TW202500748A - 用於調節pnpla3表現之組合物及方法 - Google Patents

用於調節pnpla3表現之組合物及方法 Download PDF

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Publication number
TW202500748A
TW202500748A TW113135289A TW113135289A TW202500748A TW 202500748 A TW202500748 A TW 202500748A TW 113135289 A TW113135289 A TW 113135289A TW 113135289 A TW113135289 A TW 113135289A TW 202500748 A TW202500748 A TW 202500748A
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seq
nucleotides
antisense strand
oligonucleotide
sense strand
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TW113135289A
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Chinese (zh)
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烏薩夫 薩克森納
亨立克 Ｔ 杜迪克
馬克 艾布蘭
安東 杜拉諾
鮑伯 戴爾 布朗
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美商戴瑟納製藥股份有限公司
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1137—Non-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 against enzymes
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Liquid Crystal Substances (AREA)
TW113135289A 2021-04-14 2022-04-13 用於調節pnpla3表現之組合物及方法 TW202500748A (zh)

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US202163174932P 2021-04-14 2021-04-14
US63/174,932 2021-04-14

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TW111114009A TWI857290B (zh) 2021-04-14 2022-04-13 用於調節ｐｎｐｌａ３表現之組合物及方法

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US (2) US12084662B2 (enExample)
EP (1) EP4323519A1 (enExample)
JP (1) JP7877355B2 (enExample)
KR (1) KR20230171431A (enExample)
CN (1) CN117120613A (enExample)
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AU (1) AU2022258459A1 (enExample)
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CA (1) CA3209418A1 (enExample)
IL (1) IL307625A (enExample)
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Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230171431A (ko) 2021-04-14 2023-12-20 다이서나 파마수이티컬, 인크. Pnpla3 발현을 조절하기 위한 조성물 및 방법
EP4622982A1 (en) 2022-11-23 2025-10-01 Eli Lilly and Company Methods of synthesizing a targeting ligand-conjugated nucleotide phosphoramidite
JP7812495B2 (ja) 2022-11-23 2026-02-09 イーライ リリー アンド カンパニー ４’－ホスフェート類似体ヌクレオチドホスホラミダイトを合成する方法
WO2024182446A2 (en) * 2023-02-28 2024-09-06 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
WO2026130520A1 (zh) * 2024-12-20 2026-06-25 正大天晴药业集团股份有限公司 靶向pnpla3的双链核糖核酸

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
EP1253199A1 (en) 1993-09-02 2002-10-30 Ribozyme Pharmaceuticals, Inc. Abasic moieties containing enzymatic nucleic acids
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
US7259150B2 (en) 2001-08-07 2007-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein (a) expression
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
WO2010033225A2 (en) 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
WO2010039543A2 (en) 2008-09-23 2010-04-08 Traversa Therapeutics, Inc. Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference
KR20110110776A (ko) 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
AU2012328680A1 (en) 2011-10-25 2014-05-01 Ionis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
EP2928877B1 (en) 2012-12-06 2020-01-22 Merck Sharp & Dohme Corp. Disulfide masked prodrug compositions and methods
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
AU2014360314B2 (en) 2013-12-06 2018-04-26 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of transthyretin (TTR) by double-stranded RNA
CN107109405A (zh) 2014-06-06 2017-08-29 索尔斯蒂斯生物有限公司 具有生物可逆性和非生物可逆性基团的多核苷酸构建体
EP3865576A1 (en) 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
JP2018510621A (ja) 2015-02-13 2018-04-19 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． パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
MX2019002075A (es) 2016-08-23 2019-07-01 Dicerna Pharmaceuticals Inc Composiciones que comprenden oligonucleotidos modificados reversiblemente, y sus usos.
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
CA3065523A1 (en) * 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US10675295B2 (en) * 2017-06-29 2020-06-09 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting HMGB1 expression
AR113490A1 (es) 2017-12-12 2020-05-06 Amgen Inc CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS
TWI869213B (zh) 2018-09-19 2025-01-01 美商Ionis製藥公司 Pnpla3表現之調節劑
CA3121510A1 (en) * 2018-12-10 2020-06-18 Amgen Inc. Rnai constructs for inhibiting pnpla3 expression and methods of use thereof
US20220389430A1 (en) 2019-10-02 2022-12-08 Dicema Pharmaceuticals, Inc. Chemical modifications of small interfering rna with minimal fluorine content
KR20230171431A (ko) 2021-04-14 2023-12-20 다이서나 파마수이티컬, 인크. Pnpla3 발현을 조절하기 위한 조성물 및 방법

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JP2024514880A (ja) 2024-04-03
US12084662B2 (en) 2024-09-10
MX2023012216A (es) 2023-10-26
TW202300645A (zh) 2023-01-01
IL307625A (en) 2023-12-01
KR20230171431A (ko) 2023-12-20
AU2022258459A1 (en) 2023-09-14
US20220364098A1 (en) 2022-11-17
CN117120613A (zh) 2023-11-24
AR125351A1 (es) 2023-07-12
US20240401059A1 (en) 2024-12-05
EP4323519A1 (en) 2024-02-21
WO2022221430A1 (en) 2022-10-20
BR112023021109A2 (pt) 2023-12-12
TWI857290B (zh) 2024-10-01
JP7877355B2 (ja) 2026-06-22
CA3209418A1 (en) 2022-10-20

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