JP7596148B2 - アンチセンス送達用キメラペプチド - Google Patents

アンチセンス送達用キメラペプチド Download PDF

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Publication number
JP7596148B2
JP7596148B2 JP2020549641A JP2020549641A JP7596148B2 JP 7596148 B2 JP7596148 B2 JP 7596148B2 JP 2020549641 A JP2020549641 A JP 2020549641A JP 2020549641 A JP2020549641 A JP 2020549641A JP 7596148 B2 JP7596148 B2 JP 7596148B2
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Prior art keywords
peptide
pharma
acceptable salt
oligonucleotide conjugate
chimeric peptide
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Japanese (ja)
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JP2021518361A5 (https=
JP2021518361A (ja
JPWO2019178479A5 (https=
Inventor
ブラッドリー エル． ペンテルート，
コリン エム． ファドゼン，
レベッカ エル． ホールデン，
ジャスティン エム． ウォルフ，
ジ－ニン チュ，
モニカ ヤオ，
ガンナー ジェイ． ハンソン，
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Massachusetts Institute of Technology
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Massachusetts Institute of Technology
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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
    • 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
    • A61K47/54—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
    • A61K47/545—Heterocyclic compounds
    • 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
    • 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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • 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

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)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020549641A 2018-03-16 2019-03-15 アンチセンス送達用キメラペプチド Active JP7596148B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024159928A JP2024163336A (ja) 2018-03-16 2024-09-17 アンチセンス送達用キメラペプチド

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862644202P 2018-03-16 2018-03-16
US62/644,202 2018-03-16
PCT/US2019/022475 WO2019178479A1 (en) 2018-03-16 2019-03-15 Chimeric peptides for antisense delivery

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024159928A Division JP2024163336A (ja) 2018-03-16 2024-09-17 アンチセンス送達用キメラペプチド

Publications (4)

Publication Number Publication Date
JP2021518361A JP2021518361A (ja) 2021-08-02
JP2021518361A5 JP2021518361A5 (https=) 2023-08-28
JPWO2019178479A5 JPWO2019178479A5 (https=) 2023-08-28
JP7596148B2 true JP7596148B2 (ja) 2024-12-09

Family

ID=67908050

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020549641A Active JP7596148B2 (ja) 2018-03-16 2019-03-15 アンチセンス送達用キメラペプチド
JP2024159928A Pending JP2024163336A (ja) 2018-03-16 2024-09-17 アンチセンス送達用キメラペプチド

Family Applications After (1)

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JP2024159928A Pending JP2024163336A (ja) 2018-03-16 2024-09-17 アンチセンス送達用キメラペプチド

Country Status (6)

Country Link
US (2) US12370261B2 (https=)
EP (1) EP3765030A4 (https=)
JP (2) JP7596148B2 (https=)
MA (1) MA52148A (https=)
TW (1) TW202002990A (https=)
WO (1) WO2019178479A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019079386A1 (en) * 2017-10-17 2019-04-25 Sarepta Therapeutics, Inc. CELL PENETRATION PEPTIDES FOR ANTISENSE ADMINISTRATION
WO2021150867A1 (en) * 2020-01-24 2021-07-29 Sarepta Therapeutics, Inc. Designing antisense oligonucleotide delivery peptides by interpretable machine learning
CA3211038A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
WO2026080471A2 (en) 2024-10-07 2026-04-16 Flagship Pioneering Innovations Vi, Llc Compositions of trem conjugates and uses thereof

Citations (9)

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JP2003533986A (ja) 2000-05-04 2003-11-18 エイブイアイ バイオファーマ， インコーポレイテッド スプライス領域アンチセンス組成物および方法
WO2007009094A2 (en) 2005-07-13 2007-01-18 Avi Biopharma, Inc. Antisense antibacterial method and compound
JP2008513012A (ja) 2004-09-16 2008-05-01 アヴィ バイオファーマ、インコーポレイテッド アンチセンス抗ウイルス化合物及びｓｓＲＮＡウイルス感染を治療する方法
JP2010505741A (ja) 2006-05-10 2010-02-25 エイブイアイ バイオファーマ， インコーポレイテッド カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ
WO2013017631A1 (en) 2011-08-04 2013-02-07 Msd Oss B.V. Kisspeptide-pentasaccharide conjugates
WO2014124952A1 (en) 2013-02-12 2014-08-21 Syddansk Universitet "clickable" alkyne-lna oligonucleotides
JP2015504650A (ja) 2011-11-30 2015-02-16 サレプタ セラピューティクス， インコーポレイテッド 脊髄性筋萎縮症における誘発されたエクソン包含
WO2015038933A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Phosphorylated akt-specific capture agents, compositions, and methods of using and making
JP2016521119A (ja) 2013-03-14 2016-07-21 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィを処置するためのエキソンスキッピング組成物

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KR102240217B1 (ko) 2012-09-25 2021-04-14 젠자임 코포레이션 근긴장성 이영양증의 치료용 펩티드-결합 모르폴리노 안티센스 올리고뉴클레오티드
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WO2019079386A1 (en) * 2017-10-17 2019-04-25 Sarepta Therapeutics, Inc. CELL PENETRATION PEPTIDES FOR ANTISENSE ADMINISTRATION
EP3829597A4 (en) * 2018-07-30 2022-05-18 Sarepta Therapeutics, Inc. TRIMER PEPTIDES FOR ANTISENSE ADMINISTRATION

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Publication number Priority date Publication date Assignee Title
JP2003533986A (ja) 2000-05-04 2003-11-18 エイブイアイ バイオファーマ， インコーポレイテッド スプライス領域アンチセンス組成物および方法
JP2008513012A (ja) 2004-09-16 2008-05-01 アヴィ バイオファーマ、インコーポレイテッド アンチセンス抗ウイルス化合物及びｓｓＲＮＡウイルス感染を治療する方法
WO2007009094A2 (en) 2005-07-13 2007-01-18 Avi Biopharma, Inc. Antisense antibacterial method and compound
JP2010505741A (ja) 2006-05-10 2010-02-25 エイブイアイ バイオファーマ， インコーポレイテッド カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ
WO2013017631A1 (en) 2011-08-04 2013-02-07 Msd Oss B.V. Kisspeptide-pentasaccharide conjugates
JP2015504650A (ja) 2011-11-30 2015-02-16 サレプタ セラピューティクス， インコーポレイテッド 脊髄性筋萎縮症における誘発されたエクソン包含
WO2014124952A1 (en) 2013-02-12 2014-08-21 Syddansk Universitet "clickable" alkyne-lna oligonucleotides
JP2016521119A (ja) 2013-03-14 2016-07-21 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィを処置するためのエキソンスキッピング組成物
WO2015038933A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Phosphorylated akt-specific capture agents, compositions, and methods of using and making

Also Published As

Publication number Publication date
WO2019178479A1 (en) 2019-09-19
US20250367306A1 (en) 2025-12-04
EP3765030A1 (en) 2021-01-20
JP2024163336A (ja) 2024-11-21
US20210260206A1 (en) 2021-08-26
US12370261B2 (en) 2025-07-29
TW202002990A (zh) 2020-01-16
MA52148A (fr) 2021-01-20
EP3765030A4 (en) 2022-01-26
JP2021518361A (ja) 2021-08-02

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