RU2739987C2 - Множественные олигонуклеотидные фрагменты на пептидном носителе - Google Patents

Множественные олигонуклеотидные фрагменты на пептидном носителе Download PDF

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RU2739987C2
RU2739987C2 RU2016150629A RU2016150629A RU2739987C2 RU 2739987 C2 RU2739987 C2 RU 2739987C2 RU 2016150629 A RU2016150629 A RU 2016150629A RU 2016150629 A RU2016150629 A RU 2016150629A RU 2739987 C2 RU2739987 C2 RU 2739987C2
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pmo
rxrrbr
cpp
aon
peptide
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RU2016150629A
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RU2016150629A3 (enExample
RU2016150629A (ru
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Тимоти Э. УИДЕН
Кэрол А. НЕЛЬСОН
Брюс М. ВЕНВОРТ
Николас П. КЛЕЙТОН
Эндрю ЛЕДЖЕР
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Джензим Корпорейшн
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    • 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
    • A61K47/54—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
    • 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
    • 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
    • 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
    • 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
    • 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)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (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)
RU2016150629A 2014-05-23 2015-05-22 Множественные олигонуклеотидные фрагменты на пептидном носителе RU2739987C2 (ru)

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

Related Child Applications (1)

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RU2020142530A Division RU2020142530A (ru) 2014-05-23 2015-05-22 Множественные олигонуклеотидные фрагменты на пептидном носителе

Publications (3)

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RU2016150629A RU2016150629A (ru) 2018-06-25
RU2016150629A3 RU2016150629A3 (enExample) 2018-12-24
RU2739987C2 true RU2739987C2 (ru) 2020-12-30

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RU2016150629A RU2739987C2 (ru) 2014-05-23 2015-05-22 Множественные олигонуклеотидные фрагменты на пептидном носителе
RU2020142530A RU2020142530A (ru) 2014-05-23 2015-05-22 Множественные олигонуклеотидные фрагменты на пептидном носителе

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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
SG10202004611SA (en) 2014-05-23 2020-06-29 Genzyme Corp Multiple oligonucleotide moieties on peptide carrier
AU2018311069A1 (en) 2017-08-03 2020-02-20 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
JP7441455B2 (ja) * 2017-09-22 2024-03-01 ザ リージェンツ オブ ザ ユニバーシティ オブ コロラド，ア ボディー コーポレイト 筋ジストロフィーの処置のためのチオモルホリノオリゴヌクレオチド
EP3762395A1 (en) * 2018-03-07 2021-01-13 Sanofi Nucleotide precursors, nucleotide analogs and oligomeric compounds containing the same
US10758629B2 (en) * 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
JP7667775B2 (ja) * 2019-09-05 2025-04-23 サノフイ ヌクレオチド類似体を含有するオリゴヌクレオチド
WO2021211572A1 (en) * 2020-04-14 2021-10-21 Oregon State University Antisense therapeutics for the treatment of coronavirus
US20250011777A1 (en) * 2021-09-03 2025-01-09 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides

Citations (4)

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Publication number Priority date Publication date Assignee Title
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
RU2275936C2 (ru) * 1999-07-28 2006-05-10 Санофи-Авентис Дойчланд Гмбх Конъюгаты и способ их получения, а также их применение для транспорта молекул через биологические мембраны
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
WO2014052276A1 (en) * 2012-09-25 2014-04-03 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

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US7468418B2 (en) 2003-04-29 2008-12-23 Avi Biopharma., Inc. Compositions for enhancing transport of molecules into cells
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
ES2694726T3 (es) 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Conjugados peptídicos específicos de tejido y métodos
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
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
DK3133160T3 (en) 2008-10-24 2019-04-01 Sarepta Therapeutics Inc EXON SKIP COMPOSITIONS FOR DMD
HK1200484A1 (zh) * 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
KR102287532B1 (ko) * 2014-01-30 2021-08-11 에프. 호프만-라 로슈 아게 생분해성 컨쥬게이트를 갖는 폴리 올리고머 화합물
SG10202004611SA (en) 2014-05-23 2020-06-29 Genzyme Corp Multiple oligonucleotide moieties on peptide carrier

Patent Citations (4)

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Publication number Priority date Publication date Assignee Title
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
RU2275936C2 (ru) * 1999-07-28 2006-05-10 Санофи-Авентис Дойчланд Гмбх Конъюгаты и способ их получения, а также их применение для транспорта молекул через биологические мембраны
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
WO2014052276A1 (en) * 2012-09-25 2014-04-03 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

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Pierce T.L. et al, Mini reviews in medicinal chemistry, 2005, vol. 5, N 1, 41-55. *

Also Published As

Publication number Publication date
CN106459975B (zh) 2020-03-27
AU2015263963A1 (en) 2016-12-01
AU2015263963B2 (en) 2021-02-18
CN106459975A (zh) 2017-02-22
RU2016150629A3 (enExample) 2018-12-24
MX2016015156A (es) 2017-03-27
BR112016027236A2 (pt) 2017-10-17
SG11201608880VA (en) 2016-11-29
RU2020142530A (ru) 2021-04-26
JP2021063128A (ja) 2021-04-22
CA2949104A1 (en) 2015-11-26
RU2016150629A (ru) 2018-06-25
EP3151841B1 (en) 2022-10-19
AR101542A1 (es) 2016-12-28
US20170182171A1 (en) 2017-06-29
KR20170005118A (ko) 2017-01-11
JP7269968B2 (ja) 2023-05-09
IL249064B (en) 2021-07-29
KR102487942B1 (ko) 2023-01-11
TWI726844B (zh) 2021-05-11
SG10202004611SA (en) 2020-06-29
TW201617373A (zh) 2016-05-16
IL249064A0 (en) 2017-01-31
EP3151841A1 (en) 2017-04-12
WO2015179742A1 (en) 2015-11-26
JP2017517253A (ja) 2017-06-29
US20190388547A1 (en) 2019-12-26
CA2949104C (en) 2023-09-26
JP6825914B2 (ja) 2021-02-03
MX381946B (es) 2025-03-13
US11103587B2 (en) 2021-08-31

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