Abstract: The present disclosure provides methods of treating a human having a disease or disorder that would benefit from increasing platelet counts. The method involves inhibiting the enzyme activity of biliverdin IX? reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.
Type:
Grant
Filed:
October 5, 2016
Date of Patent:
January 28, 2020
Assignee:
The Research Foundation for The State University of New York
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 of 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: Treatment of tumors, especially breast cancer or glioblastoma tumors, by silencing RAB27A and/or TRAF3IP2, compositions and methods for same.
Type:
Grant
Filed:
November 26, 2018
Date of Patent:
January 21, 2020
Assignee:
The Administrators of the Tulane Educational Fund
Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Provided herein are a double-stranded oligonucleotides of 13 to 22 nucleobases in length targeting a GGGGCC expanded repeat region in an intron of C9orf72, comprises (a) 3-5 central mismatches (within bases 9-14) within a target sequence comprising the expanded repeat sequence, or (h) 3-5 mismatches outside of the seed sequence (bases 2-8 within the guide strand complementary to the expanded repeat sequence).
Type:
Grant
Filed:
October 8, 2015
Date of Patent:
January 21, 2020
Assignee:
The Board of Regents of the University of Texas System
Abstract: The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.
Type:
Grant
Filed:
March 24, 2017
Date of Patent:
January 14, 2020
Assignee:
BioMarin Technologies B.V.
Inventors:
Judith Christina Theodora Van Deutekom, Josephus Johannes De Kimpe, Gerard Johannes Platenburg
Abstract: The present invention provides an oligodeoxy nucleotide for preparing drugs for inhibiting tumor growth and an application thereof. The core sequence of the oligodeoxy nucleotide is TTYSGGAAWT, wherein Y is C or T; S is C or G; and W is A or T. The oligodeoxy nucleotide above further comprises an antisense strand and a modified type thereof. The oligodeoxy nucleotide according to the present invention plays a role of inhibiting the tumor growth in vitro and vivo, and is expected to be used for preparing drugs for inhibiting tumor growth, with high specificity and high inhibition ratio.
Type:
Grant
Filed:
June 25, 2015
Date of Patent:
January 7, 2020
Assignees:
JIANGSU KEYGEN BIOTECH CORP., LTD, NANJING NEW INDUSTRY INVESTMENT GROUP CO., LTD
Abstract: The present invention provides an antiviral agent comprising an RNA oligonucleotide having a particular sequence and structure. Specifically, when a cell line is treated with an RNA oligonucleotide having specific sequence and helical bend structure according to the present invention, the expression of interferon-? or ISG56 is increased and anti-viral activating is exhibited. Thus, a composition comprising the RNA oligonucleotide can be used as an antiviral agent.
Type:
Grant
Filed:
August 29, 2016
Date of Patent:
December 31, 2019
Assignee:
KOREA ADVANCED INSTITUTE OF SCIENCE AND TECHNOLOGY
Abstract: The present invention relates to plasmid-based miR inhibitory systems (PMIS), miR inhibitors and methods of use of these systems and inhibitors.
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:
May 20, 2016
Date of Patent:
December 31, 2019
Assignee:
CureVac AG
Inventors:
Fabian Johannes Eber, Benyamin Yazdan Panah, Stefanie Sewig, Thomas Ketterer, Thorsten Mutzke, Tilmann Roos, Michael Sonntag, Michael Wiggenhorn, Katharina Kolland
Abstract: The present disclosure concerns aptamers of formula (I) capable of specifically binding to Factor XIa. The aptamers can be used to prevent, treat or alleviate the symptoms of thrombosis. The aptamers can also be used to detect Factor XIa in a sample and/or purify Factor XIa from a sample. The aptamers can further be used to identity putative therapeutic agents for the prevention, treatment or alleviation of symptoms associated with thrombosis.
