Nucleic Acid Expression Inhibitors Patents (Class 536/24.5)
  • Patent number: 11492620
    Abstract: Provided is a modified double-stranded oligonucleotide, in which the sense strand comprises a nucleotide sequence 1, the anti-sense strand comprises a nucleotide sequence 2, the nucleotide sequences 1 and 2 are both 19 nucleotides in length, and in the direction from 5? end to 3? end, nucleotides at positions 7, 8 and 9 of the nucleotide sequence 1 and nucleotides at positions 2, 6, 14 and 16 of the nucleotide sequence 2 are all fluoro-modified nucleotides, and each nucleotide at other positions is independently one of non-fluoro-modified nucleotides. Further provided are a pharmaceutical composition and a conjugate comprising the oligonucleotide, and pharmaceutical use thereof.
    Type: Grant
    Filed: November 29, 2018
    Date of Patent: November 8, 2022
    Assignee: SUZHOU RIBO LIFE SCIENCE CO., LTD.
    Inventors: Hongyan Zhang, Shan Gao, Daiwu Kang
  • Patent number: 11492621
    Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c. 2991+1655 A>G CEP290 mRNA.
    Type: Grant
    Filed: March 3, 2020
    Date of Patent: November 8, 2022
    Assignees: INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS), GENETHON, UNIVERSITE PARIS DESCARTES, ENSCP—CHIMIE PARISTECH—ECOLE NATIONALE SUPERIEURE DE CHIME DE PARIS, ASSISTANCE PUBLIQUE—HOPITAUX DE PARIS
    Inventors: Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman, M. Arnold Munnich
  • Patent number: 11473090
    Abstract: Linear covalently closed vectors, and compositions and methods for making same.
    Type: Grant
    Filed: May 16, 2018
    Date of Patent: October 18, 2022
    Assignee: Helix Nanotechnologies, Inc.
    Inventors: Nikolai Eroshenko, Nikhil Dhar
  • Patent number: 11446380
    Abstract: The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the HAO1 gene, and methods of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.
    Type: Grant
    Filed: November 4, 2019
    Date of Patent: September 20, 2022
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: William Querbes, Kevin Fitzgerald, Brian Bettencourt, Abigail Liebow, David V. Erbe
  • Patent number: 11447736
    Abstract: The present disclosure provides a cell separation apparatus for a bioreactor. The cell separation apparatus may be disposed outside the bioreactor and in fluid connection with the bioreactor, the cell separation apparatus may be in a shape of a box body, the cell separation apparatus may include a liquid buffer device including a first liquid cavity disposed in the box body; a filter device including a filter channel and a filter membrane disposed in the box body, the filter membrane may be disposed above the filter channel; and a first liquid channel may be configured in the box body to facilitate a fluid communication between the first liquid cavity and the filter channel. A power system for filtering and microfluidic channels are integrated in the cell separation apparatus that is of a box shape, thereby reducing the volume and production cost thereof.
    Type: Grant
    Filed: September 18, 2021
    Date of Patent: September 20, 2022
    Assignee: ALIT BIOTECH (SHANGHAI) CO., LTD.
    Inventors: Yu Liu, Rui Chen
  • Patent number: 11447773
    Abstract: The invention relates to saRNA targeting an HNF4a transcript and therapeutic compositions comprising said saRNA. Methods of using the therapeutic compositions are also provided.
    Type: Grant
    Filed: September 7, 2018
    Date of Patent: September 20, 2022
    Assignee: MINA THERAPEUTICS LIMITED
    Inventors: Hans E. Huber, David Blakey, Jon Voutila, Vikash Reebye
  • Patent number: 11434486
    Abstract: The disclosure provides polynucleotides encoding a polypeptide including a morpholino linker. In some embodiments, the polynucleotides of the invention have increased stability compared to wild-type polynucleotides.
    Type: Grant
    Filed: September 19, 2016
    Date of Patent: September 6, 2022
    Assignee: ModernaTX, Inc.
    Inventors: Gabor Butora, Andrew W. Fraley, Edward John Miracco, Jennifer Nelson, Amy Rhoden Smith, Matthew Stanton
  • Patent number: 11419942
    Abstract: The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the angiotensinogen (AGT) gene, and methods of using such RNAi agents to inhibit expression of AGT and methods of treating subjects having an AGT-associated disorder, e.g., hypertension.
