CN114616339A - 用于治疗肢端肥大症的缀合物和方法 - Google Patents

用于治疗肢端肥大症的缀合物和方法 Download PDF

Info

Publication number
CN114616339A
CN114616339A CN202080075748.1A CN202080075748A CN114616339A CN 114616339 A CN114616339 A CN 114616339A CN 202080075748 A CN202080075748 A CN 202080075748A CN 114616339 A CN114616339 A CN 114616339A
Authority
CN
China
Prior art keywords
sirna
group
compound
alkyl
formula
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.)
Pending
Application number
CN202080075748.1A
Other languages
English (en)
Chinese (zh)
Inventor
德鲁·康德拉托维茨
詹姆斯·海斯
理查德·J·霍兰德
克莉丝汀·伊索
凯文·麦克林托克
克里斯多夫·贾斯汀·帕塞特卡
马克·伍德
艾伦·D·马丁
玛格丽特·施瓦兹
大卫·克罗
史蒂文·泰勒
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.)
Gainawan Science Co ltd
Original Assignee
Gainawan 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 Gainawan Science Co ltd filed Critical Gainawan Science Co ltd
Publication of CN114616339A publication Critical patent/CN114616339A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K9/00—Peptides having up to 20 amino acids, containing saccharide radicals and having a fully defined sequence; Derivatives thereof
    • C07K9/001—Peptides having up to 20 amino acids, containing saccharide radicals and having a fully defined sequence; Derivatives thereof the peptide sequence having less than 12 amino acids and not being part of a ring structure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/06—Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH
    • A61P5/08—Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH for decreasing, blocking or antagonising the activity of the anterior pituitary hormones
    • 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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • C12N2310/35—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Endocrinology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
CN202080075748.1A 2019-10-24 2020-10-23 用于治疗肢端肥大症的缀合物和方法 Pending CN114616339A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962925659P 2019-10-24 2019-10-24
US62/925,659 2019-10-24
PCT/US2020/057185 WO2021081420A2 (en) 2019-10-24 2020-10-23 Conjugates and methods for treating acromegaly

Publications (1)

Publication Number Publication Date
CN114616339A true CN114616339A (zh) 2022-06-10

Family

ID=75620892

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080075748.1A Pending CN114616339A (zh) 2019-10-24 2020-10-23 用于治疗肢端肥大症的缀合物和方法

Country Status (9)

Country Link
US (1) US20220288214A1 (https=)
EP (1) EP4048801A4 (https=)
JP (1) JP7668032B2 (https=)
CN (1) CN114616339A (https=)
AU (1) AU2020371736A1 (https=)
BR (1) BR112022007725A2 (https=)
CA (1) CA3158917A1 (https=)
IL (1) IL292391A (https=)
WO (1) WO2021081420A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3020585A1 (en) * 2016-04-11 2017-10-19 Arbutus Biopharma Corporation Targeted nucleic acid conjugate compositions

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2322651A1 (en) * 2004-05-14 2011-05-18 Rosetta Genomics Ltd MicroRNAs and uses thereof
CA2917229A1 (en) * 2013-07-02 2015-01-08 Isis Pharmaceuticals, Inc. Modulators of growth hormone receptor
BR112016022742B1 (pt) * 2014-05-01 2022-06-14 Ionis Pharmaceuticals, Inc Composto químico, composição compreendendo o composto e uso dos mesmos
WO2015179693A1 (en) * 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3151839A4 (en) * 2014-06-06 2018-02-28 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
EP3228326A1 (en) 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate
DK3607069T3 (da) 2017-04-05 2022-11-21 Silence Therapeutics Gmbh Produkter og sammensætninger
WO2018185253A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Ligand modified double-stranded nucleic acids
JP7406793B2 (ja) * 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
EP3775209A1 (en) * 2018-04-05 2021-02-17 Silence Therapeutics GmbH Sirnas with at least two ligands at different ends

Also Published As

Publication number Publication date
IL292391A (en) 2022-06-01
EP4048801A4 (en) 2023-11-22
JP2022553056A (ja) 2022-12-21
WO2021081420A3 (en) 2021-06-03
JP7668032B2 (ja) 2025-04-24
EP4048801A2 (en) 2022-08-31
AU2020371736A1 (en) 2022-06-02
BR112022007725A2 (pt) 2022-08-16
US20220288214A1 (en) 2022-09-15
WO2021081420A2 (en) 2021-04-29
CA3158917A1 (en) 2021-04-29

Similar Documents

Publication Publication Date Title
JP7507495B2 (ja) 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
TWI805577B (zh) 靶向組合物
US11603532B2 (en) Oligonucleotide compositions and methods of use thereof
JP2024175018A (ja) 治療方法
JP7614650B2 (ja) 核酸、薬物組成物及び複合体並びに調製方法と使用
JP2023505434A (ja) 肝線維症を治療するためのコンジュゲート及び方法
JP2022535708A (ja) 核酸、薬物組成物及び複合体ならびに調製方法と使用
JP7808963B2 (ja) ＨＩＦ－２アルファ（ＥＰＡＳ１）の発現を阻害するためのＲＮＡｉ剤、その組成物及び使用方法
JP2022533419A (ja) 核酸、薬物組成物及び複合体ならびに調製方法と使用
JP2025524740A (ja) オリゴヌクレオチド送達増強剤、医薬組成物およびそれらを使用する方法
US20240052349A1 (en) Targeted conjugates comprising modified sirna
JP7668032B2 (ja) 先端巨大症を処置するためのコンジュゲート及び方法
EP4355355A1 (en) Treatment of plin1 related diseases and disorders
TW202516005A (zh) Ｓｉｒｎａ，含有ｓｉｒｎａ的綴合物，醫藥組成物及其用途
WO2025261378A1 (zh) 一种修饰的核苷化合物及其在寡核苷酸中的应用
CN121852371A (zh) 抑制CFB基因表达的siRNA及其用途
HK40029072A (en) Oligonucleotide compositions and methods of use thereof

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