JP2023515664A - 修飾された短い干渉核酸（ｓｉＮＡ）分子およびそれらの使用 - Google Patents

修飾された短い干渉核酸（ｓｉＮＡ）分子およびそれらの使用 Download PDF

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JP2023515664A
JP2023515664A JP2022552538A JP2022552538A JP2023515664A JP 2023515664 A JP2023515664 A JP 2023515664A JP 2022552538 A JP2022552538 A JP 2022552538A JP 2022552538 A JP2022552538 A JP 2022552538A JP 2023515664 A JP2023515664 A JP 2023515664A
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nucleotides
sina
nucleotide sequence
nucleotide
methyl
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バイゲルマン，レオニード
クマール ラジワンシ，ヴィヴェク
ホスバッハ，マルクス
ケー． パンデイ，ラジェンドラ
ホン，ジン
エルテプ，ラクスマン
マルティネス モンテロ，サウル
コスタ，エヌ．ティラニ エス． デ
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アリゴス セラピューティクス，インコーポレーテッド
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JP2022552538A 2020-03-06 2021-03-05 修飾された短い干渉核酸（ｓｉＮＡ）分子およびそれらの使用 Pending JP2023515664A (ja)

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US202062986150P 2020-03-06 2020-03-06
US62/986,150 2020-03-06
US202063109196P 2020-11-03 2020-11-03
US63/109,196 2020-11-03
PCT/US2021/021199 WO2021178885A1 (en) 2020-03-06 2021-03-05 Modified short interfering nucleic acid (sina) molecules and uses thereof

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Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA202090720A1 (ru) * 2017-09-14 2020-07-07 Янссен Байофарма, Инк. ПРОИЗВОДНЫЕ GalNAc
PE20221578A1 (es) * 2020-03-06 2022-10-06 Aligos Therapeutics Inc Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas
JP2023548031A (ja) 2020-10-21 2023-11-15 アリゴス セラピューティクス インコーポレイテッド 二環式化合物
WO2022184852A1 (en) * 2021-03-03 2022-09-09 Silence Therapeutics Gmbh Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell
US11957683B2 (en) 2021-06-18 2024-04-16 Aligos Therapeutics, Inc. Bicyclic compounds
IL312811A (en) 2021-11-16 2024-07-01 Shanghai Argo Biopharmaceutical Co Ltd Composition and method for inhibiting angiotensinogen (agt) protein expression
WO2023122317A2 (en) * 2021-12-23 2023-06-29 Aligos Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (SINA) MOLECULES FOR TARGETING β-CATENIN AND USES THEREOF
WO2023224979A1 (en) * 2022-05-16 2023-11-23 University Of Massachusetts Optimized sirna scaffolds
CN119421887A (zh) * 2022-06-14 2025-02-11 大睿生物医药科技(上海)有限公司 环膦酸酯修饰的核苷酸
WO2024033374A1 (en) 2022-08-11 2024-02-15 Syngenta Crop Protection Ag Novel arylcarboxamide or arylthioamide compounds
WO2024089216A1 (en) 2022-10-27 2024-05-02 Syngenta Crop Protection Ag Novel sulfur-containing heteroaryl carboxamide compounds
JP2026500298A (ja) 2022-12-15 2026-01-06 シンジェンタ クロップ プロテクション アクチェンゲゼルシャフト 殺有害生物剤として有用である新規な二環式カルボキサミド化合物
AR131541A1 (es) 2023-01-07 2025-04-09 Syngenta Crop Protection Ag Compuestos de carboxamida novedosos
EP4654973A1 (en) * 2023-01-26 2025-12-03 Aligos Therapeutics, Inc. Hepatitis b combination therapies
EP4665852A1 (en) * 2023-02-14 2025-12-24 Basecure Therapeutics Modified double stranded rna agents
WO2024254091A1 (en) * 2023-06-05 2024-12-12 Empirico Inc. Treatment of fgg related diseases and disorders
WO2025032129A1 (en) 2023-08-08 2025-02-13 Syngenta Crop Protection Ag Novel aminoindane and aminotetraline compounds
CN117024485B (zh) * 2023-08-11 2026-02-13 江苏申基生物科技有限公司 一种2’-OMe腺苷的合成方法
WO2025096970A2 (en) * 2023-11-03 2025-05-08 Basecure Therapeutics Llc Modified oligonucleotides and methods of use thereof
WO2025104032A1 (en) 2023-11-14 2025-05-22 Syngenta Crop Protection Ag Novel carboxamide compounds
WO2025109114A1 (en) 2023-11-24 2025-05-30 Syngenta Crop Protection Ag Novel carboxamide compounds
CN117534717A (zh) * 2024-01-09 2024-02-09 凯莱英生命科学技术(天津)有限公司 5′-(e)-乙烯基磷酸酯的合成方法
PY2501648A (es) 2024-01-12 2025-10-31 Syngenta Crop Protection Ag Nuevos compuestos de carboxamida
WO2025149637A1 (en) 2024-01-12 2025-07-17 Syngenta Crop Protection Ag Novel carboxamide compounds
WO2025201529A1 (en) * 2024-03-29 2025-10-02 Ractigen Therapeutics Compounds for synthesizing modified small activating rna molecules, modified small activating rna molecules and uses thereof
US20250368996A1 (en) 2024-05-14 2025-12-04 Aligos Therapeutics, Inc. Modified antisense oligonucleotides for treating hepatitis b virus
WO2025242321A1 (en) 2024-05-24 2025-11-27 Glaxosmithkline Intellectual Property (No.3) Limited Novel use
WO2026008750A1 (en) 2024-07-05 2026-01-08 Syngenta Crop Protection Ag Novel carboxamide compounds
CN120796266A (zh) * 2024-07-08 2025-10-17 北京悦康科创医药科技股份有限公司 一种降低hbv基因表达的反义寡核苷酸及其应用

