Nucleic Acid Expression Inhibitors Patents (Class 536/24.5)
  • 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: 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
  • 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: 10738307
    Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.
    Type: Grant
    Filed: October 5, 2017
    Date of Patent: August 11, 2020
    Inventors: Gene Hung, C. Frank Bennett, Janet M. Leeds, Susan M. Freier
  • Patent number: 10738309
    Abstract: The invention relates to antisense oligonucleotidic sequences (ODN) against Smad7 suitably modified, and their uses in medical field as therapeutic biological agents, in particular in the treatment of chronic inflammatory bowel disease, such as Crohn's disease and ulcerative colitis.
    Type: Grant
    Filed: March 22, 2018
    Date of Patent: August 11, 2020
    Assignee: Nogra Pharma Limited
    Inventor: Giovanni Monteleone
  • Patent number: 10711272
    Abstract: Provided herein are, inter alia, nucleic acid compounds useful for targeting CTLA-4-expressing cells and modulating cell activity of the CTLA-4-expressing cells. The compositions provided herein may be part of pharmaceutical compositions and may be used for treatment of cancer, inflammatory diseases, infectious diseases or metabolic diseases.
    Type: Grant
    Filed: January 21, 2015
    Date of Patent: July 14, 2020
    Assignee: City of Hope
    Inventors: Hua Yu, Andreas Herrmann, Marcin Tomasz Kortylewski
  • Patent number: 10662428
    Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
    Type: Grant
    Filed: April 11, 2019
    Date of Patent: May 26, 2020
    Assignee: SIRNA THERAPEUTICS, INC.
    Inventors: Leonid Beigelman, James McSwiggen, Chandra Vargeese
  • Patent number: 10570414
    Abstract: This invention provides plants having resistance to invertebrate pests. More specifically, this invention discloses a non-natural transgenic plant cell expressing at least one invertebrate miRNA in planta for suppression of a target gene of an invertebrate pest or of a symbiont associated with the invertebrate pest. Also provided are recombinant DNA constructs for expression of at least one invertebrate miRNA in planta, a non-natural transgenic plant containing the non-natural transgenic plant cell of this invention, a non-natural transgenic plant grown from the non-natural transgenic plant cell of this invention, and non-natural transgenic seed produced by the non-natural transgenic plants, as well as commodity products produced from a non-natural transgenic plant cell, plant, or seed of this invention.
    Type: Grant
    Filed: November 10, 2016
    Date of Patent: February 25, 2020
    Assignee: Monsanto Technology LLC
    Inventors: Edwards Allen, William P. Donovan, Gregory R. Heck, James K. Roberts, Virginia Ursin, Yuanji Zhang
  • Patent number: 10538763
    Abstract: The present disclosure pertains generally to double-stranded small interfering RNAs that modulate gene expression for use in research, diagnostics, and/or therapeutics. In certain embodiments, the present disclosure provides double-stranded small interfering RNAs that modulate DUX4 gene expression. In certain embodiments, the present disclosure provides methods of inhibiting DUX4 gene expression by contacting a cell with double-stranded small interfering RNAs.
    Type: Grant
    Filed: January 15, 2016
    Date of Patent: January 21, 2020
    Assignees: Ionis Pharmaceuticals, Inc., Fred Hutchinson Cancer Research Center
    Inventors: Frank Rigo, Stephen J. Tapscott
  • Patent number: 10519452
    Abstract: The present invention provides an antiviral agent comprising an RNA oligonucleotide having a particular sequence and structure. Specifically, when a cell line is treated with an RNA oligonucleotide having specific sequence and helical bend structure according to the present invention, the expression of interferon-? or ISG56 is increased and anti-viral activating is exhibited. Thus, a composition comprising the RNA oligonucleotide can be used as an antiviral agent.
