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
Other languages
English (en)
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)
  • 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)
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 (ja) 2018-06-26 2019-06-26 アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用

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

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
ECSP20083454A (es) 2021-01-29
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
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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