Patents Examined by Sean McGarry
  • Patent number: 12018257
    Abstract: The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA 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 cytidine (a non-complementary nucleotide) or a uridine in a position opposite to the target adenosine to be edited in the target RNA region.
    Type: Grant
    Filed: April 6, 2023
    Date of Patent: June 25, 2024
    Assignee: ProQR Therapeutics II B.V.
    Inventors: Janne Juha Turunen, Petra Geziena De Bruijn, Bart Klein, Roxana Simona Redis, Lenka Van Sint Fiet
  • Patent number: 12016877
    Abstract: In alternative embodiments, provided are compositions, including products of manufacture and kits, and methods, for treating or ameliorating a cancer by inhibiting expression or activity of Mouse Double Minute 2 homolog (MDM2), an APOBEC3G (A3G) protein, message (mRNA) or gene, and/or an ADAR1p150 protein, message (mRNA) or gene, e.g., by increasing the presence of in a cell or adding to a cell a molecule inhibitory to MDM2, APOBEC3G and/or ADAR1p150 expression, such as an miRNA that binds to MDM2, APOBEC3G and/or ADAR1p150 transcripts, or any molecule that can inhibit or destabilize the transcripts, resulting in decreased MDM2, APOBEC3G and/or ADAR1p150 expression, to treat a cancer such as leukemia, e.g., by inhibiting the propagation of a cancer cell, a leukemia cell, a leukemia stem cell (LSC) or a pre-leukemia cell stem cell (pre-LSC).
    Type: Grant
    Filed: August 24, 2022
    Date of Patent: June 25, 2024
    Assignee: The Regents of the University of California
    Inventors: Catriona Jamieson, Raymond Diep, Jane Isquith, Qingfei Jiang, Jessica Pham
  • Patent number: 12012601
    Abstract: Provided herein are methods of treating a cancer in a patient comprising administration of an effective amount of a nuclease-resistant polynucleotide that hybridizes to the translation initiation site of a Grb2 nucleic acid in the patient and either a Bcr-Abl tyrosine kinase inhibitor (e.g., dasatinib) or a cytidine analogue (e.g., decitabine or cytarabine). The cancer may be Ph+ and/or Bcr-Abl positive chronic myelogenous leukemia or acute myeloid leukemia or myelodysplastic syndrome.
    Type: Grant
    Filed: January 15, 2021
    Date of Patent: June 18, 2024
    Assignee: BIO-PATH HOLDINGS, INC.
    Inventor: Ana Tari Ashizawa
  • Patent number: 12006502
    Abstract: Therapeutic compounds for red blood cell-mediated delivery of an active pharmaceutical ingredient to a target cell are described. The therapeutic compounds are configured to bind CD47 on the surface of a red blood cell and to be subsequently transferred to CD47 on the surface of the target cell, the therapeutic compound ultimately being internalized by the target cell via endocytosis. The target cell may be a fibrotic cell.
    Type: Grant
    Filed: January 9, 2024
    Date of Patent: June 11, 2024
    Assignees: K2B Therapeutics, Inc., KIST (Korea Institute of Science and Technology)
    Inventors: HoWon J. Kim, In-San Kim, Jay S. Kim, Sun Hwa Kim, Ick Chan Kwon, Jong Won Lee, Yoo Soo Yang, Hong Yeol Yoon
  • Patent number: 11993776
    Abstract: The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.
    Type: Grant
    Filed: April 17, 2019
    Date of Patent: May 28, 2024
    Assignees: Ascidian Therapeutics, Inc., The Trustees of the University of Pennsylvania
    Inventors: Philip R. Johnson, Bruce C. Schnepp, Jean Bennett, Scott J. Dooley, Krishna Jawaharlal Fisher, Junwei Sun
  • Patent number: 11981894
    Abstract: The present invention provides an oligomer which allows exon 45 skipping in the human dystrophin gene.
    Type: Grant
    Filed: November 17, 2020
    Date of Patent: May 14, 2024
    Assignees: NIPPON SHINYAKU CO., LTD., NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY
    Inventors: Yukiko Enya, Yuichiro Tone, Shin'ichi Takeda, Yoshitsugu Aoki
  • Patent number: 11980672
    Abstract: mRNAs containing an exogenous open reading frame (ORF) flanked by a 5? untranslated region (UTR) and a 3? UTR is provided, wherein the 5? and 3? UTRs are derived from a naturally abundant mRNA in a tissue. Also provided are methods for identifying the 5? and 3? UTRs, and methods for making and using the mRNAs.
    Type: Grant
    Filed: June 21, 2022
    Date of Patent: May 14, 2024
    Assignee: ModernaTX, Inc.
    Inventors: David H. Burkhardt, Romesh R. Subramanian, Christian Cobaugh
  • Patent number: 11965161
    Abstract: Provided herein are methods and compositions related to the treatment or prevention of cancer (e.g., by targeting a tumor in a subject with cancer) by administering to a subject an agent that inhibits autophagy. In certain aspects, provided herein are methods of compositions related to methods of sensitizing cancer cells to tumor necrosis factor-alpha (TNF-?) mediated killing by contacting the cells or administering the agent that inhibits autophagy.
    Type: Grant
    Filed: March 3, 2021
    Date of Patent: April 23, 2024
    Assignee: Regeneron Pharmaceuticals, Inc.
    Inventors: Tara Young, Christopher Daly, Gavin Thurston
  • Patent number: 11965164
    Abstract: Compositions and methods disclosed herein can help provide improved delivery of non-natural therapeutic nucleotides for the treatment of diseases such as cancer. An example composition includes an assembly of amphiphilic polynucleotides, where each amphiphilic polynucleotide includes an aptamer portion, a first nucleotide portion, and a second nucleotide portion.
