Nucleic Acid Expression Inhibitors Patents (Class 536/24.5)
  • Patent number: 11891606
    Abstract: A method for treating a patient suffering from multiple sclerosis, including progressive forms of multiple sclerosis, comprising periodically administering a pharmaceutical composition comprising a therapeutically effective amount of OLIGONUCLEOTIDE I to the patient, thereby treating the patient.
    Type: Grant
    Filed: June 15, 2018
    Date of Patent: February 6, 2024
    Assignee: Antisense Therapeutics Ltd
    Inventor: George Tachas
  • Patent number: 11891603
    Abstract: The present invention relates to a therapeutic antisense oligonucleotide which binds to exon 51 of the human dystrophin pre-mRNA to induce exon skipping, and conjugates and compositions thereof for the treatment of DMD.
    Type: Grant
    Filed: September 3, 2021
    Date of Patent: February 6, 2024
    Assignee: THE GOVERNORS OF THE UNIVERSITY OF ALBERTA
    Inventors: Toshifumi Yokota, Yusuke Echigoya
  • Patent number: 11872239
    Abstract: The present invention relates to a composition comprising an oligomeric compound comprising one or more tricyclo-deoxyribonucleic acid (tc-DNA) nucleosides and one or more lipid moiety, wherein said one or more lipid moiety is covalently linked to said oligomeric compound either directly or via a spacer, and wherein preferably said oligomeric compound comprises from 5 to 40 monomer subunits, as well as pharmaceutical compositions thereof and their uses in the prevention or treatment of neuromuscular or musculoskeletal diseases such as Duchenne muscular dystrophy or Steinert disease.
    Type: Grant
    Filed: April 20, 2018
    Date of Patent: January 16, 2024
    Assignee: Synthena AG
    Inventors: Wolfgang Andreas Renner, Branislav Dugovic, Reto Bertolini
  • Patent number: 11860099
    Abstract: An antibody or Fab fragment, wherein at least one amino group of the N-terminal amino acid of the light chain and/or of the N-terminal amino acid of the heavy chain is bound by an amidic bond to a molecule comprising a fluorophore group A, wherein said bond constitutes at least 70% of the total binding of said molecule to said antibody or Fab.
    Type: Grant
    Filed: June 26, 2018
    Date of Patent: January 2, 2024
    Assignee: Alda S.r.l.
    Inventors: Alberto Luini, Vincenzo Manuel Marzullo, Giuseppe Palumbo, Federica Liccardo
  • Patent number: 11859185
    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: October 14, 2020
    Date of Patent: January 2, 2024
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Kevin Fitzgerald, David Erbe, Gregory Hinkle
  • Patent number: 11827882
    Abstract: Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.
    Type: Grant
    Filed: August 9, 2019
    Date of Patent: November 28, 2023
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Anastasia Khvorova, Julia Alterman, Faith Conroy, Edith Pfister, Neil Aronin, Ken Yamada
  • Patent number: 11820984
    Abstract: The present invention relates to a double-helix oligonucleotide construct comprising a double-stranded miRNA and a composition for preventing or treating cancer comprising the same. More particularly, the present invention relates to a double-helix oligonucleotide construct comprising miR-544a characterized by a method that effectively inhibits the proliferation of cancer cells or induces a voluntary death of cancer cells, and an anticancer composition comprising the construct.
    Type: Grant
    Filed: January 29, 2019
    Date of Patent: November 21, 2023
    Assignee: BIONEER CORPORATION
    Inventors: Taewoo Lee, Jiwon Ryu, Eun Ji Im
  • Patent number: 11793756
    Abstract: A non-viral delivery complex comprising a cationic core which is a nanoparticle comprising a peptide having a polycationic nucleic acid binding component, a cleavable spacer element and a cell surface receptor binding component; a nucleic acid and optionally a cationic lipid; and an anionic liposomal coating surrounding the cationic core, said coating comprising lipids from a subject's cells. Also related formulations, uses and methods.
    Type: Grant
    Filed: January 10, 2019
    Date of Patent: October 24, 2023
    Assignee: UCL Business PLC
    Inventors: Stephen Hart, Aristides Tagalakis
  • Patent number: 11786546
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GFAP RNA in a cell or subject, and in certain instances reducing the amount of GFAP in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include motor delays, cognitive delays, paroxysmal deterioration, seizures, vomiting, swallowing difficulties, ataxic gait, palatal myoclonus, autonomic dysfunction, and presence of intra-astrocytic inclusions called Rosenthal fibers. Such leukodystrophies include Alexander Disease.
    Type: Grant
    Filed: July 24, 2020
    Date of Patent: October 17, 2023
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Berit Elissa Powers, Frank Rigo
  • Patent number: 11781143
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting PNPLA3 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PNPLA3.
    Type: Grant
    Filed: July 22, 2020
    Date of Patent: October 10, 2023
    Assignee: IONIS PHARMACEUTICALS, INC.
    Inventors: Susan M. Freier, Huynh-Hoa Bui
  • Patent number: 11767527
    Abstract: Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.
    Type: Grant
    Filed: August 9, 2019
    Date of Patent: September 26, 2023
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Anastasia Khvorova, Julia Alterman, Faith Conroy, Edith Pfister, Neil Aronin, Ken Yamada
  • Patent number: 11732009
    Abstract: A nanoparticle activity sensor containing a reporter and at least one tuning domain that modifies a distribution or residence time of the activity sensor when administered to a patient. When administered to the patient, the activity sensor enters cells or tissue where it is cleaved by enzymes specific to a physiological state such as a disease to release a detectable analyte. The tuning domains include molecular structures that modulate distribution or decay by protecting the particle from premature cleavage and indiscriminate hydrolysis, shielding the particle from immune detection and clearance, or by targeting the particle to specific tissue, bodily fluids, or cell types.
