KR20200015608A - Htra1 발현을 조절하기 위한 안티센스 올리고뉴클레오티드 - Google Patents

Htra1 발현을 조절하기 위한 안티센스 올리고뉴클레오티드 Download PDF

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KR20200015608A
KR20200015608A KR1020197038723A KR20197038723A KR20200015608A KR 20200015608 A KR20200015608 A KR 20200015608A KR 1020197038723 A KR1020197038723 A KR 1020197038723A KR 20197038723 A KR20197038723 A KR 20197038723A KR 20200015608 A KR20200015608 A KR 20200015608A
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oligonucleotide
oligonucleotides
htra1
nucleosides
seq
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루벤 알바레즈 산체스
로베르토 이아콘
페테르 하게도른
주잔네 카믈러
쇠렌 오토센
신드리 트라우스타손
하이디 뤠 후들부슈
뤼케 페데르센
마르코 베레라
안드레아스 디에크만
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에프. 호프만-라 로슈 아게
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    • C12N15/113Non-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
    • C12N15/1137Non-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
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    • C12N2310/32Chemical structure of the sugar
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KR1020197038723A 2017-06-01 2018-05-30 Htra1 발현을 조절하기 위한 안티센스 올리고뉴클레오티드 Withdrawn KR20200015608A (ko)

Applications Claiming Priority (7)

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

Publications (1)

Publication Number Publication Date
KR20200015608A true KR20200015608A (ko) 2020-02-12

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KR1020197038723A Withdrawn KR20200015608A (ko) 2017-06-01 2018-05-30 Htra1 발현을 조절하기 위한 안티센스 올리고뉴클레오티드

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

Families Citing this family (4)

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Publication number Priority date Publication date Assignee Title
MA45620A (fr) 2016-07-01 2019-05-08 Hoffmann La Roche Oligonucléotides antisens pour la modulation de l'expression de htra1
AU2019218987B2 (en) 2018-02-12 2025-04-24 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
AU2021367458B2 (en) * 2020-10-30 2025-05-01 Hideaki Hara Gdf15 modulator for use in inhibition of ocular tissue fibrosis

Family Cites Families (29)

* 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
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
WO2000008134A2 (en) * 1998-08-03 2000-02-17 Novartis Ag HUMAN HtrA SERINE PROTEASE
RU2233844C2 (ru) 1999-02-12 2004-08-10 Санкио Компани Лимитед Новые нуклеозидные и олигонуклеотидные аналоги
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
DK2284269T3 (en) 2002-11-18 2017-10-23 Roche Innovation Ct Copenhagen As Antisense design
CA2609180A1 (en) * 2005-05-24 2006-11-30 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
EP1984381B1 (en) 2006-01-27 2010-09-29 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
ES2389737T3 (es) 2006-05-11 2012-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en 5'
ES2391788T3 (es) * 2006-07-26 2012-11-29 Yale University, Inc. Diagnóstico y tratamiento de degeneración macular relacionada con la edad
EP2081596A4 (en) * 2006-10-06 2010-07-21 Univ Utah Res Found METHOD FOR THE DETECTION OF EYE DISEASES AND PATHOLOGICAL CONDITIONS AND TREATMENT THEREFOR
US8278425B2 (en) 2007-05-30 2012-10-02 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
TW200911290A (en) 2007-07-02 2009-03-16 Alcon Res Ltd RNAI-mediated inhibition of HTRA1 for treatment of macular degeneration
US8278283B2 (en) 2007-07-05 2012-10-02 Isis Pharmaceuticals, Inc. 6-disubstituted or unsaturated bicyclic nucleic acid analogs
CA2704447A1 (en) * 2007-11-01 2009-05-07 University Of Iowa Research Foundation Predicting amd with snps within or near c2, factor b, plekha1, htra1, prelp, or loc387715
US8546556B2 (en) 2007-11-21 2013-10-01 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
FR2965278B1 (fr) 2010-09-23 2014-10-10 Univ Caen Basse Normandie Procede d'obtention in vitro ou ex vivo de chondrocytes et leurs utilisations
CN104136451A (zh) 2011-09-07 2014-11-05 玛瑞纳生物技术有限公司 具有构象限制的单体的核酸化合物的合成和用途
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
JP2016522674A (ja) * 2012-05-16 2016-08-04 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
CA2889044A1 (en) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
EP3538671A1 (en) * 2016-11-11 2019-09-18 Roche Innovation Center Copenhagen A/S Therapeutic oligonucleotides capture and detection

Also Published As

Publication number Publication date
CA3062590A1 (en) 2018-12-06
US20220042022A1 (en) 2022-02-10
BR112019025290A2 (pt) 2020-06-23
TW201907008A (zh) 2019-02-16
CR20190543A (es) 2020-02-10
RU2019143004A (ru) 2021-07-09
PH12019502636A1 (en) 2020-06-08
AU2018277219A1 (en) 2019-12-19
WO2018220034A1 (en) 2018-12-06
CL2019003337A1 (es) 2020-05-15
IL271039A (en) 2020-01-30
JP2020521491A (ja) 2020-07-27
MA49278A (fr) 2020-04-08
JP7169995B2 (ja) 2022-11-11
US20190055564A1 (en) 2019-02-21
CO2019013317A2 (es) 2020-01-17
CN110691849A (zh) 2020-01-14
PE20191845A1 (es) 2019-12-31
MX2019014187A (es) 2020-01-27
EP3630976A1 (en) 2020-04-08
US20200157546A1 (en) 2020-05-21

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Patent event date: 20191230

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Comment text: International Patent Application

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