TW202229557A - Enpp1或enpp3之肝臟特異性生產 - Google Patents
Enpp1或enpp3之肝臟特異性生產 Download PDFInfo
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
- C12Y301/04001—Phosphodiesterase I (3.1.4.1)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/01—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
- C12Y306/01009—Nucleotide diphosphatase (3.6.1.9), i.e. nucleotide-pyrophosphatase
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
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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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- 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
- C12N2750/14171—Demonstrated in vivo effect
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- 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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- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Investigating Or Analysing Biological Materials (AREA)
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Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
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| US202063089515P | 2020-10-08 | 2020-10-08 | |
| US63/089,515 | 2020-10-08 | ||
| US202163165650P | 2021-03-24 | 2021-03-24 | |
| US63/165,650 | 2021-03-24 | ||
| US202163248339P | 2021-09-24 | 2021-09-24 | |
| US63/248,339 | 2021-09-24 |
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| TW202229557A true TW202229557A (zh) | 2022-08-01 |
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| TW110137622A TW202229557A (zh) | 2020-10-08 | 2021-10-08 | Enpp1或enpp3之肝臟特異性生產 |
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| EP (1) | EP4225927A4 (https=) |
| JP (1) | JP2023548758A (https=) |
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| CA (1) | CA3195071A1 (https=) |
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| WO2023049864A1 (en) * | 2021-09-24 | 2023-03-30 | Inozyme Pharma, Inc. | Lyophilized enpp1 polypeptide formulations and uses thereof |
| CN119242712A (zh) * | 2024-09-27 | 2025-01-03 | 南京师范大学 | 特异性靶向骨骼肌细胞的携带miRNA-486基因的腺相关病毒载体、药物组合物 |
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| EP1736553A1 (en) * | 2005-06-17 | 2006-12-27 | Centre National De La Recherche Scientifique | ENPP1 (PC-1) gene haplotype associated with the risk of obesity and type 2 diabetes and their applications |
| CA2788682C (en) * | 2010-02-05 | 2019-03-05 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| WO2014126965A2 (en) * | 2013-02-13 | 2014-08-21 | Yale University | Compositions and methods for treating pathological calcification and ossification |
| KR20250078598A (ko) * | 2015-05-19 | 2025-06-02 | 예일 유니버시티 | 병리학적 석회화 병태를 치료하기 위한 조성물, 및 이의 사용 방법 |
| WO2017087936A1 (en) * | 2015-11-20 | 2017-05-26 | Yale University | Compositions for treating ectopic calcification disorders, and methods using same |
| KR20210142599A (ko) * | 2019-01-18 | 2021-11-25 | 이노자임 파마, 인코포레이티드 | Enpp1 또는 enpp3 결핍을 수반하는 질환의 치료 |
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| US20240148907A1 (en) | 2024-05-09 |
| AU2021356692A9 (en) | 2024-06-13 |
| EP4225927A1 (en) | 2023-08-16 |
| WO2022076848A1 (en) | 2022-04-14 |
| JP2023548758A (ja) | 2023-11-21 |
| AU2021356692A1 (en) | 2023-05-04 |
| CA3195071A1 (en) | 2022-04-14 |
| EP4225927A4 (en) | 2025-05-07 |
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