    Type: Grant
    Filed: March 15, 2019
    Date of Patent: August 22, 2023
    Assignee: GLYMPSE BIO, INC.
    Inventors: Sangeeta Bhatia, Gabriel Kwong, Eric Huang, Sirshendu Roopom Banerjee, Andrew Warren, Sophie Cazanave
  • Patent number: 11725214
    Abstract: The invention relates to methods for increasing plant yield, and in particular grain yield by reducing or abolishing the expression and/or activity of OTUB1 in a plant. Also described are genetically altered plants characterised by the above phenotype and methods of producing such plants.
    Type: Grant
    Filed: May 24, 2018
    Date of Patent: August 15, 2023
    Assignee: INSTITUTE OF GENETICS AND DEVELOPMENTAL BIOLOGY CHINESE ACADEMY OF SCIENCES
    Inventors: Xiangdong Fu, Shuansuo Wang, Kun Wu, Qian Liu, Qian Qian, Ke Huang, Penggen Duan, Baolan Zhang, Yunhai Li
  • Patent number: 11680262
    Abstract: A method of skipping a target exon of a gene of interest in a genome uses CRISPR-Cas and guide RNA. The guide RNA contains a spacer sequence such that the site of cleavage by the CRISPR-Cas is positioned within 80 bases from the splice donor site immediately before the target exon or the splice acceptor site immediately after the target exon.
    Type: Grant
    Filed: November 21, 2017
    Date of Patent: June 20, 2023
    Assignee: KYOTO UNIVERSITY
    Inventors: Hongmei Li, Noriko Sasakawa, Akitsu Hotta
  • Patent number: 11674154
    Abstract: In some aspects, the disclosure relates to compositions and methods for treating fibrodysplasia ossificans progressiva (FOP) in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that inhibit (e.g., decrease) expression of mutated AVCR1 gene in muscle cells or connective tissues.
    Type: Grant
    Filed: December 2, 2021
    Date of Patent: June 13, 2023
    Assignee: University of Massachusetts
    Inventors: Jae-Hyuck Shim, Guangping Gao, Jun Xie, Yeon-Suk Yang, Jung min Kim, Sachin Chaugule
  • Patent number: 11655472
    Abstract: The present specification provides a drug that causes highly-efficient skipping of exon 50 in the human dystrophin gene. The present specification provides an antisense oligomer which induces skipping of exon 50 in the human dystrophin gene.
    Type: Grant
    Filed: June 23, 2022
    Date of Patent: May 23, 2023
    Assignees: NIPPON SHINYAKU CO., LTD., NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY
    Inventors: Yukiko Enya, Yuta Sunadoi, Reiko Waki, Kaname Muchima, Shin'ichi Takeda, Yoshitsugu Aoki
  • Patent number: 11618773
    Abstract: The present invention relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence separated by a spacer sequence, to said polynucleotide for use in treating and/or preventing disease, and to viral particles, compositions, and uses related thereto. The present invention further relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence for use in treating and/or preventing an NFAT-mediated disease.
    Type: Grant
    Filed: January 14, 2019
    Date of Patent: April 4, 2023
    Assignee: Universität Heidelberg
    Inventors: Markus Hecker, Andreas Wagner, Andreas Jungmann, Oliver Müller, Anca Remes, Hugo Katus
  • Patent number: 11591600
    Abstract: The present technology relates, in part, to long double-stranded RNA (dsRNA) (e.g., 30 or more base pairs) that inhibits gene expression.
    Type: Grant
    Filed: February 12, 2018
    Date of Patent: February 28, 2023
    Assignee: OliX Pharmaceuticals. Inc.
    Inventor: Dong Ki Lee
  • Patent number: 11583571
    Abstract: Stromal Derived Factor-1 (SDF-1) is a small, naturally occurring, potent chemokine with inherent angiogenic, neurogenic, anti-apoptotic protein, which is also a potent stem cell chemoattractant, cardiovascular disease, and other metabolic disturbances. The present invention provides methods for treating erectile dysfunction in a male subject comprising administering to the major pelvic ganglion supplying the cavernous nerves subject compositions comprising SDF-1. SDF-1 promotes stem cell activation, to the major pelvic ganglion supplying the cavernous nerves, helps cell preservation, and prevents adverse penile remodeling. It can be administered as a protein or by gene therapy including but not limited to plasmid DNA, viral transduction, or nanoparticle delivery directly to the penis or to the neurovascular bundle or other pelvic nerve structures during the time of surgery, or before injury, or to treat existing erectile dysfunction.
    Type: Grant
    Filed: May 11, 2018
    Date of Patent: February 21, 2023
    Assignees: The Johns Hopkins University, Summa Health System
    Inventors: Nikolai Sopko, Trinity Bivalacqua, Marc Penn
  • Patent number: 11524047
    Abstract: The present invention relates to pharmaceutical compositions for preventing or treating pulmonary metastasis of cancer. More specifically, the present invention relates to compositions that enhance anti-cancer immunity of the lung rather than induce death of advanced cancer, thus being effective in inhibiting, preventing or treating pulmonary metastasis of cancer.
    Type: Grant
    Filed: December 27, 2018
    Date of Patent: December 13, 2022
    Assignee: Industry-University Cooperation Foundation Hanyang University
    Inventors: Je-Min Choi, Do-Hyun Kim
  • 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: 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: 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: 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: 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: 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: 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: 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: 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