TWI886314B - 抑制ｌｐａ表現之組合物及方法 - Google Patents

抑制ｌｐａ表現之組合物及方法 Download PDF

Info

Publication number
TWI886314B
TWI886314B TW110128888A TW110128888A TWI886314B TW I886314 B TWI886314 B TW I886314B TW 110128888 A TW110128888 A TW 110128888A TW 110128888 A TW110128888 A TW 110128888A TW I886314 B TWI886314 B TW I886314B
Authority
TW
Taiwan
Prior art keywords
lpa
oligonucleotide
galnac
nucleotides
seq
Prior art date
Application number
TW110128888A
Other languages
English (en)
Chinese (zh)
Other versions
TW202220673A (zh
Inventor
馬克 艾布蘭
包柏 戴爾 布朗
亨立克 Ｔ 杜迪克
韓雯
安東 杜拉諾
Original Assignee
美商戴瑟納製藥股份有限公司
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 美商戴瑟納製藥股份有限公司 filed Critical 美商戴瑟納製藥股份有限公司
Publication of TW202220673A publication Critical patent/TW202220673A/zh
Application granted granted Critical
Publication of TWI886314B publication Critical patent/TWI886314B/zh

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21007—Plasmin (3.4.21.7), i.e. fibrinolysin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
TW110128888A 2020-08-05 2021-08-05 抑制ｌｐａ表現之組合物及方法 TWI886314B (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202063061676P 2020-08-05 2020-08-05
US63/061,676 2020-08-05
US202063074779P 2020-09-04 2020-09-04
US63/074,779 2020-09-04

Publications (2)

Publication Number Publication Date
TW202220673A TW202220673A (zh) 2022-06-01
TWI886314B true TWI886314B (zh) 2025-06-11

Family

ID=77519885

Family Applications (2)

Application Number Title Priority Date Filing Date
TW114118397A TW202535432A (zh) 2020-08-05 2021-08-05 抑制lpa表現之組合物及方法
TW110128888A TWI886314B (zh) 2020-08-05 2021-08-05 抑制ｌｐａ表現之組合物及方法

Family Applications Before (1)

Application Number Title Priority Date Filing Date
TW114118397A TW202535432A (zh) 2020-08-05 2021-08-05 抑制lpa表現之組合物及方法

Country Status (12)

Country Link
US (2) US12435336B2 (enExample)
EP (1) EP4192954A1 (enExample)
JP (2) JP7476422B2 (enExample)
KR (2) KR20250077604A (enExample)
CN (1) CN116323943A (enExample)
AU (2) AU2021320550B2 (enExample)
BR (1) BR112023001957A2 (enExample)
CA (1) CA3185348A1 (enExample)
IL (1) IL300338A (enExample)
MX (1) MX2023001538A (enExample)
TW (2) TW202535432A (enExample)
WO (1) WO2022032288A1 (enExample)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4192954A1 (en) 2020-08-05 2023-06-14 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting lpa expression
EP4450624A4 (en) * 2021-12-16 2025-03-26 Tuojie Biotech (Shanghai) Co., Ltd. LPA-TARGETTING SIRNA AND CONJUGATE
CN114703184B (zh) * 2022-03-11 2024-06-18 厦门甘宝利生物医药有限公司 Lpa抑制剂及其用途
CA3245064A1 (en) * 2022-04-15 2023-10-19 Dicerna Pharmaceuticals, Inc. COMPOSITIONS AND METHODS OF MODULATING THE ACTIVITY OF STRUCTURAL AND CYTOSKELETON-ASSOCIATED PROTEINS
CN120076811A (zh) * 2022-08-11 2025-05-30 靖因药业有限公司 用于抑制Lp(a)的表达的多核酸分子、药物组合物及其用途
CN115851723B (zh) * 2022-10-24 2023-10-03 厦门甘宝利生物医药有限公司 一种抑制lpa基因表达的rna抑制剂及其应用
WO2024112865A1 (en) 2022-11-23 2024-05-30 Eli Lilly And Company Method of synthesizing 4'-phosphate analog nucleotide phosphoramidite
KR20250099265A (ko) 2022-11-23 2025-07-01 일라이 릴리 앤드 캄파니 표적화 리간드-접합된 뉴클레오티드 포스포르아미다이트의 합성 방법
AU2023408776A1 (en) 2022-12-22 2025-07-10 Eli Lilly And Company Oligonucleotide fragments and methods of making rnai agents using the same
KR20250131826A (ko) 2023-01-13 2025-09-03 일라이 릴리 앤드 캄파니 치료 올리고뉴클레오티드-함유 제약 조성물 및 그를 사용한 투여 요법
WO2024197065A2 (en) * 2023-03-20 2024-09-26 Verve Therapeutics, Inc. In vivo nickase-based editing of the lpa gene for treatment of cardiovascular disease
CN118685405A (zh) * 2023-06-20 2024-09-24 施能康医药科技(苏州)有限公司 靶向脂蛋白a的核酸及其用途
WO2025064821A2 (en) 2023-09-21 2025-03-27 Ionis Pharmaceuticals, Inc. Compounds and methods for inhibiting lpa
WO2025082058A1 (zh) * 2023-10-16 2025-04-24 润佳(上海)医药技术有限公司 一种抑制LPA基因表达的dsRNA及其用途
CN120344664A (zh) * 2023-10-27 2025-07-18 北京安龙生物医药有限公司 靶向脂蛋白a的寡核苷酸及其用途
CN120020252A (zh) * 2023-11-17 2025-05-20 纳肽得(青岛)生物医药有限公司 抑制LPA表达的siRNA及其用途
WO2025117709A2 (en) * 2023-11-29 2025-06-05 Sanegene Bio Usa Inc. Small interfering rna targeting lp(a) and uses thereof
WO2025119212A1 (zh) * 2023-12-05 2025-06-12 广东东阳光药业股份有限公司 一种新型的双链siRNA、其缀合物及其用途
WO2025170868A1 (en) * 2024-02-05 2025-08-14 Sirius Therapeutics, Inc. Polynucleic acid molecules for inhibiting expression of lp(a) or apoc3, pharmaceutical compositions, and uses thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017059223A2 (en) * 2015-10-01 2017-04-06 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of lpa
WO2019092283A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of lpa in a cell
WO2019118638A2 (en) * 2017-12-12 2019-06-20 Amgen Inc. Rnai constructs for inhibiting pnpla3 expression and methods of use thereof

