Limited
    Inventor: Giovanni Monteleone
  • Therapeutic targets for facioscapulohumeral muscular dystrophy
    Patent number: 11160823
    Abstract: The disclosure relates to methods and compositions for regulating expression of DUX4. In some aspects, methods described by the disclosure are useful for treating a disease associated with aberrant DUX4 expression (e.g., facioscapulohumeral muscular dystrophy).
    Type: Grant
    Filed: November 7, 2017
    Date of Patent: November 2, 2021
    Assignee: University of Massachusetts
    Inventors: Peter L. Jones, Michael R. Green
  • Method and pharmaceutical composition for treating cancer
    Patent number: 11149271
    Abstract: A method of treating a subject suffering from cancer includes administering an effective amount of a RNA molecule to the subject, wherein the RNA molecule is isolated or derived from a plant of the genus Taxus. A method of inhibiting growth or proliferation of cancer cells includes contacting cancer cells with the RNA molecule; and a pharmaceutical composition for treating cancer includes the RNA molecule and a pharmaceutically tolerable excipient. Also a double-stranded RNA molecule and a recombinant vector include the double-stranded RNA molecule.
    Type: Grant
    Filed: September 4, 2018
    Date of Patent: October 19, 2021
    Inventors: Zhi-Hong Jiang, Tong-Meng Yan, Kai-Yue Cao
  • Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
    Patent number: 11142766
    Abstract: The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.
    Type: Grant
    Filed: August 7, 2019
    Date of Patent: October 12, 2021
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Kevin Fitzgerald, William Querbes, James Butler, Stephanie Williams, Abigail Liebow, Gregory Hinkle, Martin A. Maier, Stuart Milstein, Satyanarayana Kuchimanchi, Muthiah Manoharan
  • Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof
    Patent number: 11142765
    Abstract: The present disclosure relates to RNA interference (RNAi) reagents, such as short hairpin microRNA (shmiR) and short hairpin RNA (shRNA), for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto. The present disclosure also relates to the use of the RNAi reagents in combination with PABPN1 replacement reagents, such as constructs which encode functional PABPN1 protein, for treatment of OPMD, compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.
    Type: Grant
    Filed: December 14, 2017
    Date of Patent: October 12, 2021
    Assignee: Benitec IP Holdings, Inc.
    Inventors: Vanessa Strings-Ufombah, David Suhy
  • SMN2 element 1 antisense compositions and methods and uses thereof
    Patent number: 11136580
    Abstract: The invention provides methods and compositions for treatment of spinal muscular atrophy (SMA). In one aspect of the invention, a series of compositions comprising an antisense oligonucleotide targeting the Element 1 site on the SMN2 pre-mRNA and a Morpholino backbone is disclosed. In another aspect of the invention, a method of treating SMA patients by modulating the splicing of SMN2 pre-mRNA to increase the amount of full-length SMN is disclosed. Certain embodiments of the inventive method comprise administering an E1-targeting antisense oligonucleotide, such as Morpholino based antisense oligonucleotide, to a SMA subject.
    Type: Grant
    Filed: November 11, 2019
    Date of Patent: October 5, 2021
    Assignee: The Curators of the University of Missouri
    Inventors: Christian Lorson, Erkan Osman
  • Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
    Patent number: 11123294
    Abstract: Liposomal spherical nucleic acids that function as multivalent immune modulators are provided according to the invention. The liposomal spherical nucleic acids of the invention are useful prophylactic and therapeutic applications as well as research and diagnostic indications.
    Type: Grant
    Filed: September 12, 2019
    Date of Patent: September 21, 2021
    Assignee: Exicure Operating Company
    Inventors: Aleksandar Filip Radovic-Moreno, Richard Kang, Subbarao Nallagatla, Christopher C. Mader, Sergei Gryaznov
  • Niacinamide (NAM) in ischemic tissue injury
    Patent number: 11116784
    Abstract: Methods of treating ischemic tissue injury or kidney disease, e.g., delayed graft function, that include administering a Nicotinamide adenine dinucleotide (NAD)/niacinamide (NAM) pathway agonist.
    Type: Grant
    Filed: February 21, 2017
    Date of Patent: September 14, 2021
    Assignee: Beth Israel Deaconess Medical Center, Inc.
    Inventor: Samir M. Parikh
  • Antisense compounds targeting genes associated with cystic fibrosis
    Patent number: 11116785
    Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.
    Type: Grant
    Filed: December 30, 2019
    Date of Patent: September 14, 2021
    Assignee: Rosalind Franklin University of Medicine and Science
    Inventor: Michelle L. Hastings
  • Reduced size self-delivering RNAI compounds
    Patent number: 11118178
    Abstract: The present invention relates to methods for in vivo administration of sd-rxRNA molecules.
    Type: Grant
    Filed: February 7, 2019
    Date of Patent: September 14, 2021
    Assignee: Phio Pharmaceuticals Corp.
