JP2022501040A5 - - Google Patents

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Publication number
JP2022501040A5
JP2022501040A5 JP2021515578A JP2021515578A JP2022501040A5 JP 2022501040 A5 JP2022501040 A5 JP 2022501040A5 JP 2021515578 A JP2021515578 A JP 2021515578A JP 2021515578 A JP2021515578 A JP 2021515578A JP 2022501040 A5 JP2022501040 A5 JP 2022501040A5
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JP
Japan
Prior art keywords
rnai agent
sequence
disease
agent according
nag37
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JP2021515578A
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English (en)
Japanese (ja)
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JP2022501040A (ja
JP7488254B2 (ja
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Priority claimed from PCT/US2019/051707 external-priority patent/WO2020061177A1/en
Publication of JP2022501040A publication Critical patent/JP2022501040A/ja
Publication of JP2022501040A5 publication Critical patent/JP2022501040A5/ja
Priority to JP2024076157A priority Critical patent/JP7741927B2/ja
Application granted granted Critical
Publication of JP7488254B2 publication Critical patent/JP7488254B2/ja
Priority to JP2025147403A priority patent/JP2025186315A/ja
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JP2021515578A 2018-09-19 2019-09-18 １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法 Active JP7488254B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024076157A JP7741927B2 (ja) 2018-09-19 2024-05-08 １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法
JP2025147403A JP2025186315A (ja) 2018-09-19 2025-09-05 １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862733320P 2018-09-19 2018-09-19
US62/733,320 2018-09-19
US201862773707P 2018-11-30 2018-11-30
US62/773,707 2018-11-30
US201962890220P 2019-08-22 2019-08-22
US62/890,220 2019-08-22
PCT/US2019/051707 WO2020061177A1 (en) 2018-09-19 2019-09-18 Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use

Related Child Applications (1)

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JP2024076157A Division JP7741927B2 (ja) 2018-09-19 2024-05-08 １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法

Publications (3)

Publication Number Publication Date
JP2022501040A JP2022501040A (ja) 2022-01-06
JP2022501040A5 true JP2022501040A5 (https=) 2023-11-24
JP7488254B2 JP7488254B2 (ja) 2024-05-21

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JP2021515578A Active JP7488254B2 (ja) 2018-09-19 2019-09-18 １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法
JP2024076157A Active JP7741927B2 (ja) 2018-09-19 2024-05-08 １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法
JP2025147403A Pending JP2025186315A (ja) 2018-09-19 2025-09-05 １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法

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JP2024076157A Active JP7741927B2 (ja) 2018-09-19 2024-05-08 １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法
JP2025147403A Pending JP2025186315A (ja) 2018-09-19 2025-09-05 １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法

Country Status (21)

