JP2020520643A5 - - Google Patents
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- JP2020520643A5 JP2020520643A5 JP2019563548A JP2019563548A JP2020520643A5 JP 2020520643 A5 JP2020520643 A5 JP 2020520643A5 JP 2019563548 A JP2019563548 A JP 2019563548A JP 2019563548 A JP2019563548 A JP 2019563548A JP 2020520643 A5 JP2020520643 A5 JP 2020520643A5
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- Japan
- Prior art keywords
- item
- expression cassette
- factor
- transgene
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- 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.)
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- 108090000623 proteins and genes Proteins 0.000 claims description 25
- 230000001225 therapeutic effect Effects 0.000 claims description 15
- 102100027591 Copper-transporting ATPase 2 Human genes 0.000 claims description 14
- 101000936280 Homo sapiens Copper-transporting ATPase 2 Proteins 0.000 claims description 14
- 230000001105 regulatory effect Effects 0.000 claims description 12
- 108010054218 Factor VIII Proteins 0.000 claims description 11
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- 229960000301 factor viii Drugs 0.000 claims description 10
- 239000012634 fragment Substances 0.000 claims description 10
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- 101000701363 Homo sapiens Phospholipid-transporting ATPase IC Proteins 0.000 claims description 7
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- 102100039032 Phosphatidylcholine translocator ABCB4 Human genes 0.000 claims description 7
- 102100030448 Phospholipid-transporting ATPase IC Human genes 0.000 claims description 7
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- 101000749877 Homo sapiens Contactin-associated protein-like 2 Proteins 0.000 claims description 6
- 101000945692 Homo sapiens Cyclin-dependent kinase-like 5 Proteins 0.000 claims description 6
- 101000723833 Homo sapiens Zinc finger E-box-binding homeobox 2 Proteins 0.000 claims description 6
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- 101710096438 DNA-binding protein Proteins 0.000 claims description 4
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 claims description 4
- 208000018839 Wilson disease Diseases 0.000 claims description 4
- 108010050904 Interferons Proteins 0.000 claims description 3
- 102000014150 Interferons Human genes 0.000 claims description 3
- 201000010099 disease Diseases 0.000 claims description 3
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- 210000004185 liver Anatomy 0.000 description 2
- 210000002569 neuron Anatomy 0.000 description 2
- 102100035028 Alpha-L-iduronidase Human genes 0.000 description 1
- 101001073206 Arabidopsis thaliana Peroxidase 30 Proteins 0.000 description 1
- 108010051696 Growth Hormone Proteins 0.000 description 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 description 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 description 1
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 description 1
- 108090001061 Insulin Proteins 0.000 description 1
- 102000004877 Insulin Human genes 0.000 description 1
- 108010013563 Lipoprotein Lipase Proteins 0.000 description 1
- 102000011965 Lipoprotein Receptors Human genes 0.000 description 1
- 108010061306 Lipoprotein Receptors Proteins 0.000 description 1
- 102100022119 Lipoprotein lipase Human genes 0.000 description 1
- 108060001084 Luciferase Proteins 0.000 description 1
- 239000005089 Luciferase Substances 0.000 description 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 description 1
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 description 1
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 1
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 1
- 102100038803 Somatotropin Human genes 0.000 description 1
- 108060008682 Tumor Necrosis Factor Proteins 0.000 description 1
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 description 1
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 description 1
- 210000004978 chinese hamster ovary cell Anatomy 0.000 description 1
- 210000002919 epithelial cell Anatomy 0.000 description 1
- 239000000122 growth hormone Substances 0.000 description 1
- 229940072221 immunoglobulins Drugs 0.000 description 1
- 229940125396 insulin Drugs 0.000 description 1
