ECSP20083454A - Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne - Google Patents

Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne

Info

Publication number
ECSP20083454A
ECSP20083454A ECSENADI202083454A ECDI202083454A ECSP20083454A EC SP20083454 A ECSP20083454 A EC SP20083454A EC SENADI202083454 A ECSENADI202083454 A EC SENADI202083454A EC DI202083454 A ECDI202083454 A EC DI202083454A EC SP20083454 A ECSP20083454 A EC SP20083454A
Authority
EC
Ecuador
Prior art keywords
treatment
muscular dystrophy
duchenne muscular
composition including
including anti
Prior art date
Application number
ECSENADI202083454A
Other languages
English (en)
Inventor
Takashi Natsukawa
Tomonori Uno
Youhei Satou
Youichi Egawa
Original Assignee
Nippon Shinyaku Co Ltd
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 Nippon Shinyaku Co Ltd filed Critical Nippon Shinyaku Co Ltd
Publication of ECSP20083454A publication Critical patent/ECSP20083454A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

La presente invención se refiere a una composición que contiene un oligonucleótido antisentido y al uso del mismo para tratar la distrofia muscular de Duchenne. La presente invención se refiere en particular a la composición descrita anteriormente que es eficaz para el tratamiento de la distrofia muscular de Duchenne cuando se administra en una dosis para el tratamiento y el uso de la misma.
ECSENADI202083454A 2018-06-26 2020-12-23 Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne ECSP20083454A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862690270P 2018-06-26 2018-06-26
US201862739386P 2018-10-01 2018-10-01

Publications (1)

Publication Number Publication Date
ECSP20083454A true ECSP20083454A (es) 2021-01-29

Family

ID=68987235

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSENADI202083454A ECSP20083454A (es) 2018-06-26 2020-12-23 Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne

Country Status (19)

Country Link
US (2) US20210261963A1 (es)
EP (1) EP3815696A4 (es)
JP (2) JP7345466B2 (es)
KR (1) KR20210023988A (es)
CN (1) CN112399849A (es)
AU (1) AU2019293687A1 (es)
BR (1) BR112020026542A2 (es)
CA (1) CA3101321A1 (es)
CL (1) CL2020003367A1 (es)
CO (1) CO2020015685A2 (es)
EC (1) ECSP20083454A (es)
IL (1) IL279692A (es)
MX (1) MX2020013880A (es)
PE (1) PE20210630A1 (es)
PH (1) PH12020552078A1 (es)
SG (1) SG11202011554PA (es)
TW (1) TW202035692A (es)
WO (1) WO2020004675A1 (es)
ZA (1) ZA202007682B (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0506830B1 (en) 1989-12-20 2005-12-14 Avi Biopharma, Inc. Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
ES2566628T3 (es) 2002-11-25 2016-04-14 Masafumi Matsuo Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm
EP4047096A1 (en) 2004-06-28 2022-08-24 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2006038608A1 (ja) 2004-10-05 2006-04-13 Nippon Shinyaku Co., Ltd. オリゴ二本鎖ｒｎａ及び医薬組成物
CA2609032A1 (en) 2005-05-30 2006-12-07 Nippon Shinyaku Co., Ltd. Method for producing a nucleic-acid-containing complex preparation
MX2010004955A (es) 2007-11-15 2010-06-30 Avi Biopharma Inc Metodo de sintesis de oligomeros de morfolina.
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP3428278A1 (en) 2008-10-24 2019-01-16 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
EP4043039A1 (en) * 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
CA2903872A1 (en) * 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
NZ631289A (en) 2013-03-15 2017-08-25 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
EP3015467A4 (en) * 2013-05-24 2016-11-02 Ajinomoto Kk PROCESS FOR THE PRODUCTION OF MORPHOLINO-OLIGONUCLEOTIDE
EP3355892A4 (en) * 2015-09-30 2019-05-22 Sarepta Therapeutics, Inc. METHODS OF TREATING MUSCLE DYSTROPHY
FR3044926B1 (fr) * 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine

Also Published As

Publication number Publication date
IL279692A (en) 2021-03-01
JP2023171731A (ja) 2023-12-05
MX2020013880A (es) 2021-03-09
US20210261963A1 (en) 2021-08-26
PH12020552078A1 (en) 2021-05-31
TW202035692A (zh) 2020-10-01
US20240158792A1 (en) 2024-05-16
AU2019293687A1 (en) 2021-01-07
SG11202011554PA (en) 2020-12-30
EP3815696A4 (en) 2022-11-30
JPWO2020004675A1 (ja) 2021-07-15
JP7345466B2 (ja) 2023-09-15
EP3815696A1 (en) 2021-05-05
ZA202007682B (en) 2024-04-24
KR20210023988A (ko) 2021-03-04
CL2020003367A1 (es) 2021-05-24
BR112020026542A2 (pt) 2021-04-06
WO2020004675A1 (ja) 2020-01-02
CA3101321A1 (en) 2020-01-02
CO2020015685A2 (es) 2021-04-30
CN112399849A (zh) 2021-02-23
PE20210630A1 (es) 2021-03-23

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