    Type: Grant
    Filed: August 29, 2016
    Date of Patent: December 31, 2019
    Assignee: KOREA ADVANCED INSTITUTE OF SCIENCE AND TECHNOLOGY
    Inventors: Byong-Seok Choi, Janghyun Lee
  • Patent number: 10493141
    Abstract: The disclosure provides compositions and methods involving viral RNA segments for use in modulating immune responses, including inhibition inflammation related to pathogenic T-cell activation. In addition, modification of the viral sequences responsible for modulating immune response provides for improved vaccine formulations.
    Type: Grant
    Filed: September 9, 2015
    Date of Patent: December 3, 2019
    Assignee: THE UNIVERSITY OF IOWA RESEARCH FOUNDATION
    Inventors: Jack T. Stapleton, Nirjal Bhattarai, James McLinden, Jinhau Xiang, Bev Davidson
  • Patent number: 10472628
    Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a Serum Amyloid A (SAA) gene, and methods of using the dsRNA to inhibit expression of SAA.
    Type: Grant
    Filed: December 19, 2017
    Date of Patent: November 12, 2019
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Antonin de Fougerolles, Tatiana Novobrantseva, Gregory Hinkle
  • Patent number: 10457945
    Abstract: This invention provides UNA oligomers for therapeutics having prolonged stability. The UNA oligomers can be composed of one or more 2?-3?-seco-nucleomonomers and one or more natural or non-natural nucleotide monomers. Embodiments include UNA oligomers with phosphorothioate or boranophosphate intermonomer linkages. The UNA oligomers can be used for therapeutics that target oligonucleotides, nucleic acids, or RNAs to reduce their activity.
    Type: Grant
    Filed: April 13, 2018
    Date of Patent: October 29, 2019
    Assignee: ARCTURUS THERAPEUTICS, INC.
    Inventor: Jesper Wengel
  • Patent number: 10443058
    Abstract: The present invention relates to oligomeric compounds and conjugates thereof that target Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) PCSK9 mRNA in a cell, leading to reduced expression of PCSK9. Reduction of PCSK9 expression is beneficial for a range of medical disorders, such as hypercholesterolemia and related disorders.
    Type: Grant
    Filed: December 8, 2017
    Date of Patent: October 15, 2019
    Assignee: ROCHE INNOVATION CENTER COPENHAGEN A/S
    Inventors: Nanna Albæk, Maj Hedtjärn, Marie Wickstrom Lindholm, Niels Fisker Nielsen, Andreas Petri, Jacob Ravn
  • Patent number: 10426789
    Abstract: The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject. The present embodiments provided herein are directed to compounds and compositions useful for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP). In certain embodiments, P23H rhodopsin inhibitors provided herein are allele-specific antisense compounds targeted to a P23H mutant allele that are capable of selectively inhibiting expression of P23H rhodopsin mutant protein to a greater extent than wild-type protein.
    Type: Grant
    Filed: February 26, 2016
    Date of Patent: October 1, 2019
    Inventors: Susan F. Murray, Punit P. Seth, Michael L. McCaleb, Susan M. Freier, Priyam Singh
  • Patent number: 10392620
    Abstract: This disclosure concerns nucleic acid molecules and methods of use thereof for control of pathogens through RNA interference-mediated inhibition of target coding and transcribed non-coding sequences in pathogens. The disclosure also concerns methods for applying dsRNA through formulations and/or transgenic plants that express nucleic acid molecules useful for the control of pathogens, and the plant cells and plants obtained thereby.
    Type: Grant
    Filed: November 1, 2017
    Date of Patent: August 27, 2019
    Assignee: Dow AgroSciences LLC
    Inventors: Javier A. Delgado, Justin M. Lira, Chaoxian Geng, Meghan L. Frey
  • Patent number: 10370659
    Abstract: In certain embodiments, the present disclosure provides compounds and methods for increasing the antisense activity of an antisense compound in a cell. In certain embodiments, the present disclosure provides methods for identifying antisense compounds having high activity. In certain embodiments, the present disclosure provides methods for identifying antisense compounds that bind to enhancer or repressor proteins.
