JP2023513487A5 - - Google Patents
Info
- Publication number
- JP2023513487A5 JP2023513487A5 JP2022547042A JP2022547042A JP2023513487A5 JP 2023513487 A5 JP2023513487 A5 JP 2023513487A5 JP 2022547042 A JP2022547042 A JP 2022547042A JP 2022547042 A JP2022547042 A JP 2022547042A JP 2023513487 A5 JP2023513487 A5 JP 2023513487A5
- Authority
- JP
- Japan
- Prior art keywords
- patient
- seq
- raav
- human
- 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 |
|---|---|---|---|
| US202062969142P | 2020-02-02 | 2020-02-02 | |
| US62/969,142 | 2020-02-02 | ||
| US202063007297P | 2020-04-08 | 2020-04-08 | |
| US63/007,297 | 2020-04-08 | ||
| US202063063119P | 2020-08-07 | 2020-08-07 | |
| US63/063,119 | 2020-08-07 | ||
| PCT/US2021/015988 WO2021155337A1 (en) | 2020-02-02 | 2021-02-01 | Compositions useful for treating gm1 gangliosidosis |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023513487A JP2023513487A (ja) | 2023-03-31 |
| JP2023513487A5 true JP2023513487A5 (https=) | 2024-02-08 |
Family
ID=74867612
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022547042A Pending JP2023513487A (ja) | 2020-02-02 | 2021-02-01 | Ｇｍ１ガングリオシドーシスを治療するのに有用な組成物 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230190966A1 (https=) |
| EP (1) | EP4096721A1 (https=) |
| JP (1) | JP2023513487A (https=) |
| KR (1) | KR20220145838A (https=) |
| CN (1) | CN115867323A (https=) |
| AU (1) | AU2021213826A1 (https=) |
| BR (1) | BR112022013914A2 (https=) |
| CA (1) | CA3165057A1 (https=) |
| IL (1) | IL294924A (https=) |
| MX (1) | MX2022009462A (https=) |
| TW (1) | TWI905145B (https=) |
| WO (1) | WO2021155337A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3114175A1 (en) | 2018-10-01 | 2020-04-09 | The Trustees Of The University Of Pennsylvania | Beta-galactosidase based aav for treating gm1 gangliosidosis |
| WO2023147351A1 (en) * | 2022-01-25 | 2023-08-03 | Regents Of The University Of Minnesota | Crispr-mediated human genome editing with vectors |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP2434420A3 (en) | 2003-08-01 | 2012-07-25 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| CA3012195A1 (en) * | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| PT3589730T (pt) * | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| WO2019222314A1 (en) * | 2018-05-15 | 2019-11-21 | University Of Massachusetts | Raav vectors encoding of lysosomal beta-galactorsidase (glb1) and cathepsin a |
| CA3114175A1 (en) * | 2018-10-01 | 2020-04-09 | The Trustees Of The University Of Pennsylvania | Beta-galactosidase based aav for treating gm1 gangliosidosis |
-
2021
- 2021-02-01 KR KR1020227029939A patent/KR20220145838A/ko active Pending
- 2021-02-01 IL IL294924A patent/IL294924A/en unknown
- 2021-02-01 CN CN202180025846.9A patent/CN115867323A/zh active Pending
- 2021-02-01 US US17/759,905 patent/US20230190966A1/en active Pending
- 2021-02-01 WO PCT/US2021/015988 patent/WO2021155337A1/en not_active Ceased
- 2021-02-01 AU AU2021213826A patent/AU2021213826A1/en active Pending
- 2021-02-01 TW TW110103642A patent/TWI905145B/zh active
- 2021-02-01 MX MX2022009462A patent/MX2022009462A/es unknown
- 2021-02-01 CA CA3165057A patent/CA3165057A1/en active Pending
- 2021-02-01 BR BR112022013914A patent/BR112022013914A2/pt unknown
- 2021-02-01 EP EP21711072.5A patent/EP4096721A1/en active Pending
- 2021-02-01 JP JP2022547042A patent/JP2023513487A/ja active Pending
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