CN117597442A - 用于降低pln表达的化合物和方法 - Google Patents

用于降低pln表达的化合物和方法 Download PDF

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Publication number
CN117597442A
CN117597442A CN202280027124.1A CN202280027124A CN117597442A CN 117597442 A CN117597442 A CN 117597442A CN 202280027124 A CN202280027124 A CN 202280027124A CN 117597442 A CN117597442 A CN 117597442A
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China
Prior art keywords
modified oligonucleotide
oligomeric compound
modified
sugar moiety
nucleobase
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CN202280027124.1A
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English (en)
Chinese (zh)
Inventor
A·慕利克
H-H·布维
S·M·弗赖尔
T·Y·叶
D·库布里
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of CN117597442A publication Critical patent/CN117597442A/zh
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A01K2217/07—Animals genetically altered by homologous recombination
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    • A01K2267/03—Animal model, e.g. for test or diseases
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CN202280027124.1A 2021-02-11 2022-02-10 用于降低pln表达的化合物和方法 Pending CN117597442A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163148579P 2021-02-11 2021-02-11
US63/148,579 2021-02-11
PCT/US2022/016015 WO2022173976A1 (en) 2021-02-11 2022-02-10 Compounds and methods for reducing pln expression

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CN117597442A true CN117597442A (zh) 2024-02-23

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US (1) US20240191232A1 (enExample)
EP (1) EP4291651A4 (enExample)
JP (1) JP2024506351A (enExample)
KR (1) KR20230145133A (enExample)
CN (1) CN117597442A (enExample)
AU (1) AU2022219976A1 (enExample)
BR (1) BR112023015160A2 (enExample)
CA (1) CA3210172A1 (enExample)
IL (1) IL304775A (enExample)
MX (1) MX2023009429A (enExample)
WO (1) WO2022173976A1 (enExample)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4689119A1 (en) * 2023-04-05 2026-02-11 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing pln expression
AU2024308906A1 (en) 2023-06-30 2026-02-05 Atrium Therapeutics, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
TW202528539A (zh) * 2023-09-20 2025-07-16 日商第一三共股份有限公司 抑制受磷蛋白之表現的siRNA

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Publication number Priority date Publication date Assignee Title
EP1126866B1 (en) * 1998-11-02 2007-02-07 The Regents of The University of California A mutant phospholamban molecule and its use in the treatment of cardiac disease and heart failure
EP1212416A2 (en) * 1999-08-31 2002-06-12 Ribozyme Pharmaceuticals, Inc. Nucleic acid based modulators of gene expression
US20030050259A1 (en) * 1999-12-06 2003-03-13 Lawrence Blatt Method and reagent for the treatment of cardiac disease
AU2005323437B2 (en) * 2004-04-30 2011-10-06 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a C5-modified pyrimidine
US7491813B2 (en) * 2005-12-07 2009-02-17 Monsanto Technology Llc Promoter polynucleotides identified from Zea mays for use in plants
US20100298697A1 (en) * 2009-05-19 2010-11-25 Medtronic, Inc. Method and devices for improved efficiency of rna delivery to cells
US8207138B2 (en) * 2009-05-19 2012-06-26 Medtronic, Inc. Methods and devices for improved efficiency of RNA delivery to cells
CA2954791C (en) * 2014-07-14 2025-11-18 The Regents Of The University Of California CRISPR/CAS TRANSCRIPTIONAL MODULATION
EP3585898A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders

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JP2024506351A (ja) 2024-02-13
IL304775A (en) 2023-09-01
KR20230145133A (ko) 2023-10-17
BR112023015160A2 (pt) 2023-10-03
EP4291651A4 (en) 2025-07-02
CA3210172A1 (en) 2022-08-18
WO2022173976A1 (en) 2022-08-18
EP4291651A1 (en) 2023-12-20
US20240191232A1 (en) 2024-06-13
AU2022219976A1 (en) 2023-08-10
MX2023009429A (es) 2023-09-27

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