    Type: Grant
    Filed: October 31, 2022
    Date of Patent: April 23, 2024
    Assignee: Duke University
    Inventors: Ashutosh Chilkoti, Stefan Zauscher, Lei Tang, Sonal Deshpande
  • Patent number: 11959080
    Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting PMP22 expression and for treating, preventing, or ameliorating a disease associated with PMP22.
    Type: Grant
    Filed: October 4, 2021
    Date of Patent: April 16, 2024
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Gene Hung, Holly Kordasiewicz, Hien Thuy Zhao, Eric E. Swayze
  • Patent number: 11946064
    Abstract: Described herein are compositions and methods for treating a disease in a subject by administering delivery vectors that express artificial microRNAs, artificial microRNA clusters, and/or a combination of microRNA clusters and associated non-coding RNAs to the subject. Also described herein are methods for preparing artificial microRNAs and artificial microRNA clusters.
    Type: Grant
    Filed: April 30, 2019
    Date of Patent: April 2, 2024
    Assignee: The Brigham and Women's Hospital, Inc.
    Inventors: Pierpaolo Peruzzi, Ennio Antonio Chiocca
  • Patent number: 11946046
    Abstract: Methods of minimizing dysregulation of Staufen1-associated RNA metabolism can include introducing an amount of a Staufen1-regulating agent to a target cell sufficient to minimize the dysregulation. Therapeutic compositions for treating a neurodegenerative condition associated with Staufen1-induced dysregulation of RNA metabolism can include a therapeutically effective amount of a Staufen1-regulating agent and a pharmaceutically acceptable carrier.
    Type: Grant
    Filed: June 14, 2019
    Date of Patent: April 2, 2024
    Assignee: University of Utah Research Foundation
    Inventors: Stefan M. Pulst, Daniel R. Scoles, Sharan Paul
  • Patent number: 11939363
    Abstract: A range of therapeutic mRNA molecules expressible to provide a target polypeptide or protein. The RNA molecules can contain one or more 5-methoxyuridines and 5-methylcytidines. Further provided are DNA templates, which can be transcribed to provide a target mRNA, and can have altered nucleotides, such as reduced deoxyadenosines. Also provided are processes for making the therapeutic mRNA molecules. The RNA molecules can be translated in vitro or in vivo to provide an active polypeptide or protein. The RNA molecules can be included in a composition used for preventing, treating, or ameliorating at least one symptom of a disease or condition in a subject in need thereof.
    Type: Grant
    Filed: July 14, 2022
    Date of Patent: March 26, 2024
    Assignee: ARCTURUS THERAPEUTICS, INC.
    Inventors: Pattraranee Limphong, Kiyoshi Tachikawa, Padmanabh Chivukula, Daiki Matsuda, Arisa Cale
  • Patent number: 11926828
    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: January 15, 2021
    Date of Patent: March 12, 2024
    Assignee: Phio Pharmaceuticals Corp.
    Inventor: Gerard Cauwenbergh
  • Patent number: 11926826
    Abstract: The present invention relates to compositions comprising TWIST signaling inhibitors and optionally one or more anti-cancer agents, and methods of using the compositions for the treatment of cancer.
    Type: Grant
    Filed: June 24, 2022
    Date of Patent: March 12, 2024
    Assignees: City of Hope, The Regents of the University of California
    Inventors: Carlotta A. Glackin, John Rossi, Jeffrey I. Zink, Fuyuhiko Tamanoi, Cai M. Roberts, James Finlay
  • Patent number: 11920134
    Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a RRM2 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a RRM2 gene using said pharmaceutical composition; and methods for inhibiting the expression of RRM2 in a cell.
    Type: Grant
    Filed: April 13, 2020
    Date of Patent: March 5, 2024
    Assignee: Arrowhead Pharmaceuticals, Inc.
    Inventors: John Frederick Boylan, Birgit Bramlage, Markus Hossbach, John Reidhaar-Olson
  • Patent number: 11896679
    Abstract: The presently disclosed subject matter provides compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter. In particular, compositions and methods are provided for the use of the H1 promoter to express CRISPR guide RNA (gRNA) with altered specificity of the 5? nucleotide, as well as use of the H1 promoter sequence as a bidirectional promoter to express Cas9 nuclease and the gRNA simultaneously. Compositions and methods are also provided for the expression and regulation of gRNA expression in vivo through the use of RNA ribozymes and regulatable aptazymes.
    Type: Grant
    Filed: April 17, 2020
    Date of Patent: February 13, 2024
    Assignee: The Johns Hopkins University
    Inventors: Vinod Jaskula-Ranga, Donald Zack
  • Patent number: 11898146
    Abstract: Compositions comprising optimized aptamers capable of specifically binding to a surface protein of Clostridium difficile spore are provided. A method for detecting, enriching, separating, and/or isolating Clostridium difficile spores is provided.
    Type: Grant
    Filed: December 3, 2020
    Date of Patent: February 13, 2024
    Assignee: LIV PROCESS, INC.
    Inventors: Ronald J. Shannon, Gregory Penner
  • 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: 11891607
    Abstract: The present disclosure provides methods of treating a subject having decreased bone mineral density or at risk of developing decreased bone mineral density, and methods of identifying subjects having an increased risk of developing decreased bone mineral density.
    Type: Grant
    Filed: July 29, 2022
    Date of Patent: February 6, 2024
    Assignees: Regeneron Pharmaceuticals, Inc., The Royal Institution For the Advancement of Learning/McGill University
    Inventors: Jonas Bovijn, Olukayode Sosina, Sirui Zhou, Luca Andrea Lotta, Aris Baras, John Brent Richards