    Type: Grant
    Filed: February 23, 2016
    Date of Patent: August 6, 2019
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Xue-hai Liang, Wen Shen, Stanley T. Crooke
  • Patent number: 10370661
    Abstract: Materials and methods for regulating gene expression using nanoparticles functionalized with antisense oligonucleotides are provided.
    Type: Grant
    Filed: July 19, 2017
    Date of Patent: August 6, 2019
    Assignee: NORTHWESTERN UNIVERSITY
    Inventors: Chad A. Mirkin, Nathaniel L. Rosi, C. Shad Thaxton, David A. Giljohann
  • Patent number: 10370670
    Abstract: The invention provides a double-stranded ribonucleic acid (dsRNA) having a chain length suitable for simultaneously showing low toxicity and high function in the use of an adjuvant and the like, and resisting variation of chain length even when subjected to a heating and cooling treatment, or a salt thereof; an immune potentiator, adjuvant, pharmaceutical product and the like containing the dsRNA and the like; and a production method of such dsRNA and the like. The invention is characterized in that the weight average chain length of two or more single-stranded ribonucleic acids (ssRNAs) constituting the first chain constituting dsRNA is not more than ½ of the weight average chain length of one ssRNA constituting the second chain.
    Type: Grant
    Filed: October 24, 2017
    Date of Patent: August 6, 2019
    Assignee: KYOWA HAKKO BIO CO., LTD.
    Inventors: Tetsuo Nakano, Eitora Yamamura
  • Patent number: 10363266
    Abstract: The present invention relates to antisense-oligonucleotides having a length of at least 10 nucleotides, wherein at least two of the nucleotides are LNAs, their use as inhibitors of TGF-R signaling, pharmaceutical compositions containing such antisense-oligonucleotides and the use for prophylaxis and treatment of neurological, neurodegenerative, fibrotic and hyperproliferative diseases.
    Type: Grant
    Filed: November 16, 2015
    Date of Patent: July 30, 2019
    Assignee: NeuroVision Pharma GmbH
    Inventors: Markus Hossbach, Monika Krampert, Hans-Lothar Arth
  • Patent number: 10358648
    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: February 1, 2017
    Date of Patent: July 23, 2019
    Assignee: OliX Pharmaceuticals, Inc.
    Inventors: Dong Ki Lee, Sun Woo Hong, Hanna Lee, Dayeon Yu, Ji Eom
  • Patent number: 10357509
    Abstract: The present disclosure relates to compositions and methods for treating cancers using antisense (AS) nucleic acids directed against Insulin-like Growth Factor 1 Receptor (IGF-1R). The AS may be administered to the patients systemically, or may be used to produce an autologous cancer cell vaccine. In embodiments, the AS are provided in an implantable irradiated biodiffusion chamber comprising tumor cells and an effective amount of the AS. The chambers are irradiated and implanted in the abdomen of subjects and stimulate an immune response that attacks tumors distally. The compositions and methods disclosed herein may be used to treat many different kinds of cancer, for example glioblastoma.
    Type: Grant
    Filed: March 9, 2018
    Date of Patent: July 23, 2019
    Assignee: Thomas Jefferson University
    Inventors: David W. Andrews, Douglas C. Hooper
  • Patent number: 10351855
    Abstract: The present invention relates to a pharmaceutical composition for inhibiting cancer cell metastasis, the pharmaceutical composition including at least one of: (1) si-rpS3/203 binding to a 203rd base sequence of rpS3 mRNA; (2) si-rpS3/635 binding to a base sequence corresponding to a position 635 of the rpS3 mRNA; (3) si-rpS3/747 binding to a base sequence corresponding to a position 747 of the rpS3 mRNA; and (4) si-rpS3/766 binding to a base sequence corresponding to a position 766 of the rpS3 mRNA.