Country Link
US (1) US12534728B2 (https=)
EP (1) EP3852769A4 (https=)
JP (3) JP7488254B2 (https=)
KR (1) KR102769565B1 (https=)
CN (1) CN113164509B (https=)
AU (1) AU2019342117A1 (https=)
BR (1) BR112021005137A2 (https=)
CA (1) CA3109553A1 (https=)
CL (1) CL2021000669A1 (https=)
CO (1) CO2021003446A2 (https=)
CR (1) CR20210186A (https=)
EC (1) ECSP21027262A (https=)
IL (1) IL281597A (https=)
MX (1) MX2021003077A (https=)
MY (1) MY209138A (https=)
PE (1) PE20211212A1 (https=)
PH (1) PH12021550423A1 (https=)
SG (1) SG11202101698WA (https=)
TW (2) TWI870358B (https=)
UY (1) UY38382A (https=)
WO (1) WO2020061177A1 (https=)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201901841TA (en) * 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
AU2019239971B2 (en) * 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
KR102769565B1 (ko) * 2018-09-19 2025-02-20 애로우헤드 파마슈티컬스 인코포레이티드 17베타-HSD 유형 13- (HSD17B13)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
CR20210395A (es) * 2018-12-21 2021-11-05 Ionis Pharmaceuticals Inc Moduladores de la expresión de hsd17b13
KR20240004092A (ko) * 2020-06-01 2024-01-11 암젠 인크 Hsd17b13 발현을 억제하기 위한 rnai 작제물 및 이의 사용 방법
WO2022098748A1 (en) * 2020-11-06 2022-05-12 Inipharm, Inc. Uses for hsd17b13 inhibitors
MX2023007587A (es) 2020-12-23 2023-09-21 Regeneron Pharma Tratamiento de enfermedades hepaticas con inhibidores de efectores b tipo dffa que inducen la muerte celular (cideb).
CA3216332A1 (en) * 2021-04-22 2022-10-27 Tuojie Biotech (Shanghai) Co., Ltd. Sirna targeting 17b-hydroxysteroid dehydrogenase type 13 and sirna conjugate
CN118103038A (zh) 2021-08-20 2024-05-28 英安塔制药有限公司 17β-羟基类固醇脱氢酶13型抑制剂及其使用方法
CN118234862A (zh) * 2021-09-08 2024-06-21 阿利戈斯治疗公司 经修饰的短干扰核酸分子(siNA)及其用途
KR20240103029A (ko) * 2021-11-18 2024-07-03 노파르티스 아게 샤르코 마리 투스 질환 치료용 pmp22 표적화 화합물
EP4433596A4 (en) * 2021-11-19 2026-02-11 Wave Life Sciences Ltd DOUBLE-STRANDED OLIGONUCLETIC COMPOSITIONS ASSOCIATED WITH HSD17B13 AND RELATED PROCESSES
WO2023109935A1 (zh) * 2021-12-16 2023-06-22 上海拓界生物医药科技有限公司 一种dsRNA、其制备方法及应用
US20230193392A1 (en) 2021-12-20 2023-06-22 Regeneron Pharmaceuticals, Inc. Methods Of Identifying And Evaluating Liver Inflammation And Liver Fibrosis In A Subject By Determining A Stratified Score Based On Gene Expression
WO2023138650A1 (zh) * 2022-01-20 2023-07-27 上海拓界生物医药科技有限公司 一种dsRNA、其应用及制备方法
WO2023196941A1 (en) 2022-04-08 2023-10-12 Arrowhead Pharmaceuticals, Inc. Treatment of a non-alcoholic fatty liver disease
EP4514805A4 (en) * 2022-04-28 2026-01-21 Enanta Pharm Inc TYPE 13 17-BETA-HYDROXYSTEROID DEHYDROGENASE INHIBITORS CONTAINING PYRIMIDINONE
CN116515835A (zh) * 2022-04-29 2023-08-01 北京福元医药股份有限公司 用于抑制HSD17B13表达的siRNA、其缀合物和药物组合物及其用途
TW202400791A (zh) * 2022-05-06 2024-01-01 大陸商蘇州瑞博生物技術股份有限公司 一種核酸、含有該核酸的組合物與綴合物及製備方法和用途
WO2024131916A1 (en) * 2022-12-22 2024-06-27 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of 17beta-hydroxysteroid dehydrogenase type 13 (hsd17b13)
WO2024255748A1 (zh) * 2023-06-16 2024-12-19 施能康医药科技(苏州)有限公司 靶向17-β-羟基类固醇脱氢酶13的核酸及其用途
IL326882A (en) * 2023-08-30 2026-04-01 Arrowhead Pharmaceuticals Inc RNAi agents for inhibiting expression of inhibitory subunit beta E (INHBE), their pharmaceutical compositions and methods of use
US20250120952A1 (en) * 2023-10-04 2025-04-17 Enanta Pharmaceuticals, Inc. 17-Beta-Hydroxysteroid Dehydrogenase Type 13 Inhibitors And Methods Of Use Thereof
CN121969377A (zh) 2023-10-06 2026-05-01 葛兰素史密斯克莱知识产权(第3号)有限公司 非酒精性脂肪性肝病的治疗
WO2026082165A1 (zh) * 2024-10-18 2026-04-23 正大天晴药业集团股份有限公司 修饰的双链核糖核酸

