Abstract: The present invention relates to a compound inhibiting the expression and/or the activity of a long non-coding RNA (lncRNA) selected from GADLOR 1 and GADLOR 2 for use in treating or preventing cardiac remodelling, wherein GADLOR 1 comprises or consists of a nucleic acid sequence selected from the group consisting of SEQ ID NOs 1 to 3 and sequences being at least 75% identical thereto, and GADLOR 2 comprises or consists of a nucleic acid sequence selected from the group consisting of SEQ ID NOs 4 to 6 and sequences being at least 75% identical thereto.
Abstract: Immunomodulating polynucleotides are disclosed. The immunomodulating polynucleotides may contain 5-modified uridine, 5-modified cytidine, a total of from 6 to 16 nucleotides, and/or one or more abasic spacers and/or internucleoside phosphotriesters. Also disclosed are conjugates containing a targeting moiety and one or more immunomodulating polynucleotides. The immunomodulating polynucleotides and conjugates may further contain one or more auxiliary moieties. Also disclosed are compositions containing the immunomodulating polynucleotides or the conjugates containing one or more stereochemically enriched internucleoside phosphorothioates. Further disclosed are pharmaceutical compositions containing the immunomodulating polynucleotides or the conjugates and methods of their use.
Type:
Grant
Filed:
April 13, 2018
Date of Patent:
December 21, 2021
Assignee:
TOLLNINE, INC.
Inventors:
Sukumar Sakamuri, Curt W. Bradshaw, Son Lam, Joseph Stock, Edward Hyungsuk Ha, Laxman Eltepu, Dingguo Liu, Bin Liu, Giuseppe Dello Iacono, Bryan R. Meade, Ayman Kabakibi
Abstract: The present invention relates to the use of an inhibitor of Rad18 expression or activity, in treating a tumor or in sensitizing a patient affected with a tumor, to a treatment with an antineoplastic agent that is a DNA damaging chemotherapeutic agent so to both reduce the self renewal of cancer stem cells and increase the DNA damage response thus boosting apoptotic cell death.
Type:
Grant
Filed:
March 8, 2019
Date of Patent:
December 21, 2021
Assignee:
Centre National de la Recherche Scientifique
Abstract: Disclosed herein are antisense oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA at the 5?-end of UBE3A-AS, which is downstream of SNORD115-45 snoRNA. Also disclosed are pharmaceutical compositions and methods for treatment of Angelman syndrome.
Abstract: The present invention relates to a compound inhibiting the expression and/or the activity of maternally expressed 3 (Meg3) for use in treating or preventing cardiac remodelling.
Type:
Grant
Filed:
April 26, 2017
Date of Patent:
November 30, 2021
Assignee:
MEDIZINISCHE HOCHSCHULE HANNOVER
Inventors:
Thomas Thum, Maria-Teresa Piccoli, Janika Viereck, Shashi Kumar Gupta
Abstract: Disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds. Also disclosed herein are methods for reducing expression of Tau mRNA and protein in an animal with Tau antisense compounds. Such compounds and methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration Tau antisense oligonucleotides include Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy, Chronic Traumatic Encephalopathy, Epilepsy, and Dravet's Syndrome.
Abstract: The present invention refers to human microRNA hsa-miR-665 for the treatment or prevention of heart diseases associated with cardiac hypertrophy and consequent pathological remodeling of the heart, in particular for preventing and/or treating heart failure (HF). Vectors and pharmaceutical compositions comprising said miRNA for the disclosed uses are also within the scope of the present invention.
Type:
Grant
Filed:
March 13, 2018
Date of Patent:
October 26, 2021
Assignee:
KING'S COLLEGE LONDON
Inventors:
Mauro Giacca, Luca Braga, Matteo Dal Ferro, Miguel Luis Cunha Mano, Ana Sofia Bregieiro Eulalio
Abstract: This invention relates to the field of ribonucleic acid (RNA) regulation of intracellular activity. In particular, the invention relates to compositions and methods of identifying and tracking specific intracellular RNAs. For example, a fluorescently tagged RNA probe may be tracked by in vivo live imaging throughout its intracellular lifetime in order to determine its purpose and identify regulatory targets to modify its effects. Alternatively, an RNA probe may carry a therapeutic payload for treatment of medical condition or disorder.
Type:
Grant
Filed:
July 30, 2019
Date of Patent:
October 26, 2021
Assignees:
The Regents of the University of Colorado, a body corporate, Institute of Organic Chemistry POS
Inventors:
Amy E. Palmer, Esther Braselmann, Robert T. Batey, Dorota Gryko
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:
March 9, 2017
Date of Patent:
October 5, 2021
Assignee:
Ionis Pharmaceuticals, Inc.
Inventors:
Gene Hung, Holly Kordasiewicz, Hien Thuy Zhao, Eric E. Swayze
Abstract: Disclosed herein are compounds and methods for decreasing MECP2 mRNA and protein expression. Such compounds and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders and syndromes. Such MECP2 associated disorders include MECP2 duplication syndrome.
