Patents Examined by Kimberly Chong
  • Patent number: 11987795
    Abstract: Described are methods for enhancing exon skipping in a pre-mRNA of interest, comprising contacting the pre-mRNA with interfering oligonucleotides such as antisense, siRNA, and miRNA. The exon skipping methods are associated with methods of treating a variety of diseases and conditions, including cancer.
    Type: Grant
    Filed: September 22, 2021
    Date of Patent: May 21, 2024
    Assignees: The Broad Institute, Inc., The General Hospital Corporation
    Inventors: Vamsi Krishna Mootha, Alexis André Jourdain
  • Patent number: 11976279
    Abstract: This disclosure relates to the use of miRNA-483 and its target genes, UBE2C, pVHL and HIF1alpha, in managing the treatment of cardiovascular and inflammatory diseases. In certain embodiments, this disclosure relates to pharmaceutical compositions comprising a miR-483 mimic and/or an HIF inhibitor and a pharmaceutically acceptable excipient for use in treating or preventing a vascular disease or condition. In certain embodiments, the miR-483 mimic is a double stranded nucleobase polymer or an expression vector that expresses mature human miR-483-5p and miR-483-3p sequences or operable fragments and variants.
    Type: Grant
    Filed: December 7, 2021
    Date of Patent: May 7, 2024
    Assignee: Emory University
    Inventors: Hanjoong Jo, Joan Fernandez Esmerats, Nicolas Villa-Roel
  • Patent number: 11970720
    Abstract: Provided herein are CRISPR/Cas methods and compositions for targeting RNA molecules, which can be used to detect, edit, or modify a target RNA.
    Type: Grant
    Filed: February 21, 2020
    Date of Patent: April 30, 2024
    Assignee: Salk Institute for Biological Studies
    Inventors: Patrick D. Hsu, Silvana Konermann
  • Patent number: 11970785
    Abstract: This invention relates to methods and materials for multiplexed utilization of collections of functional ligands, particularly to methods and materials for selecting for and/or utilizing particular desirable traits of functional ligands in a multiplexed manner, and more particularly to methods and materials for selecting for and/or utilizing particular structural changes of functional ligands in a multiplexed manner.
    Type: Grant
    Filed: August 30, 2021
    Date of Patent: April 30, 2024
    Assignee: BASE PAIR BIOTECHNOLOGIES, INC.
    Inventors: George W. Jackson, Robert Batchelor, Alexander S. Chiu, Rafal Drabek, Deepak Thirunavukarasu, Caitlin Bruns
  • Patent number: 11965204
    Abstract: The subject invention pertains to composition and methods of using said composition as an in vitro biosensor of small molecules in biological and/or environmental samples using enzyme-assisted nucleic acid reactions. The methods and compositions can be used to sense and/or transduce the signal of a sensing event mediated by allosteric proteins, endonucleases and nucleic acid reactions. This invention allows the rapid development and setup of one-pot assays to provide results in minutes. The methods and compositions may be used to generate an electrochemical, fluorescent, colorimetric, and/or luminescent output and the methods can be performed in different modalities, including a solution-based or paper-based assay.
    Type: Grant
    Filed: July 29, 2021
    Date of Patent: April 23, 2024
    Assignee: The Hong Kong University of Science and Technology
    Inventors: I-Ming Hsing, Alan Fernando Rodriguez Serrano
  • Patent number: 11965163
    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 28, 2021
    Date of Patent: April 23, 2024
    Assignee: MiNA Therapeutics Limited
    Inventors: Hans E. Huber, David Blakey, Jon Voutila, Monika Krampert, Markus Hossbach
  • Patent number: 11965166
    Abstract: The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the complement factor B (CFB) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a CFB gene and to methods of treating or preventing a CFB-associated disease in a subject.
    Type: Grant
    Filed: March 22, 2023
    Date of Patent: April 23, 2024
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: James D. McIninch, Mark K. Schlegel, Adam Castoreno, Elane Fishilevich, Kristina Yucius
  • Patent number: 11959081
    Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the transthyretin (TTR) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an TTR gene and to methods of preventing and treating an TTR-associated disorder, e.g., senile systemic amyloidosis (SSA), systemic familial amyloidosis, familial amyloidotic polyneuropathy (FAP), familial amyloidotic cardiomyopathy (FAC), leptomeningeal/Central Nervous System (CNS) amyloidosis, and hyperthyroxinemia.
    Type: Grant
    Filed: September 28, 2022
    Date of Patent: April 16, 2024
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Mark K. Schlegel, Adam Castoreno, James D. McIninch
  • Patent number: 11951184
    Abstract: An expression cassette for gene silencing and replacement, including, in operable communication, a promoter, an expression attenuator, a nucleotide sequence encoding a gene for a replacement target protein, and an shRNA sequence for knockdown of an endogenous variant of the target protein, wherein the promoter, the expression attenuator, the nucleotide sequence encoding the gene for the replacement target protein, and the shRNA are expressed as a single transcript. Also included are expression vectors and cells. Also included are methods of silencing and replacement of a target gene in a cell in culture by transforming the cells with the expression vectors described herein. Also included are minimal expression cassettes suitable for therapeutic methods.