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014527401A (ja) * 2011-06-30 2014-10-16 アローヘッド リサーチ コーポレイション Ｂ型肝炎ウイルスの遺伝子発現を阻害するための組成物および方法
JP2015502931A (ja) * 2011-11-18 2015-01-29 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． 修飾ＲＮＡｉ剤
WO2018185241A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
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
JP2019504112A (ja) * 2015-12-07 2019-02-14 ジェンザイム・コーポレーション Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物
WO2019113393A1 (en) * 2017-12-06 2019-06-13 Avidity Biosciences Llc Compositions and methods of treating muscle atrophy and myotonic dystrophy
WO2019217459A1 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009002944A1 (en) * 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
CN102753187A (zh) * 2009-06-03 2012-10-24 戴瑟纳制药公司 肽-dicer底物试剂及其特异性抑制基因表达的方法
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
CN106103463B (zh) * 2014-02-18 2018-11-30 国立大学法人大阪大学 桥连型核苷和核苷酸
AU2016306275A1 (en) * 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
SG11201901841TA (en) 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
HUE059718T2 (hu) 2016-09-02 2022-12-28 Dicerna Pharmaceuticals Inc 4'-foszfát analógok és azokat tartalmazó oligonukleotidok
DK3719128T3 (da) * 2017-12-01 2025-03-03 Suzhou Ribo Life Science Co Ltd Dobbelt-strenget oligonukleotid, sammensætning og konjugat bestaende dobbelt-strenget oligonukleotid, fremgangsmåde herfor og anvendelse heraf
WO2019217397A2 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for improving strand biased
PE20211783A1 (es) 2018-11-08 2021-09-08 Aligos Therapeutics Inc Polimeros oligonucleotidos que inhiben el transporte de antigenos y metodos
PE20221578A1 (es) * 2020-03-06 2022-10-06 Aligos Therapeutics Inc Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014527401A (ja) * 2011-06-30 2014-10-16 アローヘッド リサーチ コーポレイション Ｂ型肝炎ウイルスの遺伝子発現を阻害するための組成物および方法
JP2015502931A (ja) * 2011-11-18 2015-01-29 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． 修飾ＲＮＡｉ剤
JP2019504112A (ja) * 2015-12-07 2019-02-14 ジェンザイム・コーポレーション Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物
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
WO2018185241A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
WO2019113393A1 (en) * 2017-12-06 2019-06-13 Avidity Biosciences Llc Compositions and methods of treating muscle atrophy and myotonic dystrophy
WO2019217459A1 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NUCLEIC ACIDS RESEARCH, vol. 45, no. 6, JPN6025013423, 2017, pages 3528 - 3536, ISSN: 0005717369 *

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