JP2021534154A - 脊髄性筋萎縮症のための併用療法 - Google Patents

脊髄性筋萎縮症のための併用療法 Download PDF

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JP2021534154A
JP2021534154A JP2021507686A JP2021507686A JP2021534154A JP 2021534154 A JP2021534154 A JP 2021534154A JP 2021507686 A JP2021507686 A JP 2021507686A JP 2021507686 A JP2021507686 A JP 2021507686A JP 2021534154 A JP2021534154 A JP 2021534154A
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smn2
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raav
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JP2021534154A5 (enExample
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アニンディア クマール セン，
アレックス マッキャンベル，
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Biogen MA Inc
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Biogen MA Inc
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Priority to JP2024096579A priority Critical patent/JP2024111074A/ja
Priority to JP2025187453A priority patent/JP2026009384A/ja
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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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
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    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2320/31—Combination 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021507686A 2018-08-15 2019-08-15 脊髄性筋萎縮症のための併用療法 Withdrawn JP2021534154A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024096579A JP2024111074A (ja) 2018-08-15 2024-06-14 脊髄性筋萎縮症のための併用療法
JP2025187453A JP2026009384A (ja) 2018-08-15 2025-11-06 脊髄性筋萎縮症のための併用療法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862764893P 2018-08-15 2018-08-15
US62/764,893 2018-08-15
US201862783189P 2018-12-20 2018-12-20
US62/783,189 2018-12-20
PCT/US2019/046720 WO2020037161A1 (en) 2018-08-15 2019-08-15 Combination therapy for spinal muscular atrophy

Related Child Applications (1)

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JP2024096579A Division JP2024111074A (ja) 2018-08-15 2024-06-14 脊髄性筋萎縮症のための併用療法

Publications (2)

Publication Number Publication Date
JP2021534154A true JP2021534154A (ja) 2021-12-09
JP2021534154A5 JP2021534154A5 (enExample) 2022-08-22

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JP2021507686A Withdrawn JP2021534154A (ja) 2018-08-15 2019-08-15 脊髄性筋萎縮症のための併用療法
JP2024096579A Pending JP2024111074A (ja) 2018-08-15 2024-06-14 脊髄性筋萎縮症のための併用療法
JP2025187453A Pending JP2026009384A (ja) 2018-08-15 2025-11-06 脊髄性筋萎縮症のための併用療法

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JP2024096579A Pending JP2024111074A (ja) 2018-08-15 2024-06-14 脊髄性筋萎縮症のための併用療法
JP2025187453A Pending JP2026009384A (ja) 2018-08-15 2025-11-06 脊髄性筋萎縮症のための併用療法

Country Status (4)

Country Link
US (1) US20210308281A1 (enExample)
EP (1) EP3837374A4 (enExample)
JP (3) JP2021534154A (enExample)
WO (1) WO2020037161A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3999643T (pt) * 2019-07-19 2023-12-11 Biogen Ma Inc Métodos de tratamento da atrofia muscular espinhal
EP4013387A4 (en) * 2019-08-15 2023-09-27 Biogen MA Inc. COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY
WO2022022617A1 (en) * 2020-07-31 2022-02-03 Ractigen Therapeutics Combinatory treatment of sma with sarna and mrna modulators
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating spinal muscular atrophy

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012530715A (ja) * 2009-06-17 2012-12-06 アイシス ファーマシューティカルズ， インコーポレーテッド 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2018007234A (es) * 2015-12-14 2018-11-09 Univ Pennsylvania Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.
WO2019094253A1 (en) * 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
EP3801638A1 (en) * 2018-06-08 2021-04-14 Novartis AG Cell-based assay for measuring drug product potency
CN120860258A (zh) * 2018-11-30 2025-10-31 北京诺华制药有限公司 Aav病毒载体及其用途

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012530715A (ja) * 2009-06-17 2012-12-06 アイシス ファーマシューティカルズ， インコーポレーテッド 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
PASSINI M. A. ET AL., HUMAN GENE THERAPY, vol. 25, JPN6023025152, 2014, pages 619 - 630, ISSN: 0005363845 *
PASSINI M. A. ET AL., J CLIN INVEST., vol. 120(4), JPN6023025153, 2010, pages 1253 - 1264, ISSN: 0005363844 *
WOOD M. ET AL., HUMAN MOLECULAR GENETICS, vol. 26, no. 2, JPN6023025151, 2017, pages 151 - 159, ISSN: 0005363846 *

Also Published As

Publication number Publication date
EP3837374A4 (en) 2022-06-08
JP2024111074A (ja) 2024-08-16
US20210308281A1 (en) 2021-10-07
EP3837374A1 (en) 2021-06-23
JP2026009384A (ja) 2026-01-19
WO2020037161A1 (en) 2020-02-20

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