IL279692A - A preparation containing antisense oligonucleotides and their uses for the treatment of Duchenne syndrome - Google Patents

A preparation containing antisense oligonucleotides and their uses for the treatment of Duchenne syndrome

Info

Publication number
IL279692A
IL279692A IL279692A IL27969220A IL279692A IL 279692 A IL279692 A IL 279692A IL 279692 A IL279692 A IL 279692A IL 27969220 A IL27969220 A IL 27969220A IL 279692 A IL279692 A IL 279692A
Authority
IL
Israel
Prior art keywords
treatment
composition
antisense oligonucleotide
muscular dystrophy
duchenne muscular
Prior art date
Application number
IL279692A
Other languages
English (en)
Hebrew (he)
Inventor
Satou Youhei
UNO Tomonori
NATSUKAWA Takashi
EGAWA Youichi
Original Assignee
Nippon Shinyaku Co Ltd
Satou Youhei
UNO Tomonori
NATSUKAWA Takashi
EGAWA Youichi
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, Satou Youhei, UNO Tomonori, NATSUKAWA Takashi, EGAWA Youichi filed Critical Nippon Shinyaku Co Ltd
Publication of IL279692A publication Critical patent/IL279692A/en

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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)
IL279692A 2018-06-26 2020-12-22 A preparation containing antisense oligonucleotides and their uses for the treatment of Duchenne syndrome IL279692A (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
IL279692A true IL279692A (en) 2021-03-01

Family

ID=68987235

Family Applications (1)

Application Number Title Priority Date Filing Date
IL279692A IL279692A (en) 2018-06-26 2020-12-22 A preparation containing antisense oligonucleotides and their uses for the treatment of Duchenne syndrome

Country Status (20)

Country Link
US (2) US20210261963A1 (OSRAM)
EP (1) EP3815696A4 (OSRAM)
JP (3) JP7345466B2 (OSRAM)
KR (1) KR20210023988A (OSRAM)
CN (1) CN112399849A (OSRAM)
AU (1) AU2019293687B2 (OSRAM)
BR (1) BR112020026542A2 (OSRAM)
CA (1) CA3101321A1 (OSRAM)
CL (1) CL2020003367A1 (OSRAM)
CO (1) CO2020015685A2 (OSRAM)
EC (1) ECSP20083454A (OSRAM)
IL (1) IL279692A (OSRAM)
MX (2) MX2020013880A (OSRAM)
MY (1) MY209148A (OSRAM)
PE (1) PE20210630A1 (OSRAM)
PH (1) PH12020552078A1 (OSRAM)
SG (1) SG11202011554PA (OSRAM)
TW (1) TW202035692A (OSRAM)
WO (1) WO2020004675A1 (OSRAM)
ZA (1) ZA202007682B (OSRAM)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
KR20240004609A (ko) 2021-04-30 2024-01-11 사렙타 쎄러퓨틱스 인코퍼레이티드 근이영양증에 대한 치료 방법
EP4493693A1 (en) 2022-03-17 2025-01-22 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
AU2024222908A1 (en) * 2023-02-14 2025-09-04 Stoke Therapeutics, Inc. Antisense oligomer formulations
US20240368596A1 (en) * 2023-05-05 2024-11-07 Biomarin Pharmaceutical Inc. Dystrophin exon skipping oligonucleotides

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
CA3001404C (en) 2002-11-25 2020-07-28 Masafumi Matsuo Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
PL1766010T3 (pl) 2004-06-28 2011-07-29 Univ Western Australia Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
EP1811024A1 (en) 2004-10-05 2007-07-25 Nippon Shinyaku Co., Ltd. Oligo double-stranded rna and medicinal composition
US20090069260A1 (en) 2005-05-30 2009-03-12 Nippon Shinyaku Co., Ltd Method for producing a nucleic-acid-containing complex preparation
CA2704261C (en) 2007-11-15 2015-05-26 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
SMT201900159T1 (it) 2008-10-24 2019-05-10 Sarepta Therapeutics Inc Composizioni di salto di esone per dmd
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
BR112014018427B1 (pt) * 2012-01-27 2021-11-03 Biomarin Technologies B.V. Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker
KR20240094032A (ko) * 2013-03-14 2024-06-24 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
JP6477464B2 (ja) * 2013-05-24 2019-03-06 味の素株式会社 モルフォリノオリゴヌクレオチドの製造方法
JP2018529715A (ja) * 2015-09-30 2018-10-11 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための方法
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
PH12020552078A1 (en) 2021-05-31
TW202035692A (zh) 2020-10-01
SG11202011554PA (en) 2020-12-30
CN112399849A (zh) 2021-02-23
JP7345466B2 (ja) 2023-09-15
EP3815696A4 (en) 2022-11-30
CO2020015685A2 (es) 2021-04-30
AU2019293687B2 (en) 2025-10-02
ECSP20083454A (es) 2021-01-29
KR20210023988A (ko) 2021-03-04
WO2020004675A1 (ja) 2020-01-02
JP2023171731A (ja) 2023-12-05
AU2019293687A1 (en) 2021-01-07
US20210261963A1 (en) 2021-08-26
EP3815696A1 (en) 2021-05-05
CA3101321A1 (en) 2020-01-02
MY209148A (en) 2025-06-24
BR112020026542A2 (pt) 2021-04-06
CL2020003367A1 (es) 2021-05-24
JPWO2020004675A1 (ja) 2021-07-15
JP7595719B2 (ja) 2024-12-06
MX2025001486A (es) 2025-03-07
ZA202007682B (en) 2024-04-24
JP2025028961A (ja) 2025-03-05
US20240158792A1 (en) 2024-05-16
PE20210630A1 (es) 2021-03-23
MX2020013880A (es) 2021-03-09

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