KR20210023988A - 안티센스 올리고뉴클레오티드를 함유하는 조성물 및 듀시엔형 근이영양증의 치료로의 그의 사용 - Google Patents

안티센스 올리고뉴클레오티드를 함유하는 조성물 및 듀시엔형 근이영양증의 치료로의 그의 사용 Download PDF

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KR20210023988A
KR20210023988A KR1020217001037A KR20217001037A KR20210023988A KR 20210023988 A KR20210023988 A KR 20210023988A KR 1020217001037 A KR1020217001037 A KR 1020217001037A KR 20217001037 A KR20217001037 A KR 20217001037A KR 20210023988 A KR20210023988 A KR 20210023988A
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도모노리 우노
다카시 나츠카와
요우이치 에가와
요우헤이 사토우
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니뽄 신야쿠 가부시키가이샤
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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
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    • 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
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    • 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

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  • 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)
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  • 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)
KR1020217001037A 2018-06-26 2019-06-26 안티센스 올리고뉴클레오티드를 함유하는 조성물 및 듀시엔형 근이영양증의 치료로의 그의 사용 Ceased KR20210023988A (ko)

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

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KR20210023988A true KR20210023988A (ko) 2021-03-04

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KR1020217001037A Ceased KR20210023988A (ko) 2018-06-26 2019-06-26 안티센스 올리고뉴클레오티드를 함유하는 조성물 및 듀시엔형 근이영양증의 치료로의 그의 사용

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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=)

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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 (4)

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WO2004048570A1 (ja) 2002-11-25 2004-06-10 Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy ｍＲＮＡ前駆体のスプライシングを修飾するＥＮＡ核酸医薬
WO2006000057A1 (en) 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2010048586A1 (en) 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
US20100168212A1 (en) 2008-09-11 2010-07-01 Royal Holloway, University Of London Oligomers

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AU655164B2 (en) 1989-12-20 1994-12-08 Antivirals Inc. Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
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
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

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Publication number Priority date Publication date Assignee Title
WO2004048570A1 (ja) 2002-11-25 2004-06-10 Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy ｍＲＮＡ前駆体のスプライシングを修飾するＥＮＡ核酸医薬
WO2006000057A1 (en) 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US20100168212A1 (en) 2008-09-11 2010-07-01 Royal Holloway, University Of London Oligomers
WO2010048586A1 (en) 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd

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Annemieke Aartsma―Rus et al., (2002) Neuromuscular Disorders 12：S71―S77
Bladen C. L. et al., Human Mutation (2015) 36：395―402
Linda J. Popplewell et al., (2010) Neuromuscular Disorders, vol.20, no.2, p.102―10
Matsuo M., Brain Dev 1996；18：p.167―172
Monaco A. P. et al., Genomics 1988；2：p.90―95
Wilton S. D. et al., Molecular Therapy 2007：15：p.1288―96

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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
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
PE20210630A1 (es) 2021-03-23
AU2025283628A1 (en) 2026-01-22
JPWO2020004675A1 (ja) 2021-07-15
US20210261963A1 (en) 2021-08-26

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