Abstract: The present disclosure provides methods of treating a subject having metabolic disorders and/or cardiovascular diseases, methods of identifying subjects having an increased risk of developing a metabolic disorder and/or a cardiovascular disease, and methods of detecting human Inhibin Subunit Beta E variant nucleic acid molecules and variant polypeptides.
Type:
Grant
Filed:
April 1, 2022
Date of Patent:
September 19, 2023
Assignee:
Regeneron Pharmaceuticals, Inc.
Inventors:
Luca Andrea Lotta, Parsa Akbari, Olukayode Sosina, Manuel Allen Revez Ferreira, Aris Baras
Abstract: The invention provides compositions and methods for high-efficiency genome editing. In some aspects, the invention provides retron-guide RNA cassettes and vectors comprising the cassettes. Also provided are host cells that have been transformed with the vectors. In other aspects, the invention provides retron donor DNA-guide molecules. In some other aspects, methods for genome editing and the screening of genetic loci are provided. In further aspects, methods and compositions are provided for the prevention or treatment of genetic diseases. Kits for genome editing and screening are also provided.
Type:
Grant
Filed:
March 5, 2019
Date of Patent:
September 19, 2023
Assignee:
The Board of Trustees of the Leland Stanford Junior University
Abstract: The invention relates to nucleic acid products that interfere with complement component C3 gene expression or inhibit its expression. The nucleic acids are preferably for use as treatment, prevention or reduction of risk of suffering from complement component C3 associated diseases, disorders or syndromes, particularly C3 Glomerulopathy (C3G), Paroxysmal Nocturnal Hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS), Lupus nephritis, IgA nephropathy (IgA N), Cold Agglutinin Disease (CAD), Myasthenia gravis (MG), and Primary Membranous Nephropathy.
Type:
Grant
Filed:
May 26, 2022
Date of Patent:
September 12, 2023
Assignee:
SILENCE THERAPEUTICS GMBH
Inventors:
Verena Aumiller, Lucas Bethge, Judith Hauptmann, Marie Wikström Lindholm, Adrien Weingärtner
Abstract: The invention includes compositions and methods useful for the diagnosis and treatment of head or brain injury in a subject in need thereof, based upon the expression level of at least one miRNA that is associated with head or brain injury.
Type:
Grant
Filed:
June 7, 2018
Date of Patent:
August 29, 2023
Assignee:
Temple University-Of The Commonwealth System of Higher Education
Inventors:
Matthew B. Hudson, Dianne Langford, John Jeka, Ryan Tierney
Abstract: Oligonucleotide analogues conjugated to carrier peptides are provided. The disclosed compounds are useful for the treatment of various diseases, for example diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
Abstract: The present disclosure provides methods of treating patients having decreased bone mineral density, methods of identifying subjects having increased risk of developing decreased bone mineral density, methods of detecting human Zinc And Ring Finger 3 (ZNRF3) variant nucleic acid molecules and variant polypeptides, and ZNRF3 variant nucleic acid molecules and variant polypeptides.
Type:
Grant
Filed:
June 22, 2020
Date of Patent:
August 1, 2023
Assignees:
Regeneran Pharmaceuticals, Inc., University of Maryland, Baltimore
Inventors:
Da-Wei Gong, Nehal Gosalia, Alan Shuldiner, Cristopher Van Hout, James Perry
Abstract: The present invention relates to a medicine for treating disorders of glucose and/or lipid metabolism. This invention demonstrates a new therapeutic target for treatment of glucose and/or lipid metabolic disorders. The invention further provides a method of treating glucose and/or lipid metabolic disorders, administering to a subject comprising a Nogo-B inhibitor, thereby treating the glucose and/or lipid metabolic disorders.
Abstract: A photocaged DNA nano-tweezer and methods of using said photocaged DNA nano-tweezer are described. In particular, provided herein is a DNA nano-tweezer comprising a hairpin with a single-stranded loop that comprises a first arm and a second arm; and a trigger strand complementary to the single-stranded loop and comprising at least one photocaged residue with a protecting group.
Type:
Grant
Filed:
October 15, 2019
Date of Patent:
July 25, 2023
Assignee:
ARIZONA BOARD OF REGENTS ON BEHALF OF ARIZONA STATE UNIVERSITY
Abstract: Disclosed herein are circular RNAs and transfer vehicles, along with related compositions and methods of treatment. The circular RNAs can comprise group I intron fragments, spacers, an IRES, duplex forming regions, and/or an expression sequence, thereby having the features of improved expression, functional stability, low immunogenicity, ease of manufacturing, and/or extended half-life compared to linear RNA. Pharmaceutical compositions comprising such circular RNAs and transfer vehicles are particularly suitable for efficient protein expression in immune cells in vivo. Also disclosed are precursor RNAs and materials useful in producing the precursor or circular RNAs, which have improved circularization efficiency and/or are compatible with effective circular RNA purification methods.
