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:
October 10, 2018
Date of Patent:
January 18, 2022
Assignee:
Emory University
Inventors:
Hanjoong Jo, Joan Fernandez Esmerats, Nicolas Villa-Roel
Abstract: Provided are aptamers and aptamer compositions and particularly, although not exclusively, to a bi-specific aptamer capable of binding a tumor cell antigen and an immune cell surface protein.
Abstract: Disclosed herein are compositions and methods useful for the diagnosis, assessment, and characterization of endometriosis in a subject in need thereof, based upon the expression level of at least one miRNA that is associated with endometriosis.
Abstract: Provided herein are methods and kits for determining the presence or absence of certain microRNA biomarkers in a blood sample of a female patient. The microRNA biomarkers are associated with ovarian cancer. Also provided are methods for screening a female subject for the presence or absence of certain microRNA biomarkers, as well as methods for treating a female subject having an ovarian cancer.
Type:
Grant
Filed:
January 9, 2018
Date of Patent:
January 4, 2022
Assignees:
DANA-FARBER CANCER INSTITUTE, INC., THE BRIGHAM AND WOMEN'S HOSPITAL, INC., MEDICAL UNIVERSITY OF LODZ
Inventors:
Dipanjan Chowdhury, Kevin M. Elias, Wojciech Fendler, Konrad Stawiski
Abstract: The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure or an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.
Abstract: The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the coronavirus genome. The invention also relates to methods of using such RNAi agents to inhibit expression of a coronavirus genome and to methods of treating or preventing a coronavirus-associated disease in a subject.
Type:
Grant
Filed:
May 17, 2021
Date of Patent:
December 28, 2021
Assignees:
Alnylam Pharmaceuticals, Inc., Vir Biotechnology, Inc.
Inventors:
Akin Akinc, James D. McIninch, Yesseinia Anglero-Rodriguez, Mark K. Schlegel, Christy M. Hebner, Florian A. Lempp
Abstract: A method is provided for achieving transfection of host cells using sonoporation. An acoustic radiation generator is positioned in acoustic coupling relationship with respect to a reservoir containing host cells to be transfected, exogenous material to be incorporated into the host cells, and a cell-compatible fluid medium. The acoustic radiation generator is activated to generate acoustic radiation and direct the acoustic radiation into the reservoir in a manner effective to enable transfection of the host cells with the exogenous material.
Type:
Grant
Filed:
August 28, 2020
Date of Patent:
December 14, 2021
Assignee:
LABCYTE INC.
Inventors:
Jennifer M. Hardee, Richard N. Ellson, Richard G. Stearns, Babur Hadimioglu, Joseph D. Olechno, Marsha N. Blauwkamp
Abstract: Certain embodiments are directed to methods and compounds for modulating expression of SMN. In certain embodiments at least two compounds are used: a first compound for inhibiting SMN-NAT and increasing expression of SMN, and a second compound for modulating the splicing of SMN. Such methods and compounds are useful for increasing expression exon 7 containing SMN mRNA in cells and animals.
Type:
Grant
Filed:
June 16, 2017
Date of Patent:
December 14, 2021
Assignees:
Ionis Pharmaceuticals, Inc., The Johns Hopkins University
Inventors:
Frank Rigo, C. Frank Bennett, Constantin Van Outryve D'Ydewalle, Charlotte J. Sumner
Abstract: Provided herein are methods, compounds, and compositions for modulating expression of at least one member of the Notch signaling pathway in a cell or individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a respiratory disorder associated with excessive mucus production in an individual.
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:
June 4, 2020
Date of Patent:
November 30, 2021
Assignee:
CITY OF HOPE
Inventors:
Hua Yu, Andreas Herrmann, Marcin Tomasz Kortylewski
Abstract: The present invention provides compositions comprising a UBC antagonist and methods of use thereof for treating cancer in a patient. In some embodiments, the cancer patient may have a reduced expression level of a UBB gene product. Further provided are reagents and methods for detection of a UBB and/or UBC gene product.
Type:
Grant
Filed:
March 5, 2018
Date of Patent:
November 23, 2021
Assignee:
NOVARTIS AG
Inventors:
William C. Forrester, Alexia T. Kedves-Volpe
Abstract: Disclosed herein are molecules and pharmaceutical compositions that induce an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that induces an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion.
Type:
Grant
Filed:
September 12, 2018
Date of Patent:
November 23, 2021
Assignee:
AVIDITY BIOSCIENCES, INC.
Inventors:
Arthur A. Levin, Andrew John Geall, Venkata Ramana Doppalapudi, Michael Caramian Cochran, Hanhua Huang, Rob Burke
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:
November 11, 2019
Date of Patent:
November 23, 2021
Assignee:
CureVac AG
Inventors:
Fabian Johannes Eber, Benyamin Yazdan Panah, Stefanie Sewing, Thomas Ketterer, Thorsten Mutzke, Tilmann Roos, Michael Sonntag, Michael Wiggenhorn, Katharina Kolland
Abstract: Provided herein are compositions of placenta-derived adherent cell exosomes and methods of making and using the same. In one aspect, provided herein are compositions comprising exosomes produced by and/or derived from placental cells, e.g., placenta-derived adherent cells. In certain embodiments, the exosomes provided herein are produced by placenta-derived adherent cells that have been cultured in vitro for, e.g., 1, 2, 3, 4, 5, 6 or more passages.
Type:
Grant
Filed:
January 10, 2020
Date of Patent:
November 16, 2021
Assignee:
Celularity Inc.
Inventors:
Eric Law, Andrew Morschauser, Aleksander Francki, Jennifer Paredes, Kathy Karasiewicz-Mendez, Allan Reduta, Vladimir Jankovic, Ivana Djuretic, Robert J. Hariri
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 7, 2018
Date of Patent:
November 2, 2021
Assignee:
MINA THERAPEUTICS LIMITED
Inventors:
Hans E. Huber, David Blakey, Jon Voutila, Monika Krampert, Markus Hossbach
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.
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:
August 7, 2017
Date of Patent:
October 19, 2021
Assignee:
Vanderbilt University
Inventors:
Craig L. Duvall, Samantha M. Sarett, Thomas A. Werfel
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:
December 22, 2016
Date of Patent:
October 12, 2021
Assignees:
Board of Regents, The University of Texas System, Oregon State University
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:
July 20, 2018
Date of Patent:
October 12, 2021
Assignee:
THE GOVERNORS OF THE UNIVERSITY OF ALBERTA