Family Cites Families (26)

* 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
DE69431669T2 (de) 1993-09-02 2003-10-23 Ribozyme Pharmaceuticals, Inc. Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
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
DK2796553T3 (da) 2000-03-30 2019-09-30 Whitehead Inst Biomedical Res Rna-sekvensspecifikke formidlere af rna-interferens
CZ302719B6 (cs) 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
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)
EP1442137A4 (en) 2001-11-07 2005-08-31 Applera Corp 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
US20090018097A1 (en) 2005-09-02 2009-01-15 Mdrna, Inc Modification of double-stranded ribonucleic acid molecules
US20100331389A1 (en) 2008-09-22 2010-12-30 Bob Dale Brown Compositions and methods for the specific inhibition of gene expression by dsRNA containing modified nucleotides
US8691971B2 (en) 2008-09-23 2014-04-08 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference
US8513207B2 (en) 2008-12-18 2013-08-20 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
US8901098B2 (en) 2011-10-25 2014-12-02 Isis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
BR112015012840A2 (pt) 2012-12-06 2017-07-11 Merck Sharp & Dohme composto, composição, e, uso de um composto ou um sal, solvato, éster ou pró-droga farmaceuticamente aceitável do mesmo
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
JP7105065B2 (ja) 2014-12-15 2022-07-22 ダイセルナ ファーマシューティカルズ， インコーポレイテッド リガンド修飾二本鎖核酸
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
MX2022007005A (es) 2019-12-09 2022-08-25 Amgen Inc Construcciones de iarn y metodos para inhibir la expresion de lpa.
EP4192954A1 (en) 2020-08-05 2023-06-14 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting lpa expression

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017059223A2 (en) * 2015-10-01 2017-04-06 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of lpa
WO2019092283A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of lpa in a cell
WO2019118638A2 (en) * 2017-12-12 2019-06-20 Amgen Inc. Rnai constructs for inhibiting pnpla3 expression and methods of use thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
期刊 Yu, Rosie Z et al. Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys. Nucleic acid therapeutics vol. 26 Mary Ann Liebert, Inc. 2016 372-380 *

Also Published As

Publication number Publication date
TW202535432A (zh) 2025-09-16
US20240344063A1 (en) 2024-10-17
BR112023001957A2 (pt) 2023-04-25
AU2021320550A1 (en) 2023-02-16
MX2023001538A (es) 2023-03-08
KR102812602B1 (ko) 2025-05-28
EP4192954A1 (en) 2023-06-14
WO2022032288A1 (en) 2022-02-10
JP2023536974A (ja) 2023-08-30
CA3185348A1 (en) 2022-02-10
KR20250077604A (ko) 2025-05-30
US20240401053A1 (en) 2024-12-05
AU2021320550B2 (en) 2025-11-06
US12435336B2 (en) 2025-10-07
IL300338A (en) 2023-04-01
AU2025252633A1 (en) 2025-11-27
JP2024081668A (ja) 2024-06-18
US12252691B2 (en) 2025-03-18
CN116323943A (zh) 2023-06-23
KR20230043912A (ko) 2023-03-31
TW202220673A (zh) 2022-06-01
JP7476422B2 (ja) 2024-04-30

Similar Documents

Publication Publication Date Title
TWI886314B (zh) 抑制ｌｐａ表現之組合物及方法
TWI870563B (zh) 用於抑制angptl3表現之組合物及方法
US20250092403A1 (en) Compositions and methods for inhibiting ketohexokinase (khk)
TWI857290B (zh) 用於調節ｐｎｐｌａ３表現之組合物及方法
TWI868755B (zh) 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
US20230374522A1 (en) Compositions and methods for modulating scap activity
TW202330920A (zh) 用於調節ａｐｏｃ３表現之組合物及方法
EA050095B1 (ru) Композиции и способы ингибирования экспрессии angptl3
EA047953B1 (ru) Композиции для ингибирования экспрессии lpa и способы на их основе