    Type: Grant
    Filed: June 21, 2017
    Date of Patent: July 16, 2019
    Inventor: Joon Kim
  • Patent number: 10342758
    Abstract: Provided are a cationic lipid which facilitates the introduction of a nucleic acid into, for example, a cell or the like; a composition containing the cationic lipid and a nucleic acid; a method for introducing a nucleic acid into a cell by using a composition containing the cationic lipid and the nucleic acid; and the like. The cationic lipid is, for example, a cationic lipid represented by formula (A): formula (A): (wherein R1 is alkenyl or the like, R2 is alkenyl or the like, R3 and R4 are each alkyl, or are combined together to form alkylene, or R3 and R5 are combined together to form alkylene, R5 is a hydrogen atom or the like, or is combined together with R3 to form alkylene, X1 is alkylene, and X2 is a single bond or alkylene).
    Type: Grant
    Filed: July 8, 2013
    Date of Patent: July 9, 2019
    Assignee: KYOWA HAKKO KIRIN CO., LTD.
    Inventors: Takeshi Kuboyama, Tomohiro Era, Tomoyuki Naoi, Kaori Yagi, Shintaro Hosoe
  • Patent number: 10337016
    Abstract: The present disclosure provides a pharmaceutical composition for treating cancer comprising an RNA oligonucleotide having a particular sequence and structure. Specifically, when a cell line is treated with an RNA oligonucleotide having specific sequence and helical bend structure according to the present disclosure, the expression of ISG56 is increased and apoptosis of cancer cells is induced. Thus, a composition comprising the RNA oligonucleotide can be used as an anticancer agent.
    Type: Grant
    Filed: October 14, 2016
    Date of Patent: July 2, 2019
    Assignee: KOREA ADVANCED INSTITUTE OF SCIENCE AND TECHNOLOGY
    Inventors: Byong-Seok Choi, Suk-Jo Kang, Janghyun Lee, Ji Youn Min, Dongmin Chun, Si-Eun Sung
  • Patent number: 10337010
    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: August 23, 2017
    Date of Patent: July 2, 2019
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Brian Bettencourt, William Querbes, Kevin Fitzgerald, Maria Frank-Kamenetsky, Stuart Milstein, Svetlana Shulga Morskaya
  • Patent number: 10329568
    Abstract: The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended.
    Type: Grant
    Filed: September 5, 2018
    Date of Patent: June 25, 2019
    Assignee: Silence Therapeutics GMBH
    Inventors: Klaus Giese, Jörg Kaufmann, Anke Klippel-Giese
  • Patent number: 10323246
    Abstract: The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended.
    Type: Grant
    Filed: August 15, 2017
    Date of Patent: June 18, 2019
    Assignee: Silence Therapeutics GMBH
    Inventors: Klaus Giese, Jörg Kaufmann, Anke Klippel-Giese
  • Patent number: 10302644
    Abstract: The invention provides, inter alia, methods of prognosing the survival of a multiple myeloma patient based levels of TP53RK in multiple myeloma cells of the patient. Also provided are methods of screening for therapeutic agents for treating multiple myeloma based on their ability to decrease TP53RK levels or activity in a patient with multiple myeloma.
    Type: Grant
    Filed: October 20, 2015
    Date of Patent: May 28, 2019
    Assignee: Dana-Farber Cancer Institute, Inc.
    Inventors: Teru Hideshima, Kenneth C. Anderson, Francesca Cottini
  • Patent number: 10232055
    Abstract: This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.
    Type: Grant
    Filed: October 31, 2016
    Date of Patent: March 19, 2019
    Assignee: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
    Inventors: Katalin Kariko, Drew Weissman
  • Patent number: 10219492
    Abstract: Provided herein are methods for altering respiratory syncytial virus (RSV) replication in a cell using oligonucleotides derived from tRNAs, also referred to as tRFs (tRNA-derived RNA Fragments). The oligonucleotides may be used to decrease or increase replication of RSV. Also provided herein are methods for treating a subject having or at risk of having an RSV infection, and animal models for evaluating viral and host factors in RSV pathogenesis.
