KR20220024153A - 안지오포이에틴 유사 7(angptl7) 관련 질환의 치료 - Google Patents

안지오포이에틴 유사 7(angptl7) 관련 질환의 치료 Download PDF

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KR20220024153A
KR20220024153A KR1020217041991A KR20217041991A KR20220024153A KR 20220024153 A KR20220024153 A KR 20220024153A KR 1020217041991 A KR1020217041991 A KR 1020217041991A KR 20217041991 A KR20217041991 A KR 20217041991A KR 20220024153 A KR20220024153 A KR 20220024153A
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sequence
seq
antisense strand
sense strand
nucleoside
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옴리 고테스만
새넌 브루스
폴 부스크
브라이언 카제스
데이비드 르위스
데이비드 로제마
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엠피리코 인크.
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KR1020217041991A 2019-05-24 2020-05-21 안지오포이에틴 유사 7(angptl7) 관련 질환의 치료 Pending KR20220024153A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962852813P 2019-05-24 2019-05-24
US62/852,813 2019-05-24
US201962881906P 2019-08-01 2019-08-01
US62/881,906 2019-08-01
PCT/US2020/034063 WO2020242896A2 (en) 2019-05-24 2020-05-21 Treatment of angiopoietin like 7 (angptl7) related diseases

Publications (1)

Publication Number Publication Date
KR20220024153A true KR20220024153A (ko) 2022-03-03

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KR1020217041991A Pending KR20220024153A (ko) 2019-05-24 2020-05-21 안지오포이에틴 유사 7(angptl7) 관련 질환의 치료

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US (2) US10941404B2 (https=)
EP (1) EP3976059A4 (https=)
JP (2) JP2022535717A (https=)
KR (1) KR20220024153A (https=)
CN (2) CN120022289A (https=)
AU (1) AU2020285639A1 (https=)
CA (1) CA3140917A1 (https=)
WO (1) WO2020242896A2 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11845989B2 (en) 2019-01-23 2023-12-19 Regeneron Pharmaceuticals, Inc. Treatment of ophthalmic conditions with angiopoietin-like 7 (ANGPTL7) inhibitors
JP2022523301A (ja) 2019-01-23 2022-04-22 リジェネロン・ファーマシューティカルズ・インコーポレイテッド アンジオポエチン様７（ａｎｇｐｔｌ７）阻害剤による眼疾患の治療
KR20220024153A (ko) 2019-05-24 2022-03-03 엠피리코 인크. 안지오포이에틴 유사 7(angptl7) 관련 질환의 치료
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
WO2022182768A1 (en) * 2021-02-26 2022-09-01 Regeneron Pharmaceuticals, Inc. Treatment of inflammation with glucocorticoids and angiopoietin-like 7 (angptl7) inhibitors
EP4352231A4 (en) * 2021-06-07 2026-02-11 Empirico Inc TREATMENT OF DISEASES ASSOCIATED WITH ANGPTL4
US20240409943A1 (en) * 2021-10-01 2024-12-12 Alnylam Pharmaceuticals, Inc. iRNA Compositions and Methods for Targeting ANGPTL7
CN118632622A (zh) 2021-12-08 2024-09-10 瑞泽恩制药公司 突变型肌纤蛋白疾病模型及其用途
US20250197859A1 (en) * 2022-03-28 2025-06-19 Empirico Inc. Compositions and methods for the treatment of angiopoietin like 7 (angptl7) related diseases
CN115624539A (zh) * 2022-12-16 2023-01-20 首都医科大学附属北京朝阳医院 一种脂质纳米颗粒及其制备方法和应用
WO2025042862A1 (en) * 2023-08-18 2025-02-27 The Johns Hopkins University Modulation of nuclear envelope protein expression for alleviation of nuclear pore complex injury cascades and tdp- 43 dysfunction in neurodegeneration
CN118604350A (zh) * 2024-05-17 2024-09-06 上海市第一人民医院 年龄相关性黄斑变性诊断的生物标志物、检测试剂盒及其应用

