JP2021531044A5 - - Google Patents
Info
- Publication number
- JP2021531044A5 JP2021531044A5 JP2021527020A JP2021527020A JP2021531044A5 JP 2021531044 A5 JP2021531044 A5 JP 2021531044A5 JP 2021527020 A JP2021527020 A JP 2021527020A JP 2021527020 A JP2021527020 A JP 2021527020A JP 2021531044 A5 JP2021531044 A5 JP 2021531044A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide sequence
- sequence number
- hgalns
- raav
- sequence
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 | ||
| PCT/US2019/043631 WO2020023857A1 (en) | 2018-07-27 | 2019-07-26 | Treatment of mucopolysaccharidosis iva |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021531044A JP2021531044A (ja) | 2021-11-18 |
| JP2021531044A5 true JP2021531044A5 (enExample) | 2022-08-02 |
Family
ID=69180546
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021527020A Pending JP2021531044A (ja) | 2018-07-27 | 2019-07-26 | ムコ多糖症ｉｖａ型の治療 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US12247213B2 (enExample) |
| EP (1) | EP3829619A4 (enExample) |
| JP (1) | JP2021531044A (enExample) |
| KR (1) | KR20210040984A (enExample) |
| AU (1) | AU2019312301A1 (enExample) |
| BR (1) | BR112021001498A2 (enExample) |
| CA (1) | CA3107800A1 (enExample) |
| CO (1) | CO2021002505A2 (enExample) |
| IL (1) | IL280343A (enExample) |
| SG (1) | SG11202100781VA (enExample) |
| TW (1) | TW202039857A (enExample) |
| WO (1) | WO2020023857A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112021001498A2 (pt) | 2018-07-27 | 2021-04-27 | Regenxbio Inc. | vírus adeno-associado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav e métodos para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
| CN113226352B (zh) | 2018-08-24 | 2025-08-26 | 星火治疗有限公司 | 最优化启动子序列、无内含子的表达结构及使用方法 |
| EP4004214A1 (en) * | 2019-07-26 | 2022-06-01 | RegenxBio Inc. | Engineered nucleic acid regulatory element and methods of uses thereof |
| BR112022015036A2 (pt) * | 2020-01-29 | 2022-10-11 | Regenxbio Inc | Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
| EP4423285A1 (en) * | 2021-10-28 | 2024-09-04 | RegenxBio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US7863238B2 (en) * | 2004-06-10 | 2011-01-04 | Saint Louis University | Proteins with an attached short peptide of acidic amino acids |
| EP2384200B1 (en) | 2009-01-30 | 2013-07-10 | UniQure IP B.V. | Alanine-glyoxylate aminotransferase therapeutics |
| 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 |
| WO2015060722A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| 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 |
| MY204731A (en) * | 2018-05-30 | 2024-09-11 | Esteve Pharmaceuticals Sa | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a |
| BR112021001498A2 (pt) | 2018-07-27 | 2021-04-27 | Regenxbio Inc. | vírus adeno-associado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav e métodos para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
| BR112022015036A2 (pt) | 2020-01-29 | 2022-10-11 | Regenxbio Inc | Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
-
2019
- 2019-07-26 BR BR112021001498-5A patent/BR112021001498A2/pt not_active IP Right Cessation
- 2019-07-26 KR KR1020217004895A patent/KR20210040984A/ko not_active Withdrawn
- 2019-07-26 CA CA3107800A patent/CA3107800A1/en active Pending
- 2019-07-26 JP JP2021527020A patent/JP2021531044A/ja active Pending
- 2019-07-26 TW TW108126686A patent/TW202039857A/zh unknown
- 2019-07-26 EP EP19841900.4A patent/EP3829619A4/en not_active Withdrawn
- 2019-07-26 WO PCT/US2019/043631 patent/WO2020023857A1/en not_active Ceased
- 2019-07-26 AU AU2019312301A patent/AU2019312301A1/en not_active Abandoned
- 2019-07-26 US US17/263,049 patent/US12247213B2/en active Active
- 2019-07-26 SG SG11202100781VA patent/SG11202100781VA/en unknown
-
2021
- 2021-01-21 IL IL280343A patent/IL280343A/en unknown
- 2021-02-25 CO CONC2021/0002505A patent/CO2021002505A2/es unknown
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