JP2020535828A5 - - Google Patents
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- JP2020535828A5 JP2020535828A5 JP2020518812A JP2020518812A JP2020535828A5 JP 2020535828 A5 JP2020535828 A5 JP 2020535828A5 JP 2020518812 A JP2020518812 A JP 2020518812A JP 2020518812 A JP2020518812 A JP 2020518812A JP 2020535828 A5 JP2020535828 A5 JP 2020535828A5
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- JP
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- Prior art keywords
- viral vector
- seq
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- cells
- item
- Prior art date
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- 239000013603 viral vector Substances 0.000 claims description 43
- 239000002245 particle Substances 0.000 claims description 28
- 239000003623 enhancer Substances 0.000 claims description 16
- 210000004185 liver Anatomy 0.000 claims description 12
- 102100027378 Prothrombin Human genes 0.000 claims description 10
- 108010094028 Prothrombin Proteins 0.000 claims description 10
- 229940039716 prothrombin Drugs 0.000 claims description 10
- 230000001225 therapeutic effect Effects 0.000 claims description 9
- 239000013598 vector Substances 0.000 claims description 9
- 102000008088 Hepatocyte Nuclear Factors Human genes 0.000 claims description 8
- 108010049606 Hepatocyte Nuclear Factors Proteins 0.000 claims description 8
- 241000713666 Lentivirus Species 0.000 claims description 8
- 210000004027 cell Anatomy 0.000 claims description 8
- 241000700605 Viruses Species 0.000 claims description 7
- 238000003745 diagnosis Methods 0.000 claims description 6
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 4
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 4
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 4
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 4
- 230000001105 regulatory effect Effects 0.000 claims description 4
- 238000000338 in vitro Methods 0.000 claims description 3
- 238000009609 prenatal screening Methods 0.000 claims description 3
- 210000003890 endocrine cell Anatomy 0.000 claims description 2
- 210000002889 endothelial cell Anatomy 0.000 claims description 2
- 210000002919 epithelial cell Anatomy 0.000 claims description 2
- 230000003394 haemopoietic effect Effects 0.000 claims description 2
- 210000003494 hepatocyte Anatomy 0.000 claims description 2
- 210000004698 lymphocyte Anatomy 0.000 claims description 2
- 210000000663 muscle cell Anatomy 0.000 claims description 2
- 210000000066 myeloid cell Anatomy 0.000 claims description 2
- 210000004412 neuroendocrine cell Anatomy 0.000 claims description 2
- 210000002569 neuron Anatomy 0.000 claims description 2
- 210000004738 parenchymal cell Anatomy 0.000 claims description 2
- 239000002243 precursor Substances 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 238000011144 upstream manufacturing Methods 0.000 claims description 2
- 210000004291 uterus Anatomy 0.000 claims description 2
- 238000000034 method Methods 0.000 description 12
- 108091032955 Bacterial small RNA Proteins 0.000 description 6
- 108020004999 messenger RNA Proteins 0.000 description 4
- 230000000295 complement effect Effects 0.000 description 3
- 108091027967 Small hairpin RNA Proteins 0.000 description 1
- 108091036066 Three prime untranslated region Proteins 0.000 description 1
- 239000004055 small Interfering RNA Substances 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023101880A JP2023115125A (ja) | 2017-10-02 | 2023-06-21 | フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762566979P | 2017-10-02 | 2017-10-02 | |
| US62/566,979 | 2017-10-02 | ||
| PCT/US2018/053919 WO2019070674A2 (en) | 2017-10-02 | 2018-10-02 | VECTORS COMPRISING PROMOTER AND ACTIVATOR COMBINATIONS FOR THE TREATMENT OF PHENYLCETONURIA |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023101880A Division JP2023115125A (ja) | 2017-10-02 | 2023-06-21 | フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020535828A JP2020535828A (ja) | 2020-12-10 |
| JP2020535828A5 true JP2020535828A5 (enExample) | 2021-11-04 |
Family
ID=65994855
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020518812A Pending JP2020535828A (ja) | 2017-10-02 | 2018-10-02 | フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター |
| JP2023101880A Pending JP2023115125A (ja) | 2017-10-02 | 2023-06-21 | フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023101880A Pending JP2023115125A (ja) | 2017-10-02 | 2023-06-21 | フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20200318081A1 (enExample) |
| EP (1) | EP3692157A4 (enExample) |