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US5624813A (en) 1994-04-21 1997-04-29 Mahant; Vijay K. NAD(P)+ /NAD(P)H based chemiluminescent diagnostics
WO1997020942A1 (en) * 1995-12-05 1997-06-12 Piao Yun Shang Hsd17b1 promoter, enhancer, silencer and use thereof
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6391311B1 (en) 1998-03-17 2002-05-21 Genentech, Inc. Polypeptides having homology to vascular endothelial cell growth factor and bone morphogenetic protein 1
DE60040274D1 (de) 1999-03-10 2008-10-30 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
FR2801218B1 (fr) 1999-11-23 2001-12-28 Hoechst Marion Roussel Inc Compositions pharmaceutiques comprenant de la trimegestone, leurs procedes de preparation ainsi que le conditionnement primaire les renfermant
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US7754709B2 (en) 2003-06-10 2010-07-13 Solvay Pharmaceuticals Bv Tetracyclic thiophenepyrimidinone compounds as inhibitors of 17β hydroxysteroid dehydrogenase compounds
US20080299042A1 (en) 2004-04-30 2008-12-04 Biogen Idec Ma Inc. Membrane Associated Molecules
WO2005116204A1 (ja) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
FR2904000A1 (fr) 2006-07-19 2008-01-25 Galderma Res & Dev S N C Snc Modulateurs de hsd17b7 dans le traitement de l'acne ou de l'hyperseborrhee
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
WO2010028110A2 (en) 2008-09-04 2010-03-11 Board Of Regents, The University Of Texas System Sequence variations in pnpla3 associated with hepatic steatosis
SI2539451T1 (sl) 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
WO2013032829A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
CA2842041A1 (en) 2012-04-18 2013-10-24 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
US9677138B2 (en) 2012-05-20 2017-06-13 Trustees Of Boston University Methods and systems for monitoring, diagnosing, and treating chronic obstructive pulmonary disease
EP2864791A2 (en) 2012-06-20 2015-04-29 Leibniz - Institut für Analytische Wissenschaften - ISAS - E.V. Specific biomarkers for hepatocellular carcinoma (hcc)
CN103520724B (zh) * 2013-10-23 2016-05-25 江苏美迪森生物医药有限公司 Hsd17b13蛋白或其编码基因的抑制剂的新用途
CA2954791C (en) 2014-07-14 2025-11-18 The Regents Of The University Of California CRISPR/CAS TRANSCRIPTIONAL MODULATION
JP2017535552A (ja) 2014-11-17 2017-11-30 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用方法
CN104698108B (zh) 2015-03-26 2016-11-09 中国药科大学 一种利用固定化酶筛选I型17β羟类固醇脱氢酶抑制剂的方法
WO2017048620A1 (en) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof
JOP20160211B1 (ar) 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
CA3011946A1 (en) * 2016-03-07 2017-09-14 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
SG11201901841TA (en) 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
AU2017378427A1 (en) 2016-12-14 2019-06-20 Ligandal, Inc. Methods and compositions for nucleic acid and protein payload delivery
CN110268060B (zh) 2017-01-10 2024-07-26 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
US10533256B2 (en) 2017-10-09 2020-01-14 GKN Aerospace Transparency Systems, Inc. Hydrophobic coatings for metals incorporating anodic and rare-earth oxides and methods of applying same
AU2018348195B2 (en) 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
US20210000906A1 (en) 2018-03-21 2021-01-07 Ionis Pharmaceuticals, Inc. Modulation of hsd17b13 expression
AU2019239971B2 (en) * 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
KR102769565B1 (ko) 2018-09-19 2025-02-20 애로우헤드 파마슈티컬스 인코포레이티드 17베타-HSD 유형 13- (HSD17B13)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
US20240002857A1 (en) * 2022-05-09 2024-01-04 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof

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