Type:
Grant
Filed:
August 8, 2019
Date of Patent:
September 28, 2021
Assignees:
Ionis Pharmaceuticals, Inc., Baylor College of Medicine
Inventors:
Susan M. Freier, Huda Y. Zoghbi, Ezequiel Sztainberg
Abstract: Methods of using B2 or Alu nucleic acids, or antisense oligonucleotides that modulate the EZH2/B2 or EZH2/Alu interaction and have the capacity to alter cleavage of B2 and Alu RNA, for increasing or decreasing cell and organismal viability.
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:
January 26, 2021
Date of Patent:
September 28, 2021
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Brian Bettencourt, William Querbes, Kevin Fitzgerald, Maria Frank-Kamenetsky, Stuart Milstein, Svetlana Shulga Morskaya
Abstract: The invention relates to the fields of medicine and immunology. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Usher Syndrome type II and/or USH2A-associated non syndromic retina degeneration, especially by skipping a pseudo exon (PE40) between exon 40 and 41 in the human USH2Agene.
Type:
Grant
Filed:
March 6, 2020
Date of Patent:
September 21, 2021
Assignee:
ProQR Therapeutics II B.V.
Inventors:
Hester Catharina Van Diepen, Hee Lam Chan, Janne Juha Turunen
Abstract: The present invention relates to an asymmetric siRNA which inhibits an expression of male pattern hair loss target genes and a use thereof and, more particularly, to an asymmetric siRNA which inhibits an expression of 3-oxo-5-alpha-steroid-4-dehydrogenase 1 (SRD5A1) gene, 3-oxo-5-alpha-steroid-4-dehydrogenase 2 (SRD5A2) gene or androgen receptor (AR) gene, and a composition for prevention or treatment of hair loss comprising the asymmetric siRNA.
Abstract: The present invention includes compositions and methods for treating an autoimmune disorder or a cancer in a subject in need thereof, the method comprising: administering an effective amount of a composition comprising an oligonucleotide that specifically binds a complementary sequence of the Interleukin-7 receptor (IL7R) pre-mRNA that influences splicing of exon 6, wherein the SM-ASO increases or decreases inclusion of exon 6 in IL7R pre-mRNAs and respectively decreases or increases expression of the soluble isoform of IL7R (sIL7R). In certain embodiments, the oligonucleotide is an antisense oligonucleotide (ASO), or a splice-modulating antisense oligonucleotide (SM-ASO).
Type:
Grant
Filed:
September 21, 2020
Date of Patent:
September 14, 2021
Assignee:
Board of Regents, The University of Texas System
Inventors:
Mariano A. Garcia-Blanco, Gaddiel Galarza-Munoz, Shelton S. Bradrick
Abstract: The present invention relates to a vaccine/agonist combination comprising an RNA vaccine comprising at least one RNA comprising at least one open reading frame (ORF) coding for at least one antigen and a composition comprising at least one OX40 agonist. The present invention furthermore relates to a pharmaceutical composition and a kit of parts comprising the components of such a vaccine/agonist combination. Additionally the present invention relates to medical use of such a vaccine/agonist combination, the pharmaceutical composition and the kit of parts comprising such a vaccine/agonist combination, particularly for the prevention or treatment of tumor or cancer diseases or infectious diseases. Furthermore, the present invention relates to the use of an RNA vaccine in therapy in combination with an OX40 agonist.
Type:
Grant
Filed:
April 15, 2019
Date of Patent:
September 7, 2021
Assignee:
CureVac AG
Inventors:
Mariola Fotin-Mleczek, Karl-Josef Kallen, Jan C. Schmollinger
Abstract: The invention relates to a nucleic acid molecule or a composition comprising said molecule for use as an anti-inflammatory and/or anti-coagulant and/or organ-protective medicament. The invention also relates to a use of said nucleic acid molecule or said composition, for the manufacture of a medicament for preventing, treating, regressing, curing and/or delaying a disease or a condition wherein inflammation and/or coagulation and/or organ damage or failure occurs in an individual. Finally, the invention further relates to a method for alleviating one or more symptom(s) and/or characteristic(s) and/or for improving a parameter of a disease or condition wherein inflammation and/or coagulation and/or organ damage or failure occur in an individual, the method comprising administering to said individual said nucleic acid molecule or said composition.
Abstract: An object of the present invention is to provide a convenient and low-cost method for enhancing the stability of a DNA aptamer and/or its capacity to bind to a target molecule, and a DNA aptamer obtained by the method. The object is solved by substituting an internal hairpin structure (stem-loop structure) of the DNA aptamer with a structure called mini-hairpin structure and optionally increasing GC pairs in a stem portion of the DNA aptamer.
Abstract: Methods and compositions for treating inflammatory disorders of the lower airways are disclosed herein. In one aspect, a method for treating an inflammatory disorder of lower airways in a human subject in need thereof is disclosed, including administering directly to the lower airways in the human subject an effective amount of anakinra, where the effective amount of anakinra is 0.01 mg to 100 mg per day.
Abstract: The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.