MX369519B - Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica. - Google Patents

Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica.

Info

Publication number
MX369519B
MX369519B MX2015003841A MX2015003841A MX369519B MX 369519 B MX369519 B MX 369519B MX 2015003841 A MX2015003841 A MX 2015003841A MX 2015003841 A MX2015003841 A MX 2015003841A MX 369519 B MX369519 B MX 369519B
Authority
MX
Mexico
Prior art keywords
peptide
treatment
antisense oligonucleotides
linked morpholino
myotonic dystrophy
Prior art date
Application number
MX2015003841A
Other languages
English (en)
Other versions
MX2015003841A (es
Inventor
Cheng Seng
E Weeden Timothy
Leger Andrew
Wentworth Bruce
A Nelson Carol
Clayton Nicholas
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 MX2015003841A publication Critical patent/MX2015003841A/es
Publication of MX369519B publication Critical patent/MX369519B/es

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    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • C12N2310/32—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)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente invención se refiere a oligonucleótidos antisentido morfolino unidos a péptido (PPMO) que están dirigidos a la extensión de repetición poli CUG en la región no traducida 3' del gen que codifica la proteína quinasa de la distrofia miotónica (DMPK) y métodos para la administración sistémica del mismo para el tratamiento de la distrofia miotónica tipo 1 (DM1).
MX2015003841A 2012-09-25 2013-09-24 Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica. MX369519B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261705335P 2012-09-25 2012-09-25
PCT/US2013/061320 WO2014052276A1 (en) 2012-09-25 2013-09-24 Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

Publications (2)

Publication Number Publication Date
MX2015003841A MX2015003841A (es) 2015-07-17
MX369519B true MX369519B (es) 2019-11-11

Family

ID=49301675

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2015003841A MX369519B (es) 2012-09-25 2013-09-24 Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica.

Country Status (15)

Country Link
US (1) US10111962B2 (es)
EP (1) EP2900821B1 (es)
JP (3) JP6404219B2 (es)
KR (1) KR102240217B1 (es)
CN (1) CN104837997A (es)
AR (1) AR092658A1 (es)
AU (2) AU2013323820B2 (es)
BR (1) BR112015006049A2 (es)
CA (1) CA2886017C (es)
IL (1) IL237457B (es)
MX (1) MX369519B (es)
RU (2) RU2018123569A (es)
SG (2) SG10201700751XA (es)
TW (1) TWI677350B (es)
WO (1) WO2014052276A1 (es)

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PH12021552592A1 (en) * 2019-05-28 2022-06-20 Modalis Therapeutics Corp Method for treating muscular dystrophy by targeting dmpk gene
GB201911403D0 (en) * 2019-08-09 2019-09-25 Univ Oxford Innovation Ltd Conjugate and uses thereof
SI4017871T1 (sl) 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
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KR20230128050A (ko) 2020-12-31 2023-09-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 근긴장성 이영양증을 치료하기위한 그의 용도
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Also Published As

Publication number Publication date
BR112015006049A2 (pt) 2017-08-22
JP6404219B2 (ja) 2018-10-10
US20150238627A1 (en) 2015-08-27
AU2013323820A1 (en) 2015-04-16
CN104837997A (zh) 2015-08-12
CA2886017C (en) 2022-04-05
EP2900821A1 (en) 2015-08-05
WO2014052276A1 (en) 2014-04-03
RU2015115721A (ru) 2016-11-20
SG11201501544PA (en) 2015-03-30
EP2900821B1 (en) 2020-04-01
AR092658A1 (es) 2015-04-29
JP2020196751A (ja) 2020-12-10
RU2662962C2 (ru) 2018-07-31
JP2019006808A (ja) 2019-01-17
TWI677350B (zh) 2019-11-21
RU2018123569A (ru) 2019-03-07
SG10201700751XA (en) 2017-03-30
JP2015532264A (ja) 2015-11-09
AU2019203929A1 (en) 2019-07-11
IL237457B (en) 2022-02-01
IL237457A0 (en) 2015-04-30
RU2018123569A3 (es) 2021-11-09
TW201424752A (zh) 2014-07-01
AU2013323820B2 (en) 2019-06-27
MX2015003841A (es) 2015-07-17
CA2886017A1 (en) 2014-04-03
KR20150060738A (ko) 2015-06-03
KR102240217B1 (ko) 2021-04-14
US10111962B2 (en) 2018-10-30

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