KR20230118075A - 파브리병 치료를 위한 조성물 및 방법 - Google Patents
파브리병 치료를 위한 조성물 및 방법 Download PDFInfo
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- KR20230118075A KR20230118075A KR1020237015206A KR20237015206A KR20230118075A KR 20230118075 A KR20230118075 A KR 20230118075A KR 1020237015206 A KR1020237015206 A KR 1020237015206A KR 20237015206 A KR20237015206 A KR 20237015206A KR 20230118075 A KR20230118075 A KR 20230118075A
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Classifications
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- 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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- 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
- C12N2830/48—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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- 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)
- C12Y302/01022—Alpha-galactosidase (3.2.1.22)
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- Proteomics, Peptides & Aminoacids (AREA)
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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| PCT/US2021/054145 WO2022076803A1 (en) | 2020-10-09 | 2021-10-08 | Compositions and methods for treatment of fabry disease |
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| EP4634394A2 (en) | 2022-12-17 | 2025-10-22 | The Trustees of The University of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
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| CN113423419A (zh) * | 2019-01-04 | 2021-09-21 | 桑格摩生物治疗股份有限公司 | 用于治疗法布里病的方法和组合物 |
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- 2021-10-08 AU AU2021356684A patent/AU2021356684A1/en not_active Abandoned
- 2021-10-08 JP JP2023521706A patent/JP2023545433A/ja active Pending
- 2021-10-08 TW TW110137528A patent/TW202229560A/zh unknown
- 2021-10-08 CA CA3193833A patent/CA3193833A1/en active Pending
- 2021-10-08 EP EP21805710.7A patent/EP4225906A1/en active Pending
- 2021-10-08 US US18/246,872 patent/US20230365955A1/en active Pending
- 2021-10-08 WO PCT/US2021/054145 patent/WO2022076803A1/en not_active Ceased
- 2021-10-08 KR KR1020237015206A patent/KR20230118075A/ko not_active Withdrawn
Also Published As
| Publication number | Publication date |
|---|---|
| EP4225906A1 (en) | 2023-08-16 |
| AU2021356684A1 (en) | 2023-05-11 |
| US20230365955A1 (en) | 2023-11-16 |
| TW202229560A (zh) | 2022-08-01 |
| WO2022076803A1 (en) | 2022-04-14 |
| WO2022076803A8 (en) | 2022-06-09 |
| CA3193833A1 (en) | 2022-04-14 |
| JP2023545433A (ja) | 2023-10-30 |
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