公开(公告)号：US20240360452A1

公开(公告)日：2024-10-31

申请号：US18771603

申请日：2024-07-12

申请人： BioMarin Technologies B.V. ,  Academisch Ziekenhuis Leiden

发明人： Judith Christina Theodora van Deutekom

IPC分类号： C12N15/113 ,  A61K31/56 ,  A61K31/57 ,  A61K31/573 ,  A61K31/58 ,  A61K31/7088 ,  A61K38/17 ,  A61K45/06 ,  A61K48/00

CPC分类号： C12N15/113 ,  A61K31/56 ,  A61K31/57 ,  A61K31/573 ,  A61K31/58 ,  A61K31/7088 ,  A61K38/1719 ,  A61K45/06 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/31 ,  C12N2310/313 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/346 ,  C12N2320/31 ,  C12N2320/33

摘要： The invention relates to a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, or exons 50-53 of the DMD pre-mRNA in a patient, the method comprising providing the patient with the molecule. The invention also relates to the molecule as such.

公开(公告)号：US12104159B2

公开(公告)日：2024-10-01

申请号：US17808247

申请日：2022-06-22

申请人： AcuraStem Incorporated

发明人： Wen-Hsuan Chang ,  Emily Elizabeth Lee

IPC分类号： C07H21/02 ,  A61P25/00 ,  C12N15/113

CPC分类号： C12N15/1137 ,  A61P25/00 ,  C12N2310/11 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2320/32

摘要： The present invention relates to PIKFYVE antisense oligonucleotides (ASOs), pharmaceutical compositions containing them, and methods for treating, inhibiting, suppressing, and preventing neurological diseases with them.

公开(公告)号：US20240301428A1

公开(公告)日：2024-09-12

申请号：US18659737

申请日：2024-05-09

申请人： THE UNIVERSITY OF CHICAGO ,  NORTHWESTERN UNIVERSITY

发明人： Elizabeth McNally ,  Eugene Wyatt

IPC分类号： C12N15/113

CPC分类号： C12N15/1138 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3233 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3513 ,  C12N2310/3521 ,  C12N2320/33

摘要： The invention is directed to one or more antisense polynucleotides and their use in pharmaceutical compositions in a strategy to induce exon skipping in the γ-sarcoglycan gene in patients suffering from Limb-Girdle Muscular Dystrophy-20 (LGM-D)2C) or in patients at risk of such a disease. The invention also provides methods of preventing or treating muscular dystrophy, e.g., LGMD)2C, by exon skipping in the gamma sarcoglycan gene using antisense polynucleotides. Accordingly, in some aspects the in-vention provides an isolated antisense oligonucleotide, wherein the oligonucleotide specifically hybridizes to an exon target region of a γ-sarcoglycan RNA. In another aspect, the invention provides a method of inducing exon-skipping of a gamma sarcoglycan RNA, comprising delivering an antisense oligonucleotide or a composition to a cell.

公开(公告)号：US20240301422A1

公开(公告)日：2024-09-12

申请号：US18661354

申请日：2024-05-10

申请人： Biogen MA Inc.

发明人： Holly Kordasiewicz

IPC分类号： C12N15/113 ,  A61K9/00 ,  A61K31/7125 ,  A61K47/02 ,  A61K47/46 ,  A61P25/08 ,  A61P25/14 ,  A61P25/28

CPC分类号： C12N15/113 ,  A61K9/0019 ,  A61K31/7125 ,  A61P25/08 ,  A61P25/14 ,  A61P25/28 ,  A61K47/02 ,  A61K47/46 ,  C12N2310/11 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/3525

摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of Tau mRNA in a cell or animal, and in certain instances reducing the amount of Tau protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include loss of memory, loss of motor function, and increase in the number and/or volume of neurofibrillary inclusions. Such neurodegenerative diseases include tauopathies, Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy (PSP), Chronic Traumatic Encephalopathy (CTE), Corticobasal Ganglionic Degeneration (CBD), Epilepsy, and Dravet's Syndrome.

