Abstract: Disclosed are messenger RNA molecules and related compositions incorporating a 4?-thio modification in the furanose ring of at least one nucleotide residue, and methods of using these mRNAs to produce an encoded therapeutic protein in vivo and to treat or prevent diseases or disorders. In certain embodiments, the 4?-thio modified mRNA provides for enhanced stability and/or reduced immunogenicity in in vivo therapies.
Abstract: A synthetic transfer RNA with an extended anticodon loop. A synthetic suppressor transfer RNA useful for the treatment of a genetic disease like cystic fibrosis associated with a nonsense mutation. The synthetic transfer RNA contains an extended anticodon loop with two consecutive anticodon base triplets configured to base-pair to two consecutive codon base triplets on an mRNA. The first anticodon base triplet or the second anticodon base triplet is configured to base-pair to a stop codon base triplet on the mRNA.
Type:
Grant
Filed:
March 14, 2019
Date of Patent:
September 6, 2022
Assignee:
ARCTURUS THERAPEUTICS, INC.
Inventors:
Zoya Ignatova, Andrew Torda, Marco Matthies
Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with DMPK whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of DMPK gene in a cell by genome editing.
Type:
Grant
Filed:
June 27, 2017
Date of Patent:
August 30, 2022
Assignee:
Vertex Pharmaceuticals Incorporated
Inventors:
Ante Sven Lundberg, Samarth Kulkarni, Lawrence Klein, Hari Kumar Padmanabhan
Abstract: Provided herein is an aptamer specific to coagulation factor XIII (FXIII) and its uses thereof. Accordingly, the present aptamer is useful as a bio-tool to label thrombi, and/or as a targeting molecule to deliver drugs to thrombotic area. Therefore, the present disclosure also pertains to methods for treating diseases associated with FXIII, such as thrombosis.
Abstract: Provided are an siRNA for inhibiting expression of a Hepatitis B virus gene, and a pharmaceutical composition and conjugate containing the siRNA. Each nucleotide in the siRNA is an independently modified or unmodified nucleotide; the siRNA comprises a sense strand and an antisense strand; the sense strand comprises a nucleotide sequence A; the length of the nucleotide sequence A is the same as that of a nucleotide sequence as shown in SEQ ID NO: 1, and the number of the nucleotide differences is not more than three; the antisense strand comprises a nucleotide sequence B; and the length of the nucleotide sequence B is the same as that of a nucleotide sequence as shown in SEQ ID NO: 2, and number of nucleotide differences is not more than three.
Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with target gene expression or inhibit target gene expression and therapeutic uses of such products.
Type:
Grant
Filed:
April 5, 2018
Date of Patent:
August 16, 2022
Assignee:
SILENCE THERAPEUTICS GMBH
Inventors:
Judith Hauptmann, Dmitry Samarsky, Adrien Weingärtner, Lucas Bethge, Christian Frauendorf, Alison Gallafent
Abstract: The present invention relates to antisense oligonucleotide that modulate the expression of and/or function of a Sirtuin (SIRT), in particular, by targeting natural antisense polynucleotides of a Sirtuin (SIRT). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Sirtuins (SIRT)s.
Type:
Grant
Filed:
June 16, 2015
Date of Patent:
August 9, 2022
Assignee:
CURNA, INC.
Inventors:
Joseph Collard, Olga Khorkova Sherman, Carlos Coito, Belinda De Leon
Abstract: This invention relates to compounds, compositions, and methods useful for reducing ?-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Abstract: The present invention relates to antisense oligonucleotides and/or compounds that modulate the expression of and/or function of Filaggrin (FLG), in particular, by targeting natural antisense polynucleotides of Filaggrin (FLG). The invention also relates to the identification of these antisense oligonucleotides and/or compounds and their use in treating diseases and disorders associated with the expression of FLG.
Type:
Grant
Filed:
September 28, 2018
Date of Patent:
July 19, 2022
Assignee:
CURNA, INC.
Inventors:
Joseph Collard, Olga Khorkova Sherman, Carlos Coito
Abstract: The present invention is directed to an aptamer composition comprising at least one oligonucleotide consisting of: deoxyribonucleotides, ribonucleotides, derivatives of deoxyribonucleotides, derivatives of ribonucleotides, and mixtures thereof; wherein said aptamer composition has a binding affinity for one or more fungi species from the genus Malassezia.
