ECSP22074446A - Ácidos nucleicos antisentido que inducen la omisión del exón 51 - Google Patents

Ácidos nucleicos antisentido que inducen la omisión del exón 51

Info

Publication number
ECSP22074446A
ECSP22074446A ECSENADI202274446A ECDI202274446A ECSP22074446A EC SP22074446 A ECSP22074446 A EC SP22074446A EC SENADI202274446 A ECSENADI202274446 A EC SENADI202274446A EC DI202274446 A ECDI202274446 A EC DI202274446A EC SP22074446 A ECSP22074446 A EC SP22074446A
Authority
EC
Ecuador
Prior art keywords
exon
nucleic acids
antisense nucleic
induce skipping
acids induce
Prior art date
Application number
ECSENADI202274446A
Other languages
English (en)
Inventor
Takahiro Fukui
Shin'ichi Takeda
Saki Hasegawa
Yoshitsugu Aoki
Yu Honda
Kaname MUCHIMA
Original Assignee
Nippon Shinyaku Co Ltd
Nat Center Neurology & Psychiatry
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, Nat Center Neurology & Psychiatry filed Critical Nippon Shinyaku Co Ltd
Publication of ECSP22074446A publication Critical patent/ECSP22074446A/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente memoria descriptiva proporciona un fármaco que provoca una omisión altamente eficaz del exón 51 en el gen de la distrofina humana. La presente memoria descriptiva proporciona un oligómero antisentido que tiene una actividad para inducir la omisión del exón 51 en el gen de la distrofina humana.
ECSENADI202274446A 2020-02-28 2022-09-23 Ácidos nucleicos antisentido que inducen la omisión del exón 51 ECSP22074446A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
JP2020033483 2020-02-28

Publications (1)

Publication Number Publication Date
ECSP22074446A true ECSP22074446A (es) 2022-10-31

Family

ID=77491251

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSENADI202274446A ECSP22074446A (es) 2020-02-28 2022-09-23 Ácidos nucleicos antisentido que inducen la omisión del exón 51

Country Status (17)

Country Link
US (2) US20230140736A1 (es)
EP (1) EP4112083A4 (es)
JP (3) JPWO2021172498A1 (es)
KR (1) KR20220145865A (es)
CN (1) CN115210376A (es)
AU (1) AU2021226089A1 (es)
BR (1) BR112022017066A2 (es)
CA (1) CA3173049A1 (es)
CL (3) CL2022002279A1 (es)
CO (1) CO2022013685A2 (es)
EC (1) ECSP22074446A (es)
IL (1) IL295967A (es)
MX (1) MX2022010545A (es)
PE (1) PE20230237A1 (es)
TW (2) TWI901457B (es)
WO (1) WO2021172498A1 (es)
ZA (1) ZA202209294B (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL293997A (en) * 2019-12-19 2022-08-01 Nippon Shinyaku Co Ltd Antistrand nucleic acids that allow exon skipping
EP4458833A4 (en) 2021-12-27 2025-12-31 Nippon Shinyaku Co Ltd PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
AU655164B2 (en) 1989-12-20 1994-12-08 Antivirals Inc. Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
JP2924179B2 (ja) 1993-02-19 1999-07-26 日本新薬株式会社 グリセロール誘導体，デバイス及び医薬組成物
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
EP2392660A3 (en) 2002-11-25 2012-03-28 Masafumi Matsuo ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
PL2735568T3 (pl) 2006-05-10 2018-02-28 Sarepta Therapeutics, Inc. Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami
WO2009005793A2 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
RU2606627C2 (ru) 2007-11-15 2017-01-10 Серепта Терапьютикс,Инк. Способ синтеза морфолиновых олигомеров
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
AU2009310557B2 (en) 2008-10-27 2014-09-11 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CA3092114A1 (en) 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CA2861247C (en) * 2011-12-28 2021-11-16 Nippon Shinyaku Co., Ltd. Antisense nucleic acids
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
EP2870246B1 (en) * 2012-07-03 2019-09-11 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
JP6519842B2 (ja) 2013-10-04 2019-05-29 国立大学法人神戸大学 福山型筋ジストロフィー治療用アンチセンス核酸
DK3118311T3 (en) 2014-03-12 2019-03-11 Nippon Shinyaku Co Ltd Antisense nucleic acid
MX373052B (es) * 2014-06-17 2020-05-11 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido.
AU2016264471B2 (en) 2015-05-19 2022-01-27 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
AU2016302009B2 (en) 2015-08-05 2021-09-30 Eisai R&D Management Co., Ltd. Chiral reagents for preparation of homogeneous oligomers
HUE050061T2 (hu) * 2015-09-15 2020-12-28 Nippon Shinyaku Co Ltd Antiszensz nukleinsav
IL295755A (en) 2015-10-09 2022-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
KR102810425B1 (ko) * 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR20240006023A (ko) 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
PT3554552T (pt) 2016-12-19 2022-10-03 Sarepta Therapeutics Inc Conjugados de oligómero de skipping de exão para distrofia muscular
WO2019241385A2 (en) 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystropy

