CN119213124A - 靶向rna结合蛋白位点的改进的寡核苷酸 - Google Patents

靶向rna结合蛋白位点的改进的寡核苷酸 Download PDF

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Publication number
CN119213124A
CN119213124A CN202380040019.6A CN202380040019A CN119213124A CN 119213124 A CN119213124 A CN 119213124A CN 202380040019 A CN202380040019 A CN 202380040019A CN 119213124 A CN119213124 A CN 119213124A
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antisense oligonucleotide
tdp
nucleotide sequence
contiguous nucleotide
seq
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Pending
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CN202380040019.6A
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English (en)
Chinese (zh)
Inventor
J·M·A·巴斯蒂安
K·奇津斯卡
L·约恩森
李美玲
B·诺德博
J·维克萨
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F Hoffmann La Roche AG
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F Hoffmann La Roche AG
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Publication of CN119213124A publication Critical patent/CN119213124A/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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Neurology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN202380040019.6A 2022-05-18 2023-05-18 靶向rna结合蛋白位点的改进的寡核苷酸 Pending CN119213124A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP22174186 2022-05-18
EP22174186.1 2022-05-18
PCT/EP2023/063442 WO2023222858A1 (en) 2022-05-18 2023-05-18 Improved oligonucleotides targeting rna binding protein sites

Publications (1)

Publication Number Publication Date
CN119213124A true CN119213124A (zh) 2024-12-27

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CN202380040019.6A Pending CN119213124A (zh) 2022-05-18 2023-05-18 靶向rna结合蛋白位点的改进的寡核苷酸

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US (1) US20250290068A2 (enExample)
EP (1) EP4526445A1 (enExample)
JP (1) JP2025518522A (enExample)
KR (1) KR20250011958A (enExample)
CN (1) CN119213124A (enExample)
AR (1) AR129362A1 (enExample)
AU (1) AU2023270744A1 (enExample)
CA (1) CA3257367A1 (enExample)
CL (1) CL2024003521A1 (enExample)
CO (1) CO2024017326A2 (enExample)
CR (1) CR20240492A (enExample)
IL (1) IL316608A (enExample)
MX (1) MX2024014159A (enExample)
PE (1) PE20250394A1 (enExample)
TW (1) TW202409276A (enExample)
WO (1) WO2023222858A1 (enExample)

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* Cited by examiner, † Cited by third party
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US12612630B2 (en) 2024-03-05 2026-04-28 Takeda Pharmaceutical Company Limited Compositions and methods for splicing modulation of UNC13A

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JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
EP1015469B2 (en) 1997-09-12 2015-11-18 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
BRPI0008131B8 (pt) 1999-02-12 2021-05-25 Daiichi Sankyo Co Ltd composto ou um sal deste, análogo de oligonucleotídeo, composição farmacêutica, sonda para um gene,iniciador para começar a amplificação, uso de um análogo de oligonucleotídeo ou de um sal deste farmacologicamente aceitável, agente antisentido, e, agente antígeno
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
EP2284269B1 (en) 2002-11-18 2017-08-09 Roche Innovation Center Copenhagen A/S Antisense design
DE602007009487D1 (de) 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US8846637B2 (en) 2010-06-08 2014-09-30 Isis Pharmaceuticals, Inc. Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
CA2889044A1 (en) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
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EP4185696A1 (en) * 2020-07-23 2023-05-31 F. Hoffmann-La Roche AG Oligonucleotides targeting rna binding protein sites
CN118434858A (zh) * 2021-12-07 2024-08-02 豪夫迈·罗氏有限公司 靶向actl6b的反义寡核苷酸

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PE20250394A1 (es) 2025-02-11
US20250290068A2 (en) 2025-09-18
IL316608A (en) 2024-12-01
AU2023270744A1 (en) 2024-11-07
WO2023222858A1 (en) 2023-11-23
JP2025518522A (ja) 2025-06-17
MX2024014159A (es) 2024-12-06
TW202409276A (zh) 2024-03-01
US20250145996A1 (en) 2025-05-08
AR129362A1 (es) 2024-08-14
CO2024017326A2 (es) 2025-03-27
EP4526445A1 (en) 2025-03-26
CA3257367A1 (en) 2023-11-23
KR20250011958A (ko) 2025-01-22
CL2024003521A1 (es) 2025-03-21
CR20240492A (es) 2025-01-06

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