CN114615998B - 缀合物及其用途 - Google Patents

缀合物及其用途 Download PDF

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Publication number
CN114615998B
CN114615998B CN202080071067.8A CN202080071067A CN114615998B CN 114615998 B CN114615998 B CN 114615998B CN 202080071067 A CN202080071067 A CN 202080071067A CN 114615998 B CN114615998 B CN 114615998B
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CN
China
Prior art keywords
peptide
suitably
bala
mod
res
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CN202080071067.8A
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English (en)
Chinese (zh)
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CN114615998A (zh
Inventor
马修·伍德
米格尔·瓦雷拉
阿诗琳·霍兰德
理查德·拉兹
丹尼斯·弗林
阿诺·克莱因
迈克尔·盖特
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.)
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
Sorbonne Universite
Oxford University Innovation Ltd
United Kingdom Research and Innovation
Original Assignee
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
Sorbonne Universite
Oxford University Innovation Ltd
United Kingdom Research and Innovation
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.)
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Publication date
Application filed by Institut National de la Sante et de la Recherche Medicale INSERM, Association Institut de Myologie, Sorbonne Universite, Oxford University Innovation Ltd, United Kingdom Research and Innovation filed Critical Institut National de la Sante et de la Recherche Medicale INSERM
Priority to CN202510582562.XA priority Critical patent/CN120837670A/zh
Publication of CN114615998A publication Critical patent/CN114615998A/zh
Application granted granted Critical
Publication of CN114615998B publication Critical patent/CN114615998B/zh
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    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N2310/11—Antisense

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Genetics & Genomics (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202080071067.8A 2019-08-09 2020-08-07 缀合物及其用途 Active CN114615998B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202510582562.XA CN120837670A (zh) 2019-08-09 2020-08-07 缀合物及其用途

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1911403.2 2019-08-09
GBGB1911403.2A GB201911403D0 (en) 2019-08-09 2019-08-09 Conjugate and uses thereof
PCT/GB2020/051891 WO2021028666A1 (en) 2019-08-09 2020-08-07 Conjugate and uses thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202510582562.XA Division CN120837670A (zh) 2019-08-09 2020-08-07 缀合物及其用途

Publications (2)

Publication Number Publication Date
CN114615998A CN114615998A (zh) 2022-06-10
CN114615998B true CN114615998B (zh) 2025-05-23

Family

ID=67990936

Family Applications (2)

Application Number Title Priority Date Filing Date
CN202080071067.8A Active CN114615998B (zh) 2019-08-09 2020-08-07 缀合物及其用途
CN202510582562.XA Pending CN120837670A (zh) 2019-08-09 2020-08-07 缀合物及其用途

Family Applications After (1)

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Country Status (13)

Country Link
US (1) US20220275372A1 (https=)
EP (1) EP4010030A1 (https=)
JP (1) JP7678654B2 (https=)
KR (1) KR20220079524A (https=)
CN (2) CN114615998B (https=)
AU (1) AU2020327659A1 (https=)
BR (1) BR112022002309A2 (https=)
CA (1) CA3149700A1 (https=)
GB (1) GB201911403D0 (https=)
IL (1) IL290424A (https=)
MX (1) MX2022001712A (https=)
SA (1) SA522431627B1 (https=)
WO (1) WO2021028666A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20200131231A1 (en) 2017-02-17 2020-04-30 Oxford University Innovation Limited Cell penetrating peptides
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
US12465646B2 (en) 2018-12-07 2025-11-11 Oxford University Innovation Limited Linkers
WO2022171972A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
EP4304657A4 (en) * 2021-03-12 2026-04-01 Pepgen Inc METHODS OF TREATMENT OF TYPE 1 MYOTONIC DYSTROPHY USING PEPTIDE-OLIGONUCLEOTIDE CONJUGATES
KR20250116673A (ko) * 2022-11-21 2025-08-01 펩젠 인크. 세포-침투 펩티드, 그의 접합체 및 그의 사용 방법
WO2025033255A1 (ja) * 2023-08-07 2025-02-13 東亞合成株式会社 ペプチドフラグメント及びその利用
WO2025033256A1 (ja) * 2023-08-07 2025-02-13 東亞合成株式会社 ペプチドフラグメント及びその利用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103998458A (zh) * 2011-08-30 2014-08-20 医学研究理事会 具有中央疏水域的细胞穿透肽
CN104837997A (zh) * 2012-09-25 2015-08-12 建新公司 用于肌强直性营养不良治疗的肽连接吗啉代反义寡核苷酸

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2049664T1 (sl) * 2006-08-11 2012-04-30 Prosensa Technologies Bv Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev
WO2009005793A2 (en) * 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2009147368A1 (en) * 2008-06-04 2009-12-10 Medical Research Council Peptides
US20110269665A1 (en) * 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
RU2708237C2 (ru) 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103998458A (zh) * 2011-08-30 2014-08-20 医学研究理事会 具有中央疏水域的细胞穿透肽
CN104837997A (zh) * 2012-09-25 2015-08-12 建新公司 用于肌强直性营养不良治疗的肽连接吗啉代反义寡核苷酸

Also Published As

Publication number Publication date
CN114615998A (zh) 2022-06-10
US20220275372A1 (en) 2022-09-01
JP2022543320A (ja) 2022-10-11
JP7678654B2 (ja) 2025-05-16
KR20220079524A (ko) 2022-06-13
CA3149700A1 (en) 2021-02-18
AU2020327659A1 (en) 2022-02-24
EP4010030A1 (en) 2022-06-15
SA522431627B1 (ar) 2024-05-19
GB201911403D0 (en) 2019-09-25
BR112022002309A2 (pt) 2022-04-26
CN120837670A (zh) 2025-10-28
WO2021028666A1 (en) 2021-02-18
MX2022001712A (es) 2022-05-13
IL290424A (en) 2022-04-01

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