AR101542A1 - Porciones de múltiples oligonucleótidos de péptidos transportadores - Google Patents

Porciones de múltiples oligonucleótidos de péptidos transportadores

Info

Publication number
AR101542A1
AR101542A1 ARP150101604A ARP150101604A AR101542A1 AR 101542 A1 AR101542 A1 AR 101542A1 AR P150101604 A ARP150101604 A AR P150101604A AR P150101604 A ARP150101604 A AR P150101604A AR 101542 A1 AR101542 A1 AR 101542A1
Authority
AR
Argentina
Prior art keywords
peptides
conveyor
portions
multiple oligonucleotides
oligonucleotides
Prior art date
Application number
ARP150101604A
Other languages
English (en)
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of AR101542A1 publication Critical patent/AR101542A1/es

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    • 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
    • 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
    • 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
    • A61K47/62Medicinal 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
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    • 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
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    • 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
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    • 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
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    • 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

Landscapes

  • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

La presente descripción se refiere a oligonucleótidos antisentido (OAS), tales como oligonucleótidos de morfolino fosforodiamidato (PMO). La presente descripción se refiere además a la conjugación de múltiples PMO a péptidos catiónicos de penetración a la célula (CPP) para mejorar la absorción de PMO en células musculares esqueléticas y cardíacas.
ARP150101604A 2014-05-23 2015-05-22 Porciones de múltiples oligonucleótidos de péptidos transportadores AR101542A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462002296P 2014-05-23 2014-05-23

Publications (1)

Publication Number Publication Date
AR101542A1 true AR101542A1 (es) 2016-12-28

Family

ID=53366294

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP150101604A AR101542A1 (es) 2014-05-23 2015-05-22 Porciones de múltiples oligonucleótidos de péptidos transportadores

Country Status (15)

Country Link
US (2) US20170182171A1 (es)
EP (1) EP3151841B1 (es)
JP (2) JP6825914B2 (es)
KR (1) KR102487942B1 (es)
CN (1) CN106459975B (es)
AR (1) AR101542A1 (es)
AU (1) AU2015263963B2 (es)
BR (1) BR112016027236A2 (es)
CA (1) CA2949104C (es)
IL (1) IL249064B (es)
MX (1) MX2016015156A (es)
RU (2) RU2739987C2 (es)
SG (2) SG11201608880VA (es)
TW (1) TWI726844B (es)
WO (1) WO2015179742A1 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2739987C2 (ru) 2014-05-23 2020-12-30 Джензим Корпорейшн Множественные олигонуклеотидные фрагменты на пептидном носителе
CN111542606A (zh) * 2017-09-22 2020-08-14 科罗拉多州立大学董事会法人团体 硫吗啉代寡核苷酸用于治疗肌肉营养不良
US11897911B2 (en) 2018-03-07 2024-02-13 Sanofi Nucleotide precursors, nucleotide analogs and oligomeric compounds containing the same
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

Family Cites Families (13)

* Cited by examiner, † Cited by third party
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
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
ES2351976T3 (es) 2003-04-29 2011-02-14 Avi Biopharma, Inc. Composiciones para mejorar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células.
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2009005793A2 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
WO2009144481A2 (en) 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery 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
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
WO2013040429A1 (en) * 2011-09-14 2013-03-21 Rana Therapeutics Inc. Multimeric oligonucleotide compounds
RU2018123569A (ru) * 2012-09-25 2019-03-07 Джензим Корпорейшн Связанные с пептидом морфолиновые антисмысловые олигонуклеотиды для лечения миотонической дистрофии
WO2015113922A1 (en) * 2014-01-30 2015-08-06 Roche Innovation Center Copenhagen A/S Poly oligomer compound with biocleavable conjugates
RU2739987C2 (ru) 2014-05-23 2020-12-30 Джензим Корпорейшн Множественные олигонуклеотидные фрагменты на пептидном носителе

Also Published As

Publication number Publication date
CN106459975B (zh) 2020-03-27
EP3151841A1 (en) 2017-04-12
SG11201608880VA (en) 2016-11-29
US11103587B2 (en) 2021-08-31
EP3151841B1 (en) 2022-10-19
TW201617373A (zh) 2016-05-16
CN106459975A (zh) 2017-02-22
TWI726844B (zh) 2021-05-11
CA2949104C (en) 2023-09-26
RU2016150629A (ru) 2018-06-25
US20190388547A1 (en) 2019-12-26
RU2020142530A (ru) 2021-04-26
JP2021063128A (ja) 2021-04-22
CA2949104A1 (en) 2015-11-26
RU2016150629A3 (es) 2018-12-24
US20170182171A1 (en) 2017-06-29
AU2015263963A1 (en) 2016-12-01
IL249064B (en) 2021-07-29
RU2739987C2 (ru) 2020-12-30
MX2016015156A (es) 2017-03-27
KR102487942B1 (ko) 2023-01-11
WO2015179742A1 (en) 2015-11-26
JP7269968B2 (ja) 2023-05-09
SG10202004611SA (en) 2020-06-29
BR112016027236A2 (pt) 2017-10-17
JP6825914B2 (ja) 2021-02-03
JP2017517253A (ja) 2017-06-29
IL249064A0 (en) 2017-01-31
AU2015263963B2 (en) 2021-02-18
KR20170005118A (ko) 2017-01-11

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