Type:
Grant
Filed:
March 15, 2021
Date of Patent:
June 20, 2023
Assignee:
Orna Therapeutics, Inc.
Inventors:
Allen T. Horhota, Brian Goodman, Robert Alexander Wesselhoeft, Junghoon Yang
Abstract: The present invention relates to RNAi constructs and their use in gene silencing. RNAi constructs associated with the invention contain a double stranded region connected to a single stranded region of phosphorothioate modified nucleotides.
Type:
Grant
Filed:
November 11, 2019
Date of Patent:
June 6, 2023
Assignee:
Phio Pharmaceuticals Corp.
Inventors:
Tod M. Woolf, Joanne Kamens, William Salomon, Anastasia Khvorova
Abstract: This invention relates to compounds, compositions, and methods useful for reducing AT3 target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Abstract: Methods of treating cancer are provided along with nucleic acids and nucleic acid analog sequences of a long-non-coding RNA (lncRNA), and reagents useful for knocking down the lncRNA.
Type:
Grant
Filed:
February 22, 2019
Date of Patent:
June 6, 2023
Assignee:
University of Pittsburgh—of the Commonwealth System of Higher Education
Abstract: In certain embodiments, the present invention provides a modified transfer RNA (tRNA) comprising a T-arm, a D-arm, and anticodon-arm and an acceptor arm, wherein the T-arm comprises nucleotides that interact with the elongation factor 1 alpha protein, and methods of use thereof.
Abstract: Oligonucleotides are provided herein that inhibit MARC1 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with MARC1 expression.
Type:
Grant
Filed:
May 27, 2022
Date of Patent:
May 23, 2023
Assignees:
Novo Nordisk A/S, Dicerna Pharmaceuticals, Inc.
Inventors:
Henryk Dudek, Wen Han, Natalie Wayne Pursell, Chengjung Lai, William Geoffrey Haynes, Zhihao Ding
Abstract: The present disclosure provides small hairpin nucleic acid molecules capable of stimulating interferon production. The nucleic acid molecules of the present disclosure has a double-stranded section of less than 19 base pairs and at least one blunt end. In certain embodiments, the molecule comprises at least one 5?-triphosphate and/or at least one 5?-diphosphate. In certain embodiments, compounds and/or compositions of the disclosure are useful for treating, ameliorating, and/or preventing SARS-CoV-2 viral infection, and/or ameliorating, minimizing, reversing, and/or preventing persistent SARS-CoV-2 viral infection, and/or minimizing or preventing SARS-CoV-2 viral infection-derived mortality and/or lethality, in a subject. In certain embodiments, compounds and/or compositions of the disclosure are useful for treating, ameliorating, and/or preventing SARS-CoV-2 viral infection in a tumor-bearing subject.
Type:
Grant
Filed:
March 30, 2022
Date of Patent:
May 16, 2023
Assignee:
Yale University
Inventors:
Anna Marie Pyle, Akiko Iwasaki, Tianyang Mao
Abstract: The disclosure provides a method of inhibiting proliferation of a cell, inhibiting m3C formation in a cell, inhibiting activity of Mettl8 in a cell, or activating ATM and p53 in a cell, the method comprising contacting the cell with a Mettl8 inhibitor. The disclosure also provides a composition comprising a cell with a reduced expression or activity of Mettl8. In another aspect, the disclosure provides methods of rendering a tumor cell sensitive to a cancer therapy.
Abstract: The purpose of the present invention is to provide novel complexes that improve the effect of nucleic acid medicines. Provided is a complex in which a multibranched lipid(s) binds through a linker to a strand of an oligonucleotide comprising a nucleic acid medicine having suppressing activity of the target gene expression.
Abstract: Provided herein are double strand DNA molecules comprising inverted repeats, expression cassette and one or more restriction sites for nicking endonucleases, the methods of use thereof, and the methods of making therefor.
Type:
Grant
Filed:
August 17, 2021
Date of Patent:
April 25, 2023
Assignee:
Anjarium Biosciences AG
Inventors:
Joel de Beer, Monique Maurer, Nicolas Meier, Lavaniya Kunalingam, Marcello Clerici