AU2019293687B2 - 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

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Publication number
AU2019293687B2
AU2019293687B2 AU2019293687A AU2019293687A AU2019293687B2 AU 2019293687 B2 AU2019293687 B2 AU 2019293687B2 AU 2019293687 A AU2019293687 A AU 2019293687A AU 2019293687 A AU2019293687 A AU 2019293687A AU 2019293687 B2 AU2019293687 B2 AU 2019293687B2
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AU
Australia
Prior art keywords
week
treatment
viltolarsen
pharmaceutical composition
dystrophin
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
AU2019293687A
Other languages
English (en)
Other versions
AU2019293687A1 (en
Inventor
Youichi EGAWA
Takashi Natsukawa
Youhei Satou
Tomonori UNO
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nippon Shinyaku Co Ltd
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 AU2019293687A1 publication Critical patent/AU2019293687A1/en
Application granted granted Critical
Publication of AU2019293687B2 publication Critical patent/AU2019293687B2/en
Priority to AU2025283628A priority Critical patent/AU2025283628A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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
    • 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
    • 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)
AU2019293687A 2018-06-26 2019-06-26 Composition comprising antisense oligonucleotide and use thereof for treatment of Duchenne muscular dystrophy Active AU2019293687B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025283628A AU2025283628A1 (en) 2018-06-26 2025-12-19 Composition comprising antisense oligonucleotide and use thereof for treatment of Duchenne muscular dystrophy

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862690270P 2018-06-26 2018-06-26
US62/690,270 2018-06-26
US201862739386P 2018-10-01 2018-10-01
US62/739,386 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2025283628A Division AU2025283628A1 (en) 2018-06-26 2025-12-19 Composition comprising antisense oligonucleotide and use thereof for treatment of Duchenne muscular dystrophy

Publications (2)

Publication Number Publication Date
AU2019293687A1 AU2019293687A1 (en) 2021-01-07
AU2019293687B2 true AU2019293687B2 (en) 2025-10-02

Family

ID=68987235

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2019293687A Active AU2019293687B2 (en) 2018-06-26 2019-06-26 Composition comprising antisense oligonucleotide and use thereof for treatment of Duchenne muscular dystrophy
AU2025283628A Pending AU2025283628A1 (en) 2018-06-26 2025-12-19 Composition comprising antisense oligonucleotide and use thereof for treatment of Duchenne muscular dystrophy

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2025283628A Pending AU2025283628A1 (en) 2018-06-26 2025-12-19 Composition comprising antisense oligonucleotide and use thereof for treatment of Duchenne muscular dystrophy

Country Status (21)

Country Link
US (2) US20210261963A1 (https=)
EP (1) EP3815696A4 (https=)
JP (3) JP7345466B2 (https=)
KR (1) KR20210023988A (https=)
CN (1) CN112399849A (https=)
AU (2) AU2019293687B2 (https=)
BR (1) BR112020026542A2 (https=)
CA (1) CA3101321A1 (https=)
CL (1) CL2020003367A1 (https=)
CO (1) CO2020015685A2 (https=)
EC (1) ECSP20083454A (https=)
IL (1) IL279692A (https=)
MX (2) MX2020013880A (https=)
MY (1) MY209148A (https=)
NZ (1) NZ770540A (https=)
PE (1) PE20210630A1 (https=)
PH (1) PH12020552078A1 (https=)
SG (1) SG11202011554PA (https=)
TW (1) TW202035692A (https=)
WO (1) WO2020004675A1 (https=)
ZA (1) ZA202007682B (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
IL307937A (en) 2021-04-30 2023-12-01 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy
CN119013401A (zh) 2022-03-17 2024-11-22 萨勒普塔医疗公司 二氨基磷酸酯吗啉代寡聚物缀合物
KR20250006069A (ko) * 2022-05-05 2025-01-10 바이오마린 파머수티컬 인크. 뒤시엔느 근이영양증을 치료하는 방법
CN121194802A (zh) * 2023-02-14 2025-12-23 斯托克制药公司 反义寡聚物调配物
WO2024233303A1 (en) * 2023-05-05 2024-11-14 Biomarin Pharmaceutical Inc. Dystrophin exon skipping oligonucleotides

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2612917A1 (en) * 2010-09-01 2013-07-10 Nippon Shinyaku Co., Ltd. Antisense nucleic acid

Family Cites Families (14)

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Publication number Priority date Publication date Assignee Title
AU655164B2 (en) 1989-12-20 1994-12-08 Antivirals Inc. Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
EP2392660A3 (en) 2002-11-25 2012-03-28 Masafumi Matsuo ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor
EP2933332A1 (en) 2004-06-28 2015-10-21 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
EP1886688A4 (en) 2005-05-30 2013-01-09 Nippon Shinyaku Co Ltd METHOD FOR PRODUCING PREPARATION OF A NUCLEIC ACID-CONTAINING COMPLEX
RU2606627C2 (ru) 2007-11-15 2017-01-10 Серепта Терапьютикс,Инк. Способ синтеза морфолиновых олигомеров
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
NZ627896A (en) * 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
US20140315977A1 (en) * 2013-03-14 2014-10-23 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
DK3015467T3 (da) * 2013-05-24 2025-02-10 Ajinomoto Kk Morpholino-oligonukleotidfremstillingsfremgangsmåde
WO2017059131A1 (en) * 2015-09-30 2017-04-06 Sarepta Therapeutics, Inc. Methods for treating muscular 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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2612917A1 (en) * 2010-09-01 2013-07-10 Nippon Shinyaku Co., Ltd. Antisense nucleic acid

Also Published As

Publication number Publication date
CL2020003367A1 (es) 2021-05-24
BR112020026542A2 (pt) 2021-04-06
MX2025001486A (es) 2025-03-07
JP7595719B2 (ja) 2024-12-06
PH12020552078A1 (en) 2021-05-31
TW202035692A (zh) 2020-10-01
WO2020004675A1 (ja) 2020-01-02
CN112399849A (zh) 2021-02-23
SG11202011554PA (en) 2020-12-30
ECSP20083454A (es) 2021-01-29
EP3815696A1 (en) 2021-05-05
CO2020015685A2 (es) 2021-04-30
ZA202007682B (en) 2024-04-24
AU2019293687A1 (en) 2021-01-07
CA3101321A1 (en) 2020-01-02
US20240158792A1 (en) 2024-05-16
JP2025028961A (ja) 2025-03-05
MX2020013880A (es) 2021-03-09
JP7345466B2 (ja) 2023-09-15
MY209148A (en) 2025-06-24
EP3815696A4 (en) 2022-11-30
NZ770540A (en) 2025-12-19
JP2023171731A (ja) 2023-12-05
IL279692A (en) 2021-03-01
KR20210023988A (ko) 2021-03-04
PE20210630A1 (es) 2021-03-23
AU2025283628A1 (en) 2026-01-22
JPWO2020004675A1 (ja) 2021-07-15
US20210261963A1 (en) 2021-08-26

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