    Type: Grant
    Filed: September 15, 2020
    Date of Patent: August 23, 2022
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Donald Foster, Brian Bettencourt, Klaus Charisse, Gregory Hinkle, Satyanarayana Kuchimanchi, Martin A. Maier, Stuart Milstein
  • Patent number: 11377658
    Abstract: Provided are compositions comprising an oligonucleotide that targets Angiopoietin-like 4 (ANGPTL4). The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating a metabolic or cardiovascular disorder by providing an oligonucleotide that targets ANGPTL4 to a subject in need thereof.
    Type: Grant
    Filed: September 15, 2021
    Date of Patent: July 5, 2022
    Assignee: EMPIRICO INC.
    Inventors: Omri Gottesman, Shannon Bruse, Brian Cajes, David Lewis, David Rozema
  • Patent number: 11359200
    Abstract: Compositions and methods for treating cancer in a subject in need thereof are described that includes administering a therapeutically effective amount of an oligonucleotide that specifically hybridizes to MALAT1.
    Type: Grant
    Filed: April 9, 2018
    Date of Patent: June 14, 2022
    Assignee: THE CLEVELAND CLINIC FOUNDATION
    Inventors: JianJun Zhao, Yi Hu, Jing Fang
  • Patent number: 11312963
    Abstract: Disclosed herein are compounds, compositions, and methods for decreasing TIGIT mRNA and protein expression. Such methods are useful to treat, prevent, or ameliorate TIGIT associated diseases, disorders, and conditions.
    Type: Grant
    Filed: October 17, 2019
    Date of Patent: April 26, 2022
    Assignee: SynerK Inc.
    Inventors: Weiwen Jiang, Jimmy X. Tang, Daqing Wang, Dong Yu
  • Patent number: 11312957
    Abstract: The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a lipophilic moiety. e.g., cholesterol, is is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents.
    Type: Grant
    Filed: July 26, 2019
    Date of Patent: April 26, 2022
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Muthiah Manoharan, Venkitasamy Kesavan, Kallanthottathil Rajeev
  • Patent number: 11242543
    Abstract: The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.
    Type: Grant
    Filed: October 23, 2020
    Date of Patent: February 8, 2022
    Assignees: The Regents of the University of California, University of Vienna
    Inventors: Jennifer A. Doudna, Martin Jinek, Krzysztof Chylinski, Emmanuelle Charpentier
  • Patent number: 11186849
    Abstract: The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.
    Type: Grant
    Filed: October 29, 2020
    Date of Patent: November 30, 2021
    Assignees: The Regents of the University of California, University of Vienna, Emmanuelle Charpentier
    Inventors: Jennifer A. Doudna, Martin Jinek, Krzysztof Chylinski, Emmanuelle Charpentier
  • Patent number: 11186538
    Abstract: A compound of general formula (I) or (II), wherein Rn and Rm independently represent a C3-C27 aliphatic group, R1 to R4, which may be the same or different at each occurrence, represent hydrogen or a C1-C8 alkyl group, X1 and X2, which may be the same or different at each occurrence, represent a linear or branched divalent hydrocarbon radical with 1 to 24 carbon atoms which can be optionally substituted and/or interrupted by one or more heteroatoms or heteroatom containing groups, and R5 and R6, which may be the same or different at each occurrence, represent a group selected from —O?, -Alk-CH(OH)—CH2—SO3— and -Alk-CO2— wherein Alk represents an alkylene group.
    Type: Grant
    Filed: November 8, 2017
    Date of Patent: November 30, 2021
    Assignee: RHODIA OPERATIONS
    Inventors: Olivier Back, Roberto Company, Hong Liu, Rémy Leroy, Philippe Marion
  • Patent number: 11180524
    Abstract: The present disclosure relates to compounds and compositions containing 5?-phosphoramidite nucleoside monomers of formulae (I) and (II), and methods of making and use, wherein the substituents are as defined in the appended claims.
    Type: Grant
    Filed: September 14, 2018
    Date of Patent: November 23, 2021
    Assignee: Janssen BioPharma, Inc.
