JPWO2020243717A5 - - Google Patents
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- JPWO2020243717A5 JPWO2020243717A5 JP2021570364A JP2021570364A JPWO2020243717A5 JP WO2020243717 A5 JPWO2020243717 A5 JP WO2020243717A5 JP 2021570364 A JP2021570364 A JP 2021570364A JP 2021570364 A JP2021570364 A JP 2021570364A JP WO2020243717 A5 JPWO2020243717 A5 JP WO2020243717A5
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- JP
- Japan
- Prior art keywords
- sequence
- seq
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- variant
- 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
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- 239000013603 viral vector Substances 0.000 claims description 37
- 210000004185 liver Anatomy 0.000 claims description 10
- 239000003623 enhancer Substances 0.000 claims description 8
- 230000001225 therapeutic effect Effects 0.000 claims description 7
- 239000002245 particle Substances 0.000 claims description 6
- 201000011252 Phenylketonuria Diseases 0.000 claims description 5
- 239000013598 vector Substances 0.000 claims description 4
- 108091032955 Bacterial small RNA Proteins 0.000 claims description 2
- 101710091045 Envelope protein Proteins 0.000 claims description 2
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 2
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 2
- 101710188315 Protein X Proteins 0.000 claims description 2
- 102100027378 Prothrombin Human genes 0.000 claims description 2
- 108010094028 Prothrombin Proteins 0.000 claims description 2
- 102100021696 Syncytin-1 Human genes 0.000 claims description 2
- 238000004806 packaging method and process Methods 0.000 claims description 2
- 229940039716 prothrombin Drugs 0.000 claims description 2
- 239000003814 drug Substances 0.000 description 1
- 238000000034 method Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962855506P | 2019-05-31 | 2019-05-31 | |
| US62/855,506 | 2019-05-31 | ||
| PCT/US2020/035584 WO2020243717A1 (en) | 2019-05-31 | 2020-06-01 | Optimized phenylalanine hydroxylase expression |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022535745A JP2022535745A (ja) | 2022-08-10 |
| JPWO2020243717A5 true JPWO2020243717A5 (https=) | 2023-06-08 |
| JP2022535745A5 JP2022535745A5 (https=) | 2023-06-08 |
Family
ID=73553945
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021570364A Pending JP2022535745A (ja) | 2019-05-31 | 2020-06-01 | 最適化されたフェニルアラニンヒドロキシラーゼ発現 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20220162643A1 (https=) |
| EP (1) | EP3976076A4 (https=) |
| JP (1) | JP2022535745A (https=) |
| KR (1) | KR20220068954A (https=) |
| CN (1) | CN113905768A (https=) |
| AU (1) | AU2020283069A1 (https=) |
| BR (1) | BR112021024124A2 (https=) |
| CA (1) | CA3137698A1 (https=) |
| IL (1) | IL288400A (https=) |
| WO (1) | WO2020243717A1 (https=) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010045659A1 (en) | 2008-10-17 | 2010-04-22 | American Gene Technologies International Inc. | Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules |
| JP6890831B2 (ja) | 2015-07-08 | 2021-06-18 | アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド | Ｈｉｖ予備免疫化および免疫療法 |
| US10137144B2 (en) | 2016-01-15 | 2018-11-27 | American Gene Technologies International Inc. | Methods and compositions for the activation of gamma-delta T-cells |
| DK3402483T3 (da) | 2016-01-15 | 2024-01-02 | American Gene Tech Int Inc | Fremgangsmåder og sammensætninger til aktivering af gamma-delta-T-celler |
| US10888613B2 (en) | 2016-02-08 | 2021-01-12 | American Gene Technologies International Inc. | Method of producing cells resistant to HIV infection |
| DK3426777T3 (da) | 2016-03-09 | 2022-04-25 | American Gene Tech Int Inc | Kombinationsvektorer og fremgangsmåder til behandling af cancer |
| US11976292B2 (en) | 2016-06-08 | 2024-05-07 | American Gene Technologies International Inc. | Non-integrating viral delivery system and methods related thereto |
| IL284348B2 (en) | 2016-07-08 | 2025-06-01 | American Gene Tech Int Inc | Hiv pre-immunization and immunotherapy |
| JP7176756B2 (ja) | 2016-07-21 | 2022-11-22 | アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド | パーキンソン病を処置するためのウイルスベクター |
| CA3057142A1 (en) | 2017-04-03 | 2018-10-11 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| CA3064442A1 (en) | 2017-06-16 | 2018-12-20 | American Gene Technologies International Inc. | Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells |
| JP7423521B2 (ja) | 2017-11-22 | 2024-01-29 | モダーナティエックス・インコーポレイテッド | フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド |
| US20240026374A1 (en) * | 2020-09-16 | 2024-01-25 | Generation Bio Co. | Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) |
| IL311240A (en) * | 2021-09-16 | 2024-05-01 | Generation Bio Co | Liver-specific expression cassettes, vectors and their uses for the expression of therapeutic proteins |
| CN115772503B (zh) * | 2022-08-16 | 2023-10-17 | 广东省南山医药创新研究院 | 一种表达pah的基因修饰细胞药物及其制备方法与应用 |
| WO2025064507A1 (en) * | 2023-09-19 | 2025-03-27 | Poseida Therapeutics, Inc. | Compositions and methods for integration of viral vectors |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| EA034103B1 (ru) * | 2013-10-22 | 2019-12-27 | Транслейт Био, Инк. | СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК |
| CN115074366A (zh) * | 2015-04-16 | 2022-09-20 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| US20180363000A1 (en) * | 2015-12-14 | 2018-12-20 | The Trustees Of The University Of Pennsylvania | Aav-anti pcsk9 antibody constructs and uses thereof |
| WO2017177169A1 (en) * | 2016-04-08 | 2017-10-12 | Rana Therapeutics, Inc. | Multimeric coding nucleic acid and uses thereof |
| KR102738752B1 (ko) * | 2016-12-30 | 2024-12-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
| CA3057142A1 (en) * | 2017-04-03 | 2018-10-11 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| WO2019070674A2 (en) * | 2017-10-02 | 2019-04-11 | American Gene Technologies International Inc. | VECTORS COMPRISING PROMOTER AND ACTIVATOR COMBINATIONS FOR THE TREATMENT OF PHENYLCETONURIA |
-
2020
- 2020-06-01 BR BR112021024124A patent/BR112021024124A2/pt unknown
- 2020-06-01 CA CA3137698A patent/CA3137698A1/en active Pending
- 2020-06-01 KR KR1020217042390A patent/KR20220068954A/ko active Pending
- 2020-06-01 CN CN202080040373.5A patent/CN113905768A/zh active Pending
- 2020-06-01 EP EP20814445.1A patent/EP3976076A4/en active Pending
- 2020-06-01 AU AU2020283069A patent/AU2020283069A1/en not_active Abandoned
- 2020-06-01 JP JP2021570364A patent/JP2022535745A/ja active Pending
- 2020-06-01 WO PCT/US2020/035584 patent/WO2020243717A1/en not_active Ceased
- 2020-06-01 US US17/610,111 patent/US20220162643A1/en active Pending
-
2021
- 2021-11-25 IL IL288400A patent/IL288400A/en unknown
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