JP2017512466A5 - - Google Patents
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- JP2017512466A5 JP2017512466A5 JP2016556768A JP2016556768A JP2017512466A5 JP 2017512466 A5 JP2017512466 A5 JP 2017512466A5 JP 2016556768 A JP2016556768 A JP 2016556768A JP 2016556768 A JP2016556768 A JP 2016556768A JP 2017512466 A5 JP2017512466 A5 JP 2017512466A5
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- JP
- Japan
- Prior art keywords
- nucleic acid
- hotc
- sequence
- seq
- acid sequence
- Prior art date
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020054236A JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461950157P | 2014-03-09 | 2014-03-09 | |
| US61/950,157 | 2014-03-09 | ||
| PCT/US2015/019513 WO2015138348A1 (en) | 2014-03-09 | 2015-03-09 | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020054236A Division JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017512466A JP2017512466A (ja) | 2017-05-25 |
| JP2017512466A5 true JP2017512466A5 (https=) | 2018-03-29 |
| JP6822841B2 JP6822841B2 (ja) | 2021-01-27 |
Family
ID=52737408
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016556768A Active JP6822841B2 (ja) | 2014-03-09 | 2015-03-09 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
| JP2020054236A Active JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020054236A Active JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Country Status (22)
| Country | Link |
|---|---|
| US (6) | US10167454B2 (https=) |
| EP (2) | EP3116900B1 (https=) |
| JP (2) | JP6822841B2 (https=) |
| KR (1) | KR102390075B1 (https=) |
| AU (2) | AU2015229750A1 (https=) |
| BR (2) | BR122023023004A2 (https=) |
| CA (1) | CA2939950C (https=) |
| CL (2) | CL2016002235A1 (https=) |
| CY (1) | CY1123378T1 (https=) |
| DK (1) | DK3116900T3 (https=) |
| ES (1) | ES2821938T3 (https=) |
| HR (1) | HRP20201544T1 (https=) |
| HU (1) | HUE051311T2 (https=) |
| IL (2) | IL247329B (https=) |
| LT (1) | LT3116900T (https=) |
| MX (1) | MX377804B (https=) |
| PL (1) | PL3116900T3 (https=) |
| PT (1) | PT3116900T (https=) |
| RS (1) | RS60902B1 (https=) |
| SI (1) | SI3116900T1 (https=) |
| SM (1) | SMT202000531T1 (https=) |
| WO (2) | WO2015138348A1 (https=) |
Families Citing this family (43)
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| BR122020023215B1 (pt) | 2013-05-03 | 2022-11-22 | Selecta Biosciences, Inc | Composição e kit de nanocarreadores sintéticos tolerogênicos para reduzir ou prevenir anafilaxia em resposta a um antígeno não alergênico |
| AU2014296278C1 (en) | 2013-07-30 | 2023-02-02 | Genevant Sciences Gmbh | Block copolymers and their conjugates or complexes with oligonucleotides |
| SMT202000531T1 (it) | 2014-03-09 | 2020-11-10 | Univ Pennsylvania | Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc) |
| JP7242180B2 (ja) | 2014-09-07 | 2023-03-20 | セレクタ バイオサイエンシーズ インコーポレーテッド | 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物 |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| JP2018509387A (ja) | 2015-01-21 | 2018-04-05 | フェーズアールエックス インコーポレイテッド | 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム |
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| ES3036482T3 (en) | 2017-03-11 | 2025-09-19 | Cartesian Therapeutics Inc | Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant |
| JP7119009B2 (ja) | 2017-05-31 | 2022-08-16 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
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| NZ762100A (en) | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
| CA3079428A1 (en) * | 2017-11-22 | 2019-05-31 | Modernatx, Inc. | Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders |
| TW202016314A (zh) | 2018-05-09 | 2020-05-01 | 美商拜奧馬林製藥公司 | 治療苯丙酮尿症之方法 |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| AU2019271132B2 (en) * | 2018-05-15 | 2026-02-05 | Translate Bio, Inc. | Subcutaneous delivery of messenger RNA |
| KR20210019996A (ko) | 2018-05-15 | 2021-02-23 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 조성물 및 방법 |
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| EP3833766A1 (en) * | 2018-08-09 | 2021-06-16 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| AU2019394996B2 (en) * | 2018-12-06 | 2025-11-06 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| WO2020160508A1 (en) | 2019-01-31 | 2020-08-06 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of aav capsids |
| AU2020287627A1 (en) | 2019-06-04 | 2021-11-18 | Selecta Biosciences, Inc. | Formulations and doses of PEGylated uricase |
| US12351834B2 (en) | 2020-03-03 | 2025-07-08 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| AU2021230361A1 (en) * | 2020-03-04 | 2022-09-08 | Poseida Therapeutics, Inc. | Compositions and methods for the treatment of Metabolic Liver Disorders |
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| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2022224372A1 (ja) * | 2021-04-21 | 2022-10-27 | 学校法人自治医科大学 | オルニチントランスカルバミラーゼ欠損症の治療用アデノ随伴ウイルスビリオン |
| EP4392445A1 (en) * | 2021-08-23 | 2024-07-03 | Bioverativ Therapeutics Inc. | Closed-end dna production with inverted terminal repeat sequences |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2023140971A1 (en) * | 2022-01-21 | 2023-07-27 | The Trustees Of The University Ofpennsylvania | Methods for treatment of ornithine transcarbamylase (otc) deficiency |
| EP4602157A2 (en) * | 2022-10-11 | 2025-08-20 | Pacific Northwest Research Institute | Constructs relating to genetic disorders |
| WO2025229995A1 (ja) * | 2024-04-30 | 2025-11-06 | 学校法人自治医科大学 | 核酸導入用ベクター、核酸導入方法、ポリヌクレオチド、及びベクター |
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| SMT202000531T1 (it) | 2014-03-09 | 2020-11-10 | Univ Pennsylvania | Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc) |
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