JPWO2021172498A1 - - Google Patents

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Publication number
JPWO2021172498A1
JPWO2021172498A1 JP2022503739A JP2022503739A JPWO2021172498A1 JP WO2021172498 A1 JPWO2021172498 A1 JP WO2021172498A1 JP 2022503739 A JP2022503739 A JP 2022503739A JP 2022503739 A JP2022503739 A JP 2022503739A JP WO2021172498 A1 JPWO2021172498 A1 JP WO2021172498A1
Authority
JP
Japan
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2022503739A
Other languages
Japanese (ja)
Other versions
JPWO2021172498A5 (https=
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 filed Critical
Publication of JPWO2021172498A1 publication Critical patent/JPWO2021172498A1/ja
Priority to JP2022141656A priority Critical patent/JP7292636B2/ja
Publication of JPWO2021172498A5 publication Critical patent/JPWO2021172498A5/ja
Priority to JP2025157620A priority patent/JP2026001070A/ja
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
    • C07K14/4708Duchenne dystrophy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration 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)
JP2022503739A 2020-02-28 2021-02-26 Pending JPWO2021172498A1 (https=)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022141656A JP7292636B2 (ja) 2020-02-28 2022-09-06 エクソン51のスキッピングを誘導するアンチセンス核酸
JP2025157620A JP2026001070A (ja) 2020-02-28 2025-09-24 エクソン51のスキッピングを誘導するアンチセンス核酸

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2020033483 2020-02-28
PCT/JP2021/007286 WO2021172498A1 (ja) 2020-02-28 2021-02-26 エクソン51のスキッピングを誘導するアンチセンス核酸

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2022141656A Division JP7292636B2 (ja) 2020-02-28 2022-09-06 エクソン51のスキッピングを誘導するアンチセンス核酸
JP2025157620A Division JP2026001070A (ja) 2020-02-28 2025-09-24 エクソン51のスキッピングを誘導するアンチセンス核酸

Publications (2)

Publication Number Publication Date
JPWO2021172498A1 true JPWO2021172498A1 (https=) 2021-09-02
JPWO2021172498A5 JPWO2021172498A5 (https=) 2024-03-04

Family

ID=77491251

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2022503739A Pending JPWO2021172498A1 (https=) 2020-02-28 2021-02-26
JP2022141656A Active JP7292636B2 (ja) 2020-02-28 2022-09-06 エクソン51のスキッピングを誘導するアンチセンス核酸
JP2025157620A Pending JP2026001070A (ja) 2020-02-28 2025-09-24 エクソン51のスキッピングを誘導するアンチセンス核酸

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022141656A Active JP7292636B2 (ja) 2020-02-28 2022-09-06 エクソン51のスキッピングを誘導するアンチセンス核酸
JP2025157620A Pending JP2026001070A (ja) 2020-02-28 2025-09-24 エクソン51のスキッピングを誘導するアンチセンス核酸

Country Status (17)

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

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
TW202200162A (zh) 2022-01-01
CL2024001471A1 (es) 2024-09-27
JP7292636B2 (ja) 2023-06-19
ECSP22074446A (es) 2022-10-31
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

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