JP7169995B2 - Ｈｔｒａ１発現を調節するためのアンチセンスオリゴヌクレオチド - Google Patents

Ｈｔｒａ１発現を調節するためのアンチセンスオリゴヌクレオチド Download PDF

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JP7169995B2
JP7169995B2 JP2019566236A JP2019566236A JP7169995B2 JP 7169995 B2 JP7169995 B2 JP 7169995B2 JP 2019566236 A JP2019566236 A JP 2019566236A JP 2019566236 A JP2019566236 A JP 2019566236A JP 7169995 B2 JP7169995 B2 JP 7169995B2
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pharmaceutically acceptable
htra1
oligonucleotide
nucleosides
acceptable salt
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JP2020521491A (ja
JP2020521491A5 (https=
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サンチェス，ルーベン・アルバレス
イアコーネ，ロベルト
ハーゲドルン，ペーター
カムラ，スサネ
オトセン，セーアン
トラウスタソン，シンドリ
フルレブッシュ，ハイディ・リュ
ピーダスン，リュケ
ベレラ，マルコ
ディークマン，アンドレアス
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F Hoffmann La Roche AG
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    • C12N15/09—Recombinant DNA-technology
    • 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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21108—HtrA2 peptidase (3.4.21.108)
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    • C12N2310/30—Chemical structure
    • 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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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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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)
JP2019566236A 2017-06-01 2018-05-30 Ｈｔｒａ１発現を調節するためのアンチセンスオリゴヌクレオチド Active JP7169995B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
EP17173964.2 2017-06-01
EP17173964 2017-06-01
EP17209407.0 2017-12-21
EP17209407 2017-12-21
EP17209535 2017-12-21
EP17209535.8 2017-12-21
PCT/EP2018/064221 WO2018220034A1 (en) 2017-06-01 2018-05-30 Antisense oligonucleotides for modulating htra1 expression

Publications (3)

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JP2020521491A JP2020521491A (ja) 2020-07-27
JP2020521491A5 JP2020521491A5 (https=) 2021-07-26
JP7169995B2 true JP7169995B2 (ja) 2022-11-11

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JP2019566236A Active JP7169995B2 (ja) 2017-06-01 2018-05-30 Ｈｔｒａ１発現を調節するためのアンチセンスオリゴヌクレオチド

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US (3) US20190055564A1 (https=)
EP (1) EP3630976A1 (https=)
JP (1) JP7169995B2 (https=)
KR (1) KR20200015608A (https=)
CN (1) CN110691849A (https=)
AU (1) AU2018277219A1 (https=)
BR (1) BR112019025290A2 (https=)
CA (1) CA3062590A1 (https=)
CL (1) CL2019003337A1 (https=)
CO (1) CO2019013317A2 (https=)
CR (1) CR20190543A (https=)
IL (1) IL271039A (https=)
MA (1) MA49278A (https=)
MX (1) MX2019014187A (https=)
PE (1) PE20191845A1 (https=)
PH (1) PH12019502636A1 (https=)
RU (1) RU2019143004A (https=)
TW (1) TW201907008A (https=)
WO (1) WO2018220034A1 (https=)

Families Citing this family (4)

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Publication number Priority date Publication date Assignee Title
MX2018015722A (es) 2016-07-01 2019-05-27 Hoffmann La Roche Oligonucleotidos antisentido para modular expresion del requisito de alta temperatura a 1 (htra1).
WO2019157531A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
CN116507741A (zh) * 2020-08-20 2023-07-28 基因组评论股份有限公司 年龄相关性黄斑变性诊断用生物标记物及其用途
KR20230095097A (ko) * 2020-10-30 2023-06-28 히데아키 하라 눈 조직 섬유화의 저해에 사용하기 위한 gdf15 조절 물질

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009006460A1 (en) 2007-07-02 2009-01-08 Alcon Research, Ltd. Rnai-mediated inhibition of htra1 for treatment of macular degeneration
WO2012038668A1 (fr) 2010-09-23 2012-03-29 Universite De Caen Basse Normandie Procédé d'obtention in vitro ou ex vivo de chondrocytes articulaires différenciés et leurs utilisations

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
WO2000008134A2 (en) * 1998-08-03 2000-02-17 Novartis Ag HUMAN HtrA SERINE PROTEASE
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
WO2004046160A2 (en) 2002-11-18 2004-06-03 Santaris Pharma A/S Amino-lna, thio-lna and alpha-l-oxy-ln
EP2462937A1 (en) * 2005-05-24 2012-06-13 Isis Pharmaceuticals, Inc. Modulation of LMW-PTPase Expression
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
ES2391788T3 (es) * 2006-07-26 2012-11-29 Yale University, Inc. Diagnóstico y tratamiento de degeneración macular relacionada con la edad
WO2008067040A2 (en) * 2006-10-06 2008-06-05 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
WO2009059321A2 (en) * 2007-11-01 2009-05-07 University Of Iowa Research Foundation Rca locus analysis to assess susceptibility to amd and mpgnii
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
DK2356129T3 (da) 2008-09-24 2013-05-13 Isis Pharmaceuticals Inc Substituerede alpha-L-bicykliske nukleosider
US9012421B2 (en) 2009-08-06 2015-04-21 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2753631A1 (en) 2011-09-07 2014-07-16 Marina Biotech, Inc. Synthesis and uses of nucleic acid compounds with conformationally restricted monomers
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
CN104837996A (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
US20190284621A1 (en) * 2016-11-11 2019-09-19 Roche Innovation Center Copenhagen A/S Therapeutic oligonucleotides capture and detection

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009006460A1 (en) 2007-07-02 2009-01-08 Alcon Research, Ltd. Rnai-mediated inhibition of htra1 for treatment of macular degeneration
WO2012038668A1 (fr) 2010-09-23 2012-03-29 Universite De Caen Basse Normandie Procédé d'obtention in vitro ou ex vivo de chondrocytes articulaires différenciés et leurs utilisations

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
KURRECK J. et al.，Nucleic Acids Research，2002年，Vol.30 No.9，p.1911-1918

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Publication number Publication date
TW201907008A (zh) 2019-02-16
CL2019003337A1 (es) 2020-05-15
CO2019013317A2 (es) 2020-01-17
RU2019143004A (ru) 2021-07-09
CN110691849A (zh) 2020-01-14
MX2019014187A (es) 2020-01-27
PH12019502636A1 (en) 2020-06-08
CR20190543A (es) 2020-02-10
PE20191845A1 (es) 2019-12-31
MA49278A (fr) 2020-04-08
CA3062590A1 (en) 2018-12-06
WO2018220034A1 (en) 2018-12-06
KR20200015608A (ko) 2020-02-12
JP2020521491A (ja) 2020-07-27
US20220042022A1 (en) 2022-02-10
US20200157546A1 (en) 2020-05-21
BR112019025290A2 (pt) 2020-06-23
US20190055564A1 (en) 2019-02-21
IL271039A (en) 2020-01-30
AU2018277219A1 (en) 2019-12-19
EP3630976A1 (en) 2020-04-08

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