    Type: Grant
    Filed: November 21, 2014
    Date of Patent: March 5, 2019
    Assignee: The Board of Regents of the University of Texas System
    Inventors: Xiaoyong Bao, Yong Sun Lee
  • Patent number: 10208309
    Abstract: The present invention relates to a novel siRNA, and a high-efficiency double-stranded oligo RNA structure containing the same, and a nanoparticle containing the high-efficiency double-stranded oligo RNA structure. The double-stranded oligo RNA structure has a structure in which a hydrophilic material and a hydrophobic material are conjugated to both ends of a double-stranded oligo RNA (siRNA) via a simple covalent bond or linker-mediated covalent bond in order to be efficiently delivered into cells, and may be converted into a nanoparticle form in an aqueous solution by hydrophobic interactions of double-stranded oligo RNA structures. It is preferable that the siRNA contained in the double-stranded oligo RNA structure is an siRNA specific for fibrosis or respiratory disease-related gene, particularly, amphiregulin or stratifin.
    Type: Grant
    Filed: April 6, 2015
    Date of Patent: February 19, 2019
    Assignee: BIONEER CORPORATION
    Inventors: Jeiwook Chae, Pyoung Oh Yoon, Boram Han, Mi Na Kim, Youngho Ko, Han Oh Park
  • Patent number: 10202599
    Abstract: The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.
    Type: Grant
    Filed: August 8, 2012
    Date of Patent: February 12, 2019
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Punit P. Seth, Michael Oestergaard, Eric E. Swayze
  • Patent number: 10174327
    Abstract: The present disclosure provides an RNA oligonucleotide having a helical bend structure and a use thereof. Specifically, double strands formed by a complementary binding of two sequences have a helical bend structure in the RNA oligonucleotide. The RNA oligonucleotide can increase the expression of interferon-? or ISG56 and thus can be used as an immune system enhancer.
    Type: Grant
    Filed: May 26, 2016
    Date of Patent: January 8, 2019
    Assignee: KOREA ADVANCED INSTITUTE OF SCIENCE AND TECHNOLOGY
    Inventors: Byong-Seok Choi, Mi Kyung Lee, Janghyun Lee, Suk-Jo Kang, Eun Byeol Park
  • Patent number: 10138485
    Abstract: Neutral lipid formulations for nucleic acid delivery are provided according to the invention. The neutral lipid formulations include hydrophobically modified polynucleotides and fat mixtures. Methods of using the neutral lipid formulations are also provided.
    Type: Grant
    Filed: September 22, 2009
    Date of Patent: November 27, 2018
    Assignee: RXi Pharmaceuticals Corporation
    Inventors: Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Michelle Miller, Karen G. Bulock
  • Patent number: 10131910
    Abstract: The invention relates to the fields of medicine and immunology. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Usher syndrome type 2A and/or USH2A-associated non syndromic retina degeneration.
    Type: Grant
    Filed: July 9, 2015
    Date of Patent: November 20, 2018
    Assignee: STICHTING KATHOLIEKE UNIVERSITEIT
    Inventor: Hendrikus Antonius Rudolfus Van Wyk
  • Patent number: 10119136
    Abstract: One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene in vivo. The dsRNA duplex comprises one or more 4?-modifications in one or both strand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for in vivo therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
    Type: Grant
    Filed: January 9, 2015
    Date of Patent: November 6, 2018
    Assignee: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Muthiah Manoharan, Shigeo Matsuda, Kallanthottathil G. Rajeev, Ivan Zlatev
  • Patent number: 10106852
    Abstract: This invention is directed to mutated Activin A type I receptor proteins (ACVR1) and isolated nucleic acids encoding same. The invention also relates to the use of mutated ACVR1 in the diagnosis and treatment of Fibrodysplasia Ossificans Progressiva (FOP).
