CN110945130A - 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 - Google Patents

一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 Download PDF

Info

Publication number
CN110945130A
CN110945130A CN201880048600.1A CN201880048600A CN110945130A CN 110945130 A CN110945130 A CN 110945130A CN 201880048600 A CN201880048600 A CN 201880048600A CN 110945130 A CN110945130 A CN 110945130A
Authority
CN
China
Prior art keywords
sirna
nucleotide
seq
nucleotide sequence
group
Prior art date
Legal status (The legal status 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 status listed.)
Granted
Application number
CN201880048600.1A
Other languages
English (en)
Other versions
CN110945130B (zh
Inventor
张鸿雁
高山
康代武
孔丽娜
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Suzhou Ribo Life Science Co Ltd
Original Assignee
Suzhou Ribo Life Science Co Ltd
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 Suzhou Ribo Life Science Co Ltd filed Critical Suzhou Ribo Life Science Co Ltd
Publication of CN110945130A publication Critical patent/CN110945130A/zh
Application granted granted Critical
Publication of CN110945130B publication Critical patent/CN110945130B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Obesity (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

提供了一种抑制血管生成素样蛋白3(ANGPTL3)基因表达的siRNA,以及含有该siRNA的药物组合物和缀合物。该siRNA中的每个核苷酸各自独立地为修饰或未修饰的核苷酸,该siRNA含有正义链和反义链,所述正义链含有核苷酸序列A,所述核苷酸序列A与SEQ ID NO:1所示的核苷酸序列长度相等,且不多于3个核苷酸差异,所述反义链含有核苷酸序列B,所述核苷酸序列B与SEQ ID NO:2所示的核苷酸序列长度相等,且不多于3个核苷酸差异。

Description

PCT国内申请,说明书已公开。

Claims (75)

  1. PCT国内申请,权利要求书已公开。
CN201880048600.1A 2017-12-01 2018-11-29 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 Active CN110945130B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
CN2017112493569 2017-12-01
CN201711249356 2017-12-01
CN201711482915 2017-12-29
CN2017114829150 2017-12-29
PCT/CN2018/118191 WO2019105414A1 (zh) 2017-12-01 2018-11-29 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202410311316.6A Division CN118291457A (zh) 2017-12-01 2018-11-29 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途

Publications (2)

Publication Number Publication Date
CN110945130A true CN110945130A (zh) 2020-03-31
CN110945130B CN110945130B (zh) 2024-04-09

Family

ID=66664357

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201880048600.1A Active CN110945130B (zh) 2017-12-01 2018-11-29 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途

Country Status (6)

Country Link
US (2) US11414661B2 (zh)
EP (1) EP3719126A4 (zh)
JP (2) JP7261494B2 (zh)
CN (1) CN110945130B (zh)
TW (1) TWI810226B (zh)
WO (1) WO2019105414A1 (zh)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023284763A1 (zh) * 2021-07-16 2023-01-19 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
WO2024027518A1 (zh) * 2022-08-04 2024-02-08 北京福元医药股份有限公司 用于抑制angptl3基因表达的双链核糖核酸及其修饰物、缀合物和用途

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2923573T3 (es) 2011-06-21 2022-09-28 Alnylam Pharmaceuticals Inc Composiciones de ARNi de proteína 3 de tipo angiopoyetina (ANGPTL3) y métodos de uso de las mismas
CA3083968C (en) 2017-12-01 2024-04-23 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
WO2019105419A1 (zh) * 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
US11633482B2 (en) 2017-12-29 2023-04-25 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
WO2020038377A1 (zh) 2018-08-21 2020-02-27 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
JP7376952B2 (ja) 2018-09-30 2023-11-09 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド siRNA複合体及びその調製方法と使用
WO2021254360A1 (zh) * 2020-06-16 2021-12-23 上海拓界生物医药科技有限公司 一种碳水化合物分子簇及其制备方法和医药用途
CN114621951A (zh) * 2020-12-10 2022-06-14 施能康生物科技有限公司 靶向乙肝病毒的核酸及其用途
CN114763547A (zh) * 2021-01-14 2022-07-19 施能康生物科技有限公司 靶向血管紧张素原的核酸及其用途
WO2022187435A1 (en) * 2021-03-04 2022-09-09 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
CN115572241A (zh) * 2021-06-21 2023-01-06 润佳(苏州)医药科技有限公司 双价化合物、偶联物及其用途
US20230159930A1 (en) * 2021-11-19 2023-05-25 Sanegene Bio Usa Inc. Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof

Citations (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030206887A1 (en) * 1992-05-14 2003-11-06 David Morrissey RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA)
WO2005116204A1 (ja) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
CN102140458A (zh) * 2010-01-29 2011-08-03 苏州瑞博生物技术有限公司 小干扰核酸和药物组合物及其制药应用
CN102344477A (zh) * 2010-07-27 2012-02-08 苏州瑞博生物技术有限公司 一种核苷酸和/或寡核苷酸及其制备方法
US20120184595A1 (en) * 2009-01-26 2012-07-19 Protive Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
CN103890000A (zh) * 2011-06-21 2014-06-25 阿尔尼拉姆医药品有限公司 血管生成素样3(ANGPTL3)iRNA组合物及其使用方法
WO2015006740A2 (en) * 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
CN105392488A (zh) * 2013-05-01 2016-03-09 Isis制药公司 用于调节载脂蛋白c-iii表达的组合物和方法
WO2016206626A1 (zh) * 2015-06-26 2016-12-29 苏州瑞博生物技术有限公司 一种siRNA、含有该siRNA的药物组合物和缀合物及它们的应用
CN107075516A (zh) * 2014-08-20 2017-08-18 阿尔尼拉姆医药品有限公司 经修饰的双链rna试剂
CN107250362A (zh) * 2014-11-17 2017-10-13 阿尔尼拉姆医药品有限公司 载脂蛋白C3(APOC3)iRNA组合物及其使用方法
CN108064313A (zh) * 2015-03-17 2018-05-22 箭头药业股份有限公司 用于抑制因子xii的基因表达的组合物和方法

Family Cites Families (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン, インコーポレイテッド 機能的siRNAおよび超機能的siRNA
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
EP1791567B1 (en) 2004-08-10 2015-07-29 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
CA2910760C (en) 2007-12-04 2019-07-09 Muthiah Manoharan Targeting lipids
AU2014208251B2 (en) 2007-12-04 2016-07-14 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CN102083983B (zh) 2008-08-01 2014-04-16 苏州瑞博生物技术有限公司 乙型肝炎病毒基因的小核酸干扰靶位点序列和小干扰核酸及组合物和应用
US8975389B2 (en) 2009-03-02 2015-03-10 Alnylam Pharmaceuticals, Inc. Nucleic acid chemical modifications
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
AU2011203986C1 (en) 2010-01-08 2015-03-05 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
CN102140461B (zh) 2010-01-29 2012-12-05 苏州瑞博生物技术有限公司 小干扰核酸和药物组合物及其制药应用
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
CA2807307C (en) 2010-08-17 2021-02-09 Merck Sharp & Dohme Corp. Rna interference mediated inhibition of hepatitis b virus (hbv) gene expression using short interfering nucleic acid (sina)
US9290760B2 (en) 2010-09-15 2016-03-22 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
CN103380113B (zh) 2010-11-15 2018-03-30 生命科技公司 含胺的转染试剂及其制备和使用方法
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
KR102481317B1 (ko) 2011-03-29 2022-12-26 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
MX342731B (es) 2011-06-30 2016-10-11 Arrowhead Res Corp Composiciones y metodos para inhibir la expresion genica del virus de hepatitis b.
KR102272498B1 (ko) 2011-10-27 2021-07-06 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, n-말단 상에 관능화된 아미노산 유도체
CN104717982B (zh) 2012-08-06 2018-08-28 阿尔尼拉姆医药品有限公司 碳水化合物共轭物及其制备改进工艺
HUE035887T2 (en) 2012-12-05 2018-05-28 Alnylam Pharmaceuticals Inc PCSK9 iRNA preparations and methods for their use
SI2970974T1 (sl) 2013-03-14 2017-12-29 Alnylam Pharmaceuticals, Inc. Komplementarna komponenta C5 sestavkov IRNA in postopki njene uporabe
US10246708B2 (en) 2013-05-06 2019-04-02 Alnylam Pharmaceuticals, Inc. Dosages and methods for delivering lipid formulated nucleic acid molecules
EP3087183B1 (en) 2013-12-24 2020-08-26 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
EP3137091B1 (en) 2014-05-01 2020-12-02 Ionis Pharmaceuticals, Inc. Conjugates of modified antisense oligonucleotides and their use for modulating pkk expression
WO2015168589A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
US20170114341A1 (en) 2014-06-06 2017-04-27 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
WO2016154127A2 (en) * 2015-03-20 2016-09-29 Protiva Biotherapeutics, Inc. Compositions and methods for treating hypertriglyceridemia
CN115927335A (zh) 2015-04-13 2023-04-07 阿尔尼拉姆医药品有限公司 类血管生成素3(ANGPTL3)iRNA组合物及其使用方法
WO2016179342A2 (en) 2015-05-06 2016-11-10 Alnylam Pharmaceuticals, Inc. Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof
US20180216114A1 (en) 2015-07-27 2018-08-02 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof
KR20180038465A (ko) 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 B형 간염 바이러스 감염에 대한 rnai 치료법
CN114939124A (zh) 2015-08-25 2022-08-26 阿尔尼拉姆医药品有限公司 治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物
US10883107B2 (en) 2016-01-08 2021-01-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting factor XII (hageman factor) (F12) and methods of use thereof
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
EP3500581A4 (en) 2016-08-17 2021-10-06 Solstice Biologics, Ltd. POLYNUCLEOTIDE CONSTRUCTS
CN108220293B (zh) 2016-12-21 2021-09-24 苏州瑞博生物技术股份有限公司 一种小干扰核酸和药物组合物及其用途
CA3048661A1 (en) 2017-01-30 2018-08-02 Arrowhead Pharmaceuticals Inc. Compositions and methods for inhibition of factor xii gene expression
JP2022506517A (ja) 2018-11-02 2022-01-17 アルブータス・バイオファーマー・コーポレイション 標的指向化二価結合体

