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| JP2025006785A Withdrawn JP2025066765A (ja) | 2018-05-16 | 2025-01-17 | コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法 |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| JP2025006785A Withdrawn JP2025066765A (ja) | 2018-05-16 | 2025-01-17 | コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法 |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 |
|---|---|---|---|---|
| AU2018281280B2 (en) | 2017-06-07 | 2024-12-19 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| CN112040985A (zh) | 2018-02-07 | 2020-12-04 | 瑞泽恩制药公司 | 用于递送治疗性蛋白质的方法和组合物 |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| 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 |
| CN113226352B (zh) | 2018-08-24 | 2025-08-26 | 星火治疗有限公司 | 最优化启动子序列、无内含子的表达结构及使用方法 |
| EP3996696A1 (en) * | 2019-07-09 | 2022-05-18 | Genethon | Treatment of glycogen storage disease (gsd) |
| MX2022007399A (es) | 2019-12-20 | 2022-09-19 | Codexis Inc | Variantes de alfa-glucosidasa ácida diseñadas. |
| PE20240915A1 (es) * | 2021-01-14 | 2024-04-30 | Spark Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de fabry |
| CA3229580A1 (en) * | 2021-08-20 | 2023-02-23 | Anna Nikolaevna STRELKOVA | Method for producing modified adeno-associated virus capsid |
| WO2023028567A2 (en) * | 2021-08-25 | 2023-03-02 | Canbridge Pharmaceuticals, Inc. | Aav particles comprising a liver-tropic capsid protein and acid alpha-glucosidase (gaa) and their use to treat pompe disease |
| EP4392569A4 (en) * | 2021-08-25 | 2025-07-16 | Canbridge Pharmaceuticals Inc | AAV PARTICLES COMPRISING A LIVER-TROPIC CAPSID PROTEIN AND AN ALPHA-GALACTOSIDASE AND THEIR USE FOR TREATING FABRY DISEASE |
| IL314482A (en) * | 2022-02-02 | 2024-09-01 | Regeneron Pharma | 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 |
|---|---|---|---|---|
| EP2687597B1 (en) | 2011-03-16 | 2018-12-26 | Amano Enzyme Inc. | Modified alpha-glucosidase and applications of same |
| WO2013151664A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| 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 |
| JP6342886B2 (ja) * | 2012-04-18 | 2018-06-13 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法 |
| US10077436B2 (en) * | 2013-09-17 | 2018-09-18 | Edeniq, Inc. | Beta-glucosidase enzymes for increased biomass saccharification |
| JP6649265B2 (ja) * | 2013-11-26 | 2020-02-19 | ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 糖原病の処置のためのアデノ随伴ウイルスベクター |
| US10781459B2 (en) * | 2014-06-20 | 2020-09-22 | University Of Florida Research Foundation, Incorporated | Methods of packaging multiple adeno-associated virus vectors |
| WO2016063331A1 (ja) | 2014-10-20 | 2016-04-28 | 昭和産業株式会社 | 新規α－グルコシダーゼ |
| EP3293259A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293203A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| KR102617502B1 (ko) * | 2016-09-12 | 2023-12-26 | 제네똥 | 산-알파 글루코시다제 변이체 및 이의 용도 |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
-
2019
- 2019-05-15 SG SG11202011191WA patent/SG11202011191WA/en unknown
- 2019-05-15 US US17/055,523 patent/US12084693B2/en active Active
- 2019-05-15 KR KR1020207036017A patent/KR20210021310A/ko active Pending
- 2019-05-15 MY MYPI2020005992A patent/MY207355A/en unknown
- 2019-05-15 BR BR112020023393-5A patent/BR112020023393A2/pt not_active Application Discontinuation
- 2019-05-15 PE PE2020001867A patent/PE20210337A1/es unknown
- 2019-05-15 CA CA3100213A patent/CA3100213A1/en active Pending
- 2019-05-15 WO PCT/US2019/032502 patent/WO2019222411A1/en not_active Ceased
- 2019-05-15 EP EP19803504.0A patent/EP3794043A4/en active Pending
- 2019-05-15 MX MX2020012332A patent/MX2020012332A/es unknown
- 2019-05-15 AU AU2019268363A patent/AU2019268363A1/en not_active Abandoned
- 2019-05-15 CN CN201980040391.0A patent/CN112334489A/zh active Pending
- 2019-05-15 JP JP2020564115A patent/JP2021523718A/ja not_active Ceased
- 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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