    Inventors: Sergei Gryaznov, Jin Hong, Vivek Kumar Rajwanshi, Leonid Beigelman
  • Patent number: 11174483
    Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the TMPRSS6 gene expression or inhibits its expression and therapeutic uses such as for the treatment of hemochromatosis, porphyria and blood disorders such as ?-thalassemias, sickle cell disease and transfusional iron overload or myelodysplastic syndrome.
    Type: Grant
    Filed: April 5, 2018
    Date of Patent: November 16, 2021
    Assignee: Silence Therapeutics GmbH
    Inventors: Sibylle Dames, Ute Schaeper, Judith Hauptmann, Christian Frauendorf, Lucas Bethge, Adrien Weingärtner
  • Patent number: 11162099
    Abstract: The invention relates to saRNA targeting an HNF4a transcript and therapeutic compositions comprising said saRNA. Methods of using the therapeutic compositions are also provided.
    Type: Grant
    Filed: September 7, 2018
    Date of Patent: November 2, 2021
    Assignee: MINA THERAPEUTICS LIMITED
    Inventors: Hans E. Huber, David Blakey, Jon Voutila, Monika Krampert, Markus Hossbach
  • Patent number: 11160877
    Abstract: The present invention relates to a targeting-type capsule for drug delivery systems. The present invention addresses the problem of providing a capsule for drug delivery systems by utilizing the reactivity of a thiol with an alkyl halide, wherein the capsule comprises a silole-containing carbosilane dendrimer and a labeling protein containing a target recognition site (e.g., green fluorescent protein), can include a biological polymer or another molecule therein, and can deliver the biological polymer or the like selectively into a target cell.
    Type: Grant
    Filed: November 27, 2018
    Date of Patent: November 2, 2021
    Assignees: Saitama University, QUARRYMEN & Co. Inc.
    Inventors: Miho Suzuki, Ken Hatano, Shojiro Yoshida, Yasuhiro Yamashita
  • 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: 11155819
    Abstract: The present invention relates to the field of biomedicine, particularly to double-stranded RNA molecules targeting CKIP-1 and uses thereof, particularly to use of the double-stranded RNA molecules for the treatment of inflammatory diseases such as arthritis, particularly rheumatoid arthritis.
    Type: Grant
    Filed: September 7, 2018
    Date of Patent: October 26, 2021
    Assignee: BEIJING TIDE PHARMACEUTICAL CO., LTD.
    Inventors: Yanping Zhao, Hongjun Wang, Yuanyuan Jiang, Weiting Zhong, Jianmei Pang, Gong Li, Xiang Li, Yixin He, Liying Zhou, Yanan Liu
  • Patent number: 11149264
    Abstract: The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.
    Type: Grant
    Filed: February 10, 2021
    Date of Patent: October 19, 2021
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Punit P. Seth, Michael Oestergaard, Michael T. Migawa, Xue-hai Liang, Wen Shen, Stanley T. Crooke, Eric E. Swayze
  • Patent number: 11142764
    Abstract: Provided are antisense oligomers targeted against bacterial mRNAs and other macromolecules associated with a biochemical pathway and/or cellular process, and related compositions and methods of using the oligomers and compositions to treat an infected mammalian subject, for example, as primary antimicrobials or as adjunctive therapies with classic antimicrobials.
    Type: Grant
    Filed: December 22, 2016
    Date of Patent: October 12, 2021
    Assignees: Board of Regents, The University of Texas System, Oregon State University
    Inventors: David Greenberg, Bruce L. Geller
  • Patent number: 11130951
    Abstract: Methods of using B2 or Alu nucleic acids, or antisense oligonucleotides that modulate the EZH2/B2 or EZH2/Alu interaction and have the capacity to alter cleavage of B2 and Alu RNA, for increasing or decreasing cell and organismal viability.