Also Published As

Publication number Publication date
ZA202209294B (en) 2024-04-24
JP2026001070A (ja) 2026-01-06
PE20230237A1 (es) 2023-02-07
TWI868323B (zh) 2025-01-01
CA3173049A1 (en) 2021-09-02
US20230140736A1 (en) 2023-05-04
CO2022013685A2 (es) 2022-10-11
WO2021172498A1 (ja) 2021-09-02
KR20220145865A (ko) 2022-10-31
BR112022017066A2 (pt) 2022-11-16
AU2021226089A1 (en) 2022-09-15
JP2022180420A (ja) 2022-12-06
EP4112083A4 (en) 2024-06-26
MX2022010545A (es) 2022-09-21
US20230097387A1 (en) 2023-03-30
CN115210376A (zh) 2022-10-18
JPWO2021172498A1 (es) 2021-09-02
TW202200162A (zh) 2022-01-01
CL2024001471A1 (es) 2024-09-27
JP7292636B2 (ja) 2023-06-19
IL295967A (en) 2022-10-01
CL2022002279A1 (es) 2023-02-10
TWI901457B (zh) 2025-10-11
EP4112083A1 (en) 2023-01-04
TW202515585A (zh) 2025-04-16
CL2024001472A1 (es) 2024-09-27
US11781140B2 (en) 2023-10-10

Similar Documents

Publication Publication Date Title
ECSP22074446A (es) Ácidos nucleicos antisentido que inducen la omisión del exón 51
CO2022008664A2 (es) Ácido nucleico antisentido que induce la omisión del exón 50
CY1122167T1 (el) Αντινοηματικα νουκλεϊνικα οξεα
CO2017000357A2 (es) Ácidos nucleicos antisentido
CL2020000889A1 (es) Oligonucleótidos para inducir la expresión paterna de ube3a (divisional solicitud 201801189)
CY1123119T1 (el) Αντινοηματικο νουκλεϊκο οξυ
ECSP17055491A (es) Ácidos 3-alquil-4-amido-bicíclico [4,5,0] hidroxámico como inhibidores de hdac
CO2017007335A2 (es) Supresión del gen de la huntingtina inducida por la arni
CL2016003041A1 (es) Ácidos ribonucleicos bicatenarios aislados, composiciones farmacéuticas que los comprenden y método para inhibir la expresión del gen alas1 basado en la administración de dicha composición. divisional de solicitud 2725-2014.
CL2021002585A1 (es) Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central
BR112013000391A2 (pt) emulsões de óleo em água catiônicas
PH12016501761B1 (en) Antisense nucleic acids
MX2022001767A (es) Construcciones de vesículas-oligonucleótidos antisentido (aso) extracelulares que se dirigen a transductor de señal y activador de la transcripción 6 (stat6).
CL2022002779A1 (es) Compuestos y métodos para modular el proceso de corte y empalme
IN2014DN06220A (es)
ECSP16072034A (es) Inhibidores de diacilglicerol aciltransferasa 2 para alteraciones metabólicas y desórdenes relacionados
MX2018002998A (es) Derivado de acido nucleico que tiene actividad inmunoestimuladora.
AR100946A1 (es) Composiciones y métodos para regular la expresión génica por medio de la interferencia de arn
CL2020000676A1 (es) Composiciones y métodos para modular el crecimiento del cabello.
BR112023000428A2 (pt) Métodos e composições para tratar epilepsia
MX2021013418A (es) Moleculas inhibidoras de acido nucleico bicatenario con cadenas de sentido acortadas.
CL2019000920A1 (es) Métodos de preservar la actividad biológica de los ácidos ribonucleicos.
CO2020016201A2 (es) Metodos y formulaciones para el tratamiento de obesidad y enfermedades metabolicas relacionadas con la obesidad
CL2021000618A1 (es) Compuesto que contiene oxadiazol y composición farmacéutica que contiene el mismo
MX2022003499A (es) Composicion de lubricante interno y uso.