Patents Examined by Brian Whiteman
  • Patent number: 11230709
    Abstract: The present invention aims at establishing a molecular therapy for Alport syndrome. The present invention provides an oligonucleotide of 15-30 bp comprising a nucleotide sequence complementary to the cDNA of COL4A5 gene, wherein the oligonucleotide is capable of inducing skipping of an exon which has a truncating mutation found in COL4A5 gene in Alport syndrome patients and whose nucleotide number is a multiple of 3, a pharmaceutically acceptable salt thereof, or a solvate thereof. Also provided is a pharmaceutical drug comprising the above oligonucleotide, a pharmaceutically acceptable salt thereof, or a solvate thereof (therapeutic drug for Alport syndrome).
    Type: Grant
    Filed: December 25, 2017
    Date of Patent: January 25, 2022
    Assignees: NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY, DAIICHI SANKYO COMPANY LIMITED
    Inventors: Kazumoto Iijima, Kandai Nozu, Akemi Shono, Makoto Koizumi, Yoshiyuki Onishi, Kiyosumi Takaishi, Tomomi Adachi
  • Patent number: 11214802
    Abstract: Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an alpha-ENaC (SCNN1A) gene. The alpha-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an alpha-ENaC gene. Pharmaceutical compositions that include one or more alpha-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described alpha-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of alpha-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects.
    Type: Grant
    Filed: January 31, 2020
    Date of Patent: January 4, 2022
    Assignee: Arrowhead Pharmaceuticals, Inc.
    Inventors: Zhen Li, Rui Zhu, Tao Pei, Anthony Nicholas, Erik W Bush
  • Patent number: 11214801
    Abstract: The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, capable of inhibiting Apolipoprotein C-III (also called APOC3, apoC-III, APOC-III, and APO C-III) gene expression, and compositions that include APOC3 RNAi agents. The APOC3 RNAi agents disclosed herein may be conjugated to targeting ligands, including ligands that include N-acetyl-galactosamine, to facilitate the delivery to cells, including to hepatocytes. Pharmaceutical compositions that include one or more APOC3 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the APOC3 RNAi agents in vivo provides for inhibition of APOC3 gene expression, and can result in lower triglycerides and/or cholesterol levels in the subject. The APOC3 RNAi agents can be used in methods of treatment of APOC3-related diseases and disorders, including hypertriglyceridemia, cardiovascular disease, and other metabolic-related disorders and diseases.
    Type: Grant
    Filed: January 31, 2020
    Date of Patent: January 4, 2022
    Assignee: Arrowhead Pharmaceuticals, Inc.
    Inventors: Zhen Li, Rui Zhu, Tao Pei, Steven Kanner, So Wong
  • Patent number: 11198873
    Abstract: The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of modulatory polynucleotides.
    Type: Grant
    Filed: January 9, 2020
    Date of Patent: December 14, 2021
    Assignee: VOYAGER THERAPEUTICS, INC.
    Inventors: Jinzhao Hou, Mathieu E. Nonnenmacher, Pengcheng Zhou
  • Patent number: 11198868
    Abstract: Disclosed are double stranded RNA (dsRNA) molecules that are toxic to insect pests. In particular, interfering RNA molecules capable of modulating expression of a pest insect target gene and that are toxic to the insect pest are provided. Further, methods of making and using the interfering RNA, for example as the active ingredient in an insecticidal composition or in a transgenic plant, to confer protection from insect damage are disclosed.
    Type: Grant
    Filed: November 6, 2018
    Date of Patent: December 14, 2021
    Assignee: DEVGEN NV
    Inventors: Yann Naudet, Myriam Beghyn, Lien De Schrijver, Isabelle Maillet, Annelies Philips
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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