JP2022525848A - クラッベ病の治療に有用な組成物 - Google Patents

クラッベ病の治療に有用な組成物 Download PDF

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JP2022525848A
JP2022525848A JP2021549986A JP2021549986A JP2022525848A JP 2022525848 A JP2022525848 A JP 2022525848A JP 2021549986 A JP2021549986 A JP 2021549986A JP 2021549986 A JP2021549986 A JP 2021549986A JP 2022525848 A JP2022525848 A JP 2022525848A
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aav
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JP2022525848A5 (enExample
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ウイルソン，ジェームス・エム
オルドー，ジュリエット
カッツ，ネイサン
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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
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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
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    • 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
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2468—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on beta-galactose-glycoside bonds, e.g. carrageenases (3.2.1.83; 3.2.1.157); beta-agarase (3.2.1.81)
    • C12N9/2471—Beta-galactosidase (3.2.1.23), i.e. exo-(1-->4)-beta-D-galactanase
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01046—Galactosylceramidase (3.2.1.46)

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Biophysics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021549986A 2019-02-26 2020-02-26 クラッベ病の治療に有用な組成物 Pending JP2022525848A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024208739A JP2025032175A (ja) 2019-02-26 2024-11-29 クラッベ病の治療に有用な組成物

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962810708P 2019-02-26 2019-02-26
US62/810,708 2019-02-26
US201962817482P 2019-03-12 2019-03-12
US62/817,482 2019-03-12
US201962877707P 2019-07-23 2019-07-23
US62/877,707 2019-07-23
US201962916652P 2019-10-17 2019-10-17
US62/916,652 2019-10-17
PCT/US2020/019794 WO2020176562A1 (en) 2019-02-26 2020-02-26 Compositions useful in treatment of krabbe disease

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JP2024208739A Division JP2025032175A (ja) 2019-02-26 2024-11-29 クラッベ病の治療に有用な組成物

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JP2022525848A true JP2022525848A (ja) 2022-05-20
JP2022525848A5 JP2022525848A5 (enExample) 2023-02-22

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JP2021549986A Pending JP2022525848A (ja) 2019-02-26 2020-02-26 クラッベ病の治療に有用な組成物
JP2024208739A Pending JP2025032175A (ja) 2019-02-26 2024-11-29 クラッベ病の治療に有用な組成物

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US (1) US20220118108A1 (enExample)
EP (1) EP3931337A1 (enExample)
JP (2) JP2022525848A (enExample)
KR (1) KR20210132095A (enExample)
CN (1) CN114040980A (enExample)
AU (1) AU2020229772A1 (enExample)
BR (1) BR112021016566A2 (enExample)
CA (1) CA3130055A1 (enExample)
CL (1) CL2021002248A1 (enExample)
CO (1) CO2021011090A2 (enExample)
IL (1) IL285776A (enExample)
MX (1) MX2021010266A (enExample)
PE (1) PE20212072A1 (enExample)
SG (1) SG11202108480UA (enExample)
TW (1) TW202045728A (enExample)
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CA3177924A1 (en) * 2020-05-12 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
IL307633A (en) * 2021-04-12 2023-12-01 Univ Pennsylvania Useful preparations in the treatment of spinal muscular atrophy (SBMA)
WO2024215493A2 (en) * 2023-04-10 2024-10-17 M6P Therapeutics, Inc. Compositions comprising modified human galactocerebrosidase

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WO2018136710A1 (en) * 2017-01-20 2018-07-26 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Treatment of krabbe disease with umbilical cord blood transplantion (ucbt) and increased galactocerebrosidase (galc) expression
WO2018160582A1 (en) * 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor

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WO2018136710A1 (en) * 2017-01-20 2018-07-26 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Treatment of krabbe disease with umbilical cord blood transplantion (ucbt) and increased galactocerebrosidase (galc) expression
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AU2020229772A1 (en) 2021-08-26
CN114040980A (zh) 2022-02-11
JP2025032175A (ja) 2025-03-11
CL2021002248A1 (es) 2022-03-04
IL285776A (en) 2021-10-31
BR112021016566A2 (pt) 2021-11-03
CA3130055A1 (en) 2020-09-03
KR20210132095A (ko) 2021-11-03
MX2021010266A (es) 2021-09-23
US20220118108A1 (en) 2022-04-21
PE20212072A1 (es) 2021-10-26
TW202045728A (zh) 2020-12-16
SG11202108480UA (en) 2021-09-29
WO2020176562A1 (en) 2020-09-03
CO2021011090A2 (es) 2021-09-09
EP3931337A1 (en) 2022-01-05

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