JP2022533448A5 - - Google Patents
Info
- Publication number
- JP2022533448A5 JP2022533448A5 JP2021569494A JP2021569494A JP2022533448A5 JP 2022533448 A5 JP2022533448 A5 JP 2022533448A5 JP 2021569494 A JP2021569494 A JP 2021569494A JP 2021569494 A JP2021569494 A JP 2021569494A JP 2022533448 A5 JP2022533448 A5 JP 2022533448A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide
- vector
- expression cassette
- expression
- human
- 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.)
- Granted
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962851411P | 2019-05-22 | 2019-05-22 | |
| US62/851,411 | 2019-05-22 | ||
| PCT/US2020/034171 WO2020237130A1 (en) | 2019-05-22 | 2020-05-22 | Ube3a genes and expression cassettes and their use |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022533448A JP2022533448A (ja) | 2022-07-22 |
| JP2022533448A5 true JP2022533448A5 (https=) | 2023-10-31 |
| JP7616668B2 JP7616668B2 (ja) | 2025-01-17 |
Family
ID=73458642
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021569494A Active JP7616668B2 (ja) | 2019-05-22 | 2020-05-22 | Ｕｂｅ３ａ遺伝子および発現カセットならびにそれらの使用 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US12465655B2 (https=) |
| EP (1) | EP3973059A4 (https=) |
| JP (1) | JP7616668B2 (https=) |
| KR (1) | KR20210158859A (https=) |
| CN (1) | CN114127296B (https=) |
| AU (1) | AU2020279387A1 (https=) |
| CA (1) | CA3139678A1 (https=) |
| EA (1) | EA202193214A1 (https=) |
| IL (1) | IL287943A (https=) |
| MX (1) | MX2021014248A (https=) |
| SG (1) | SG11202112470YA (https=) |
| WO (1) | WO2020237130A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL303239A (en) * | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | Compositions and their uses for the treatment of Engelmann syndrome |
| AU2023285684A1 (en) * | 2022-06-07 | 2025-01-02 | Kicho Inc. | Agents for modulating expression |
| EP4661916A1 (en) * | 2023-02-08 | 2025-12-17 | Ginkgo Bioworks, Inc. | Gene therapy for angelman syndrome |
| WO2025128816A1 (en) * | 2023-12-13 | 2025-06-19 | Kicho Inc. | Methods of modulating expression of ube3a gene |
| WO2025213123A1 (en) * | 2024-04-05 | 2025-10-09 | Encoded Therapeutics, Inc. | Methods and compositions for increasing expression of ube3a |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| WO1998011244A2 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| AU774706B2 (en) | 1998-09-22 | 2004-07-08 | Johns Hopkins University, The | Methods for large-scale production of recombinant AAV vectors |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| WO2014004572A2 (en) * | 2012-06-25 | 2014-01-03 | Isis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| EP2900686B1 (en) | 2012-09-28 | 2020-06-10 | The University of North Carolina At Chapel Hill | Aav vectors targeted to oligodendrocytes |
| MX374532B (es) * | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| CN105745326A (zh) * | 2013-10-24 | 2016-07-06 | 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 |
| EP4344741A3 (en) | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
| EP3291843B1 (en) | 2015-05-07 | 2023-03-22 | University of South Florida | Modified ube3a gene for a gene therapy approach for angelman syndrome |
| WO2017048466A1 (en) * | 2015-09-15 | 2017-03-23 | The Regents Of The University Of California | Compositions and methods for delivering biotherapeutics |
| LT3374509T (lt) | 2015-11-12 | 2021-03-10 | F. Hoffmann-La Roche Ag | Oligonukleotidai, skirti sukelti iš tėvo paveldėto ube3a raišką |
| BR112019027692A2 (pt) * | 2017-06-28 | 2020-11-24 | University Of South Florida | gene ube3a modificado para uma abordagem terapêutica genética para a síndrome de angelman |
| CN114206393A (zh) * | 2019-03-21 | 2022-03-18 | Ptc医疗公司 | 用于治疗天使综合征的载体和方法 |
| EP4017523A4 (en) * | 2019-08-22 | 2024-01-17 | The Regents Of The University Of California | UBE3A FOR THE TREATMENT OF ANGELMAN SYNDROME |
-
2020
- 2020-05-22 US US17/612,833 patent/US12465655B2/en active Active
- 2020-05-22 WO PCT/US2020/034171 patent/WO2020237130A1/en not_active Ceased
- 2020-05-22 CN CN202080050918.0A patent/CN114127296B/zh active Active
- 2020-05-22 JP JP2021569494A patent/JP7616668B2/ja active Active
- 2020-05-22 MX MX2021014248A patent/MX2021014248A/es unknown
- 2020-05-22 CA CA3139678A patent/CA3139678A1/en active Pending
- 2020-05-22 KR KR1020217041626A patent/KR20210158859A/ko not_active Withdrawn
- 2020-05-22 AU AU2020279387A patent/AU2020279387A1/en active Pending
- 2020-05-22 EP EP20809370.8A patent/EP3973059A4/en active Pending
- 2020-05-22 EA EA202193214A patent/EA202193214A1/ru unknown
- 2020-05-22 SG SG11202112470YA patent/SG11202112470YA/en unknown
-
2021
- 2021-11-09 IL IL287943A patent/IL287943A/en unknown
-
2025
- 2025-10-15 US US19/359,449 patent/US20260102519A1/en active Pending