CN106459975B - 肽载体上的多个寡核苷酸部分 - Google Patents

肽载体上的多个寡核苷酸部分 Download PDF

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Publication number
CN106459975B
CN106459975B CN201580027369.4A CN201580027369A CN106459975B CN 106459975 B CN106459975 B CN 106459975B CN 201580027369 A CN201580027369 A CN 201580027369A CN 106459975 B CN106459975 B CN 106459975B
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pmo
peptide
seq
cpp
rxrrbr
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Chinese (zh)
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CN106459975A (zh
Inventor
T·E·威登
C·A·纳尔逊
B·M·文特沃斯
N·P·克莱顿
A·莱杰
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Genzyme Corp
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Genzyme Corp
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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
    • C12N15/113—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
    • A61K47/54—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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • 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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CN201580027369.4A 2014-05-23 2015-05-22 肽载体上的多个寡核苷酸部分 Expired - Fee Related CN106459975B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462002296P 2014-05-23 2014-05-23
US62/002,296 2014-05-23
PCT/US2015/032142 WO2015179742A1 (en) 2014-05-23 2015-05-22 Multiple oligonucleotide moieties on peptide carrier

Publications (2)

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CN106459975A CN106459975A (zh) 2017-02-22
CN106459975B true CN106459975B (zh) 2020-03-27

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CN201580027369.4A Expired - Fee Related CN106459975B (zh) 2014-05-23 2015-05-22 肽载体上的多个寡核苷酸部分

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US (2) US20170182171A1 (enExample)
EP (1) EP3151841B1 (enExample)
JP (2) JP6825914B2 (enExample)
KR (1) KR102487942B1 (enExample)
CN (1) CN106459975B (enExample)
AR (1) AR101542A1 (enExample)
AU (1) AU2015263963B2 (enExample)
BR (1) BR112016027236A2 (enExample)
CA (1) CA2949104C (enExample)
IL (1) IL249064B (enExample)
MX (1) MX381946B (enExample)
RU (2) RU2739987C2 (enExample)
SG (2) SG10202004611SA (enExample)
TW (1) TWI726844B (enExample)
WO (1) WO2015179742A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
WO2019060522A2 (en) * 2017-09-22 2019-03-28 The Regents Of The University Of Colorado, A Body Corporate OLIGONUCLEOTIDES THIOMORPHOLINO FOR THE TREATMENT OF MUSCLE DYSTROPHY
CN112105625B (zh) * 2018-03-07 2024-12-31 赛诺菲 核苷酸前体、核苷酸类似物以及含其的寡聚化合物
US10765760B2 (en) * 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US20220372063A1 (en) * 2019-09-05 2022-11-24 Sanofi Oligonucleotides containing nucleotide analogs
WO2021211572A1 (en) * 2020-04-14 2021-10-21 Oregon State University Antisense therapeutics for the treatment of coronavirus
WO2023034515A2 (en) * 2021-09-03 2023-03-09 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides

Citations (1)

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WO2014052276A1 (en) * 2012-09-25 2014-04-03 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

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WO1995006056A1 (en) * 1993-08-20 1995-03-02 University Of Medicine & Dentistry Of New Jersey Bridged polycationic polymer-oligonucleotide conjugates and methods for preparing same
DE19935302A1 (de) 1999-07-28 2001-02-08 Aventis Pharma Gmbh Konjugate und Verfahren zu deren Herstellung sowie deren Verwendung zum Transport von Molekülen über biologische Membranen
AU2004235396B2 (en) 2003-04-29 2010-11-25 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
CA2691673A1 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
WO2009147368A1 (en) 2008-06-04 2009-12-10 Medical Research Council Peptides
EP3133160B1 (en) 2008-10-24 2018-12-12 Sarepta Therapeutics, Inc. Exon skipping compositions for dmd
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
HK1200484A1 (zh) 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
US10358643B2 (en) * 2014-01-30 2019-07-23 Hoffmann-La Roche, Inc. Poly oligomer compound with biocleavable conjugates
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

Patent Citations (1)

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WO2014052276A1 (en) * 2012-09-25 2014-04-03 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

Non-Patent Citations (2)

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Tracie L. Pierce 等.Peptide-Oligonucleotide Hybrids in Antisense Therapy.《Mini Reviews in Medicinal Chemistry》.2005,第5卷(第1期),第41-55页. *

Also Published As

Publication number Publication date
AU2015263963B2 (en) 2021-02-18
SG10202004611SA (en) 2020-06-29
JP2021063128A (ja) 2021-04-22
BR112016027236A2 (pt) 2017-10-17
MX381946B (es) 2025-03-13
CN106459975A (zh) 2017-02-22
JP6825914B2 (ja) 2021-02-03
US20170182171A1 (en) 2017-06-29
AR101542A1 (es) 2016-12-28
MX2016015156A (es) 2017-03-27
JP2017517253A (ja) 2017-06-29
RU2739987C2 (ru) 2020-12-30
IL249064A0 (en) 2017-01-31
US20190388547A1 (en) 2019-12-26
AU2015263963A1 (en) 2016-12-01
JP7269968B2 (ja) 2023-05-09
EP3151841B1 (en) 2022-10-19
RU2020142530A (ru) 2021-04-26
WO2015179742A1 (en) 2015-11-26
US11103587B2 (en) 2021-08-31
TW201617373A (zh) 2016-05-16
KR20170005118A (ko) 2017-01-11
CA2949104A1 (en) 2015-11-26
RU2016150629A3 (enExample) 2018-12-24
EP3151841A1 (en) 2017-04-12
RU2016150629A (ru) 2018-06-25
IL249064B (en) 2021-07-29
SG11201608880VA (en) 2016-11-29
TWI726844B (zh) 2021-05-11
KR102487942B1 (ko) 2023-01-11
CA2949104C (en) 2023-09-26

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