    Inventors: Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia
  • Multifunctional RNA nanoparticles and methods for treating cancer and therapeutic resistant cancer
    Patent number: 11110182
    Abstract: RNA nanoparticles functionalized with a HER2-targeting RNA aptamer and at least one MED1 siRNA for targeted delivery of MED1 siRNA to human cancer cells via human epidermal growth factor receptor 2 (HER2) receptors, pharmaceutical compositions of the inventive RNA nanoparticles, and methods for treating breast, HER2-implicated cancers, and in particular therapeutic-resistant cancer such as tamoxifen-resistant breast cancer, by administering pharmaceutical compositions of the inventive RNA nanoparticles.
    Type: Grant
    Filed: December 8, 2017
    Date of Patent: September 7, 2021
    Assignees: University of Cincinnati, Ohio State Innovation Foundation
    Inventors: Xiaoting Zhang, Peixuan Guo
  • Splice inhibiting oligonucleotides
    Patent number: 11104902
    Abstract: MicroRNAs embedded within an intron, which are called ‘mirtrons,’ can be used as a platform for expressing one or more shRNA or miRNA mimics in a lentiviral vector. The inventors developed a strategy to improve lentiviral titering by reducing the production of shRNA/miRNA from the vector during packaging through the introduction of splice-inhibiting antisense oligonucleotides during vector packaging, which inhibit the splicing of the mirtron and subsequent processing of the shRNAs/miRNAs. In an aspect is provided a kit comprising an oligonucleotide comprising a mirtron splice site binding sequence and a lentiviral packaging system. In an aspect is provided a method for producing a lentivirus. The method comprises the step of transfecting a cell with an oligonucleotide comprising a mirtron splice site binding sequence and a lentiviral packaging system; thereby producing the lentivirus.
    Type: Grant
    Filed: September 21, 2018
    Date of Patent: August 31, 2021
    Assignee: City of Hope
    Inventors: John C. Burnett, Elizabeth Epps, John J. Rossi
  • Antisense oligomers and uses thereof
    Patent number: 11096956
    Abstract: Provided herein are methods and compositions for increasing the expression of a protein, and for treating a subject in need thereof, e.g., a subject with deficient protein expression or a subject having a disease described herein.
    Type: Grant
    Filed: June 13, 2018
    Date of Patent: August 24, 2021
    Assignees: STOKE THERAPEUTICS, INC., COLD SPRING HARBOR LABORATORY
    Inventors: Isabel Aznarez, Huw M. Nash, Adrian Krainer
  • Methods and compositions relating to proteasome inhibitor resistance
    Patent number: 11092602
    Abstract: In some aspects, the disclosure provides methods of modulating the level of proteasome inhibitor resistance of a cell, the methods comprising manipulating the level of expression or activity of a subunit of the 19S proteasome in the cell. In some aspects, cells in which the level of a 19S subunit is modulated, e.g., reduced, are provided. In some aspects, methods of identifying agents that reduce proteasome inhibitor resistance are provided. In some aspects, methods of classifying cancers according to predicted proteasome inhibitor resistance are provided. In some aspects, methods of killing or inhibiting proliferation of cancer cells, e.g., proteasome inhibitor resistant cancer cells, are provided. In some aspects, methods of treating cancer, e.g., proteasome inhibitor resistant cancer, are provided.
    Type: Grant
    Filed: July 6, 2016
    Date of Patent: August 17, 2021
    Assignees: Whitehead Institute for Biomedical Research, The Brigham and Women's Hospital, Inc.
    Inventors: Peter Tsvetkov, Sandro Santagata, Susan Lindquist
  • Organic compositions to treat HSF1-related diseases
    Patent number: 11091761
    Abstract: The present disclosure relates to methods of treating heat shock factor 1 (HSF1)-related diseases such as cancer and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.
    Type: Grant
    Filed: May 14, 2019
    Date of Patent: August 17, 2021
    Assignee: Arrowhead Pharmaceuticals, Inc.
    Inventors: Gregory Hinkle, Satyanarayana Kuchimanchi, Stuart Milstein, Markus Warmuth, Wenlai Zhou, Ping Zhu, Tracy S. Zimmermann
  • Presymptomatic micro RNA targets for treatment of neurodegeneration pathology
    Patent number: 11078535
    Abstract: Compositions and methods are provided for the early detection of neuroinflammation in a patient in a presymptomatic stage of a neurodegenerative disease. The method comprises identifying patients with elevated expression levels of a micro RNA selected from the group consisting of miR142-3p, miR142-5p, miR181a, miR181b, miR219-3p and miR219-5p. The identified patients can then be treated with a therapeutic pharmaceutical composition comprising antimiR oligonucleotides.
    Type: Grant
    Filed: July 7, 2018
    Date of Patent: August 3, 2021
    Assignee: The Trustees of Indiana University
    Inventors: Salil Sharma, Hui-Chen Lu
  • Compounds and methods for reducing ATXN2 expression
    Patent number: 11078486
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.
    Type: Grant
    Filed: July 25, 2019
    Date of Patent: August 3, 2021
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Paymaan Jafar-nejad