TWI874898B - 用於高歇氏(gaucher)病之基因療法 - Google Patents

用於高歇氏(gaucher)病之基因療法 Download PDF

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TWI874898B
TWI874898B TW112102851A TW112102851A TWI874898B TW I874898 B TWI874898 B TW I874898B TW 112102851 A TW112102851 A TW 112102851A TW 112102851 A TW112102851 A TW 112102851A TW I874898 B TWI874898 B TW I874898B
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Taiwan
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aav
sequence
gene
gcase
genome
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TW112102851A
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TW202338093A (zh
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羅伯特 Ｄ 貝爾
鄭婷文
賈汀 那魯拉
克拉克 坤 潘
蘇雅納拉洋 索瑪納桑
大衛 韋恩 蘇薩
里卡多斯 塔貝特
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美商輝瑞大藥廠
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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
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    • 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
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    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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    • C12N2750/14011—Parvoviridae
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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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  • 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)
TW112102851A 2022-01-21 2023-01-19 用於高歇氏(gaucher)病之基因療法 TWI874898B (zh)

Applications Claiming Priority (4)

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

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TW202338093A TW202338093A (zh) 2023-10-01
TWI874898B true TWI874898B (zh) 2025-03-01

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TW112102851A TWI874898B (zh) 2022-01-21 2023-01-19 用於高歇氏(gaucher)病之基因療法

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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=)

Families Citing this family (1)

* 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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021195218A1 (en) * 2020-03-24 2021-09-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
US20210332385A1 (en) * 2017-10-03 2021-10-28 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

Family Cites Families (6)

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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
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210332385A1 (en) * 2017-10-03 2021-10-28 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
WO2021195218A1 (en) * 2020-03-24 2021-09-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
期刊 Miranda CJ., et al., "Liver-Directed AAV Gene Therapy for Gaucher Disease", Blood, Vol. 134, Supplement 1, 13 November 2019, Page 3354. *

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KR20240134991A (ko) 2024-09-10
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
JP2025504478A (ja) 2025-02-12

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