JP2022530095A5 - - Google Patents
Info
- Publication number
- JP2022530095A5 JP2022530095A5 JP2021563234A JP2021563234A JP2022530095A5 JP 2022530095 A5 JP2022530095 A5 JP 2022530095A5 JP 2021563234 A JP2021563234 A JP 2021563234A JP 2021563234 A JP2021563234 A JP 2021563234A JP 2022530095 A5 JP2022530095 A5 JP 2022530095A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- raav
- seq
- mirna target
- vector
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962837947P | 2019-04-24 | 2019-04-24 | |
| US62/837,947 | 2019-04-24 | ||
| PCT/US2020/029642 WO2020219766A1 (en) | 2019-04-24 | 2020-04-23 | Compositions useful in treatment of rett syndrome |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022530095A JP2022530095A (ja) | 2022-06-27 |
| JP2022530095A5 true JP2022530095A5 (https=) | 2023-04-28 |
Family
ID=72941315
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021563234A Pending JP2022530095A (ja) | 2019-04-24 | 2020-04-23 | レット症候群の治療に有用な組成物 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220202960A1 (https=) |
| EP (1) | EP3973060A4 (https=) |
| JP (1) | JP2022530095A (https=) |
| KR (1) | KR20220003553A (https=) |
| CN (1) | CN114026236A (https=) |
| AU (1) | AU2020261051A1 (https=) |
| CA (1) | CA3133889A1 (https=) |
| SG (1) | SG11202111279QA (https=) |
| WO (1) | WO2020219766A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2022014258A (es) * | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
| WO2022094078A1 (en) * | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| MX2024004723A (es) * | 2021-10-18 | 2024-07-19 | Univ Pennsylvania | Composiciones útiles en el tratamiento del trastorno por deficiencia de cdkl5 (cdd). |
| US20250312486A1 (en) * | 2022-04-18 | 2025-10-09 | City Of Hope | Compositions for restoring mecp2 gene function and methods of use thereof |
| JP2025536852A (ja) * | 2022-10-10 | 2025-11-07 | パッセージ バイオ， インコーポレイテッド | 完全な組換えａａｖ粒子を精製する方法 |
| EP4715058A1 (en) * | 2023-05-15 | 2026-03-25 | Genecombio Ltd. | Gene therapy for rett syndrome |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1838870A2 (en) * | 2004-12-29 | 2007-10-03 | Exiqon A/S | NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF MICRORNAS AND THEIR TARGET MRNAs |
| CA2609142C (en) * | 2005-05-27 | 2016-02-09 | Fondazione Centro San Raffaele Del Monte Tabor | Therapeutic gene vectors comprising mirna target sequences |
| SI2010563T1 (sl) * | 2006-04-07 | 2011-07-29 | Univ Goettingen Georg August | Sintetična MeCP2 sekvenca za proteinsko nadomestno terapijo |
| US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| UY37620A (es) * | 2017-02-28 | 2018-08-31 | Univ Pennsylvania | Vacunas contra la gripe mediadas por aav novedosas |
| PT3589730T (pt) * | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| BR112019019833A2 (pt) * | 2017-03-24 | 2020-04-22 | The University Court Of The University Of Edinburgh | terapia baseada em mecp2 |
| US11680275B2 (en) * | 2017-06-06 | 2023-06-20 | University Of Massachusetts | Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome |
-
2020
- 2020-04-23 CA CA3133889A patent/CA3133889A1/en active Pending
- 2020-04-23 EP EP20795847.1A patent/EP3973060A4/en not_active Withdrawn
- 2020-04-23 AU AU2020261051A patent/AU2020261051A1/en not_active Abandoned
- 2020-04-23 WO PCT/US2020/029642 patent/WO2020219766A1/en not_active Ceased
- 2020-04-23 JP JP2021563234A patent/JP2022530095A/ja active Pending
- 2020-04-23 CN CN202080046692.7A patent/CN114026236A/zh active Pending
- 2020-04-23 KR KR1020217037510A patent/KR20220003553A/ko active Pending
- 2020-04-23 US US17/605,827 patent/US20220202960A1/en not_active Abandoned
- 2020-04-23 SG SG11202111279QA patent/SG11202111279QA/en unknown
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