公开(公告)号：US20240287522A1

公开(公告)日：2024-08-29

申请号：US18637337

申请日：2024-04-16

申请人： Avidity Biosciences, Inc.

发明人： Barbora MALECOVA ,  Rob BURKE ,  David SALA CANO ,  Beatrice Diana DARIMONT

IPC分类号： C12N15/113 ,  A61K31/7125 ,  A61K47/68 ,  A61K48/00 ,  A61P21/00 ,  A61P21/06 ,  C07K16/28 ,  C07K19/00

CPC分类号： C12N15/113 ,  A61K31/7125 ,  A61K47/6807 ,  A61K47/6849 ,  A61K47/6889 ,  A61K48/005 ,  A61P21/00 ,  A61P21/06 ,  C07K16/2881 ,  C07K19/00 ,  C07K2317/55 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/313 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3513 ,  C12N2320/32

摘要： Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.

公开(公告)号：US12037586B2

公开(公告)日：2024-07-16

申请号：US17859781

申请日：2022-07-07

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Weimin Wang ,  Qingyi Li ,  Naim Nazef

IPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K45/06 ,  A61K47/54 ,  A61K47/60 ,  C07H19/10 ,  C07H21/02 ,  C07H21/04

CPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K45/06 ,  A61K47/549 ,  A61K47/60 ,  C07H19/10 ,  C07H21/02 ,  C07H21/04 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/322 ,  C12N2310/344 ,  C12N2310/3515

摘要： Disclosed herein are oligonucleotides, such as nucleic acid inhibitor molecules, having a 4′-phosphate analog and methods of using the same, for example, to modulate the expression of a target gene in a cell. The phosphate analogs are bound to the 4′-carbon of the sugar moiety (e.g., a ribose or deoxyribose or analog thereof) of the 5′-terminal nucleotide of an oligonucleotide. Typically, the phosphate analog is an oxymethylphosphonate, where the oxygen atom of the oxymethyl group is bound to the 4′-carbon of the sugar moiety or analog thereof.

公开(公告)号：US12012601B2

公开(公告)日：2024-06-18

申请号：US17150151

申请日：2021-01-15

申请人： Bio-Path Holdings, Inc.

发明人： Ana Tari Ashizawa

IPC分类号： C12N15/113 ,  A61K9/00 ,  A61K9/127 ,  A61K31/506 ,  A61K31/706 ,  A61K31/7068 ,  A61K31/7088 ,  A61P35/02 ,  C12Q1/68

CPC分类号： C12N15/1138 ,  A61K9/0019 ,  A61K9/127 ,  A61K31/506 ,  A61K31/706 ,  A61K31/7068 ,  A61K31/7088 ,  A61P35/02 ,  C12N2310/11 ,  C12N2310/31 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2320/35

摘要： Provided herein are methods of treating a cancer in a patient comprising administration of an effective amount of a nuclease-resistant polynucleotide that hybridizes to the translation initiation site of a Grb2 nucleic acid in the patient and either a Bcr-Abl tyrosine kinase inhibitor (e.g., dasatinib) or a cytidine analogue (e.g., decitabine or cytarabine). The cancer may be Ph+ and/or Bcr-Abl positive chronic myelogenous leukemia or acute myeloid leukemia or myelodysplastic syndrome.