Type:
Grant
Filed:
February 8, 2019
Date of Patent:
July 12, 2022
Assignee:
The Procter and Gamble Company
Inventors:
Juan Esteban Velasquez, Amy Violet Trejo, Laurie Ellen Breyfogle, James Patrick Henry, Douglas Joseph Dobrozsi, Gregory Allen Penner
Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least 5 one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.
Abstract: The application of PDCD4 as a drug treatment target for anti-depression and/or anti-anxiety disorders has been proved by experimental research that the increase of PDCD4 is an important factor leading to depression in the process of stress; PDCD4 as a target to inhibit its expression or function can play a good antidepressant role, and has no effect on normal physiological state. Therefore, PDCD4 can be used as a target in the preparation and screening of antidepressant and/or anxiolytic drugs, which has a broad application prospect.
Abstract: This invention relates to compounds, compositions, and methods useful for reducing ?-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Abstract: This invention relates to compounds, compositions, and methods useful for reducing ?-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Abstract: Provided are antisense oligonucleotides targeted against bacterial genes involved in biochemical pathways and/or cellular processes, and related compositions and methods of using the oligonucleotides and compositions, alone or in combination with other antimicrobial agents, for instance, in the treatment of an infected mammalian subject.
Type:
Grant
Filed:
December 23, 2015
Date of Patent:
April 5, 2022
Assignees:
Board of Regents, The University of Texas System, Oregon State University
Abstract: The present invention relates to use of miR-18b for prevention, treatment, or diagnosis of muscle diseases or neuromuscular diseases, and specifically it was confirmed that, in a muscle disease by gene mutations model, gene mutations reduce miR-18b expression, cause dysregulation of miR-18b signaling pathways, and thus induce calcium signaling, cell differentiation inhibition, and apoptosis. Therefore, miR-18b of the present invention may be used as a target factor for diagnosing and treating muscle diseases caused by gene mutations such as ALS and DMD.
Abstract: The invention relates to the treatment and prevention of cancer by administering agents that inhibit the activity of microRNAs that modulate tumor suppressor genes, which can include PTEN, p53, and INPP4B, among others. Inhibitors can include oligonucleotides that are at least partially complementary to these miRNAs. In some embodiments, these inhibitors are chemically modified oligonucleotides, including locked nucleic acids (LNAs).
Type:
Grant
Filed:
October 6, 2017
Date of Patent:
March 15, 2022
Assignee:
BETH ISRAEL DEACONESS MEDICAL CENTER
Inventors:
Pier Paolo Pandolfi, Laura Poliseno, Yvonne Tay, Leonardo Salmena
Abstract: Provided are non-aggregating immunostimulatory oligonucleotides. The present invention also provides a delivery agent for an immunostimulatory-oligonucleotide nucleic acid medicine, said delivery agent including a nucleic acid that contains a phosphorothioated nucleotide. According to another aspect, the present invention further provides an oligonucleotide including a bioactive core, and a nucleic acid that contains a phosphorothioated nucleotide. The present invention yet further provides an immunostimulator including the bioactive core of a type A/D, type B/K, or type C immunostimulatory oligonucleotide.
Type:
Grant
Filed:
December 24, 2015
Date of Patent:
March 8, 2022
Assignees:
National Institutes of Biomedical Innovation, Health and Nutrition, GeneDesign, Inc.
Abstract: The invention is directed to methods and compositions for collecting a non-placental biological samples of cells and quantifying and comparing levels of expression of microRNAs to characterize a preeclampsia-related condition. The samples may be collected before or after an intervention or may be collected over a period of time. One of the samples may be a control sample. Patients may then be treated according to their response.
Abstract: Provided herein, inter alia, are compounds capable of binding HSP70 (e.g. mHSP70) on a cell and internalizing into said cell. The compositions provided herein may be useful for delivering therapeutic and diagnostic agents to a cell. Further provided are pharmaceutical compositions and methods of treatment using nucleic acid compounds provided herein.