    Type: Grant
    Filed: June 14, 2021
    Date of Patent: April 9, 2024
    Assignee: Wisconsin Alumni Research Foundation
    Inventors: Matthew Michael Millette, Erik Wolfe Dent
  • Patent number: 11946050
    Abstract: This disclosure concerns an engineered polynucleotide that interacts with a pre-mRNA and a spliceosome to regulate gene expression. The engineered polynucleotide may have stem-loop structure that recruits the spliceosome and targeting sequences that are complementary to a target sequence at an exon-intron splice junction and may include nucleotides with 2? modifications and phorphorothioate linkages.
    Type: Grant
    Filed: July 18, 2022
    Date of Patent: April 2, 2024
    Assignee: APTAH BIO, INC.
    Inventor: Caio Bruno Quinta De Souza Leal
  • Patent number: 11944636
    Abstract: This invention discloses a medicinal composition includes a non-coding RNA molecule and an antibody targeting a tumor antigen for preventing and/or treating cancer. This invention uses the synergistic combination of a non-coding RNA molecule or its functional variant or homologue, and an antibody targeting a tumor antigen to prevent and/or treat cancer, thereby providing a novel and effective method in preventing and/or treating various cancers.
    Type: Grant
    Filed: November 11, 2020
    Date of Patent: April 2, 2024
    Assignee: Macau University of Science and Technology
    Inventors: Zhi-Hong Jiang, Lee-Fong Yau, Tian-Tian Tong, Hao Huang, Kua Hu, Elaine Lai-Han Leung
  • Patent number: 11939578
    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 14, 2021
    Date of Patent: March 26, 2024
    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: 11939580
    Abstract: A self-circularization RNA construct that can be expressed in a DNA vector and simultaneously circularized through a self-targeting and splicing reaction to form a circRNA is disclosed. The circRNA can consist only of a gene of interest which can be a coding, non-coding, or a combination thereof. The gene of interest has the advantage of being able to rapidly express a peptide or protein. The formed circRNA has a circular structure and has a stable and high half-life because 5? and 3? ends are not exposed. Accordingly, functional RNA such as miRNA, anti-miRNA, siRNA, shRNA, aptamer, functional RNA for gene/RNA editing, ADAR (adenosine deaminase acting on the RNA)-recruiting RNA, mRNA vaccine, mRNA therapeutic agent, vaccine adjuvant, and CAR-T mRNA can be produced as a stable circRNA in cells.
    Type: Grant
    Filed: October 12, 2022
    Date of Patent: March 26, 2024
    Assignee: Rznomics Inc.
    Inventors: Seong-Wook Lee, Kyung Hyun Lee, Seung Ryul Han, Ji Hyun Kim, Seongcheol Kim
  • Patent number: 11912995
    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: October 25, 2021
    Date of Patent: February 27, 2024
    Assignee: City of Hope
    Inventors: Hua Yu, Andreas Herrmann, Marcin Tomasz Kortylewski, Piotr Marek Swiderski
  • Patent number: 11903960
    Abstract: Described herein are methods for treating fibrosis, e.g., kidney fibrosis, using agents that target Secreted Modular Calcium-binding protein 2 (SMOC2).
    Type: Grant
    Filed: December 23, 2021
    Date of Patent: February 20, 2024
    Assignee: The Brigham and Women's Hospital, Inc.
    Inventors: Vishal S. Vaidya, Casimiro Gerarduzzi
  • 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: 11883471
    Abstract: Methods of treating an age-related disorder in a subject are provided. Aspects of the methods include administering to the subject a nucleic acid vector including a coding sequence for telomerase reverse transcriptase (TERT) and/or telomerase RNA (TR). Gene therapy methods are also provided. Aspects of the invention further include compositions, e.g., nucleic acid vectors and kits, etc., that find use in methods of the invention.
    Type: Grant
    Filed: May 26, 2021
    Date of Patent: January 30, 2024
    Assignee: Sierra Sciences, LLC
    Inventors: William H. Andrews, Lancer K. Brown, Hamid Mohammadpour, Laura A. Briggs
  • Patent number: 11872287
    Abstract: Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.
    Type: Grant
    Filed: September 27, 2022
    Date of Patent: January 16, 2024
    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: 11866689
    Abstract: The present disclosure provides methods and compositions for the treatment of cancer. In some aspects, the present disclosure provides splice-switching oligonucleotides that downregulate AR expression and methods of using these splice-switching oligonucleotides to treat cancer, specifically castrate resistant prostate cancer.
    Type: Grant
    Filed: March 22, 2019
    Date of Patent: January 9, 2024
    Assignee: Duke University
    Inventors: Jennifer Freedman, Steven Patierno, Daniel George, Bruce Sullenger, Bonnie LaCroix, Brendon Patierno
  • Patent number: 11866710
    Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS6 gene and to methods of preventing and treating a TMPRSS6-associated disorder, e.g., a disorder associated with iron overload and/or a disorder of ineffective erythropoiesis, e.g., hereditary hemochromatosis, ?-thalassemia (e.g., ?-thalassemia major and ?-thalassemia intermedia), polycythemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, Parkinson's Disease, Alzheimer's Disease or Friedreich's Ataxia.
    Type: Grant
    Filed: January 6, 2023
    Date of Patent: January 9, 2024
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Aimee M. Deaton, John Michael Gansner, James D. McIninch, Mark K. Schlegel, Benjamin P. Garfinkel