CL2020002401A1 - Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas. - Google Patents

Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas.

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Publication number
CL2020002401A1
CL2020002401A1 CL2020002401A CL2020002401A CL2020002401A1 CL 2020002401 A1 CL2020002401 A1 CL 2020002401A1 CL 2020002401 A CL2020002401 A CL 2020002401A CL 2020002401 A CL2020002401 A CL 2020002401A CL 2020002401 A1 CL2020002401 A1 CL 2020002401A1
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CL
Chile
Prior art keywords
hsd17b13
compositions
methods
arni
hydroxysteroid dehydrogenases
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CL2020002401A
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English (en)
Inventor
Jesper Gromada
Gregory Hinkle
Frederic Tremblay
Noura S Abul-Husn
Omri Gottesman
Alexander Li
Xiping Cheng
Yurong Xin
Frederick E Dewey
Aris Baras
Alan Shuldiner
Stuart Milstein
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Regeneron Pharma
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Publication date
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of CL2020002401A1 publication Critical patent/CL2020002401A1/es

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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
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Abstract

Composiciones de ácidos ribonucleicos de hebra doble (ARNhd) dirigidos al gen HSD17B13, así como métodos para inhibir la expresión de HSD17B13, y métodos para tratar a sujetos que pudieran verse beneficiados con una reducción de la expresión de HSD17B13, tales como sujetos que padecen una enfermedad, trastorno o condición asociada con HSD17B13, mediante el uso de dichas composiciones de ARNhd.
CL2020002401A 2018-03-21 2020-09-16 Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas. CL2020002401A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862645941P 2018-03-21 2018-03-21
US201862770298P 2018-11-21 2018-11-21
US201862775590P 2018-12-05 2018-12-05

Publications (1)

Publication Number Publication Date
CL2020002401A1 true CL2020002401A1 (es) 2021-01-29

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Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020002401A CL2020002401A1 (es) 2018-03-21 2020-09-16 Composiciones de arni de 17 b-hidroxiesteroide deshidrogenasas tipo 13 (hsd17b13) y métodos de uso de las mismas.

Country Status (16)

