JP2024506099A - 細胞透過性のペプチド複合体およびその使用方法 - Google Patents

細胞透過性のペプチド複合体およびその使用方法 Download PDF

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JP2024506099A
JP2024506099A JP2023548577A JP2023548577A JP2024506099A JP 2024506099 A JP2024506099 A JP 2024506099A JP 2023548577 A JP2023548577 A JP 2023548577A JP 2023548577 A JP2023548577 A JP 2023548577A JP 2024506099 A JP2024506099 A JP 2024506099A
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peptide
complex
oligonucleotide
terminus
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JP2024506099A5 (enExample
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ウッド マシュー
マンサノ ラケル
ゴドフリー キャロライン
マクローリー グラハム
ラズ リチャード
ゲート マイケル
アルズマノフ アンドレー
オドノヴァン リズ
ヘイゼル ガレス
ホーランド アシュリング
ヴァレラ ミゲル
バナジー サバシス
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Oxford University Innovation Ltd
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    • 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
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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
    • A61K47/64Drug-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
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic 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
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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
    • A61K47/64Drug-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K19/00Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/08Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • CCHEMISTRY; METALLURGY
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    • 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
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    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • General Health & Medical Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • 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)
JP2023548577A 2021-02-12 2022-02-11 細胞透過性のペプチド複合体およびその使用方法 Pending JP2024506099A (ja)

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

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JP2024506099A true JP2024506099A (ja) 2024-02-08
JP2024506099A5 JP2024506099A5 (enExample) 2025-02-17

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

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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

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WO2019209764A2 (en) * 2018-04-26 2019-10-31 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
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WO2020257489A1 (en) * 2019-06-19 2020-12-24 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
JP2021500016A (ja) * 2017-10-18 2021-01-07 サレプタ セラピューティクス, インコーポレイテッド アンチセンスオリゴマー化合物

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JP2020503010A (ja) * 2016-12-19 2020-01-30 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コンジュゲート
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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
AU2021427595A1 (en) 2023-09-28
EP4291244A1 (en) 2023-12-20
EP4291244B1 (en) 2025-12-10
CA3211042A1 (en) 2022-08-18
CN117295525A (zh) 2023-12-26
MX2023009446A (es) 2023-10-05

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