Patent Citations (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030206887A1 (en) * 1992-05-14 2003-11-06 David Morrissey RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA)
WO2005116204A1 (ja) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
US20120184595A1 (en) * 2009-01-26 2012-07-19 Protive Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
CN102140458A (zh) * 2010-01-29 2011-08-03 苏州瑞博生物技术有限公司 小干扰核酸和药物组合物及其制药应用
CN102344477A (zh) * 2010-07-27 2012-02-08 苏州瑞博生物技术有限公司 一种核苷酸和/或寡核苷酸及其制备方法
CN103890000A (zh) * 2011-06-21 2014-06-25 阿尔尼拉姆医药品有限公司 血管生成素样3(ANGPTL3)iRNA组合物及其使用方法
CN105392488A (zh) * 2013-05-01 2016-03-09 Isis制药公司 用于调节载脂蛋白c-iii表达的组合物和方法
WO2015006740A2 (en) * 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
CN107075516A (zh) * 2014-08-20 2017-08-18 阿尔尼拉姆医药品有限公司 经修饰的双链rna试剂
CN107250362A (zh) * 2014-11-17 2017-10-13 阿尔尼拉姆医药品有限公司 载脂蛋白C3(APOC3)iRNA组合物及其使用方法
CN108064313A (zh) * 2015-03-17 2018-05-22 箭头药业股份有限公司 用于抑制因子xii的基因表达的组合物和方法
WO2016206626A1 (zh) * 2015-06-26 2016-12-29 苏州瑞博生物技术有限公司 一种siRNA、含有该siRNA的药物组合物和缀合物及它们的应用

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DONALD J. FOSTER等: "Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates", 《MOL THER》, vol. 26, no. 3, 4 January 2018 (2018-01-04), pages 711 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023284763A1 (zh) * 2021-07-16 2023-01-19 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
WO2024027518A1 (zh) * 2022-08-04 2024-02-08 北京福元医药股份有限公司 用于抑制angptl3基因表达的双链核糖核酸及其修饰物、缀合物和用途

Also Published As

Publication number Publication date
JP2021503940A (ja) 2021-02-15
US11414661B2 (en) 2022-08-16
TW201925470A (zh) 2019-07-01
US20220356474A1 (en) 2022-11-10
JP7261494B2 (ja) 2023-04-20
TWI810226B (zh) 2023-08-01
CN110945130B (zh) 2024-04-09
EP3719126A4 (en) 2021-10-20
EP3719126A1 (en) 2020-10-07
WO2019105414A1 (zh) 2019-06-06
US20210032623A1 (en) 2021-02-04
JP2023082151A (ja) 2023-06-13

Similar Documents

Publication Publication Date Title
CN110997917B (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN110959011B (zh) 缀合物及其制备方法和用途
CN110945130B (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN110944675B (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN110945131B (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN110945132B (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN110997919A (zh) 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
CN112423794A (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN111050807B (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN112423795A (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN111973618B (zh) 核酸、药物组合物与siRNA缀合物及制备方法和用途
CN111973619B (zh) 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
CN113795582B (zh) 核酸、药物组合物与缀合物及制备方法和用途
CN111973617A (zh) 核酸、药物组合物与缀合物及制备方法和用途
CN111655849A (zh) 一种核酸、含有该核酸的药物组合物和缀合物及其用途
CN113330117A (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN113795280A (zh) 核酸、药物组合物与缀合物及制备方法和用途
CN113891939A (zh) 核酸、药物组合物与缀合物及制备方法和用途
WO2020233651A1 (zh) 核酸、药物组合物与缀合物及制备方法和用途
CN111377984A (zh) 化合物和缀合物及其制备方法和用途
CN112876534A (zh) 肝靶向化合物及缀合物
CN113811613B (zh) 核酸、药物组合物与缀合物及制备方法和用途
CN118291457A (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN118291456A (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN118252843A (zh) 核酸、药物组合物与缀合物及制备方法和用途

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
CB02 Change of applicant information

Address after: 215300, No. 168, Feng Feng Road, Yushan Town, Kunshan, Jiangsu, Suzhou

Applicant after: Suzhou Ruibo Biotechnology Co.,Ltd.

Address before: 215300, No. 168, Feng Feng Road, Yushan Town, Kunshan, Jiangsu, Suzhou

Applicant before: SUZHOU RIBO LIFE SCIENCE Co.,Ltd.

CB02 Change of applicant information
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40019792

Country of ref document: HK

GR01 Patent grant
GR01 Patent grant