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 |
|---|---|---|---|---|
| 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 |
| EP4004214A1 (en) * | 2019-07-26 | 2022-06-01 | RegenxBio Inc. | Engineered nucleic acid regulatory element and methods of uses thereof |
| WO2021154956A1 (en) * | 2020-01-29 | 2021-08-05 | Regenxbio Inc. | Treatment of mucopolysaccharidosis iva |
| WO2023077092A1 (en) * | 2021-10-28 | 2023-05-04 | 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 |
| US7863238B2 (en) * | 2004-06-10 | 2011-01-04 | Saint Louis University | Proteins with an attached short peptide of acidic amino acids |
| 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 |
| CA2750811C (en) | 2009-01-30 | 2017-08-29 | Amsterdam Molecular Therapeutics (Amt) 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 |
| CA2995733A1 (en) | 2015-08-31 | 2017-03-09 | The Trustees Of The University Of Pennsylvania | Aav-epo for treating companion animals |
| US12146169B2 (en) * | 2018-05-30 | 2024-11-19 | Esteve Pharmaceuticals, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type IV A |
| 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 |
| WO2021154956A1 (en) | 2020-01-29 | 2021-08-05 | Regenxbio Inc. | Treatment of mucopolysaccharidosis iva |
-
2019
- 2019-07-26 TW TW108126686A patent/TW202039857A/zh unknown
- 2019-07-26 US US17/263,049 patent/US12247213B2/en active Active
- 2019-07-26 BR BR112021001498-5A patent/BR112021001498A2/pt not_active IP Right Cessation
- 2019-07-26 JP JP2021527020A patent/JP2021531044A/ja active Pending
- 2019-07-26 SG SG11202100781VA patent/SG11202100781VA/en unknown
- 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 CA CA3107800A patent/CA3107800A1/en active Pending
- 2019-07-26 EP EP19841900.4A patent/EP3829619A4/en not_active Withdrawn
- 2019-07-26 KR KR1020217004895A patent/KR20210040984A/ko not_active Withdrawn
-
2021
- 2021-01-21 IL IL280343A patent/IL280343A/en unknown
- 2021-02-25 CO CONC2021/0002505A patent/CO2021002505A2/es unknown
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