    Type: Grant
    Filed: June 9, 2017
    Date of Patent: September 28, 2021
    Assignee: The General Hospital Corporation
    Inventors: Jeannie T. Lee, Athanasios Zovoilis
  • Patent number: 11110174
    Abstract: The present invention provides a phosphorothioate-modified oligonucleotide comprising a structure shown below: The present invention also provides a phosphorothioate-modified oligonucleotide comprising a structure having formula (CIII):
    Type: Grant
    Filed: April 29, 2020
    Date of Patent: September 7, 2021
    Assignee: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Muthiah Manoharan, Kallanthottathil G. Rajeev, Jayaprakash Nair, Martin Maier
  • Patent number: 11111493
    Abstract: The present disclosure provides methods and compositions related to the modification of immune effector cells to increase therapeutic efficacy. In some embodiments, immune effector cells modified to reduce expression of one or more endogenous target genes, or to reduce one or more functions of an endogenous protein to enhance effector functions of the immune cells are provided. In some embodiments, immune effector cells further modified by introduction of transgenes conferring antigen specificity, such as exogenous T cell receptors (TCRs) or chimeric antigen receptors (CARs) are provided. Methods of treating a cell proliferative disorder, such as a cancer, using the modified immune effector cells described herein are also provided.
    Type: Grant
    Filed: February 14, 2020
    Date of Patent: September 7, 2021
    Assignee: KSQ Therapeutics, Inc.
    Inventors: Micah Benson, Jason Merkin, Gregory V. Kryukov, Solomon Martin Shenker, Michael Schlabach, Noah Tubo
  • Patent number: 11104887
    Abstract: In certain aspects, the disclosure relates to compositions comprising modified Ornithine transcarbamylase (OTC) polyribonucleotides and methods of use.
    Type: Grant
    Filed: December 15, 2017
    Date of Patent: August 31, 2021
    Assignee: ethris GmbH
    Inventors: Carsten Rudolph, Rebekka Kubisch-Dohmen, Manish Kumar Aneja, Johannes Geiger, Marino Schuhmacher
  • Patent number: 11083745
    Abstract: Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
    Type: Grant
    Filed: December 14, 2016
    Date of Patent: August 10, 2021
    Assignees: COLD SPRING HARBOR LABORATORY, STOKE THERAPEUTICS, INC.
    Inventors: Isabel Aznarez, Huw M. Nash, Adrian Krainer
  • Patent number: 11078483
    Abstract: The invention describes a novel system for identifying optimized gRNAs for use in CRISPR/Cas9 genome editing platforms. The invention allows for the determination of specific gene alterations rendered by a particular gRNA, thereby permitting the generation of optimized gRNA libraries.
    Type: Grant
    Filed: September 1, 2017
    Date of Patent: August 3, 2021
    Assignee: KSQ Therapeutics, Inc.
    Inventors: Gregory V. Kryukov, Michael R. Schlabach, Jason J. Merkin
  • 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
  • Patent number: 11033571
    Abstract: The present invention relates to a pharmaceutical composition for treating or preventing hepatitis B, comprising: at least one oligonucleotide selected from the group consisting an oligonucleotide represented by a nucleic acid sequence of SEQ ID NO: 1, 2, or 6 or a nucleic acid sequence complementary thereto; and an oligonucleotide having at least one chemical modification on the oligonucleotide, as an active ingredient, and a method for screening a therapeutic agent for hepatitis B according to the formation of a G-quadruplex by HBV and a candidate material. The pharmaceutical composition forms a G-quadruplex with HBV and reduces cccDNA (covalently closed circular DNA) and thus can be used in the treatment or prevention of hepatitis B.
    Type: Grant
    Filed: December 13, 2017
    Date of Patent: June 15, 2021
    Assignee: AM SCIENCES INC
    Inventors: Kyun Hwan Kim, Doo Hyun Kim, Yeong Min Park
  • Patent number: 11015194
    Abstract: The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.
    Type: Grant
    Filed: July 23, 2018
    Date of Patent: May 25, 2021
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Muthiah Manoharan, Kallanthottathil G. Rajeev
  • Patent number: 11015197
    Abstract: The present invention relates to a novel anti-miRNA single-stranded nucleic acid maleimide derivative, which comprises an anti-miRNA single-stranded nucleic acid having a nucleic acid sequence complementary to a nucleic acid sequence of an miRNA. Further, the present invention provides an anti-miRNA single-stranded nucleic acid-serum albumin conjugate in which serum albumin is covalently bonded to the anti-miRNA single-stranded nucleic acid maleimide derivative via the maleimide group.
