JP2021523718A5 - - Google Patents
Info
- Publication number
- JP2021523718A5 JP2021523718A5 JP2020564115A JP2020564115A JP2021523718A5 JP 2021523718 A5 JP2021523718 A5 JP 2021523718A5 JP 2020564115 A JP2020564115 A JP 2020564115A JP 2020564115 A JP2020564115 A JP 2020564115A JP 2021523718 A5 JP2021523718 A5 JP 2021523718A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- gaa
- acid encoding
- sequence
- aav
- 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.)
- Ceased
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025006785A JP2025066765A (ja) | 2018-05-16 | 2025-01-17 | コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862672419P | 2018-05-16 | 2018-05-16 | |
| US62/672,419 | 2018-05-16 | ||
| US201862734454P | 2018-09-21 | 2018-09-21 | |
| US62/734,454 | 2018-09-21 | ||
| PCT/US2019/032502 WO2019222411A1 (en) | 2018-05-16 | 2019-05-15 | Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025006785A Division JP2025066765A (ja) | 2018-05-16 | 2025-01-17 | コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021523718A JP2021523718A (ja) | 2021-09-09 |
| JP2021523718A5 true JP2021523718A5 (enExample) | 2022-04-11 |
Family
ID=68540790
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020564115A Ceased JP2021523718A (ja) | 2018-05-16 | 2019-05-15 | コドン最適化された酸性α−グルコシダーゼ発現カセット及びそれを使用する方法 |
| JP2025006785A Withdrawn JP2025066765A (ja) | 2018-05-16 | 2025-01-17 | コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025006785A Withdrawn JP2025066765A (ja) | 2018-05-16 | 2025-01-17 | コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法 |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US12084693B2 (enExample) |
| EP (1) | EP3794043A4 (enExample) |
| JP (2) | JP2021523718A (enExample) |
| KR (1) | KR20210021310A (enExample) |
| CN (1) | CN112334489A (enExample) |
| AU (1) | AU2019268363A1 (enExample) |
| BR (1) | BR112020023393A2 (enExample) |
| CA (1) | CA3100213A1 (enExample) |
| CL (1) | CL2020002973A1 (enExample) |
| CO (1) | CO2020015707A2 (enExample) |
| MX (1) | MX2020012332A (enExample) |
| MY (1) | MY207355A (enExample) |
| PE (1) | PE20210337A1 (enExample) |
| PH (1) | PH12020551957A1 (enExample) |
| SG (1) | SG11202011191WA (enExample) |
| TW (1) | TW202016310A (enExample) |
| WO (1) | WO2019222411A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK3635009T3 (da) | 2017-06-07 | 2026-03-30 | Regeneron Pharma | Sammensætninger og fremgangsmåder til internalisering af enzymer |
| US12258597B2 (en) | 2018-02-07 | 2025-03-25 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| BR112021003399A2 (pt) | 2018-08-24 | 2021-05-18 | Spark Therapeutics, Inc. | sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso |
| EP3996696A1 (en) * | 2019-07-09 | 2022-05-18 | Genethon | Treatment of glycogen storage disease (gsd) |
| JP2023507575A (ja) | 2019-12-20 | 2023-02-24 | コデクシス， インコーポレイテッド | 操作された酸性アルファ－グルコシダーゼバリアント |
| MX2023008362A (es) * | 2021-01-14 | 2023-10-04 | Spark Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de fabry. |
| CR20240137A (es) * | 2021-08-20 | 2024-07-29 | Biocad Joint Stock Co | Método para obtener una cápside de virus adenoasociado modificada |
| CA3229998A1 (en) * | 2021-08-25 | 2023-03-02 | Canbridge Pharmaceuticals, Inc. | Aav particles comprising a liver-tropic capsid protein and alpha-galactosidase and their use to treat fabry disease |
| AR126877A1 (es) * | 2021-08-25 | 2023-11-22 | Canbridge Pharmaceuticals Inc | PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GLUCOSIDASA ÁCIDA (GAA) Y SU USO PARA TRATAR LA ENFERMEDAD DE POMPE |
| CA3242731A1 (en) * | 2022-02-02 | 2023-08-10 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease |
| CN118667797A (zh) * | 2023-03-14 | 2024-09-20 | 北京据德医药科技有限公司 | 一种重组酸性α-葡萄糖苷酶及其用途 |
| WO2024231820A1 (en) * | 2023-05-05 | 2024-11-14 | Takeda Pharmaceutical Company Limited | Treatment of pompe disease |
| IL326018A (en) * | 2023-07-28 | 2026-03-01 | Regeneron Pharma | Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN103443273B (zh) | 2011-03-16 | 2016-10-26 | 天野酶制品株式会社 | 修饰型α-葡萄糖苷酶及其用途 |
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| CN104411338A (zh) * | 2012-04-02 | 2015-03-11 | 现代治疗公司 | 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 |
| NZ701693A (en) * | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| WO2015042064A1 (en) * | 2013-09-17 | 2015-03-26 | Edeniq, Inc. | Improved beta-glucosidase enzymes for increased biomass saccharification |
| AU2014354839B2 (en) * | 2013-11-26 | 2020-06-04 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Adeno-associated virus vectors for treatment of glycogen storage disease |
| US10781459B2 (en) * | 2014-06-20 | 2020-09-22 | University Of Florida Research Foundation, Incorporated | Methods of packaging multiple adeno-associated virus vectors |
| EP3211076A4 (en) | 2014-10-20 | 2018-03-21 | Showa Sangyo Co., Ltd. | Novel alpha-glucosidase |
| WO2018046774A1 (en) * | 2016-09-12 | 2018-03-15 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293203A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| JP2022513067A (ja) * | 2018-11-16 | 2022-02-07 | アスクレピオス バイオファーマシューティカル， インコーポレイテッド | ポンペ病を処置するための治療用アデノ随伴ウイルス |
-
2019
- 2019-05-15 MY MYPI2020005992A patent/MY207355A/en unknown
- 2019-05-15 PE PE2020001867A patent/PE20210337A1/es unknown
- 2019-05-15 AU AU2019268363A patent/AU2019268363A1/en not_active Abandoned
- 2019-05-15 WO PCT/US2019/032502 patent/WO2019222411A1/en not_active Ceased
- 2019-05-15 KR KR1020207036017A patent/KR20210021310A/ko active Pending
- 2019-05-15 SG SG11202011191WA patent/SG11202011191WA/en unknown
- 2019-05-15 US US17/055,523 patent/US12084693B2/en active Active
- 2019-05-15 CA CA3100213A patent/CA3100213A1/en active Pending
- 2019-05-15 JP JP2020564115A patent/JP2021523718A/ja not_active Ceased
- 2019-05-15 EP EP19803504.0A patent/EP3794043A4/en active Pending
- 2019-05-15 CN CN201980040391.0A patent/CN112334489A/zh active Pending
- 2019-05-15 BR BR112020023393-5A patent/BR112020023393A2/pt unknown
- 2019-05-15 MX MX2020012332A patent/MX2020012332A/es unknown
- 2019-05-16 TW TW108116960A patent/TW202016310A/zh unknown
-
2020
- 2020-11-16 PH PH12020551957A patent/PH12020551957A1/en unknown
- 2020-11-16 CL CL2020002973A patent/CL2020002973A1/es unknown
- 2020-12-15 CO CONC2020/0015707A patent/CO2020015707A2/es unknown
-
2024
- 2024-09-09 US US18/828,493 patent/US20250101396A1/en active Pending
-
2025
- 2025-01-17 JP JP2025006785A patent/JP2025066765A/ja not_active Withdrawn
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