CL2020003367A1 - 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
CL2020003367A1
CL2020003367A1 CL2020003367A CL2020003367A CL2020003367A1 CL 2020003367 A1 CL2020003367 A1 CL 2020003367A1 CL 2020003367 A CL2020003367 A CL 2020003367A CL 2020003367 A CL2020003367 A CL 2020003367A CL 2020003367 A1 CL2020003367 A1 CL 2020003367A1
Authority
CL
Chile
Prior art keywords
treatment
muscular dystrophy
duchenne muscular
composition
antisense oligonucleotide
Prior art date
Application number
CL2020003367A
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 CL2020003367A1 publication Critical patent/CL2020003367A1/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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (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.
CL2020003367A 2018-06-26 2020-12-23 Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne CL2020003367A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862690270P 2018-06-26 2018-06-26
US201862739386P 2018-10-01 2018-10-01

Publications (1)

Publication Number Publication Date
CL2020003367A1 true CL2020003367A1 (es) 2021-05-24

Family

ID=68987235

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020003367A CL2020003367A1 (es) 2018-06-26 2020-12-23 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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240004609A (ko) 2021-04-30 2024-01-11 사렙타 쎄러퓨틱스 인코퍼레이티드 근이영양증에 대한 치료 방법
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
WO2024173582A2 (en) * 2023-02-14 2024-08-22 Stoke Therapeutics, Inc. Antisense oligomer formulations

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991009033A1 (en) 1989-12-20 1991-06-27 Anti-Gene Development Group Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
ES2509140T3 (es) 2002-11-25 2014-10-17 Masafumi Matsuo Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm
USRE47769E1 (en) 2004-06-28 2019-12-17 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
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
DK2207779T3 (da) 2007-11-15 2014-07-14 Sarepta Therapeutics Inc Fremgangsmåde til syntese af morpholinooligomerer
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2350281B1 (en) 2008-10-24 2014-05-14 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CA2862628C (en) * 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
TR201903009T4 (tr) * 2013-03-14 2019-03-21 Sarepta Therapeutics Inc Musküler distrofi tedavisine yönelik ekson atlama bileşimleri.
CA2906812A1 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
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
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
SG11202011554PA (en) 2020-12-30
CO2020015685A2 (es) 2021-04-30
WO2020004675A1 (ja) 2020-01-02
EP3815696A4 (en) 2022-11-30
EP3815696A1 (en) 2021-05-05
IL279692A (en) 2021-03-01
JP7345466B2 (ja) 2023-09-15
BR112020026542A2 (pt) 2021-04-06
CA3101321A1 (en) 2020-01-02
US20210261963A1 (en) 2021-08-26
MX2020013880A (es) 2021-03-09
ZA202007682B (en) 2024-04-24
US20240158792A1 (en) 2024-05-16
JPWO2020004675A1 (ja) 2021-07-15
CN112399849A (zh) 2021-02-23
AU2019293687A1 (en) 2021-01-07
ECSP20083454A (es) 2021-01-29
PE20210630A1 (es) 2021-03-23
KR20210023988A (ko) 2021-03-04
PH12020552078A1 (en) 2021-05-31
TW202035692A (zh) 2020-10-01
JP2023171731A (ja) 2023-12-05

Similar Documents

Publication Publication Date Title
CL2020003367A1 (es) Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne
CL2019000107A1 (es) Composiciones de arni contra el componente c5 del complemento y métodos para su uso.(divisional solicitud 201702014)
CL2017002636A1 (es) Composiciones que comprenden una combinación de un anticuerpo anti muerte programada 1 (pd-1) y otro anticuerpo.
CL2017002230A1 (es) Tratamiento combinado con un agonista de tlr7 y un inhibidor del ensamblaje de la cápsida del vhb.
MX2020004467A (es) Metodos para el tratamiento o la prevencion del asma mediante la administracion de antagonista de il-4r.
CL2017001488A1 (es) Benzamidas sustituidas con 1,3–tiazol–2–ilo
CO2017001493A2 (es) Kits que comprenden inhibidores tigit y agentes anticáncer
CL2020002512A1 (es) Tratamiento de la hidradenitis supurativa mediante el uso de inhibidores de jak.
CU20150096A7 (es) Compuestos de amida y composición farmacéutica para el tratamiento de vih
AR101312A1 (es) Combinaciones de inhibidores de bet e inhibidores de tirosina quinasa de bruton
CO2019009215A2 (es) Sal hemi-sulfato de nucleótido para el tratamiento del virus de la hepatitis c
CL2016002276A1 (es) Composiciones tópicas que comprenden un extracto de coriolus versicolor para aumentar la autoinmunidad
CO2020004034A2 (es) Métodos para el tratamiento de distrofia muscular
UY35328A (es) Tratamiento de formas progresivas de esclerosis múltiple con laquinimod
CL2019001746A1 (es) Amidas aromáticas de ácido carboxílico
CO2020007601A2 (es) Cepa vacunal de brucella modificada para el tratamiento de brucelosis
CL2019003463A1 (es) Composición farmacéutica y forma de dosis farmacéutica que comprenden (e)-4-(2-(aminometil)-3-fluoroaliloxi)-n-ter-butilbenzamida, proceso para su preparación, métodos para tratamiento y usos de estas.
CO2017001076A2 (es) Composiciones farmacéuticas antihiperalgésicas, antialodínicas y antiinflamatorias que contienen pregabalina y meloxicam
BR112021006388A2 (pt) composto, composição farmacêutica, uso de um composto, e, método para tratamento de uma infecção viral em um mamífero
CL2022001079A1 (es) Métodos y composiciones para el tratamiento del síndrome de rett
CO2021004612A2 (es) Derivados de 4 pirazin 2 ilmetil-morfolina y su uso como medicamento
BR112017005241A2 (pt) composto, e, composição farmacêutica.
PE20142020A1 (es) Metodos para tratar el cancer usando inhibidor de pi3k e inhibidor de mek
CL2017001815A1 (es) Composición fungicida efectiva contra alternaria en cítricos
TR201819816T4 (tr) Tümörlerin tedavi edilmesine yönelik parvovirüs formülasyonu.