CO2020015685A2 - Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne - Google Patents

Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne

Info

Publication number
CO2020015685A2
CO2020015685A2 CONC2020/0015685A CO2020015685A CO2020015685A2 CO 2020015685 A2 CO2020015685 A2 CO 2020015685A2 CO 2020015685 A CO2020015685 A CO 2020015685A CO 2020015685 A2 CO2020015685 A2 CO 2020015685A2
Authority
CO
Colombia
Prior art keywords
treatment
muscular dystrophy
duchenne muscular
composition
antisense oligonucleotide
Prior art date
Application number
CONC2020/0015685A
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 CO2020015685A2 publication Critical patent/CO2020015685A2/es

Links

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
    • 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)

Abstract

La presente invención se refiere a una composición que contiene un oligonucleótido antisentido y al uso del mismo para tratar la distrofia muscular de Duchenne. La presente invención se refiere en particular a la composición descrita anteriormente que es eficaz para el tratamiento de la distrofia muscular de Duchenne cuando se administra en una dosis para el tratamiento y el uso de la misma.
CONC2020/0015685A 2018-06-26 2020-12-14 Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne CO2020015685A2 (es)

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
CO2020015685A2 true CO2020015685A2 (es) 2021-04-30

Family

ID=68987235

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2020/0015685A CO2020015685A2 (es) 2018-06-26 2020-12-14 Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne

Country Status (19)

Country Link
US (2) US20210261963A1 (es)
EP (1) EP3815696A4 (es)
JP (2) JP7345466B2 (es)
KR (1) KR20210023988A (es)
CN (1) CN112399849A (es)
AU (1) AU2019293687A1 (es)
BR (1) BR112020026542A2 (es)
CA (1) CA3101321A1 (es)
CL (1) CL2020003367A1 (es)
CO (1) CO2020015685A2 (es)
EC (1) ECSP20083454A (es)
IL (1) IL279692A (es)
MX (1) MX2020013880A (es)
PE (1) PE20210630A1 (es)
PH (1) PH12020552078A1 (es)
SG (1) SG11202011554PA (es)
TW (1) TW202035692A (es)
WO (1) WO2020004675A1 (es)
ZA (1) ZA202007682B (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3216839A1 (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
KR0167574B1 (ko) 1989-12-20 1999-01-15 제임스 섬머톤 소단위 사이에 인-함유 키랄결합을 가지는 비전하 모르폴리노기저 중합체
ES2561851T3 (es) 2002-11-25 2016-03-01 Masafumi Matsuo Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm
ES2361325T3 (es) 2004-06-28 2011-06-16 The University Of Western Australia Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos.
EP1811024A1 (en) 2004-10-05 2007-07-25 Nippon Shinyaku Co., Ltd. Oligo double-stranded rna and medicinal composition
JP5056413B2 (ja) 2005-05-30 2012-10-24 日本新薬株式会社 核酸含有複合体製剤の製造方法
BR122019021332A8 (pt) 2007-11-15 2022-12-06 Avi Biopharma Inc Processo de síntese de oligômeros de morfolino
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
PL3133160T3 (pl) 2008-10-24 2019-06-28 Sarepta Therapeutics, Inc. Kompozycje do pomijania eksonów w DMD
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
JP2015509922A (ja) * 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
PL2970964T3 (pl) * 2013-03-14 2019-06-28 Sarepta Therapeutics, Inc. Kompozycje pomijające eksony do leczenia dystrofii mięśniowej
KR20200139271A (ko) 2013-03-15 2020-12-11 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 개선된 조성물
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
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
ECSP20083454A (es) 2021-01-29
AU2019293687A1 (en) 2021-01-07
US20240158792A1 (en) 2024-05-16
EP3815696A4 (en) 2022-11-30
CL2020003367A1 (es) 2021-05-24
WO2020004675A1 (ja) 2020-01-02
BR112020026542A2 (pt) 2021-04-06
EP3815696A1 (en) 2021-05-05
CA3101321A1 (en) 2020-01-02
CN112399849A (zh) 2021-02-23
PH12020552078A1 (en) 2021-05-31
TW202035692A (zh) 2020-10-01
JPWO2020004675A1 (ja) 2021-07-15
PE20210630A1 (es) 2021-03-23
MX2020013880A (es) 2021-03-09
JP7345466B2 (ja) 2023-09-15
ZA202007682B (en) 2024-04-24
JP2023171731A (ja) 2023-12-05
SG11202011554PA (en) 2020-12-30
KR20210023988A (ko) 2021-03-04
US20210261963A1 (en) 2021-08-26
IL279692A (en) 2021-03-01

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