CA2949104C - Multiple oligonucleotide moieties on peptide carrier - Google Patents

Multiple oligonucleotide moieties on peptide carrier Download PDF

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Publication number
CA2949104C
CA2949104C CA2949104A CA2949104A CA2949104C CA 2949104 C CA2949104 C CA 2949104C CA 2949104 A CA2949104 A CA 2949104A CA 2949104 A CA2949104 A CA 2949104A CA 2949104 C CA2949104 C CA 2949104C
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CA
Canada
Prior art keywords
aon
cpp
peptide
conjugate
rxrrbr
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Active
Application number
CA2949104A
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English (en)
French (fr)
Other versions
CA2949104A1 (en
Inventor
Timothy E. Weeden
Carol A. Nelson
Bruce M. Wentworth
Nicholas P. CLAYTON
Andrew Leger
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
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Genzyme Corp
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Publication of CA2949104A1 publication Critical patent/CA2949104A1/en
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Active legal-status Critical Current
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Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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)
  • 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)
CA2949104A 2014-05-23 2015-05-22 Multiple oligonucleotide moieties on peptide carrier Active CA2949104C (en)

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)

Publication Number Publication Date
CA2949104A1 CA2949104A1 (en) 2015-11-26
CA2949104C true CA2949104C (en) 2023-09-26

Family

ID=53366294

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2949104A Active CA2949104C (en) 2014-05-23 2015-05-22 Multiple oligonucleotide moieties on peptide carrier

Country Status (15)

Country Link
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

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
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
US10111962B2 (en) * 2012-09-25 2018-10-30 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
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

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
CN106459975B (zh) 2020-03-27

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