BR112020022512A2 - métodos e composições para tratamento de doença de armazenamento de éster de colesteril - Google Patents
métodos e composições para tratamento de doença de armazenamento de éster de colesteril Download PDFInfo
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- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- C12N2320/33—Alteration of splicing
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- C12Q2600/00—Oligonucleotides characterized by their use
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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Applications Claiming Priority (3)
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| US201862667205P | 2018-05-04 | 2018-05-04 | |
| US62/667,205 | 2018-05-04 | ||
| PCT/US2019/030605 WO2019213525A1 (en) | 2018-05-04 | 2019-05-03 | Methods and compositions for treatment of cholesteryl ester storage disease |
Publications (1)
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| BR112020022512A2 true BR112020022512A2 (pt) | 2021-05-04 |
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| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| WO2020117715A1 (en) | 2018-12-03 | 2020-06-11 | Board Of Regents, The University Of Texas System | Oligo-benzamide analogs and their use in cancer treatment |
| WO2021117122A1 (ja) * | 2019-12-10 | 2021-06-17 | 株式会社リボルナバイオサイエンス | ライソゾーム酸性リパーゼ欠損症の予防または治療剤 |
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2019
- 2019-05-03 CA CA3099280A patent/CA3099280A1/en active Pending
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2024
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| CA3099280A1 (en) | 2019-11-07 |
| US12060558B2 (en) | 2024-08-13 |
| WO2019213525A1 (en) | 2019-11-07 |
| JP2021523227A (ja) | 2021-09-02 |
| MX2020011695A (es) | 2021-02-26 |
| EP3788169A1 (en) | 2021-03-10 |
| JP2024079818A (ja) | 2024-06-11 |
| US20210371866A1 (en) | 2021-12-02 |
| EP3788169A4 (en) | 2022-08-10 |
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