TW202039857A - 黏多醣病iva之治療 - Google Patents
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06004—N-Acetylgalactosamine-6-sulfatase (3.1.6.4)
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Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
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| US201862711238P | 2018-07-27 | 2018-07-27 | |
| US62/711,238 | 2018-07-27 | ||
| US201862756880P | 2018-11-07 | 2018-11-07 | |
| US62/756,880 | 2018-11-07 | ||
| US201962799834P | 2019-02-01 | 2019-02-01 | |
| US62/799,834 | 2019-02-01 |
Publications (1)
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| TW202039857A true TW202039857A (zh) | 2020-11-01 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| TW108126686A TW202039857A (zh) | 2018-07-27 | 2019-07-26 | 黏多醣病iva之治療 |
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|---|---|
| US (1) | US12247213B2 (https=) |
| EP (1) | EP3829619A4 (https=) |
| JP (1) | JP2021531044A (https=) |
| KR (1) | KR20210040984A (https=) |
| AU (1) | AU2019312301A1 (https=) |
| BR (1) | BR112021001498A2 (https=) |
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| CO (1) | CO2021002505A2 (https=) |
| IL (1) | IL280343A (https=) |
| SG (1) | SG11202100781VA (https=) |
| TW (1) | TW202039857A (https=) |
| WO (1) | WO2020023857A1 (https=) |
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| TW202039857A (zh) | 2018-07-27 | 2020-11-01 | 美商銳進科斯生物股份有限公司 | 黏多醣病iva之治療 |
| US12290574B2 (en) | 2018-08-24 | 2025-05-06 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| JP2022544004A (ja) * | 2019-07-26 | 2022-10-17 | リジェネックスバイオ インコーポレイテッド | 操作された核酸調節エレメントならびにその使用方法 |
| AR121201A1 (es) * | 2020-01-29 | 2022-04-27 | Regenxbio Inc | Tratamiento de mucopolisacaridosis iva |
| EP4423285A1 (en) * | 2021-10-28 | 2024-09-04 | RegenxBio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
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| US7863238B2 (en) * | 2004-06-10 | 2011-01-04 | Saint Louis University | Proteins with an attached short peptide of acidic amino acids |
| US7972593B2 (en) | 2004-06-10 | 2011-07-05 | Saint Louis University | Delivery of therapeutic agents to the bone |
| US20070207139A1 (en) | 2004-06-10 | 2007-09-06 | Saint Louis University, A Non-Profit Organization | Enhancing the effect of therapeutic proteins on the central nervous system |
| ES2429142T3 (es) | 2009-01-30 | 2013-11-13 | Uniqure Ip B.V. | Agentes terapéuticos de alanina-glioxilato aminotransferasa |
| US20140271550A1 (en) | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| CN120174012A (zh) * | 2013-10-24 | 2025-06-20 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| EP3101125A1 (en) * | 2015-06-05 | 2016-12-07 | Laboratorios Del Dr. Esteve, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
| HK1257024A1 (zh) | 2015-08-31 | 2019-10-11 | 宾夕法尼亚大学 | 用於治疗伴侣动物的aav-epo |
| UA128788C2 (uk) | 2018-05-30 | 2024-10-23 | Естеве Фармасьютікалз, С.А. | Вектор аденоасоційованого вірусу для лікування мукополісахаридозу типу iva |
| TW202039857A (zh) | 2018-07-27 | 2020-11-01 | 美商銳進科斯生物股份有限公司 | 黏多醣病iva之治療 |
| AR121201A1 (es) | 2020-01-29 | 2022-04-27 | Regenxbio Inc | Tratamiento de mucopolisacaridosis iva |
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2019
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2021
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| EP3829619A1 (en) | 2021-06-09 |
| CA3107800A1 (en) | 2020-01-30 |
| AU2019312301A1 (en) | 2021-02-18 |
| SG11202100781VA (en) | 2021-02-25 |
| US12247213B2 (en) | 2025-03-11 |
| US20210292789A1 (en) | 2021-09-23 |
| CO2021002505A2 (es) | 2021-03-08 |
| IL280343A (en) | 2021-03-25 |
| JP2021531044A (ja) | 2021-11-18 |
| KR20210040984A (ko) | 2021-04-14 |
| EP3829619A4 (en) | 2022-05-18 |
| BR112021001498A2 (pt) | 2021-04-27 |
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