Patents Examined by Kimberly Chong
  • Patent number: 12275938
    Abstract: One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2?-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
    Type: Grant
    Filed: May 13, 2022
    Date of Patent: April 15, 2025
    Assignee: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Shigeo Matsuda, Mark K. Schlegel, Maja Janas, Vasant R. Jadhav, Martin Maier, Klaus Charisse, Muthiah Manoharan, Kallanthottathil Rajeev, Jayaprakash K. Nair
  • Patent number: 12270029
    Abstract: The disclosure provides, e.g., compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell's genome at one or more locations in a DNA sequence in a cell, tissue, or subject. Gene modifying systems for treating cystic fibrosis, e.g., in subjects having a mutation resulting in F508del, are described.
    Type: Grant
    Filed: September 20, 2023
    Date of Patent: April 8, 2025
    Assignee: Flagship Pioneering Innovations VI, LLC
    Inventors: Robert Charles Altshuler, Anne Helen Bothmer, Daniel Raymond Chee, Cecilia Giovanna Silvia Cotta-Ramusino, Kyusik Kim, Randi Michelle Kotlar, Gregory David McAllister, Aamir Mir, Ananya Ray, Nathaniel Roquet, Carlos Sanchez, Barrett Ethan Steinberg, Robert James Citorik, William Edward Salomon, William Querbes, Luciano Henrique Apponi, Zhan Wang
  • Patent number: 12263224
    Abstract: Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.
    Type: Grant
    Filed: November 17, 2022
    Date of Patent: April 1, 2025
    Assignee: AVIDITY BIOSCIENCES, INC.
    Inventors: Andrew John Geall, Venkata Doppalapudi, David Sai-Ho Chu, Michael Caramian Cochran, Michael Hood, Beatrice Diana Darimont, Rob Burke, Yunyu Shi, Gulin Erdogan Marelius, Barbora Malecova
  • Patent number: 12263232
    Abstract: Some embodiments of the present disclosure relate to one or more compositions that upregulate the production of one or more sequences of mRNA. The sequences of mRNA may encode for translation of a target biomolecule, thereby causing an increase in bioavailability of the target biomolecule within a subject who is administered the one or more compositions. The target biomolecule is a precursor protein such as a precursor protein of CLTA4.
    Type: Grant
    Filed: June 10, 2024
    Date of Patent: April 1, 2025
    Assignee: Wyvern Pharmaceuticals Inc.
    Inventors: Jill L. Thompson, Bradley G. Thompson
  • Patent number: 12263233
    Abstract: Among the various aspects herein is the provision of compositions and methods for modulating MYCT1. Methods are provided for regulating tumor angiogenesis (anti-angiogenesis, Myct1-targeted vascular control) and/or immunostimulation, which inhibit tumor growth, in a subject. Methods are provided to quantify MYCT1 to predict responsiveness of a subject having a cancer or tumor to a treatment, guide treatment decisions, select subjects for clinical trials, and evaluate the clinical efficacy of certain therapeutic interventions.
    Type: Grant
    Filed: October 19, 2021
    Date of Patent: April 1, 2025
    Assignee: Washington University
    Inventors: Kyunghee Choi, Ashraf Ul Kabir, Karen Krchma
  • Patent number: 12258565
    Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid, pemphigus, e.g., pemphigus vulgaris (PV) and pemphigus foliaceus (PF), and C3 glomerulopathy.
    Type: Grant
    Filed: March 4, 2024
    Date of Patent: March 25, 2025
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Mark Keating, James D. McIninch, Elane Fishilevich, Kristina Yucius, Sarah Solomon, Mark K. Schlegel, Adam Castoreno, Charalambos Kaittanis
  • Patent number: 12247203
    Abstract: One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2?-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
    Type: Grant
    Filed: May 16, 2019
    Date of Patent: March 11, 2025
    Inventors: Shigeo Matsuda, Mark K. Schlegel, Maja Janas, Vasant R. Jadhav, Martin Maier, Klaus Charisse, Muthiah Manoharan, Kallanthottathil Rajeev, Jayaprakash K. Nair
  • Patent number: 12239710
    Abstract: The present disclosure relates to compositions, such as siRNA molecules and FN3 domains conjugated to the same, as well as methods of making and using the molecules.
