JP7678654B2 - 複合体及びその使用 - Google Patents

複合体及びその使用 Download PDF

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Publication number
JP7678654B2
JP7678654B2 JP2022507761A JP2022507761A JP7678654B2 JP 7678654 B2 JP7678654 B2 JP 7678654B2 JP 2022507761 A JP2022507761 A JP 2022507761A JP 2022507761 A JP2022507761 A JP 2022507761A JP 7678654 B2 JP7678654 B2 JP 7678654B2
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Prior art keywords
seq
peptide
cag
pmo
complex
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Japanese (ja)
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JP2022543320A (ja
JP2022543320A5 (https=
Inventor
ウッド マシュー
ヴァレラ ミゲル
ホーランド アシュリング
ラズ リチャード
フュルラン ドニ
クラン アルノー
ゲート マイケル
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Association Institut de Myologie
Sorbonne Universite
Oxford University Innovation Ltd
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Association Institut de Myologie
Sorbonne Universite
Oxford University Innovation Ltd
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Publication of JP2022543320A5 publication Critical patent/JP2022543320A5/ja
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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

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  • 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)
JP2022507761A 2019-08-09 2020-08-07 複合体及びその使用 Active JP7678654B2 (ja)

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

Publications (3)

Publication Number Publication Date
JP2022543320A JP2022543320A (ja) 2022-10-11
JP2022543320A5 JP2022543320A5 (https=) 2023-08-14
JP7678654B2 true JP7678654B2 (ja) 2025-05-16

Family

ID=67990936

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022507761A Active JP7678654B2 (ja) 2019-08-09 2020-08-07 複合体及びその使用

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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010532168A (ja) 2007-06-29 2010-10-07 エーブイアイ バイオファーマ インコーポレイテッド 組織特異的ペプチドコンジュゲートおよび方法
JP2011523557A (ja) 2008-06-04 2011-08-18 メディカル リサーチ カウンシル ペプチド
JP2014526238A (ja) 2011-08-30 2014-10-06 メディカル リサーチ カウンシル ペプチド
JP2015532264A (ja) 2012-09-25 2015-11-09 ジェンザイム・コーポレーション 筋強直性ジストロフィーを治療するためのペプチド結合モルホリノアンチセンスオリゴヌクレオチド

Family Cites Families (4)

* 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
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010532168A (ja) 2007-06-29 2010-10-07 エーブイアイ バイオファーマ インコーポレイテッド 組織特異的ペプチドコンジュゲートおよび方法
JP2011523557A (ja) 2008-06-04 2011-08-18 メディカル リサーチ カウンシル ペプチド
JP2014526238A (ja) 2011-08-30 2014-10-06 メディカル リサーチ カウンシル ペプチド
JP2015532264A (ja) 2012-09-25 2015-11-09 ジェンザイム・コーポレーション 筋強直性ジストロフィーを治療するためのペプチド結合モルホリノアンチセンスオリゴヌクレオチド

Also Published As

Publication number Publication date
CN114615998A (zh) 2022-06-10
US20220275372A1 (en) 2022-09-01
JP2022543320A (ja) 2022-10-11
KR20220079524A (ko) 2022-06-13
CA3149700A1 (en) 2021-02-18
AU2020327659A1 (en) 2022-02-24
EP4010030A1 (en) 2022-06-15
CN114615998B (zh) 2025-05-23
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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