JP2025504478A - ゴーシェ病の遺伝子療法 - Google Patents

ゴーシェ病の遺伝子療法 Download PDF

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JP2025504478A
JP2025504478A JP2024543149A JP2024543149A JP2025504478A JP 2025504478 A JP2025504478 A JP 2025504478A JP 2024543149 A JP2024543149 A JP 2024543149A JP 2024543149 A JP2024543149 A JP 2024543149A JP 2025504478 A JP2025504478 A JP 2025504478A
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aav
sequence
gcase
gene
aav vector
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JP2025504478A5 (https=
Inventor
ロバート ディー． ベル，
ティン－ウェン チェン，
ジャティン ナルラ，
クラーク チュン パン，
スリヤナラヤン ソマナサン，
デイビッド ウェイン ソウザ，
リカルドス タベット，
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AstraZeneca Ireland Ltd
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AstraZeneca Ireland Ltd
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Publication of JP2025504478A publication Critical patent/JP2025504478A/ja
Publication of JP2025504478A5 publication Critical patent/JP2025504478A5/ja
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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    • 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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    • C12N2830/50—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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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2024543149A 2022-01-21 2023-01-18 ゴーシェ病の遺伝子療法 Pending JP2025504478A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263267046P 2022-01-21 2022-01-21
US63/267,046 2022-01-21
US202263382502P 2022-11-05 2022-11-05
US63/382,502 2022-11-05
PCT/IB2023/050426 WO2023139496A1 (en) 2022-01-21 2023-01-18 Gene therapy for gaucher disease

Publications (2)

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JP2025504478A true JP2025504478A (ja) 2025-02-12
JP2025504478A5 JP2025504478A5 (https=) 2026-01-26

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JP2024543149A Pending JP2025504478A (ja) 2022-01-21 2023-01-18 ゴーシェ病の遺伝子療法

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US (1) US20250257367A1 (https=)
EP (1) EP4465998A1 (https=)
JP (1) JP2025504478A (https=)
KR (1) KR20240134991A (https=)
AU (1) AU2023208274A1 (https=)
CA (1) CA3248631A1 (https=)
CO (1) CO2024011137A2 (https=)
IL (1) IL314294A (https=)
MX (1) MX2024009003A (https=)
TW (1) TWI874898B (https=)
WO (1) WO2023139496A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250152747A1 (en) * 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating gaucher disease and gba-parkinson’s disease

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
AU2018346104B2 (en) * 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
TW202005978A (zh) * 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
WO2020012149A1 (en) * 2018-07-13 2020-01-16 Ucl Business Ltd Glucocerebrosidase gene therapy
JP7664848B2 (ja) * 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
KR20220098384A (ko) * 2019-11-19 2022-07-12 아스클레피오스 바이오파마슈티컬, 인크. 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스
JP2023520763A (ja) * 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー ゴーシェ治療薬を発現するための非ウイルス性ｄｎａベクター及びその使用

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KR20240134991A (ko) 2024-09-10
TWI874898B (zh) 2025-03-01
CA3248631A1 (en) 2023-07-27
TW202338093A (zh) 2023-10-01
IL314294A (en) 2024-09-01
MX2024009003A (es) 2024-09-17
AU2023208274A1 (en) 2024-08-01
WO2023139496A1 (en) 2023-07-27
US20250257367A1 (en) 2025-08-14
EP4465998A1 (en) 2024-11-27
CO2024011137A2 (es) 2024-08-30

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