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US543227A (en) 1895-07-23 Core-drilling machinery
IE922292A1 (en) 1991-07-15 1993-01-27 Jolla Pharma Modified phosphorous intermediates for providing functional¹groups on the 5' end of oligonucleotides
US20060148743A1 (en) * 2001-05-18 2006-07-06 Vasant Jadhav RNA interference mediated inhibition of histone deacetylase (HDAC) gene expression using short interfering nucleic acid (siNA)
US20090137507A1 (en) * 2002-02-20 2009-05-28 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF ANGIOPOIETIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US20040198640A1 (en) * 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
US7601501B2 (en) * 2006-08-11 2009-10-13 The Scripps Research Institute Controlling osteogenesis by inhibition of osteogenic suppressors
JP2010505897A (ja) 2006-10-11 2010-02-25 マックス−プランク−ゲゼルシャフト・ツア・フェルデルング・デア・ヴィッセンシャフテン・エー・ファオ インフルエンザターゲット
US20110301228A1 (en) 2009-02-11 2011-12-08 The University Of North Carolina At Chapel Hill Gene therapy vector for treatment of steroid glaucoma
EP3028707A1 (en) 2009-05-01 2016-06-08 Ophthotech Corporation Methods for treating or preventing ophthalmological diseases
EP2494351B1 (en) * 2009-10-26 2016-06-08 Externautics S.p.A. Colon and rectal tumor markers and methods of use thereof
EP3260540A1 (en) * 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
EP2780454A2 (en) 2011-11-18 2014-09-24 Alnylam Pharmaceuticals, Inc. Modified rnai agents
WO2013120497A1 (en) * 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
US20150174203A1 (en) * 2012-05-30 2015-06-25 Icahn School Of Medicine At Mount Sinai Compositions And Methods For Modulating Pro-Inflammatory Immune Response
US9206423B2 (en) * 2012-12-30 2015-12-08 The Regents Of The University Of California Methods of modulating compliance of the trabecular meshwork
WO2014167529A1 (en) * 2013-04-10 2014-10-16 Institut De Cardiologie De Montreal Methods and compositions for preventing and treating atherosclerosis
CN103509101B (zh) * 2013-08-12 2016-11-02 中国科学院广州生物医药与健康研究院 一种扩增脐带血造血干细胞的细胞因子及其培养基
WO2015106128A2 (en) 2014-01-09 2015-07-16 Alnylam Pharmaceuticals, Inc. MODIFIED RNAi AGENTS
JP6708630B2 (ja) * 2015-04-17 2020-06-10 国立大学法人 東京大学 角膜障害の予防又は治療剤
WO2016205488A1 (en) * 2015-06-17 2016-12-22 The California Institute For Biomedical Research Modified therapeutic agents and compositions thereof
US11696954B2 (en) 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
JP7420727B2 (ja) 2018-02-17 2024-01-23 アローヘッド ファーマシューティカルズ インコーポレイテッド トリアルキン結合剤及び使用方法
US11452738B2 (en) 2019-01-04 2022-09-27 Empirico Inc. Treatment of thymic stromal lymphopoietin (TSLP) related diseases by inhibition of long-form TSLP transcripts
JP2022523301A (ja) 2019-01-23 2022-04-22 リジェネロン・ファーマシューティカルズ・インコーポレイテッド アンジオポエチン様７（ａｎｇｐｔｌ７）阻害剤による眼疾患の治療
US20220089664A1 (en) 2019-01-23 2022-03-24 Regeneron Pharmaceuticals, Inc. Treatment Of Ophthalmic Conditions With Angiopoietin-Like 7 (ANGPTL7) Inhibitors
KR20220024153A (ko) 2019-05-24 2022-03-03 엠피리코 인크. 안지오포이에틴 유사 7(angptl7) 관련 질환의 치료
IL300283A (en) 2020-08-04 2023-04-01 Dicerna Pharmaceuticals Inc Systemic administration of oligonucleotides
WO2022072356A1 (en) 2020-09-29 2022-04-07 Empirico Inc. Compositions and methods for the treatment of angiopoietin like 7 (angptl7) related diseases
US20250197859A1 (en) 2022-03-28 2025-06-19 Empirico Inc. Compositions and methods for the treatment of angiopoietin like 7 (angptl7) related diseases

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EP3976059A4 (en) 2024-09-11
US12378556B2 (en) 2025-08-05
AU2020285639A1 (en) 2021-12-23
WO2020242896A3 (en) 2021-02-04
EP3976059A2 (en) 2022-04-06
US10941404B2 (en) 2021-03-09
CN114375194A (zh) 2022-04-19
US20210163950A1 (en) 2021-06-03
CA3140917A1 (en) 2020-12-03
JP2022535717A (ja) 2022-08-10
CN114375194B (zh) 2024-12-13
CN120022289A (zh) 2025-05-23
WO2020242896A2 (en) 2020-12-03
US20200399640A1 (en) 2020-12-24
JP2025128091A (ja) 2025-09-02

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