    Type: Grant
    Filed: April 14, 2022
    Date of Patent: March 4, 2025
    Assignee: ARO BIOTHERAPEUTICS COMPANY
    Inventors: Swapnil Kulkarni, Russell C. Addis, Steven G. Nadler, Yao Xin, Zhanna Druzina, Karyn T. O'Neil, Robert V. Kolakowski, Stephen J. Anderson
  • Patent number: 12234447
    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: March 17, 2022
    Date of Patent: February 25, 2025
    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: 12227748
    Abstract: A lipid-binding drug for binding to a membrane-based or membrane lipid comprises at least one aptamer having a sequence selected from the group consisting of GGCGGCAAAAAAAAAA (SEQ ID NO: 1), GGCGGCAAAAAAAAAAA (SEQ ID NO: 2), GGCGGCAAAAAAAAAAAA (SEQ ID NO: 3), GGCGGCAAAAAAAAAAAAA (SEQ ID NO: 4), GGCGGCGGGGGGGGGG (SEQ ID NO: 5), GGCGGCGGGGGGGGGGG (SEQ ID NO: 6), GGCGGCGGGGGGGGGGG (SEQ ID NO: 7), GGCGGCGGGGGGGGGGG (SEQ ID NO: 8), CAAAAGGAGCAAAAAAAAAAAAAAAAAAAAAAA (SEQ ID NO: 9) CAAAAGGAGCAAAAAAAAAAAAAAAAAAAAAAAA (SEQ ID NO: 10), CAAAAGGAGCAAAAAAAAAAAAAAAAAAAAAAAAA (SEQ ID NO: 11), CAAAAGGAGCAAAAAAAAAAAAAAAAAAAAAAAAAA (SEQ ID NO: 12), CAAAAGGAGCGGGGGGGGGGGGGGGGGGGGGGG (SEQ ID NO: 13), CAAAAGGAGCGGGGGGGGGGGGGGGGGGGGGGGG (SEQ ID NO: 14), CAAAAGGAGCGGGGGGGGGGGGGGGGGGGGGGGGG (SEQ ID NO: 15), and CAAAAGGAGCGGGGGGGGGGGGGGGGGGGGGGGGGG (SEQ ID NO: 16).
    Type: Grant
    Filed: April 25, 2024
    Date of Patent: February 18, 2025
    Assignee: KING SAUD UNIVERSITY
    Inventor: Md. Ashrafuzzaman
  • Patent number: 12221607
    Abstract: The present invention relates, in general to, compounds, compositions and methods useful for modulating gene expression of multiple target nucleic acids by a single chemical entity.
    Type: Grant
    Filed: February 22, 2021
    Date of Patent: February 11, 2025
    Assignee: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Vasant Jadhav, Martin Maier, Ivan Zlatev, Akin Akinc, Kallanthottathil G. Rajeev, Jayaprakash K. Nair, Pachamuthu Kandasamy, Muthiah Manoharan
  • Patent number: 12215323
    Abstract: Embodiments of the disclosure concern the use of expression constructs in which at least one polyA signal is embedded upstream of an expressible transcript, such as within a 5? UTR for the transcript, for example. In certain embodiments, the polyA signal is comprised within a ligand-binding aptamer, and the binding of the ligand to the aptamer, or lack thereof, dictates the outcome for the expressible transcript. In specific embodiments, absence of the ligand causes the expressed transcript having a polyA in its 5? UTR to be expressed but then degraded, whereas presence of the ligand causes inhibition of degradation upon expression of the expressible transcript. More than one ligand-binding aptamer may be present on the same expression construct.
    Type: Grant
    Filed: January 8, 2021
    Date of Patent: February 4, 2025
    Assignee: Baylor College of Medicine
    Inventors: Laising Yen, Liming Luo, Pei-Wen Chao
  • Patent number: 12209240
    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: October 12, 2021
    Date of Patent: January 28, 2025
    Assignees: Board of Regents, The University of Texas System, Oregon State University
    Inventors: David Greenberg, Bruce L. Geller
  • Patent number: 12208165
    Abstract: The present invention is directed to a storage-stable formulation of long-chain RNA. In particular, the invention concerns a dry powder composition comprising a long-chain RNA molecule. The present invention is furthermore directed to methods for preparing a dry powder composition comprising a long-chain RNA molecule by spray-drying. The invention further concerns the use of such a dry powder composition comprising a long-chain RNA molecule in the preparation of pharmaceutical compositions and vaccines, to a method of treating or preventing a disorder or a disease, to first and second medical uses of such a dry powder composition comprising a long-chain RNA molecule and to kits, particularly to kits of parts, comprising such a dry powder composition comprising a long-chain RNA molecule.