    Type: Grant
    Filed: October 22, 2014
    Date of Patent: October 23, 2018
    Assignee: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
    Inventors: Frederick S. Kaplan, Eileen M. Shore
  • Patent number: 10106792
    Abstract: The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.
    Type: Grant
    Filed: December 12, 2016
    Date of Patent: October 23, 2018
    Assignees: CITY OF HOPE, INTEGRATED DNA TECHNOLOGIES, INC.
    Inventors: John J. Rossi, Mark A. Behlke, Dongho Kim
  • Patent number: 10100004
    Abstract: Here described are compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.
    Type: Grant
    Filed: June 8, 2016
    Date of Patent: October 16, 2018
    Assignee: Nitto Denko Corporation
    Inventors: Yoshiro Niitsu, Joseph E. Payne, John A. Gaudette, Zheng Hou, Victor Knopov, Richard P. Witte, Mohammad Ahmadian, Loren A. Perelman, Yasunobu Tanaka, Violetta Akopian
  • Patent number: 10100310
    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: April 11, 2017
    Date of Patent: October 16, 2018
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Susan M. Freier, Huynh-Hoa Bui
  • Patent number: 10036022
    Abstract: The invention relates to antisense oligonucleotidic sequences (ODN) against Smad7 suitably modified, and their uses in medical field as therapeutic biological agents, in particular in the treatment of chronic inflammatory bowel disease, such as Crohn's disease and ulcerative colitis.
    Type: Grant
    Filed: November 18, 2016
    Date of Patent: July 31, 2018
    Assignee: Nogra Pharma Limited
    Inventor: Giovanni Monteleone
  • Patent number: 10004814
    Abstract: The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.
    Type: Grant
    Filed: November 10, 2014
    Date of Patent: June 26, 2018
    Assignee: SIRNA THERAPEUTICS, INC.
    Inventors: Marija Tadin-Strapps, Tayeba Khan, Walter Richard Strapps, Laura Sepp-Lorenzino, Vasant Jadhav, Duncan Brown
  • Patent number: 10000766
    Abstract: Provided is a strategy for generating transgenic plants with concurrent resistance to DNA and RNA viruses at one construction, so as to develop an RNA-directed DNA methylation (RdDM) transgenic system using a hairpin construct of Ageratum yellow vein virus (AYVV) promoter region residing in an intron to resist DNA virus infection by RdDM. Furthermore, the hairpin construct of the AYVV promoter region coupled with an untranslatable nucleocapsid protein (NP) fragment of Melon yellow sport virus (MYSV) is created to induce post-transcriptional gene silencing (PTGS) against MYSV. A method for providing transgenic plants conferring concurrent resistance to both AYVV and MYSV for control of DNA and RNA virus at the same time, and underlying RdDM and PTGS mechanisms, respectively, is also provided.
    Type: Grant
    Filed: July 17, 2015
    Date of Patent: June 19, 2018
    Assignee: NATIONAL CHUNG HSING UNIVERSITY
    Inventors: Shyi-Dong Yeh, Ching-Fu Yang, Kuan-Chun Chen, Ya-Ling Huang
  • Patent number: RE47691
    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 202.
    Type: Grant
    Filed: July 10, 2017
    Date of Patent: November 5, 2019
    Assignee: The University of Western Australia
    Inventors: Stephen Donald Wilton, Sue Fletcher, Graham McClorey
  • Patent number: RE47751
    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 202.
    Type: Grant
    Filed: July 20, 2017
    Date of Patent: December 3, 2019
    Assignee: The University of Western Australia
    Inventors: Stephen Donald Wilton, Sue Fletcher, Graham McClorey
  • Patent number: RE47769
    Abstract: Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.
    Type: Grant
    Filed: November 11, 2016
    Date of Patent: December 17, 2019
    Assignee: The University of Western Australia
    Inventors: Stephen Donald Wilton, Sue Fletcher, Graham McClorey