JP2024515612A - 球脊髄性筋萎縮症（ｓｂｍａ）の治療に有用な組成物 - Google Patents

球脊髄性筋萎縮症（ｓｂｍａ）の治療に有用な組成物 Download PDF

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JP2024515612A
JP2024515612A JP2023562714A JP2023562714A JP2024515612A JP 2024515612 A JP2024515612 A JP 2024515612A JP 2023562714 A JP2023562714 A JP 2023562714A JP 2023562714 A JP2023562714 A JP 2023562714A JP 2024515612 A JP2024515612 A JP 2024515612A
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aav
sequence
mirna
mice
seq
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JPWO2022221276A5 (https=
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ウイルソン，ジェームス・エム
ヒンデラー，クリスチャン
ワークマン，アイリーン
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0083—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 administration regime
    • 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
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • 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]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2023562714A 2021-04-12 2022-04-12 球脊髄性筋萎縮症（ｓｂｍａ）の治療に有用な組成物 Pending JP2024515612A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163173885P 2021-04-12 2021-04-12
US63/173,885 2021-04-12
US202163187883P 2021-05-12 2021-05-12
US63/187,883 2021-05-12
US202163293505P 2021-12-23 2021-12-23
US63/293,505 2021-12-23
PCT/US2022/024415 WO2022221276A1 (en) 2021-04-12 2022-04-12 Compositions useful for treating spinal and bulbar muscular atrophy (sbma)

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JP2024515612A true JP2024515612A (ja) 2024-04-10
JPWO2022221276A5 JPWO2022221276A5 (https=) 2025-04-21
JP2024515612A5 JP2024515612A5 (https=) 2025-04-21

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US (1) US20240191258A1 (https=)
EP (1) EP4323520A1 (https=)
JP (1) JP2024515612A (https=)
KR (1) KR20230170022A (https=)
AU (1) AU2022258312A1 (https=)
BR (1) BR112023021245A2 (https=)
CA (1) CA3215141A1 (https=)
IL (1) IL307633A (https=)
MX (1) MX2023012052A (https=)
WO (1) WO2022221276A1 (https=)

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WO2020176562A1 (en) * 2019-02-26 2020-09-03 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
WO2020227166A1 (en) * 2019-05-03 2020-11-12 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
WO2021044175A1 (en) * 2019-09-06 2021-03-11 Babraham Institute Novel method

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WO2020227166A1 (en) * 2019-05-03 2020-11-12 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
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KR20230170022A (ko) 2023-12-18
CA3215141A1 (en) 2022-10-20
IL307633A (en) 2023-12-01
AU2022258312A1 (en) 2023-10-26
EP4323520A1 (en) 2024-02-21
MX2023012052A (es) 2024-03-15
US20240191258A1 (en) 2024-06-13
WO2022221276A1 (en) 2022-10-20
AU2022258312A9 (en) 2023-11-09
BR112023021245A2 (pt) 2023-12-19

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