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US (2) US20210380985A1 (es)
EP (1) EP3768838A1 (es)
JP (1) JP7507093B2 (es)
KR (1) KR20200133373A (es)
CN (1) CN112020556A (es)
AU (1) AU2019239971A1 (es)
BR (1) BR112020018758A2 (es)
CA (1) CA3091146A1 (es)
CL (1) CL2020002401A1 (es)
CO (1) CO2020010342A2 (es)
IL (1) IL277329A (es)
MX (1) MX2020009812A (es)
PH (1) PH12020500622A1 (es)
SG (1) SG11202007583SA (es)
TW (1) TW202003848A (es)
WO (1) WO2019183164A1 (es)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102619197B1 (ko) 2017-01-23 2024-01-03 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
AU2018250727A1 (en) 2017-04-11 2019-10-31 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (HSD17B) family
EP3695012B1 (en) 2017-10-11 2023-03-08 Regeneron Pharmaceuticals, Inc. Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation
CN112020556A (zh) 2018-03-21 2020-12-01 瑞泽恩制药公司 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法
CN113164509A (zh) * 2018-09-19 2021-07-23 箭头药业股份有限公司 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法
US20220273691A1 (en) 2018-12-21 2022-09-01 Ionis Pharmaceuticals, Inc. Modulators of hsd17b13 expression
MX2022015169A (es) * 2020-06-01 2023-07-03 Amgen Inc Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos.
WO2022098748A1 (en) * 2020-11-06 2022-05-12 Inipharm, Inc. Uses for hsd17b13 inhibitors
CA3201624A1 (en) 2020-12-23 2022-06-30 Regeneron Pharmaceuticals, Inc. Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors
TW202302852A (zh) * 2021-04-22 2023-01-16 大陸商上海拓界生物醫藥科技有限公司 靶向第13型17β-羥基類固醇脫氫酶的siRNA和siRNA綴合物
CA3230382A1 (en) * 2021-09-08 2023-03-16 Aligos Therapeutics, Inc. Modified short interfering nucleic acid (sina) molecules and uses thereof
WO2023109945A1 (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
WO2023208109A1 (zh) * 2022-04-29 2023-11-02 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
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
WO2024059165A1 (en) * 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5624813A (en) 1994-04-21 1997-04-29 Mahant; Vijay K. NAD(P)+ /NAD(P)H based chemiluminescent diagnostics
AU1067697A (en) 1995-12-05 1997-06-27 Jouko Antero Oikarinen Hsd17b1 promoter, enhancer, silencer and use thereof
US6391311B1 (en) 1998-03-17 2002-05-21 Genentech, Inc. Polypeptides having homology to vascular endothelial cell growth factor and bone morphogenetic protein 1
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
US20050119211A1 (en) * 2001-05-18 2005-06-02 Sirna Therapeutics, Inc. RNA mediated inhibition connexin gene expression using short interfering nucleic acid (siNA)
US20050182007A1 (en) * 2001-05-18 2005-08-18 Sirna Therapeutics, Inc. RNA interference mediated inhibition of interleukin and interleukin receptor gene expression using short interfering nucleic acid (SINA)
WO2005014811A2 (en) * 2003-08-08 2005-02-17 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF XIAP GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
JP2006507841A (ja) * 2002-11-14 2006-03-09 ダーマコン, インコーポレイテッド 機能的siRNAおよび超機能的siRNA
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
US7754709B2 (en) 2003-06-10 2010-07-13 Solvay Pharmaceuticals Bv Tetracyclic thiophenepyrimidinone compounds as inhibitors of 17β hydroxysteroid dehydrogenase compounds
WO2005108415A2 (en) 2004-04-30 2005-11-17 Biogen Idec Ma Inc. Membrane associated molecules
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
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
EP2087110A2 (en) * 2006-10-11 2009-08-12 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Influenza targets
US20100056384A1 (en) 2008-09-04 2010-03-04 Board Of Regents, The University Of Texas System Sequence Variations in PNPLA3 Associated with Hepatic Steatosis
WO2013177060A2 (en) 2012-05-20 2013-11-28 Trustees Of Boston University Methods and systems for monitoring, diagnosing, and treating chronic obstructive polmonary 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
EP3256587A2 (en) * 2015-02-13 2017-12-20 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
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
EP3350328A1 (en) 2015-09-14 2018-07-25 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof
TWI788312B (zh) * 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
KR20210035022A (ko) 2016-12-14 2021-03-31 리간달 인코포레이티드 핵산 및/또는 단백질 적재물 전달을 위한 조성물 및 방법
KR102619197B1 (ko) 2017-01-23 2024-01-03 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
CN112020556A (zh) 2018-03-21 2020-12-01 瑞泽恩制药公司 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法
US20210000906A1 (en) 2018-03-21 2021-01-07 Ionis Pharmaceuticals, Inc. Modulation of hsd17b13 expression
CN113164509A (zh) * 2018-09-19 2021-07-23 箭头药业股份有限公司 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法
EP4055167A2 (en) * 2019-11-08 2022-09-14 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides targeting bromodomain containing protein 4 (brd4) for immunotherapy
EP4110919A4 (en) * 2020-02-28 2024-06-05 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
AU2021373262A1 (en) * 2020-11-04 2023-06-01 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of pros1 in a cell

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TW202003848A (zh) 2020-01-16
AU2019239971A1 (en) 2020-09-03
EP3768838A1 (en) 2021-01-27
KR20200133373A (ko) 2020-11-27
US11180757B1 (en) 2021-11-23
WO2019183164A1 (en) 2019-09-26
BR112020018758A2 (pt) 2021-01-26
MX2020009812A (es) 2021-01-08
CA3091146A1 (en) 2019-09-26
JP2021518125A (ja) 2021-08-02
SG11202007583SA (en) 2020-09-29
JP7507093B2 (ja) 2024-06-27
CO2020010342A2 (es) 2020-08-31
IL277329A (en) 2020-10-29
PH12020500622A1 (en) 2021-03-15
US20210380985A1 (en) 2021-12-09
CN112020556A (zh) 2020-12-01

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