SG11202011554PA - Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy - Google Patents

Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy

Info

Publication number
SG11202011554PA
SG11202011554PA SG11202011554PA SG11202011554PA SG11202011554PA SG 11202011554P A SG11202011554P A SG 11202011554PA SG 11202011554P A SG11202011554P A SG 11202011554PA SG 11202011554P A SG11202011554P A SG 11202011554PA SG 11202011554P A SG11202011554P A SG 11202011554PA
Authority
SG
Singapore
Prior art keywords
treatment
composition
antisense oligonucleotide
muscular dystrophy
duchenne muscular
Prior art date
Application number
SG11202011554PA
Inventor
Tomonori Uno
Takashi Natsukawa
Youichi Egawa
Youhei Satou
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 SG11202011554PA publication Critical patent/SG11202011554PA/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
    • 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
    • C12N2320/35Special 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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (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)
SG11202011554PA 2018-06-26 2019-06-26 Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy SG11202011554PA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862690270P 2018-06-26 2018-06-26
US201862739386P 2018-10-01 2018-10-01
PCT/JP2019/026393 WO2020004675A1 (en) 2018-06-26 2019-06-26 Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy

Publications (1)

Publication Number Publication Date
SG11202011554PA true SG11202011554PA (en) 2020-12-30

Family

ID=68987235

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202011554PA SG11202011554PA (en) 2018-06-26 2019-06-26 Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy

Country Status (19)

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

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240004609A (en) 2021-04-30 2024-01-11 사렙타 쎄러퓨틱스 인코퍼레이티드 Treatment Methods for Muscular Dystrophy
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
WO2024173582A2 (en) * 2023-02-14 2024-08-22 Stoke Therapeutics, Inc. Antisense oligomer formulations

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991009033A1 (en) 1989-12-20 1991-06-27 Anti-Gene Development Group Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
ES2509140T3 (en) 2002-11-25 2014-10-17 Masafumi Matsuo ENA nucleic acid drugs that modify splicing in mRNA precursors
USRE47769E1 (en) 2004-06-28 2019-12-17 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2006038608A1 (en) 2004-10-05 2006-04-13 Nippon Shinyaku Co., Ltd. Oligo double-stranded rna and medicinal composition
JP5056413B2 (en) 2005-05-30 2012-10-24 日本新薬株式会社 Method for producing nucleic acid-containing complex preparation
DK2207779T3 (en) 2007-11-15 2014-07-14 Sarepta Therapeutics Inc Process for the Synthesis of Morpholine Oligomers
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2350281B1 (en) 2008-10-24 2014-05-14 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
TWI541024B (en) * 2010-09-01 2016-07-11 日本新藥股份有限公司 Antisense nucleic acid
CA2862628C (en) * 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
TR201903009T4 (en) * 2013-03-14 2019-03-21 Sarepta Therapeutics Inc Exon skipping compositions for the treatment of muscular dystrophy.
CA2906812A1 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
JP6477464B2 (en) * 2013-05-24 2019-03-06 味の素株式会社 Method for producing morpholino oligonucleotide
WO2017059131A1 (en) * 2015-09-30 2017-04-06 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
FR3044926B1 (en) * 2015-12-09 2020-01-31 Genethon EFFICIENT GENE THERAPY TOOLS FOR JUMPING DYSTROPHIN EXON 53

Also Published As

Publication number Publication date
CO2020015685A2 (en) 2021-04-30
WO2020004675A1 (en) 2020-01-02
EP3815696A4 (en) 2022-11-30
EP3815696A1 (en) 2021-05-05
IL279692A (en) 2021-03-01
JP7345466B2 (en) 2023-09-15
BR112020026542A2 (en) 2021-04-06
CA3101321A1 (en) 2020-01-02
US20210261963A1 (en) 2021-08-26
MX2020013880A (en) 2021-03-09
ZA202007682B (en) 2024-04-24
US20240158792A1 (en) 2024-05-16
JPWO2020004675A1 (en) 2021-07-15
CN112399849A (en) 2021-02-23
AU2019293687A1 (en) 2021-01-07
CL2020003367A1 (en) 2021-05-24
ECSP20083454A (en) 2021-01-29
PE20210630A1 (en) 2021-03-23
KR20210023988A (en) 2021-03-04
PH12020552078A1 (en) 2021-05-31
TW202035692A (en) 2020-10-01
JP2023171731A (en) 2023-12-05

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