CA3101321A1 - 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 Download PDF

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Publication number
CA3101321A1
CA3101321A1 CA3101321A CA3101321A CA3101321A1 CA 3101321 A1 CA3101321 A1 CA 3101321A1 CA 3101321 A CA3101321 A CA 3101321A CA 3101321 A CA3101321 A CA 3101321A CA 3101321 A1 CA3101321 A1 CA 3101321A1
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CA
Canada
Prior art keywords
week
pharmaceutical composition
treatment
patients
time
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Pending
Application number
CA3101321A
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English (en)
French (fr)
Inventor
Tomonori UNO
Takashi Natsukawa
Youichi EGAWA
Youhei Satou
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
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Nippon Shinyaku Co Ltd
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Application filed by Nippon Shinyaku Co Ltd filed Critical Nippon Shinyaku Co Ltd
Publication of CA3101321A1 publication Critical patent/CA3101321A1/en
Pending legal-status Critical Current

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    • 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)
CA3101321A 2018-06-26 2019-06-26 Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy Pending CA3101321A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3101321A1 true CA3101321A1 (en) 2020-01-02

Family

ID=68987235

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3101321A Pending CA3101321A1 (en) 2018-06-26 2019-06-26 Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy

Country Status (21)

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

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

Family Cites Families (15)

* Cited by examiner, † Cited by third party
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
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
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

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
AU2019293687B2 (en) 2025-10-02
EP3815696A1 (en) 2021-05-05
CO2020015685A2 (es) 2021-04-30
ZA202007682B (en) 2024-04-24
AU2019293687A1 (en) 2021-01-07
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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