    Type: Grant
    Filed: July 11, 2022
    Date of Patent: January 28, 2025
    Assignee: CureVac SE
    Inventors: Fabian Johannes Eber, Benyamin Yazdan Panah, Stefanie Sewing, Thomas Ketterer, Thorsten Mutzke, Tilmann Roos, Michael Sonntag, Michael Wiggenhorn, Katharina Kolland
  • Patent number: 12203069
    Abstract: The present disclosure is related to an engineered nucleic acid encoding a post-poly A signal RNA 3? to a terminator for expression of protein, and/or non-coding RNA. Also provided herein are methods for reducing epigenetic silencing, genetic modification, transcriptional regulation of the engineered nucleic acid described herein.
    Type: Grant
    Filed: May 15, 2020
    Date of Patent: January 21, 2025
    Assignee: Massachusetts Institute of Technology
    Inventors: Ron Weiss, James J. Collins, Elvira Vitu, Casper Enghuus, Jeremy Jonathan Gam
  • Patent number: 12203073
    Abstract: A nanoparticle includes an icosahedral-shaped plant virus particle; an RNAi construct; and one or more endolysosomal release agents, wherein the RNAi construct is noncovalently loaded within the icosahedral-shaped plant virus particle.
    Type: Grant
    Filed: December 21, 2020
    Date of Patent: January 21, 2025
    Assignee: CASE WESTERN RESERVE UNIVERSITY
    Inventor: Nicole F. Steinmetz
  • Patent number: 12201644
    Abstract: Provided herein are compounds and methods for gene silencing. The compound includes a RNA directly conjugated to an albumin-binding group. The method includes administering a compound comprising a RNA directly conjugated to an albumin-binding group to a subject in need thereof.
    Type: Grant
    Filed: September 14, 2021
    Date of Patent: January 21, 2025
    Assignee: Vanderbilt University
    Inventors: Craig L. Duvall, Samantha M. Sarett, Thomas A. Werfel
  • Patent number: 12203068
    Abstract: In an embodiment, a strand displacement system including a DNA/PNA complex, an input DNA strand, where the DNA/PNA complex binds to the input DNA strand forming a complex and displaces a PNA intermediate and forms an activated domain on the PNA, and a Bi complex that reacts with the activated domain on the PNA to thereby release an output DNA strand. In an additional embodiment, a fluorogenic sensor including a heteroduplex between an achiral PNA strand and a fluorogenic aptamer, where the fluorogenic aptamer includes L-RNA, an input DNA strand, where the input DNA strand binds to the heteroduplex and displaces an incumbent fluorogenic aptamer strand and forms an activated domain on that strand, and a dye.
    Type: Grant
    Filed: February 4, 2019
    Date of Patent: January 21, 2025
    Assignee: The Texas A&M University System
    Inventors: Jonathan T. Sczepanski, Adam M. Kabza, Brian E. Young
  • Patent number: 12203071
    Abstract: The present invention relates to oligonucleotides which up-regulate or restore the expression of progranulin in cells, and their use in the treatment of neurological disorders, and disorders associated with progranulin haploinsufficiency.
    Type: Grant
    Filed: May 13, 2021
    Date of Patent: January 21, 2025
    Assignee: Hoffmann-La Roche Inc.
    Inventors: Ravi Jagasia, Lars Joenson, Søren Rasmussen, Disa Tehler, Dorthe Vang Larsen, Jesper Worm
  • Patent number: 12194148
    Abstract: The present invention is directed to the field of RNA formulation, in particular to lyophilization of RNA. The invention provides a method for lyophilization of RNA. The present invention further concerns a lyophilized composition obtainable by the inventive method, a pharmaceutical composition, a vaccine and a kit or kit of parts. Moreover, the present invention provides a novel use of a lyoprotectant for lyophilizing RNA, the use of the inventive method in the manufacture of a medicament as well as the first and second medical use of the composition obtainable by the inventive method, the pharmaceutical composition, the vaccine or the kit or kit of parts according to the invention.
    Type: Grant
    Filed: August 26, 2022
    Date of Patent: January 14, 2025
    Assignee: CureVac Manufacturing GmbH
    Inventors: Thomas Ketterer, Thorsten Mutzke, Michael Wiggenhorn, Frank Schaubhut, Florian Von Der Mülbe