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
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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2021523718A JP2021523718A (ja) | 2021-09-09 |
| JP2021523718A5 true JP2021523718A5 (https=) | 2022-04-11 |
| JPWO2019222411A5 JPWO2019222411A5 (https=) | 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| JP2025006785A Withdrawn JP2025066765A (ja) | 2018-05-16 | 2025-01-17 | コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法 |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| JP2025006785A Withdrawn JP2025066765A (ja) | 2018-05-16 | 2025-01-17 | コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法 |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 (https=) |
| EP (1) | EP3794043A4 (https=) |
| JP (2) | JP2021523718A (https=) |
| KR (1) | KR20210021310A (https=) |
| CN (1) | CN112334489A (https=) |
| AU (1) | AU2019268363A1 (https=) |
| BR (1) | BR112020023393A2 (https=) |
| CA (1) | CA3100213A1 (https=) |
| CL (1) | CL2020002973A1 (https=) |
| CO (1) | CO2020015707A2 (https=) |
| MX (1) | MX2020012332A (https=) |
| MY (1) | MY207355A (https=) |
| PE (1) | PE20210337A1 (https=) |
| PH (1) | PH12020551957A1 (https=) |
| SG (1) | SG11202011191WA (https=) |
| TW (1) | TW202016310A (https=) |
| WO (1) | WO2019222411A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FI3635009T3 (fi) | 2017-06-07 | 2026-04-07 | Regeneron Pharma | Koostumuksia ja menetelmiä entsyymien internalisoimiseksi |
| JP7779653B2 (ja) | 2018-02-07 | 2025-12-03 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 治療用タンパク質送達のための方法および組成物 |
| 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 |
| US12290574B2 (en) | 2018-08-24 | 2025-05-06 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| EP3996696A1 (en) * | 2019-07-09 | 2022-05-18 | Genethon | Treatment of glycogen storage disease (gsd) |
| KR20220129547A (ko) * | 2019-12-20 | 2022-09-23 | 코덱시스, 인코포레이티드 | 조작된 산성 알파-글루코시다제 변이체 |
| EP4277988A4 (en) * | 2021-01-14 | 2024-12-11 | Spark Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF FABRY'S DISEASE |
| IL310949A (en) * | 2021-08-20 | 2024-04-01 | Biocad Joint Stock Co | A method for preparing an adapted adeno-associated virus envelope |
| TW202338095A (zh) * | 2021-08-25 | 2023-10-01 | 美商北海康成製藥有限公司 | 包含肝臟向性衣殼蛋白質及酸α－葡萄糖苷酶（ＧＡＡ）之ＡＡＶ顆粒及其用於治療龐貝氏病（ＰＯＭＰＥ ＤＩＳＥＡＳＥ）之用途 |
| KR20240073025A (ko) * | 2021-08-25 | 2024-05-24 | 캔브리지 파마슈티컬스, 인크. | 간-향성 캡시드 단백질 및 알파-갈락토시다제를 포함하는 aav 입자 및 파브리 질환을 치료하기 위한 이의 용도 |
| EP4473103A2 (en) * | 2022-02-02 | 2024-12-11 | 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 |
| AU2024317483A1 (en) * | 2023-07-28 | 2026-01-29 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2687597T3 (en) | 2011-03-16 | 2019-02-25 | Amano Enzyme Inc | MODIFIED ALPHA-GLUCOSIDASE AND USES OF SAME |
| CA2868391A1 (en) * | 2012-04-02 | 2013-10-10 | Stephane Bancel | Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same |
| AU2013243948A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| AU2013249202B2 (en) * | 2012-04-18 | 2018-08-09 | The Children's Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using AAV capsid variants |
| CN105745322A (zh) * | 2013-09-17 | 2016-07-06 | 易登尼有限公司 | 用于增加生物质糖化的改良的β-葡萄糖苷酶 |
| WO2015081101A1 (en) * | 2013-11-26 | 2015-06-04 | 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 |
| JP6469127B2 (ja) | 2014-10-20 | 2019-02-13 | 昭和産業株式会社 | 新規α−グルコシダーゼ |
| 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 |
| MX2019002842A (es) * | 2016-09-12 | 2019-08-29 | Genethon | Variantes de alfa-glucosidasa acida y usos de las mismas. |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
-
2019
- 2019-05-15 CA CA3100213A patent/CA3100213A1/en active Pending
- 2019-05-15 SG SG11202011191WA patent/SG11202011191WA/en unknown
- 2019-05-15 EP EP19803504.0A patent/EP3794043A4/en active Pending
- 2019-05-15 KR KR1020207036017A patent/KR20210021310A/ko active Pending
- 2019-05-15 CN CN201980040391.0A patent/CN112334489A/zh active Pending
- 2019-05-15 US US17/055,523 patent/US12084693B2/en active Active
- 2019-05-15 JP JP2020564115A patent/JP2021523718A/ja not_active Ceased
- 2019-05-15 WO PCT/US2019/032502 patent/WO2019222411A1/en not_active Ceased
- 2019-05-15 AU AU2019268363A patent/AU2019268363A1/en not_active Abandoned
- 2019-05-15 MX MX2020012332A patent/MX2020012332A/es unknown
- 2019-05-15 PE PE2020001867A patent/PE20210337A1/es unknown
- 2019-05-15 MY MYPI2020005992A patent/MY207355A/en unknown
- 2019-05-15 BR BR112020023393-5A patent/BR112020023393A2/pt 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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