公开(公告)号：US11932666B2

公开(公告)日：2024-03-19

申请号：US17965125

申请日：2022-10-13

申请人： TOKYO UNIVERSITY OF SCIENCE FOUNDATION

发明人： Takeshi Wada ,  Tatsuya Saito ,  Yuka Ishii ,  Yohei Nukaga

IPC分类号： C07H19/073 ,  C07F5/02 ,  C07F9/6558 ,  C07F9/6584 ,  C07H19/173 ,  C07H21/00 ,  C12N15/113

CPC分类号： C07H19/173 ,  C07F5/02 ,  C07F9/6558 ,  C07F9/6584 ,  C07H19/073 ,  C07H21/00 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/31 ,  C12N2310/321 ,  Y02P20/55

摘要： Provided is a polymerizable compound represented by a Formula E-1 or Formula E-2 shown in the description. In Formula E-1 or Formula E-2, R1 represents an alkoxy group, —NRN2, a hydroxy group, an aryl group, or an alkyl group, wherein RN each independently represents a hydrogen atom or an alkyl group having 1 to 10 carbon atoms; n represents an integer from 1 to 5; R3 represents a hydrogen atom, an acetyl group, a phenoxyacetyl group, a pivaloyl group, a benzyl group, a 4-methoxybenzyl group, a benzoyl group, a triphenylmethyl group, a 4,4′-dimethoxytrityl (DMTr) group, a 4-methoxytrityl (MMTr) group, a 9-phenylxanthenyl group, a trimethylsilyl group, a cyanomethoxymethyl group, a 2-(cyanoethoxy)ethyl group, or a cyanoethoxymethyl group; and X represents a structure represented by any one of Formula B-1 to Formula B-5 shown in the description.

公开(公告)号：US20230407305A1

公开(公告)日：2023-12-21

申请号：US18322487

申请日：2023-05-23

申请人： Universitat de València ,  ARTHEX BIOTECH S.L.

发明人： Rubén ARTERO ALLEPUZ ,  Nerea MORENO CERVERA ,  Irene GONZÁLEZ MARTÍNEZ ,  Estefanía CERRO HERREROS ,  Eric G. MARCUSSON ,  María Beatriz LLAMUSÍ TROISI

IPC分类号： C12N15/113 ,  A61P21/00

CPC分类号： C12N15/113 ,  C12N2310/31 ,  C12N2310/141 ,  A61P21/00

摘要： The invention provides oligonucleotide and/or oligonucleotide analogue molecules that are antagonists of a microRNA, preferably antagonists of human microRNAs hsa-miR-23b-3p and hsa-miR-218-5p, that comprise a mixture of phosphorothioate and phosphodiester linkages, and that are conjugated to at least one oleic acid molecule. Inhibiting these microRNAs allows to increase the endogenous levels of the corresponding proteins MBNL1 and/or MBNL2. The present invention further provides compositions comprising said oligonucleotides and/or oligonucleotide analogue molecules and their uses for the treatment and prevention of DM in a subject in need thereof.

公开(公告)号：US11814645B2

公开(公告)日：2023-11-14

申请号：US17220693

申请日：2021-04-01

申请人： The Regents of the University of California ,  University of Vienna ,  Emmanuelle Charpentier

发明人： Jennifer A. Doudna ,  Martin Jinek ,  Krzysztof Chylinski ,  Emmanuelle Charpentier

IPC分类号： C12N15/11 ,  C12N15/63 ,  C12N9/22 ,  C12N15/113 ,  C12N15/10 ,  C12N15/90 ,  C12Q1/686 ,  H01L33/00 ,  H01L21/02 ,  C12N15/70 ,  C12N15/74 ,  A61K38/46 ,  A01H6/46 ,  A01K67/027 ,  A61K48/00

CPC分类号： C12N15/907 ,  A01H6/4684 ,  A01K67/027 ,  A61K38/465 ,  C12N9/22 ,  C12N15/102 ,  C12N15/111 ,  C12N15/113 ,  C12N15/63 ,  C12N15/70 ,  C12N15/746 ,  C12N15/90 ,  C12N15/902 ,  C12Q1/686 ,  H01L21/02312 ,  H01L21/02365 ,  H01L33/0075 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/13 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2310/31 ,  C12N2310/32 ,  C12N2310/33 ,  C12N2310/3519 ,  C12N2310/531 ,  C12N2800/80 ,  C12Y301/04

摘要： The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.