JP2024543918A - Ａａｄｃ、ｇｄｎｆポリヌクレオチド及びそのパーキンソン病を治療するための使用 - Google Patents

Ａａｄｃ、ｇｄｎｆポリヌクレオチド及びそのパーキンソン病を治療するための使用 Download PDF

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JP2024543918A
JP2024543918A JP2024531366A JP2024531366A JP2024543918A JP 2024543918 A JP2024543918 A JP 2024543918A JP 2024531366 A JP2024531366 A JP 2024531366A JP 2024531366 A JP2024531366 A JP 2024531366A JP 2024543918 A JP2024543918 A JP 2024543918A
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polynucleotide
polya
sequence
seq
aav
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JP2024543918A5 (https=
JPWO2023093905A5 (https=
Inventor
▲軍▼ 姜
明月 ▲呉▼
威 ▲寧▼
成 廖
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Shanghai Regenelead Therapies Co Ltd
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Shanghai Regenelead Therapies Co Ltd
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    • C12N9/88—Lyases (4.)
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • 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
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    • 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
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    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01015—Glutamate decarboxylase (4.1.1.15)
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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
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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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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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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    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01028—Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase

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  • General Chemical & Material Sciences (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024531366A 2021-11-29 2022-11-29 Ａａｄｃ、ｇｄｎｆポリヌクレオチド及びそのパーキンソン病を治療するための使用 Pending JP2024543918A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN202111434384.4 2021-11-29
CN202111434384 2021-11-29
PCT/CN2022/134993 WO2023093905A1 (zh) 2021-11-29 2022-11-29 Aadc、gdnf多核苷酸及其用于治疗帕金森病

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JP2024543918A true JP2024543918A (ja) 2024-11-26
JP2024543918A5 JP2024543918A5 (https=) 2025-12-10
JPWO2023093905A5 JPWO2023093905A5 (https=) 2025-12-10

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US (1) US20250051750A1 (https=)
EP (1) EP4442830A4 (https=)
JP (1) JP2024543918A (https=)
KR (1) KR20240110849A (https=)
CN (1) CN118525098A (https=)
AU (1) AU2022397707A1 (https=)
CA (1) CA3238867A1 (https=)
TW (1) TW202325850A (https=)
WO (1) WO2023093905A1 (https=)

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WO2023093905A1 (zh) 2023-06-01
AU2022397707A1 (en) 2024-06-13
US20250051750A1 (en) 2025-02-13
EP4442830A1 (en) 2024-10-09
TW202325850A (zh) 2023-07-01
CA3238867A1 (en) 2023-06-01
KR20240110849A (ko) 2024-07-16
CN118525098A (zh) 2024-08-20

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