CN117295525A - 细胞穿透肽缀合物及其使用方法 - Google Patents

细胞穿透肽缀合物及其使用方法 Download PDF

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Publication number
CN117295525A
CN117295525A CN202280028220.8A CN202280028220A CN117295525A CN 117295525 A CN117295525 A CN 117295525A CN 202280028220 A CN202280028220 A CN 202280028220A CN 117295525 A CN117295525 A CN 117295525A
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CN
China
Prior art keywords
seq
conjugate
peptide
oligonucleotide
terminus
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
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CN202280028220.8A
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English (en)
Chinese (zh)
Inventor
马修·伍德
拉克尔·曼扎诺
卡罗琳·戈弗雷
格雷厄姆·麦克洛里
理查德·拉兹
迈克尔·盖特
安德烈·阿尔祖马诺夫
利兹·奥多诺万
加雷思·哈泽尔
阿诗琳·霍兰德
米格尔·瓦雷拉
苏哈什斯·班纳吉
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.)
Oxford University Innovation Ltd
United Kingdom Research and Innovation
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Oxford University Innovation Ltd
United Kingdom Research and Innovation
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Filing date
Publication date
Application filed by Oxford University Innovation Ltd, United Kingdom Research and Innovation filed Critical Oxford University Innovation Ltd
Publication of CN117295525A publication Critical patent/CN117295525A/zh
Pending legal-status Critical Current

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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
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/3513—Protein; Peptide
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
CN202280028220.8A 2021-02-12 2022-02-11 细胞穿透肽缀合物及其使用方法 Pending CN117295525A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBPCT/GB2021/050357 2021-02-12
PCT/GB2021/050357 WO2022171972A1 (en) 2021-02-12 2021-02-12 Cell-penetrating peptide conjugates and methods of their use
PCT/GB2022/050371 WO2022172019A1 (en) 2021-02-12 2022-02-11 Cell-penetrating peptide conjugates and methods of their use

Publications (1)

Publication Number Publication Date
CN117295525A true CN117295525A (zh) 2023-12-26

Family

ID=74669201

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280028220.8A Pending CN117295525A (zh) 2021-02-12 2022-02-11 细胞穿透肽缀合物及其使用方法

Country Status (12)

Country Link
US (2) US20240299563A1 (https=)
EP (2) EP4291244B1 (https=)
JP (1) JP2024506099A (https=)
KR (1) KR20240006019A (https=)
CN (1) CN117295525A (https=)
AU (2) AU2021427595A1 (https=)
BR (1) BR112023016183A2 (https=)
CA (2) CA3211038A1 (https=)
ES (1) ES3058927T3 (https=)
IL (1) IL305117A (https=)
MX (1) MX2023009446A (https=)
WO (2) WO2022171972A1 (https=)

Families Citing this family (10)

* 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
WO2022192749A2 (en) * 2021-03-12 2022-09-15 Pepgen Inc. Methods of treating duchenne muscular dystrophy using peptide-oligonucleotide conjugates
EP4304657A4 (en) * 2021-03-12 2026-04-01 Pepgen Inc METHODS OF TREATMENT OF TYPE 1 MYOTONIC DYSTROPHY USING PEPTIDE-OLIGONUCLEOTIDE CONJUGATES
TW202442246A (zh) 2023-04-27 2024-11-01 美商薩羅塔治療公司 用於治療慢性腎病之反義寡聚物
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies
GB202405980D0 (en) * 2024-04-29 2024-06-12 Univ Oxford Innovation Ltd Conjugate for treating repeat expansion disorders
WO2025264554A1 (en) 2024-06-17 2025-12-26 Pepgen Inc. Antisense oligonucleotide conjugates targeting peripheral myelin protein 22 (pmp22) and methods of use thereof

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2009147368A1 (en) * 2008-06-04 2009-12-10 Medical Research Council Peptides
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CA3092114A1 (en) * 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US20140315977A1 (en) * 2013-03-14 2014-10-23 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
PT3554552T (pt) * 2016-12-19 2022-10-03 Sarepta Therapeutics Inc Conjugados de oligómero de skipping de exão para distrofia muscular
KR20240006023A (ko) * 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR102810425B1 (ko) * 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
US20200131231A1 (en) * 2017-02-17 2020-04-30 Oxford University Innovation Limited Cell penetrating peptides
EP3687547A1 (en) * 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
WO2019067979A1 (en) * 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
WO2019067981A1 (en) * 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
US11555189B2 (en) * 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
EP3765030A4 (en) * 2018-03-16 2022-01-26 Sarepta Therapeutics, Inc. CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION
JP2021521794A (ja) * 2018-04-26 2021-08-30 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート
JP7579147B2 (ja) * 2018-06-14 2024-11-07 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート
TW202449155A (zh) * 2018-07-27 2024-12-16 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812980D0 (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
WO2020214763A1 (en) * 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
JP2022536495A (ja) * 2019-06-19 2022-08-17 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを治療する方法
GB201911403D0 (en) * 2019-08-09 2019-09-25 Univ Oxford Innovation Ltd Conjugate and uses thereof

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IL305117A (en) 2023-10-01
CA3211038A1 (en) 2022-08-18
AU2022220252A1 (en) 2023-09-28
EP4717702A2 (en) 2026-04-01
US20250177543A1 (en) 2025-06-05
ES3058927T3 (en) 2026-03-13
WO2022172019A1 (en) 2022-08-18
EP4291244C0 (en) 2025-12-10
US20240299563A1 (en) 2024-09-12
WO2022171972A1 (en) 2022-08-18
BR112023016183A2 (pt) 2023-12-12
KR20240006019A (ko) 2024-01-12
JP2024506099A (ja) 2024-02-08
AU2021427595A1 (en) 2023-09-28
EP4291244A1 (en) 2023-12-20
EP4291244B1 (en) 2025-12-10
CA3211042A1 (en) 2022-08-18
MX2023009446A (es) 2023-10-05

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