CN113825839A - 用固醇调节元件结合蛋白切割激活蛋白(scap)抑制剂治疗脂质水平升高 - Google Patents

用固醇调节元件结合蛋白切割激活蛋白(scap)抑制剂治疗脂质水平升高 Download PDF

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Publication number
CN113825839A
CN113825839A CN202080036542.8A CN202080036542A CN113825839A CN 113825839 A CN113825839 A CN 113825839A CN 202080036542 A CN202080036542 A CN 202080036542A CN 113825839 A CN113825839 A CN 113825839A
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China
Prior art keywords
seq
scap
positions
nucleotide sequence
nucleic acid
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CN202080036542.8A
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English (en)
Chinese (zh)
Inventor
龚大为
J·A·佩里
A·舒尔迪纳
N·戈萨利亚
C·范霍特
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Regeneron Pharmaceuticals Inc
University of Maryland Baltimore
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Regeneron Pharmaceuticals Inc
University of Maryland Baltimore
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Publication of CN113825839A publication Critical patent/CN113825839A/zh
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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CN202080036542.8A 2019-03-20 2020-03-19 用固醇调节元件结合蛋白切割激活蛋白(scap)抑制剂治疗脂质水平升高 Pending CN113825839A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962820895P 2019-03-20 2019-03-20
US62/820,895 2019-03-20
PCT/US2020/023564 WO2020191163A1 (en) 2019-03-20 2020-03-19 Treatment of increased lipid levels with sterol regulatory element binding protein cleavage-activating protein (scap) inhibitors

Publications (1)

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CN113825839A true CN113825839A (zh) 2021-12-21

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CN202080036542.8A Pending CN113825839A (zh) 2019-03-20 2020-03-19 用固醇调节元件结合蛋白切割激活蛋白(scap)抑制剂治疗脂质水平升高

Country Status (6)

Country Link
US (2) US11331332B2 (https=)
EP (1) EP3942047A1 (https=)
JP (1) JP2022525546A (https=)
CN (1) CN113825839A (https=)
CA (1) CA3133002A1 (https=)
WO (1) WO2020191163A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117757790A (zh) * 2022-11-17 2024-03-26 北京福元医药股份有限公司 抑制SCAP基因表达的siRNA、其缀合物和药物组合物及用途

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017100542A1 (en) * 2015-12-10 2017-06-15 Alnylam Pharmaceuticals, Inc. Sterol regulatory element binding protein (srebp) chaperone (scap) irna compositions and methods of use thereof

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5688787A (en) 1991-07-23 1997-11-18 Schering Corporation Substituted β-lactam compounds useful as hypochlesterolemic agents and processes for the preparation thereof
US5631356A (en) 1992-04-03 1997-05-20 Gist-Brocades, N.V. Selective N-acylation of amino alcohols
LT3595B (en) 1993-01-21 1995-12-27 Schering Corp Spirocycloalkyl-substituted azetidinones useful as hypocholesterolemic agents
US5631365A (en) 1993-09-21 1997-05-20 Schering Corporation Hydroxy-substituted azetidinone compounds useful as hypocholesterolemic agents
FR2710337B1 (fr) 1993-09-23 1995-12-08 Gerard Coquerel Procédé de dédoublement de deux antipodes optiques par entraînement polythermique programmé et autoensemencé.
US5624920A (en) 1994-11-18 1997-04-29 Schering Corporation Sulfur-substituted azetidinone compounds useful as hypocholesterolemic agents
US5633246A (en) 1994-11-18 1997-05-27 Schering Corporation Sulfur-substituted azetidinone compounds useful as hypocholesterolemic agents
US5656624A (en) 1994-12-21 1997-08-12 Schering Corporation 4-[(heterocycloalkyl or heteroaromatic)-substituted phenyl]-2-azetidinones useful as hypolipidemic agents
US5756470A (en) 1996-10-29 1998-05-26 Schering Corporation Sugar-substituted 2-azetidinones useful as hypocholesterolemic agents
HRP20030498A2 (en) 2000-12-21 2005-04-30 Aventis Pharma Deutschland Gmbh Novel 1,2-diphenzylazetidinones, method for producing the same, medicaments containing said compounds, and the use thereof for treating disorders of the lipid metabolism
TWI291957B (en) 2001-02-23 2008-01-01 Kotobuki Pharmaceutical Co Ltd Beta-lactam compounds, process for repoducing the same and serum cholesterol-lowering agents containing the same
US20080249040A1 (en) 2001-05-18 2008-10-09 Sirna Therapeutics, Inc. RNA interference mediated inhibition of sterol regulatory element binding protein 1 (SREBP1) gene expression using short interfering nucleic acid (siNA)
US20050014177A1 (en) 2003-05-19 2005-01-20 Koustubh Ranade Human sterol response element binding protein 1 (SREBP1) single nucleotide polymorphisms
AU2007297388A1 (en) * 2006-09-18 2008-03-27 Alnylam Pharmaceuticals, Inc. RNAi modulation of scap and therapeutic uses thereof
JP2018507234A (ja) * 2015-03-04 2018-03-15 メディベイション テクノロジーズ エルエルシー 肝臓線維症、コレステロールの上昇およびインスリン抵抗性を処置する際における使用のためのｓｒｅｂｐ遮断薬
WO2019141723A1 (en) 2018-01-18 2019-07-25 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting srebp1

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017100542A1 (en) * 2015-12-10 2017-06-15 Alnylam Pharmaceuticals, Inc. Sterol regulatory element binding protein (srebp) chaperone (scap) irna compositions and methods of use thereof

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WO2020191163A1 (en) 2020-09-24
CA3133002A1 (en) 2020-09-24
US20200297753A1 (en) 2020-09-24
US20220323483A1 (en) 2022-10-13
EP3942047A1 (en) 2022-01-26
US11331332B2 (en) 2022-05-17
JP2022525546A (ja) 2022-05-17

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