    Type: Grant
    Filed: May 29, 2019
    Date of Patent: May 25, 2021
    Assignee: KOREA INSTITUTE OF SCIENCE AND TECHNOLOGY
    Inventors: Sun Hwa Kim, Ick Chan Kwon, Kwangmeyung Kim, Hong Yeol Yoon, Gi-Jung Kwak, Juho Park
  • Patent number: 10995337
    Abstract: An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 214.
    Type: Grant
    Filed: May 22, 2020
    Date of Patent: May 4, 2021
    Assignee: The University of Western Australia
    Inventors: Stephen Donald Wilton, Sue Fletcher, Graham McClorey
  • Patent number: 10968450
    Abstract: An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 214.
    Type: Grant
    Filed: July 31, 2019
    Date of Patent: April 6, 2021
    Assignee: The University of Western Australia
    Inventors: Stephen Donald Wilton, Sue Fletcher, Graham McClorey
  • Patent number: 10947541
    Abstract: In certain aspects, provided herein are RNA complexes (e.g., asymmetric RNA complexes, such as asiRNAs or cell penetrating asiRNAs) that inhibit IL4R?, TRPA1, and/or F2RL1 expression and are therefore useful for treating atopic dermatitis or asthma.
    Type: Grant
    Filed: July 17, 2019
    Date of Patent: March 16, 2021
    Assignee: OliX Pharmaceuticals, Inc.
    Inventors: Dong-Ki Lee, Sun Woo Hong, Hanna Lee, Dayeon Yu, Ji Eom
  • Patent number: 10947544
    Abstract: The invention provides nucleic acid therapeutics and methods for using these nucleic acid therapeutics in the treatment of complement-related disorders.
    Type: Grant
    Filed: February 28, 2019
    Date of Patent: March 16, 2021
    Assignee: Archemix LLC
    Inventors: Claude Benedict, David Epstein, Charles Wilson, Dilara McCauley, Jeffrey Kurz, Markus Kurz, Thomas Greene McCauley, James Rottman
  • Patent number: 10941402
    Abstract: The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA sequence 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 non-complementary nucleotide in a position opposite to the nucleotide to be edited in the target RNA sequence.
    Type: Grant
    Filed: August 31, 2017
    Date of Patent: March 9, 2021
    Assignee: PROQR THERAPEUTICS II B.V.
    Inventors: Janne Juha Turunen, Antti Aalto, Bart Klein, Lenka Van Sint Fiet, Julien Auguste Germain Boudet
  • Patent number: 10941404
    Abstract: Provided herein are oligonucleotide compositions that inhibit ANGPTL7 and reduce intraocular pressure when administered to an eye. The oligonucleotide compositions contain nucleoside modifications.
    Type: Grant
    Filed: September 4, 2020
    Date of Patent: March 9, 2021
    Assignee: EMPIRICO INC.
    Inventors: Omri Gottesman, Shannon Bruse, Paul Buske, Brian Cajes, David Lewis, David Rozema
  • Patent number: 10934550
    Abstract: Immunogenic modulators and compositions comprising oligonucleotide agents capable of inhibiting suppression of immune response by reducing expression of one or more gene involved with an immune suppression mechanism.
    Type: Grant
    Filed: December 2, 2014
    Date of Patent: March 2, 2021
    Assignee: Phio Pharmaceuticals Corp.
    Inventors: Alexey Wolfson, Alexey Eliseev, Taisia Shmushkovich
  • Patent number: 10927372
    Abstract: Provided herein are oligomeric compounds with conjugate groups targeting apolipoprotein C-III (ApoCIII). In certain embodiments, the ApoCIII targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting ApoCIII for use in decreasing ApoCIII to treat, prevent, or ameliorate diseases, disorders or conditions related to ApoCIII. Certain diseases, disorders or conditions related to ApoCIII include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.
    Type: Grant
    Filed: February 7, 2018
    Date of Patent: February 23, 2021
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Mark J. Graham
  • Patent number: 10900039
    Abstract: The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal applications.
    Type: Grant
    Filed: September 4, 2015
    Date of Patent: January 26, 2021
    Assignee: Phio Pharmaceuticals Corp.
    Inventor: Gerard Cauwenbergh
  • Patent number: 10889813
    Abstract: The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the programmed cell death 1 ligand 1 (PD-L1) gene, and methods of using such RNAi agents to inhibit expression of a PD-L1 gene and methods of treating subjects having a PD-L1-associated disorder.