Type:
Grant
Filed:
May 4, 2017
Date of Patent:
December 3, 2019
Assignee:
CANADIAN BLOOD SERVICES
Inventors:
William Peter Sheffield, David Apraku Donkor
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 31, 2018
Date of Patent:
November 26, 2019
Assignee:
Sierra Sciences, LLC
Inventors:
William H. Andrews, Lancer K. Brown, Hamid Mohammadpour, Laura A. Briggs
Abstract: The present invention relates to the combined use of antisense oligonucleotides and viral vectors for the treatment of Duchenne muscular dystrophy.
Type:
Grant
Filed:
June 10, 2016
Date of Patent:
November 12, 2019
Assignees:
ASSOCIATION INSTITUT DE MYOLOGIE, SORBONNE UNIVERSITE, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
Inventors:
Stephanie Lorain, Thomas Voit, Matthew Wood, Graham McClorey
Abstract: The present invention relates to methods for treating a tumor, including a metastatic tumor, with TLR9 agonist in combination with an immune checkpoint inhibitor therapy.
Type:
Grant
Filed:
September 15, 2017
Date of Patent:
November 5, 2019
Assignee:
IDERA PHARMACEUTICALS, INC.
Inventors:
Sudhir Agrawal, Daqing Wang, Wayne Jiang
Abstract: The inventions relate to compositions and articles of manufacture comprising connexin modulators, pannexin modulators, gap junction modulators, hemichannel modulators, and pannexin channel modulators and their use, alone or in combination, in treating ocular and other disorders.
Abstract: Compositions and methods for treating cancer in a subject in need thereof are described that includes administering a therapeutically effective amount of an oligonucleotide that inhibits the binding of splicing regulator SRSF1 or SRSF2 to MDM2 exon 4 or 11.
Type:
Grant
Filed:
May 12, 2017
Date of Patent:
November 5, 2019
Assignee:
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Inventors:
Dawn Suzan Chandler, Daniel Forrest Comiskey, Jr.
Abstract: Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.
Type:
Grant
Filed:
April 10, 2018
Date of Patent:
October 29, 2019
Assignee:
Royal Holloway, University of London
Inventors:
Linda Popplewell, Ian Graham, John George Dickson
Abstract: Provided is an immunostimulating oligonucleotide that is suitable for industrial production and has an excellent type-I IFN inducing activity even when not modified to become a phosphorothioate. This linear double-stranded oligonucleotide contains 10-100 base pairs, wherein the single-stranded oligonucleotides constituting the double-stranded oligonucleotide each contain 2-20 phosphodiester-mediated cytosine-guanine (CpG) sequences, and at least 90% of the internucleotide bonds in each single-stranded oligonucleotide are phosphodiester bonds.
Type:
Grant
Filed:
July 8, 2016
Date of Patent:
October 22, 2019
Assignees:
NATIONAL INSTITUTE FOR MATERIALS SCIENCE, DENKA COMPANY LIMITED
Abstract: Described herein is a previously unknown function of XBP1 in controlling anti-tumor immunity. It is shown that inhibiting XBP1 in tumor-associated dendritic cells inhibits tumor growth and induces protective anti-tumor immune responses.
Type:
Grant
Filed:
March 14, 2018
Date of Patent:
October 22, 2019
Assignee:
Cornell University
Inventors:
Juan R. Cubillos-Ruiz, Laurie H. Glimcher
Abstract: The present invention lies in the field of molecules known as “small interfering RNA” with therapeutic applications. siRNAs have the ability to reduce gene expression in an extremely specific way (1). These are small sequences of double-strand RNA, normally used in laboratory to modify cell function, which revolutionized cell biology by allowing previously precluded molecular manipulations.
Type:
Grant
Filed:
May 21, 2015
Date of Patent:
October 22, 2019
Assignee:
Università degli Studi dell'Aquila
Inventors:
Anna Maria Teti, Nadia Rucci, Mattia Capulli, Antonio Maurizi