RU2020142530A - Множественные олигонуклеотидные фрагменты на пептидном носителе - Google Patents

Множественные олигонуклеотидные фрагменты на пептидном носителе Download PDF

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Publication number
RU2020142530A
RU2020142530A RU2020142530A RU2020142530A RU2020142530A RU 2020142530 A RU2020142530 A RU 2020142530A RU 2020142530 A RU2020142530 A RU 2020142530A RU 2020142530 A RU2020142530 A RU 2020142530A RU 2020142530 A RU2020142530 A RU 2020142530A
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RU
Russia
Prior art keywords
aon
peptide
rxrrbr
glu
naturally occurring
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RU2020142530A
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English (en)
Russian (ru)
Inventor
Тимоти Э. Уиден
Кэрол А. НЕЛЬСОН
Брюс М. ВЕНВОРТ
Николас П. КЛЕЙТОН
Эндрю ЛЕДЖЕР
Original Assignee
Джензим Корпорейшн
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Application filed by Джензим Корпорейшн filed Critical Джензим Корпорейшн
Publication of RU2020142530A publication Critical patent/RU2020142530A/ru

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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
    • A61K47/64Drug-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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K19/00Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • C12N2310/3145Phosphoramidates with the nitrogen in 3' or 5'-position
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special 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)
  • 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)
RU2020142530A 2014-05-23 2015-05-22 Множественные олигонуклеотидные фрагменты на пептидном носителе RU2020142530A (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462002296P 2014-05-23 2014-05-23
US62/002,296 2014-05-23

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
RU2016150629A Division RU2739987C2 (ru) 2014-05-23 2015-05-22 Множественные олигонуклеотидные фрагменты на пептидном носителе

Publications (1)

Publication Number Publication Date
RU2020142530A true RU2020142530A (ru) 2021-04-26

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

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Country Status (15)

Country Link
US (2) US20170182171A1 (https=)
EP (1) EP3151841B1 (https=)
JP (2) JP6825914B2 (https=)
KR (1) KR102487942B1 (https=)
CN (1) CN106459975B (https=)
AR (1) AR101542A1 (https=)
AU (1) AU2015263963B2 (https=)
BR (1) BR112016027236A2 (https=)
CA (1) CA2949104C (https=)
IL (1) IL249064B (https=)
MX (1) MX381946B (https=)
RU (2) RU2020142530A (https=)
SG (2) SG11201608880VA (https=)
TW (1) TWI726844B (https=)
WO (1) WO2015179742A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112016027236A2 (pt) 2014-05-23 2017-10-17 Genzyme Corp porções de múltiplos oligonucleotídeos em veículo de peptídeo
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Aavを送達するための組成物および方法
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
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
WO2023034515A2 (en) * 2021-09-03 2023-03-09 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides

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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
CA2523672C (en) 2003-04-29 2012-07-17 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
EP3443976A1 (en) 2007-06-29 2019-02-20 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
US8575305B2 (en) 2008-06-04 2013-11-05 Medical Research Council Cell penetrating peptides
CN112574988A (zh) 2008-10-24 2021-03-30 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
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
KR102240217B1 (ko) * 2012-09-25 2021-04-14 젠자임 코포레이션 근긴장성 이영양증의 치료용 펩티드-결합 모르폴리노 안티센스 올리고뉴클레오티드
WO2015113922A1 (en) * 2014-01-30 2015-08-06 Roche Innovation Center Copenhagen A/S Poly oligomer compound with biocleavable conjugates
BR112016027236A2 (pt) 2014-05-23 2017-10-17 Genzyme Corp porções de múltiplos oligonucleotídeos em veículo de peptídeo

Also Published As

Publication number Publication date
US20190388547A1 (en) 2019-12-26
CN106459975A (zh) 2017-02-22
RU2016150629A (ru) 2018-06-25
EP3151841B1 (en) 2022-10-19
KR102487942B1 (ko) 2023-01-11
AU2015263963B2 (en) 2021-02-18
AU2015263963A1 (en) 2016-12-01
JP7269968B2 (ja) 2023-05-09
MX381946B (es) 2025-03-13
TW201617373A (zh) 2016-05-16
IL249064B (en) 2021-07-29
JP2017517253A (ja) 2017-06-29
EP3151841A1 (en) 2017-04-12
AR101542A1 (es) 2016-12-28
SG11201608880VA (en) 2016-11-29
JP6825914B2 (ja) 2021-02-03
WO2015179742A1 (en) 2015-11-26
SG10202004611SA (en) 2020-06-29
TWI726844B (zh) 2021-05-11
MX2016015156A (es) 2017-03-27
BR112016027236A2 (pt) 2017-10-17
RU2016150629A3 (https=) 2018-12-24
RU2739987C2 (ru) 2020-12-30
CA2949104A1 (en) 2015-11-26
US11103587B2 (en) 2021-08-31
IL249064A0 (en) 2017-01-31
US20170182171A1 (en) 2017-06-29
CN106459975B (zh) 2020-03-27
CA2949104C (en) 2023-09-26
JP2021063128A (ja) 2021-04-22
KR20170005118A (ko) 2017-01-11

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