    Type: Grant
    Filed: August 22, 2016
    Date of Patent: January 12, 2021
    Assignee: ALNYLAM PHARMACEUTICALS, INC.
    Inventor: Gregory Hinkle
  • Patent number: 10844384
    Abstract: The invention relates to double stranded ribonucleic acid (dsRNA) compositions targeting a glucokinase (GCK) gene, as well as methods of inhibiting expression of a glucokinase (GCK) gene, and methods of treating subjects having a glycogen storage disease (GSD), e.g., type Ia GSD.
    Type: Grant
    Filed: December 21, 2017
    Date of Patent: November 24, 2020
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Kevin Fitzgerald, David Erbe, Gregory Hinkle
  • Patent number: 10799591
    Abstract: Provided herein are branched oligonucleotides exhibiting efficient and specific tissue distribution, cellular uptake, minimum immune response and off-target effects, without formulation.
    Type: Grant
    Filed: April 22, 2019
    Date of Patent: October 13, 2020
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Anastasia Khvorova, Matthew Hassler, Julia Alterman, Bruno Miguel da Cruz Godinho
  • Patent number: 10786572
    Abstract: The present invention relates to an apparatus for inhibiting oncogene-induced malignant carcinogenesis, including an irradiation unit for applying low-dose radiation to a subject, and to a method of inhibiting oncogene-induced malignant carcinogenesis, including applying low-dose radiation to a subject, whereby oncogene-induced malignant carcinogenesis can be inhibited by means of the apparatus and method of the present invention.
    Type: Grant
    Filed: August 23, 2016
    Date of Patent: September 29, 2020
    Assignee: KOREA HYDRO & NUCLEAR POWER CO., LTD.
    Inventors: Seonyoung Nam, Kwang Hee Yang, Su-Jae Lee, Rae-Kwon Kim
  • Patent number: 10774327
    Abstract: This disclosure relates to novel huntingtin targets. Novel oligonucleotides for the treatment of Huntington's disease are also provided.
    Type: Grant
    Filed: January 31, 2019
    Date of Patent: September 15, 2020
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Anastasia Khvorova, Neil Aronin, Julia Alterman
  • Patent number: 10772907
    Abstract: The present invention relates to methods for treating a tumor, including a metastatic tumor, with TLR9 agonist in combination with an immune checkpoint inhibitor therapy.
    Type: Grant
    Filed: August 30, 2019
    Date of Patent: September 15, 2020
    Assignee: IDERA PHARMACEUTICALS, INC.
    Inventors: Sudhir Agrawal, Daqing Wang, Wayne Jiang
  • Patent number: 10760098
    Abstract: The present invention is directed to non-naturally occurring peptides containing a membrane-penetrating amino acid sequence and further at least one polycationic moiety or peptide sequence. The peptides are suitable for use in delivery a cargo to the interior of a cell. Suitable cargo includes nucleic acid molecules (including DNA, RNA or PNA), polypeptides, or other biologically active molecules. The present invention is further directed to transfection complexes containing the non-naturally occurring peptides of the present invention in non-covalent association with at least one cationic lipid and a cargo to be delivered to the interior of a cell. The invention further relates to methods for the preparation and use of the non-naturally occurring peptides for the formation of transfection complexes and the delivery of a cargo to the interior of a cell in culture, an animal or a human. The invention also relates to compositions and kits useful for transfecting cells.
    Type: Grant
    Filed: November 13, 2017
    Date of Patent: September 1, 2020
    Assignee: LIFE TECHNOLOGIES CORPORATION
    Inventor: Xavier de Mollerat du Jeu
  • Patent number: 10758558
    Abstract: Provided herein are hybrid oligonucleotides comprising a region that promotes cleavage of a nucleic acid and a region that protects a nucleic acid from exonuclease activity. Such hybrid oligonucleotides are useful for modulating the expression of genes. Related compositions and methods are also provided. In some embodiments, methods are provided for treating a disease, such as by administering a hybrid oligonucleotide.
    Type: Grant
    Filed: February 12, 2016
    Date of Patent: September 1, 2020
    Assignee: Translate Bio MA, Inc.
    Inventor: Fatih Ozsolak