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
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
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
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
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
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
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.
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
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.
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
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.
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.
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.
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
Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
Type:
Grant
Filed:
November 22, 2021
Date of Patent:
January 9, 2024
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Brian Bettencourt, William Querbes, Kevin Fitzgerald, Maria Frank-Kamenetsky, Stuart Milstein, Svetlana Shulga Morskaya
Abstract: Compounds and methods for regulation of exonic splicing enhancers and exonic splicing silencers. Compounds include polynucleotides targeted to aberrant exonic splicing enhancers and exonic splicing silencers. Compounds and methods for the diagnosis of diseases and conditions associated with aberrant exonic splicing enhancers and exonic splicing silencers. Methods for identifying splicing-sensitive disease mutations, and functional RNA elements as targets for amelioration of aberrant pre-mRNA splicing.
Type:
Grant
Filed:
April 23, 2021
Date of Patent:
January 9, 2024
Assignee:
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
Abstract: An aptamer composition is disclosed which has one or more oligonucleotides that include at least one of deoxyribonucleotides, ribonucleotides, derivatives of deoxyribonucleotides, derivatives of ribonucleotides, or mixtures thereof. The aptamer composition has a binding affinity for one or more cellular membrane glycoproteins selected from the group consisting of: intercellular adhesion molecule 1 (ICAM-1), low-density lipoprotein receptor (LDLR) family members, and cadherin-related family member 3 (CDHR3), preferably intercellular adhesion molecule 1 (ICAM-1), and is configured to reduce the binding of one or more human rhinoviruses to the intercellular adhesion molecule 1 (ICAM-1).
Type:
Grant
Filed:
June 24, 2021
Date of Patent:
January 2, 2024
Assignee:
The Procter & Gamble Company
Inventors:
Juan Esteban Velasquez, Spencer Christopher Rupard, Amy Violet Trejo, Adam Michael Pitz, Kelly Lee Schmeichel, Erin Nicole Swigart, Gregory Allen Penner
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.
Abstract: Disclosed herein is a composition including a recombinant nucleic acid sequence that encodes an anti-influenza-hemagglutinin synthetic antibody. The disclosure also provides a method of preventing and/or treating influenza in a subject using said composition and method of generation.
Type:
Grant
Filed:
May 5, 2017
Date of Patent:
December 26, 2023
Assignees:
The Trustees of the University of Pennsylvania, The Wistar Institute of Anatomy and Biology, Inovio Pharmaceuticals, Inc.
Inventors:
David B. Weiner, Ami Patel, Jian Yan, Sarah Elliott
Abstract: BANF1, PPP2CA, and ANKLE2 were identified as genes that promote tau aggregation when disrupted. Improved tauopathy models such as cells, tissues, or animals having mutations in or inhibition of expression of BANF1 and/or PPP2CA and/or ANKLE2 are provided. Methods of using such improved tauopathy models for assessing therapeutic candidates for the treatment of a tauopathy, methods of making the improved tauopathy models, and methods of accelerating or exacerbating tau aggregation in a tauopathy model are also provided.
Type:
Grant
Filed:
June 12, 2020
Date of Patent:
December 19, 2023
Assignee:
Regeneron Pharmaceuticals, Inc.
Inventors:
Marine Prissette, Matthew Koss, Mathieu Desclaux, John McWhirter, Arijit Bhowmick, David Frendewey, Brian Zambrowicz, Claudia Racioppi