JP7763104B2 - ＳＣＡＰ発現を阻害するためのＲＮＡｉコンストラクト及びその使用方法 - Google Patents

ＳＣＡＰ発現を阻害するためのＲＮＡｉコンストラクト及びその使用方法

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JP7763104B2
JP7763104B2 JP2021570170A JP2021570170A JP7763104B2 JP 7763104 B2 JP7763104 B2 JP 7763104B2 JP 2021570170 A JP2021570170 A JP 2021570170A JP 2021570170 A JP2021570170 A JP 2021570170A JP 7763104 B2 JP7763104 B2 JP 7763104B2
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scap
rnai construct
seq
nucleotides
expression
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JP2022534402A (ja
JP2022534402A5 (https=
JPWO2020243702A5 (https=
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ダス，アムリタ
ハーバリッチ，ブラッドリー・ジェイ
マレー，ジャスティン・ケイ
ホーマン，オリバー
オルマン，マイケル
リン，ダニエル・シー・エイチ
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アムジエン・インコーポレーテツド
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JP2021570170A 2019-05-30 2020-06-01 ＳＣＡＰ発現を阻害するためのＲＮＡｉコンストラクト及びその使用方法 Active JP7763104B2 (ja)

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US201962854433P 2019-05-30 2019-05-30
US62/854,433 2019-05-30
PCT/US2020/035545 WO2020243702A2 (en) 2019-05-30 2020-06-01 Rnai constructs for inhibiting scap expression and methods of use thereof

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JP2025176137A Pending JP2026016500A (ja) 2019-05-30 2025-10-20 ＳＣＡＰ発現を阻害するためのＲＮＡｉコンストラクト及びその使用方法

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US (1) US20220307022A1 (https=)
EP (1) EP3976786A2 (https=)
JP (2) JP7763104B2 (https=)
KR (1) KR20220016138A (https=)
CN (2) CN113924368B (https=)
AR (1) AR119061A1 (https=)
AU (1) AU2020284254A1 (https=)
BR (1) BR112021024080A2 (https=)
CA (1) CA3141902A1 (https=)
CL (2) CL2021003169A1 (https=)
EA (1) EA202193296A1 (https=)
IL (1) IL288398A (https=)
MX (2) MX2021014465A (https=)
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Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20250005108A (ko) * 2022-04-15 2025-01-09 다이서나 파마수이티컬, 인크. Scap 활성을 조절하기 위한 조성물 및 방법
US20250313834A1 (en) * 2022-05-25 2025-10-09 Amgen Inc. Rnai constructs for inhibiting scap expression and methods of use thereof
IL317824A (en) * 2022-07-11 2025-02-01 Sanegene Bio Usa Inc Ribose derivatives modified at the '2' position and methods of use
AU2023313321A1 (en) * 2022-07-27 2025-01-09 E-Therapeutics Plc Nucleic acid compounds
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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010503415A (ja) 2006-09-18 2010-02-04 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 ＳＣＡＰのＲＮＡｉ調節およびその治療的使用
WO2018039647A1 (en) 2016-08-26 2018-03-01 Amgen Inc. Rnai constructs for inhibiting asgr1 expression and methods of use thereof
JP2018536689A (ja) 2015-12-10 2018-12-13 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． ステロール調節エレメント結合タンパク質（ＳＲＥＢＰ）シャペロン（ＳＣＡＰ）ｉＲＮＡ組成物およびその使用方法

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5744101A (en) 1989-06-07 1998-04-28 Affymax Technologies N.V. Photolabile nucleoside protecting groups
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
DE69233501T2 (de) 1991-11-22 2006-02-23 Affymetrix, Inc. (n.d.Ges.d.Staates Delaware), Santa Clara Kombinatorische Strategien für die Polymersynthese
US5981505A (en) 1993-01-26 1999-11-09 The Trustees Of The University Of Pennsylvania Compositions and methods for delivery of genetic material
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US5556752A (en) 1994-10-24 1996-09-17 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
US5840710A (en) 1994-12-09 1998-11-24 Genzyme Corporation Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5545531A (en) 1995-06-07 1996-08-13 Affymax Technologies N.V. Methods for making a device for concurrently processing multiple biological chip assays
ATE285477T1 (de) 1995-06-07 2005-01-15 Inex Pharmaceutical Corp Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US6217900B1 (en) 1997-04-30 2001-04-17 American Home Products Corporation Vesicular complexes and methods of making and using the same
DE69834038D1 (de) 1997-07-01 2006-05-18 Isis Pharmaceutical Inc Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre
WO2000003683A2 (en) 1998-07-20 2000-01-27 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US6693187B1 (en) 2000-10-17 2004-02-17 Lievre Cornu Llc Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2003093449A2 (en) 2002-05-06 2003-11-13 Nucleonics, Inc. Methods for delivery of nucleic acids
US8017762B2 (en) 2003-04-17 2011-09-13 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
DK1620544T3 (en) 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
CA2685127C (en) 2007-04-23 2019-01-08 Alnylam Pharmaceuticals, Inc. Glycoconjugates of rna interference agents
WO2009082607A2 (en) 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
AR090905A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
EP3564374A1 (en) 2013-06-21 2019-11-06 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
BR112019014282A2 (pt) * 2017-01-10 2020-03-03 Arrowhead Pharmaceuticals, Inc. Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010503415A (ja) 2006-09-18 2010-02-04 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 ＳＣＡＰのＲＮＡｉ調節およびその治療的使用
JP2018536689A (ja) 2015-12-10 2018-12-13 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． ステロール調節エレメント結合タンパク質（ＳＲＥＢＰ）シャペロン（ＳＣＡＰ）ｉＲＮＡ組成物およびその使用方法
WO2018039647A1 (en) 2016-08-26 2018-03-01 Amgen Inc. Rnai constructs for inhibiting asgr1 expression and methods of use thereof

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