Patents Examined by Jane J Zara
  • Regulation of RAN translation by PKR and eIF2A-P pathways
    Patent number: 11345911
    Abstract: Methods and compositions for modulating repeat non-ATG protein (RAN protein) translation are provided. In some aspects, the disclosure provides methods of inhibiting RAN protein translation by contacting a cell with an effective amount of an inhibitor of eIF2 phosphorylation or an inhibitor of protein kinase R (PKR). In some embodiments, methods described by the disclosure are useful for treating diseases associated with RAN protein translation, such as certain neurodegenerative diseases.
    Type: Grant
    Filed: April 17, 2018
    Date of Patent: May 31, 2022
    Assignee: University of Florida Research Foundation, Incorporated
    Inventors: Laura Ranum, Tao Zu
  • Promoter for cell-specific gene expression and uses thereof
    Patent number: 11344631
    Abstract: The present invention refers to nucleotide sequences used for driving the expression of a therapeutic gene, preferably FVIII and/or its variants specifically in endothelial cells and/or hematopoietic, preferably myeloid cells. The sequences are useful for gene and/or cell therapy, preferably for treating hemophilia, more preferably type A hemophilia.
    Type: Grant
    Filed: June 12, 2017
    Date of Patent: May 31, 2022
    Assignee: Universita' del Piemonte Orientale
    Inventors: Antonia Follenzi, Diego Zanolini, Rosella Fama', Simone Merlin
  • Cell line with METTL3 gene knocked out, its construction method and interference vector
    Patent number: 11339370
    Abstract: A pig intestinal tract epithelium cell line with METTL3 gene knocked out and a construction method therefor. A gene interference vector for METTL3 form a pig is also provided.
    Type: Grant
    Filed: July 21, 2018
    Date of Patent: May 24, 2022
    Assignee: ZHEJIANG UNIVERSITY
    Inventors: Xin Zong, Yizhen Wang, Jing Zhao
  • Compositions and methods for inhibiting TIGIT gene expression
    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
  • Modulators of diacyglycerol acyltransferase 2 (DGAT2)
    Patent number: 11312962
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting DGAT2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DGAT2.
    Type: Grant
    Filed: June 28, 2019
    Date of Patent: April 26, 2022
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Sanjay Bhanot, Susan M. Freier, Eric E. Swayze
  • RNA interference for the treatment of gain-of-function disorders
    Patent number: 11299734
    Abstract: The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.
    Type: Grant
    Filed: May 17, 2019
    Date of Patent: April 12, 2022
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Neil Aronin, Phillip D. Zamore
  • Switchable CAS9 nucleases and uses thereof
    Patent number: 11299755
    Abstract: Some aspects of this disclosure provide compositions, methods, systems, and kits for controlling the activity and/or improving the specificity of RNA-programmable endonucleases, such as Cas9. For example, provided are guide RNAs (gRNAs) that are engineered to exist in an “on” or “off” state, which control the binding and hence cleavage activity of RNA-programmable endonucleases. Some aspects of this disclosure provide mRNA-sensing gRNAs that modulate the activity of RNA-programmable endonucleases based on the presence or absence of a target mRNA. Some aspects of this disclosure provide gRNAs that modulate the activity of an RNA-programmable endonuclease based on the presence or absence of an extended DNA (xDNA).
    Type: Grant
    Filed: February 20, 2020
    Date of Patent: April 12, 2022
    Assignee: President and Fellows of Harvard College
    Inventors: David R. Liu, Johnny Hao Hu
  • Compositions and methods for delivery of agents
    Patent number: 11285222
    Abstract: This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
    Type: Grant
    Filed: October 11, 2019
    Date of Patent: March 29, 2022
    Assignee: ModernaTX, Inc.
    Inventors: Gilles Besin, Stephen Hoge, Joseph Senn, Kerry Benenato, Staci Sabnis
  • Integrin alpha 9 blockade suppresses lymphatic valve formation and promotes transplant survival
    Patent number: 11279763
    Abstract: In certain embodiments, the present invention provides methods of suppressing valvulogenesis (VG) in a lymphatic vessel in an inflamed or transplanted tissue or organ in a mammal in need thereof comprising administering an effective amount of an anti-integrin alpha 9 (Itga-9) therapeutic agent to the mammal, and optionally by administering anti-VEGFR-3 agent.
    Type: Grant
    Filed: October 26, 2017
    Date of Patent: March 22, 2022
    Assignee: The Regents of the University of California
    Inventor: Lu Chen
  • Exosomes from clonal progenitor cells
    Patent number: 11274281
    Abstract: The invention provides methods, compositions, uses and kits relating to exosomes isolated from progenitor cells.
    Type: Grant
    Filed: February 7, 2019
    Date of Patent: March 15, 2022
    Assignee: ReCyte Therapeutics, Inc.
    Inventors: Dana Larocca, Mohammad Hassanipour, Paola A. Bignone
  • Organic compositions to treat EPAS1-related diseases
    Patent number: 11261444
    Abstract: The present disclosure relates to methods of treating EPAS1-related diseases such as cancer, metastases, astrocytoma, bladder cancer, breast cancer, chondrosarcoma, colorectal carcinoma, gastric carcinoma, glioblastoma, head and neck squamous cell carcinoma, hepatocellular carcinoma, lung adenocarcinoma, neuroblastoma, non-small cell lung cancer, melanoma, multiple myeloma, ovarian cancer, rectal cancer, renal cancer, clear cell renal cell carcinoma (and metastases of this and other cancers), gingivitis, psoriasis, Kaposi's sarcoma-associated herpesvirus, preeclampsia, inflammation, chronic inflammation, neovascular diseases, and rheumatoid arthritis, using a therapeutically effective amount of a RNAi agent to EPAS1.
