JP7320500B2 - アンチセンス送達のための細胞透過性ペプチド - Google Patents

アンチセンス送達のための細胞透過性ペプチド Download PDF

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JP7320500B2
JP7320500B2 JP2020521453A JP2020521453A JP7320500B2 JP 7320500 B2 JP7320500 B2 JP 7320500B2 JP 2020521453 A JP2020521453 A JP 2020521453A JP 2020521453 A JP2020521453 A JP 2020521453A JP 7320500 B2 JP7320500 B2 JP 7320500B2
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peptide
pharmaceutically acceptable
acceptable salt
alkyl
oligonucleotide
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JP2020537654A5 (enExample
JP2020537654A (ja
Inventor
ジャスティン エム． ウォルフ，
コリン エム． ファドゼン，
ジ－ニン チュ，
レベッカ エル． ホールデン，
モニカ ヤオ，
ガンナー ジェイ． ハンソン，
ブラッドリー エル． ペンテルート，
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Massachusetts Institute of Technology
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Massachusetts Institute of Technology
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A61P31/06—Antibacterial agents for tuberculosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
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    • 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
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
JP2020521453A 2017-10-17 2018-10-17 アンチセンス送達のための細胞透過性ペプチド Active JP7320500B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023071470A JP7702444B2 (ja) 2017-10-17 2023-04-25 アンチセンス送達のための細胞透過性ペプチド
JP2025032837A JP2025087783A (ja) 2017-10-17 2025-03-03 アンチセンス送達のための細胞透過性ペプチド

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762573379P 2017-10-17 2017-10-17
US62/573,379 2017-10-17
PCT/US2018/056205 WO2019079386A1 (en) 2017-10-17 2018-10-17 CELL PENETRATION PEPTIDES FOR ANTISENSE ADMINISTRATION

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JP2023071470A Division JP7702444B2 (ja) 2017-10-17 2023-04-25 アンチセンス送達のための細胞透過性ペプチド

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JP2020537654A JP2020537654A (ja) 2020-12-24
JP2020537654A5 JP2020537654A5 (enExample) 2021-11-11
JP7320500B2 true JP7320500B2 (ja) 2023-08-03

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JP2020521453A Active JP7320500B2 (ja) 2017-10-17 2018-10-17 アンチセンス送達のための細胞透過性ペプチド
JP2023071470A Active JP7702444B2 (ja) 2017-10-17 2023-04-25 アンチセンス送達のための細胞透過性ペプチド
JP2025032837A Pending JP2025087783A (ja) 2017-10-17 2025-03-03 アンチセンス送達のための細胞透過性ペプチド

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JP2023071470A Active JP7702444B2 (ja) 2017-10-17 2023-04-25 アンチセンス送達のための細胞透過性ペプチド
JP2025032837A Pending JP2025087783A (ja) 2017-10-17 2025-03-03 アンチセンス送達のための細胞透過性ペプチド

Country Status (6)

Country Link
US (2) US12138312B2 (enExample)
EP (1) EP3697422A4 (enExample)
JP (3) JP7320500B2 (enExample)
MA (1) MA50834A (enExample)
TW (2) TW202423453A (enExample)
WO (1) WO2019079386A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12370261B2 (en) 2018-03-16 2025-07-29 Sarepta Therapeutics, Inc. Chimeric peptides for antisense delivery
EP4093441A4 (en) * 2020-01-24 2025-02-12 Sarepta Therapeutics, Inc. DESIGN OF ANTISENSE OLIGONUCLEOTIDE DELIVERY PEPTIDES BY INTERPRETABLE MACHINE LEARNING
US20240041050A1 (en) * 2020-12-16 2024-02-08 Pioneer Hi-Bred International, Inc. Cell penetrating peptide mediated rna transduction within insect cells
WO2023034515A2 (en) * 2021-09-03 2023-03-09 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides
CN114380886B (zh) * 2022-01-26 2022-10-21 深圳深创生物药业有限公司 一种肿瘤靶向多肽、多肽偶联药物及其应用
EP4230196A1 (en) 2022-02-21 2023-08-23 Som Innovation Biotech, S.A. Compounds for use in the treatment of dystrophinopathies
EP4612292A1 (en) * 2022-11-02 2025-09-10 Sarepta Therapeutics, Inc. Formulation of an antisense oligomer conjugate
US20250171776A1 (en) * 2023-08-02 2025-05-29 Sarepta Therapeutics, Inc. Non-canonical cell-penetrating peptides for antisense oligomer delivery

Citations (3)

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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
WO2014124952A1 (en) 2013-02-12 2014-08-21 Syddansk Universitet "clickable" alkyne-lna oligonucleotides
JP2016185991A (ja) 2011-05-05 2016-10-27 サレプタ セラピューティクス， インコーポレイテッド ペプチドオリゴヌクレオチドコンジュゲート

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PL2735568T3 (pl) * 2006-05-10 2018-02-28 Sarepta Therapeutics, Inc. Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR102272498B1 (ko) 2011-10-27 2021-07-06 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, ｎ-말단 상에 관능화된 아미노산 유도체
RU2018123569A (ru) * 2012-09-25 2019-03-07 Джензим Корпорейшн Связанные с пептидом морфолиновые антисмысловые олигонуклеотиды для лечения миотонической дистрофии
WO2017066789A1 (en) * 2015-10-16 2017-04-20 Modernatx, Inc. Mrna cap analogs with modified sugar
US12370261B2 (en) * 2018-03-16 2025-07-29 Sarepta Therapeutics, Inc. Chimeric peptides for antisense delivery

Patent Citations (3)

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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
JP2016185991A (ja) 2011-05-05 2016-10-27 サレプタ セラピューティクス， インコーポレイテッド ペプチドオリゴヌクレオチドコンジュゲート
WO2014124952A1 (en) 2013-02-12 2014-08-21 Syddansk Universitet "clickable" alkyne-lna oligonucleotides

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Publication number Publication date
JP7702444B2 (ja) 2025-07-03
TWI837102B (zh) 2024-04-01
MA50834A (fr) 2020-08-26
JP2025087783A (ja) 2025-06-10
US20200316210A1 (en) 2020-10-08
JP2023083557A (ja) 2023-06-15
TW202423453A (zh) 2024-06-16
TW201928056A (zh) 2019-07-16
US20250135015A1 (en) 2025-05-01
EP3697422A1 (en) 2020-08-26
US12138312B2 (en) 2024-11-12
EP3697422A4 (en) 2021-08-11
JP2020537654A (ja) 2020-12-24
WO2019079386A1 (en) 2019-04-25

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