| JP (2) | JP2020535828A (enExample) |
| KR (1) | KR20200057766A (enExample) |
| CN (1) | CN111433368A (enExample) |
| AU (1) | AU2018345745A1 (enExample) |
| BR (1) | BR112020006672A2 (enExample) |
| CA (1) | CA3077355A1 (enExample) |
| IL (1) | IL273670B2 (enExample) |
| WO (1) | WO2019070674A2 (enExample) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20100183558A1 (en) | 2008-10-17 | 2010-07-22 | Zhennan Lai | Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules |
| WO2017007994A1 (en) | 2015-07-08 | 2017-01-12 | American Gene Technologies International Inc. | Hiv pre-immunization and immunotherapy |
| CN116064678A (zh) | 2016-01-15 | 2023-05-05 | 美国基因技术国际有限公司 | 用于活化γ-δT细胞的方法和组合物 |
| US10137144B2 (en) | 2016-01-15 | 2018-11-27 | American Gene Technologies International Inc. | Methods and compositions for the activation of gamma-delta T-cells |
| US10888613B2 (en) | 2016-02-08 | 2021-01-12 | American Gene Technologies International Inc. | Method of producing cells resistant to HIV infection |
| WO2017156311A2 (en) | 2016-03-09 | 2017-09-14 | American Gene Technologies International Inc. | Combination vectors and methods for treating cancer |
| JP7173548B2 (ja) | 2016-06-08 | 2022-11-16 | 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 | 非組み込みウイルス送達系およびその関連方法 |
| IL300730A (en) | 2016-07-08 | 2023-04-01 | American Gene Tech Int Inc | Pre-HIV vaccine and immunotherapy |
| US11583562B2 (en) | 2016-07-21 | 2023-02-21 | American Gene Technologies International Inc. | Viral vectors for treating Parkinson's disease |
| WO2018187231A2 (en) | 2017-04-03 | 2018-10-11 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| AU2020283069A1 (en) * | 2019-05-31 | 2022-01-06 | American Gene Technologies International Inc. | Optimized phenylalanine hydroxylase expression |
| CA3165015A1 (en) * | 2020-01-16 | 2021-07-22 | Matthias Klugmann | Adeno associated virus based gene therapy for phenylketonuria |
| TW202208632A (zh) * | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| US12173308B2 (en) | 2020-10-01 | 2024-12-24 | Genzyme Corporation | Human PAH expression cassette for treatment of PKU by liver-directed gene replacement therapy |
| IL316839A (en) * | 2022-05-17 | 2025-01-01 | Umoja Biopharma Inc | Production of viral particles |
| 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 |
| TWI893802B (zh) * | 2023-05-11 | 2025-08-11 | 大陸商四川至善唯新生物科技有限公司 | 工程化的肝臟特異性增強子、核心啟動子及其應用 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE360082T1 (de) * | 1999-11-16 | 2007-05-15 | Genzyme Corp | Vektoren und transgene mit regulatorischen elementen zur genverabreichung in leber |
| US20030198620A1 (en) * | 2002-04-16 | 2003-10-23 | Keiya Ozawa | Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions |
| US20050138677A1 (en) * | 2003-09-16 | 2005-06-23 | Pfister Herbert J. | Transgenic animal model for the treatment of skin tumors |
| EP2008656A1 (en) * | 2007-06-28 | 2008-12-31 | Bergen Teknologioverforing AS | Compositions for the treatment of hyperphenylalaninemia |
| AU2013216920B2 (en) * | 2012-02-07 | 2016-09-22 | Global Bio Therapeutics, Inc. | Compartmentalized method of nucleic acid delivery and compositions and uses thereof |
| CN114395559A (zh) * | 2014-04-25 | 2022-04-26 | 吉尼松公司 | 高胆红素血症的治疗 |
| US11389546B2 (en) * | 2015-12-09 | 2022-07-19 | Modernatx, Inc. | Heterologous UTR sequences for enhanced mRNA expression |
| WO2018187231A2 (en) * | 2017-04-03 | 2018-10-11 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| EP3692141A1 (en) * | 2017-10-03 | 2020-08-12 | Children's Hospital and Clinics of Minnesota | Vectors and methods of use |
| WO2019217513A2 (en) * | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
-
2018
- 2018-10-02 BR BR112020006672-9A patent/BR112020006672A2/pt not_active Application Discontinuation
- 2018-10-02 JP JP2020518812A patent/JP2020535828A/ja active Pending
- 2018-10-02 WO PCT/US2018/053919 patent/WO2019070674A2/en not_active Ceased
- 2018-10-02 IL IL273670A patent/IL273670B2/en unknown
- 2018-10-02 EP EP18864047.8A patent/EP3692157A4/en active Pending
- 2018-10-02 AU AU2018345745A patent/AU2018345745A1/en not_active Abandoned
- 2018-10-02 KR KR1020207012276A patent/KR20200057766A/ko not_active Ceased
- 2018-10-02 US US16/652,867 patent/US20200318081A1/en active Pending
- 2018-10-02 CN CN201880077904.0A patent/CN111433368A/zh active Pending
- 2018-10-02 CA CA3077355A patent/CA3077355A1/en active Pending
-
2023
- 2023-06-21 JP JP2023101880A patent/JP2023115125A/ja active Pending
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