JP2024536721A - 鏡像ペプチドによるアンチセンスオリゴマーの送達 - Google Patents

鏡像ペプチドによるアンチセンスオリゴマーの送達 Download PDF

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JP2024536721A
JP2024536721A JP2024514047A JP2024514047A JP2024536721A JP 2024536721 A JP2024536721 A JP 2024536721A JP 2024514047 A JP2024514047 A JP 2024514047A JP 2024514047 A JP2024514047 A JP 2024514047A JP 2024536721 A JP2024536721 A JP 2024536721A
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peptide
alkyl
oligonucleotide conjugate
cells
pmo
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JP2024536721A5 (cg-RX-API-DMAC10.html
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カーリー・ケー・シッセル
シャーロット・イー・ファーカー
アンニカ・ビー・マルムバーグ
アンドレイ・ロアス
ブラッドリー・エル・ペンテルート
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サレプタ セラピューティクス， インコーポレイテッド
マサチューセッツ・インスティトュート・オブ・テクノロジー
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Publication of JP2024536721A publication Critical patent/JP2024536721A/ja
Publication of JP2024536721A5 publication Critical patent/JP2024536721A5/ja
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    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
JP2024514047A 2021-09-03 2022-09-01 鏡像ペプチドによるアンチセンスオリゴマーの送達 Pending JP2024536721A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163240472P 2021-09-03 2021-09-03
US63/240,472 2021-09-03
US202263314855P 2022-02-28 2022-02-28
US63/314,855 2022-02-28
PCT/US2022/042365 WO2023034515A2 (en) 2021-09-03 2022-09-01 Delivery of anitsense oligomers by mirror image peptides

Publications (2)

Publication Number Publication Date
JP2024536721A true JP2024536721A (ja) 2024-10-08
JP2024536721A5 JP2024536721A5 (cg-RX-API-DMAC10.html) 2025-10-16

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JP2024514047A Pending JP2024536721A (ja) 2021-09-03 2022-09-01 鏡像ペプチドによるアンチセンスオリゴマーの送達

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US (1) US20250011777A1 (cg-RX-API-DMAC10.html)
EP (1) EP4387676A2 (cg-RX-API-DMAC10.html)
JP (1) JP2024536721A (cg-RX-API-DMAC10.html)
WO (1) WO2023034515A2 (cg-RX-API-DMAC10.html)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2026526969A (ja) * 2023-08-02 2026-08-10 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送達のための非標準細胞透過性ペプチド
WO2026080471A2 (en) 2024-10-07 2026-04-16 Flagship Pioneering Innovations Vi, Llc Compositions of trem conjugates and uses thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020537654A (ja) * 2017-10-17 2020-12-24 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達のための細胞透過性ペプチド

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US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン，ジエ−ムス ポリヌクレオチド測定試薬と方法
AU2004235396B2 (en) 2003-04-29 2010-11-25 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
LT2735568T (lt) 2006-05-10 2017-11-27 Sarepta Therapeutics, Inc. Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų
CA2691673A1 (en) * 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
MX2010004955A (es) 2007-11-15 2010-06-30 Avi Biopharma Inc Metodo de sintesis de oligomeros de morfolina.
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
TWI726844B (zh) * 2014-05-23 2021-05-11 美商健臻公司 胜肽載體上之多重寡核苷酸部分
EA201891419A1 (ru) * 2015-12-15 2019-01-31 Сарепта Терапьютикс, Инк. Пептид-олигонуклеотидные конъюгаты

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020537654A (ja) * 2017-10-17 2020-12-24 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達のための細胞透過性ペプチド

Non-Patent Citations (3)

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Title
JAE SAM LEE ET AL.: ""Lipo-oligoarginines as effective delivery vectors to promote cellular uptake"", MOLECULAR BIOSYSTEMS, vol. 6, no. 10, JPN6026024156, 9 August 2010 (2010-08-09), pages 2049 - 2055, ISSN: 0005886052 *
R． P. WU ET AL.: ""Cell-penetrating peptides as transporters for morpholino oligomers: effects of amino acid composit", NUCLEIC ACIDS RESEARCH, vol. 35, no. 15, JPN6026024155, 11 July 2007 (2007-07-11), pages 5182 - 5191, ISSN: 0005886051 *
WOUTER P.R. VERDURMEN ET AL.: ""Preferential Uptake of L- versus D-Amino Acid Cell-Penetrating Peptides in a Cell Type-Dependent M", CHEMISTRY & BIOLOGY, vol. 18, no. 8, JPN6026024157, August 2011 (2011-08-01), pages 1000 - 1010, ISSN: 0005886053 *

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US20250011777A1 (en) 2025-01-09
WO2023034515A2 (en) 2023-03-09
EP4387676A2 (en) 2024-06-26

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