MX2016015156A - Restos oligonucleotidicos multiples sobre soporte peptidico. - Google Patents

Restos oligonucleotidicos multiples sobre soporte peptidico.

Info

Publication number
MX2016015156A
MX2016015156A MX2016015156A MX2016015156A MX2016015156A MX 2016015156 A MX2016015156 A MX 2016015156A MX 2016015156 A MX2016015156 A MX 2016015156A MX 2016015156 A MX2016015156 A MX 2016015156A MX 2016015156 A MX2016015156 A MX 2016015156A
Authority
MX
Mexico
Prior art keywords
pmos
peptide carrier
multiple oligonucleotide
oligonucleotide moieties
present disclosure
Prior art date
Application number
MX2016015156A
Other languages
English (en)
Inventor
E Weeden Timothy
Leger Andrew
A Nelson Carol
P Clayton Nicholas
M Wentworth Bruce
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 MX2016015156A publication Critical patent/MX2016015156A/es

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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
    • 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

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)
  • 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)

Abstract

La presente descripción se refiere a oligonucleótidos antisentido (OAS), tal 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 cardiacas.
MX2016015156A 2014-05-23 2015-05-22 Restos oligonucleotidicos multiples sobre soporte peptidico. MX2016015156A (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
MX2016015156A true MX2016015156A (es) 2017-03-27

Family

ID=53366294

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016015156A MX2016015156A (es) 2014-05-23 2015-05-22 Restos oligonucleotidicos multiples sobre soporte peptidico.

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) SG10202004611SA (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
SG10202004611SA (en) 2014-05-23 2020-06-29 Genzyme Corp Multiple oligonucleotide moieties on peptide carrier
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
US10765760B2 (en) * 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2021044004A1 (en) * 2019-09-05 2021-03-11 Sanofi Oligonucleotides containing nucleotide analogs
WO2021211572A1 (en) * 2020-04-14 2021-10-21 Oregon State University Antisense therapeutics for the treatment of coronavirus
EP4387676A2 (en) * 2021-09-03 2024-06-26 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
AU7634694A (en) * 1993-08-20 1995-03-21 University Of Medicine And 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
WO2004097017A2 (en) 2003-04-29 2004-11-11 Avi Biopharma, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
EP2170363B1 (en) 2007-06-29 2018-08-08 Sarepta Therapeutics, 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
EP2283029A1 (en) 2008-06-04 2011-02-16 Medical Research Council Peptides
EP3428278A1 (en) 2008-10-24 2019-01-16 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
CA2848753C (en) * 2011-09-14 2022-07-26 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
TWI677350B (zh) * 2012-09-25 2019-11-21 美商健臻公司 治療強直性肌失養症之經胜肽連結之嗎啉基反義寡核苷酸
EP3099797B1 (en) * 2014-01-30 2019-08-21 F. Hoffmann-La Roche AG Poly oligomer compound with biocleavable conjugates
SG10202004611SA (en) 2014-05-23 2020-06-29 Genzyme Corp Multiple oligonucleotide moieties on peptide carrier

Also Published As

Publication number Publication date
KR20170005118A (ko) 2017-01-11
SG10202004611SA (en) 2020-06-29
AU2015263963A1 (en) 2016-12-01
RU2016150629A (ru) 2018-06-25
AR101542A1 (es) 2016-12-28
EP3151841B1 (en) 2022-10-19
TWI726844B (zh) 2021-05-11
BR112016027236A2 (pt) 2017-10-17
US20170182171A1 (en) 2017-06-29
SG11201608880VA (en) 2016-11-29
RU2016150629A3 (es) 2018-12-24
CN106459975A (zh) 2017-02-22
JP7269968B2 (ja) 2023-05-09
JP6825914B2 (ja) 2021-02-03
JP2017517253A (ja) 2017-06-29
TW201617373A (zh) 2016-05-16
RU2020142530A (ru) 2021-04-26
AU2015263963B2 (en) 2021-02-18
CA2949104A1 (en) 2015-11-26
RU2739987C2 (ru) 2020-12-30
EP3151841A1 (en) 2017-04-12
JP2021063128A (ja) 2021-04-22
CA2949104C (en) 2023-09-26
IL249064A0 (en) 2017-01-31
IL249064B (en) 2021-07-29
US20190388547A1 (en) 2019-12-26
CN106459975B (zh) 2020-03-27
KR102487942B1 (ko) 2023-01-11
WO2015179742A1 (en) 2015-11-26
US11103587B2 (en) 2021-08-31

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