    Type: Grant
    Filed: January 21, 2020
    Date of Patent: March 1, 2022
    Assignee: Arrowhead Pharmaceuticals, Inc.
    Inventors: Brian Bettencourt, Shanthi Ganesh, Elizabeth George, Dieter Huesken, Stuart Milstein, Jonathan Solomon, Emily Thomas, Ivanka Toudjarska, Jennifer Tullai, Jan Weiler
  • Agents for use in the treatment of glioma
    Patent number: 11246927
    Abstract: The present invention relates to agents for use in the treatment of glioma, in particular astrocytoma WHO I? and 111°, as well as IV0 (glioblastoma), in a subject.
    Type: Grant
    Filed: July 6, 2016
    Date of Patent: February 15, 2022
    Assignee: DC EUROPA LIMITED
    Inventors: Matthias Osswald, Wolfgang Wick, Frank Winkler, Erik Jung, Jonas Blaes
  • Self-assembled 3D RNA cage nanoparticles
    Patent number: 11242532
    Abstract: Disclosed herein are three-dimensional cage molecules, wherein the cage molecule is composed of RNA. Also disclosed is a composition including the three-dimensional cage molecule, as well as a pharmaceutical composition containing the three-dimensional cage molecule. Also disclosed herein are methods of administering a cage molecule, composition, or formulation thereof to a subject in need thereof.
    Type: Grant
    Filed: May 10, 2017
    Date of Patent: February 8, 2022
    Assignees: Ohio State Innovation Foundation, Ball State University
    Inventors: Peixuan Guo, Daniel Jasinski, Hui Li, Emil Khisamutdinov
  • Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof
    Patent number: 11234994
    Abstract: The present disclosure relates to RNA interference (RNAi) reagents for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.
    Type: Grant
    Filed: April 13, 2017
    Date of Patent: February 1, 2022
    Assignee: Benitec Biopharma Limited
    Inventors: David Suhy, Michael Graham, Capucine Trollet, Alberto Malerba, John George Dickson
  • Methods for diagnosing and treating metastatic cancer
    Patent number: 11236331
    Abstract: Disclosed are methods of diagnosing and treating metastatic cancer in a subject. The methods involve detecting or modulating the expression of at least one of Kif3b, ACTB, SRPK1, TM EM 229b, Cl4orf142, KB-1460A1.5, ACTC1, Nr2f1, KIAA0922, KDELR3, APBA2, miRNA 130b, miRNA 374b, or miRNA 122 in a biological sample from the subject.
    Type: Grant
    Filed: June 14, 2017
    Date of Patent: February 1, 2022
    Assignee: ENTOS PHARMACEUTICALS INC.
    Inventors: John Lewis, Konstantin Stoletov, Lian Willetts
  • Peptide oligonucleotide conjugates
    Patent number: 11225662
    Abstract: Oligonucleotide analogues conjugated to carrier peptides are provided. The disclosed compounds are useful for the treatment of various diseases, for example diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
    Type: Grant
    Filed: October 4, 2019
    Date of Patent: January 18, 2022
    Assignee: Sarepta Therapeutics, Inc.
    Inventor: Gunnar J. Hanson
  • Compounds and compositions for intracellular delivery of therapeutic agents
    Patent number: 11220476
    Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
    Type: Grant
    Filed: August 29, 2019
    Date of Patent: January 11, 2022
    Assignee: ModernaTX, Inc.
    Inventors: Kerry E. Benenato, Mark Cornebise
  • Sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
    Patent number: 11213593
    Abstract: Spherical nucleic acids (SNAs), consisting of densely packed, highly oriented polynucleotide strands attached to the surface of nanoparticles, are able to overcome the typical challenges of nucleic acid delivery. The present disclosure demonstrates that G-rich SNAs exhibit several-fold higher uptake into cells relative to SNAs rich in other nucleotides. This disclosure provides an effective strategy to maximize the intracellular delivery of SNAs, which is applicable to other nanoparticle systems, thus establishing an important design consideration for nanoparticle-based intracellular delivery of therapeutics.
    Type: Grant
    Filed: November 20, 2015
    Date of Patent: January 4, 2022
    Assignee: Northwestern University
    Inventors: Chad A. Mirkin, Chung Hang J. Choi, Suguna P. Narayan, Liangliang Hao, Evelyn Auyeung
  • Methods and compositions for the treatment of cancer or other diseases
    Patent number: 11208654
    Abstract: The present invention relates to methods and compositions for the treatment of diseases, including cancer, infectious diseases and autoimmune diseases. The present invention also relates to methods and compositions for improving immune function. More particularly, the present invention relates to multifunctional molecules that are capable of being delivered to cells of interest for the treatment of diseases and for the improvement in immune function.
    Type: Grant
    Filed: March 6, 2019
    Date of Patent: December 28, 2021
    Assignee: CITY OF HOPE
    Inventors: Hua Yu, Marcin Kortylewski, Richard Jove, Piotr Marek Swiderski, John J. Rossi
  • Compositions and methods for internalizing enzymes
    Patent number: 11208458
    Abstract: Compositions and methods for treating enzyme-deficiency diseases are disclosed. Multidomain therapeutic proteins containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The multidomain therapeutic proteins are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.
    Type: Grant
    Filed: June 6, 2018
    Date of Patent: December 28, 2021
    Assignee: Regeneron Pharmaceuticals, Inc.
    Inventors: Andrew Baik, Katherine Cygnar, Christopher Schoenherr, Christos Kyratsous, Cheng Wang