- 229940047124 interferons Drugs 0.000 description 1
- 230000000968 intestinal effect Effects 0.000 description 1
- 210000003292 kidney cell Anatomy 0.000 description 1
- 210000005229 liver cell Anatomy 0.000 description 1
- 208000019423 liver disease Diseases 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 description 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 description 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 description 1
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 description 1
- 210000000663 muscle cell Anatomy 0.000 description 1
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- 102000003390 tumor necrosis factor Human genes 0.000 description 1
- 230000003612 virological effect Effects 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022152422A JP2022173348A (ja) | 2017-05-19 | 2022-09-26 | 高活性制御エレメント |
| JP2023211093A JP2024015408A (ja) | 2017-05-19 | 2023-12-14 | 高活性制御エレメント |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762508968P | 2017-05-19 | 2017-05-19 | |
| US62/508,968 | 2017-05-19 | ||
| PCT/US2018/033515 WO2018213786A1 (en) | 2017-05-19 | 2018-05-18 | High activity regulatory elements |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022152422A Division JP2022173348A (ja) | 2017-05-19 | 2022-09-26 | 高活性制御エレメント |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020520643A JP2020520643A (ja) | 2020-07-16 |
| JP2020520643A5 true JP2020520643A5 (enExample) | 2021-07-26 |
| JP7315475B2 JP7315475B2 (ja) | 2023-07-26 |
Family
ID=64274721
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019563548A Active JP7315475B2 (ja) | 2017-05-19 | 2018-05-18 | 高活性制御エレメント |
| JP2022152422A Pending JP2022173348A (ja) | 2017-05-19 | 2022-09-26 | 高活性制御エレメント |
| JP2023211093A Pending JP2024015408A (ja) | 2017-05-19 | 2023-12-14 | 高活性制御エレメント |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022152422A Pending JP2022173348A (ja) | 2017-05-19 | 2022-09-26 | 高活性制御エレメント |
| JP2023211093A Pending JP2024015408A (ja) | 2017-05-19 | 2023-12-14 | 高活性制御エレメント |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US12104175B2 (enExample) |
| EP (1) | EP3624858A4 (enExample) |
| JP (3) | JP7315475B2 (enExample) |
| KR (2) | KR20240024326A (enExample) |
| CN (2) | CN110944674B (enExample) |
| AU (1) | AU2018269050A1 (enExample) |
| BR (1) | BR112019024222A2 (enExample) |
| CA (1) | CA3063464A1 (enExample) |
| MA (1) | MA49153A (enExample) |
| TW (1) | TWI811219B (enExample) |
| WO (1) | WO2018213786A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| CN110944674B (zh) | 2017-05-19 | 2024-07-19 | 编码治疗公司 | 高活性调控元件 |
| BR112020010883A2 (pt) | 2017-12-01 | 2020-11-10 | Encoded Therapeutics, Inc. | proteínas de ligação a dna engenheiradas |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| WO2020150832A1 (en) * | 2019-01-24 | 2020-07-30 | Valorisation-Hsj, Limited Partnership | Cell-specific transcriptional regulatory sequences and uses thereof |
| US20220168449A1 (en) * | 2019-04-12 | 2022-06-02 | Encoded Therapeutics, Inc. | Compositions and methods for administration of therapeutics |
| SG11202113048SA (en) | 2019-05-29 | 2021-12-30 | Encoded Therapeutics Inc | Compositions and methods for selective gene regulation |
| JP7759311B2 (ja) | 2019-07-19 | 2025-10-23 | フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー | リコンビナーゼ組成物及び使用方法 |
| CN113088519B (zh) * | 2019-12-23 | 2024-02-02 | 苏州华毅乐健生物科技有限公司 | 一种分离的核酸分子及其应用 |
| IL294781A (en) * | 2020-02-14 | 2022-09-01 | Ultragenyx Pharmaceutical Inc | Gene therapy for treating cdkl5 deficiency disorder |
| BR112022021762A2 (pt) * | 2020-04-27 | 2023-01-17 | Univ Pennsylvania | Composições úteis em tratamento de distúrbio de deficiência de cdkl5 (cdd) |
| AU2022325165A1 (en) * | 2021-08-05 | 2024-01-18 | Insmed Incorporated | Adeno-associated virus particles and methods of use thereof |
| WO2024079661A1 (en) * | 2022-10-11 | 2024-04-18 | Meiragtx Uk Ii Limited | Atp7b gene therapy |
| CN117925624B (zh) * | 2024-03-22 | 2024-05-31 | 上海凌医生物科技有限公司 | 一种金属响应调控元件 |
| CN117947035A (zh) * | 2024-03-25 | 2024-04-30 | 上海凌医生物科技有限公司 | 一种增强基因表达的启动子 |
Family Cites Families (13)
| 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 |
| US6436703B1 (en) * | 2000-03-31 | 2002-08-20 | Hyseq, Inc. | Nucleic acids and polypeptides |
| EP1783645A1 (en) * | 2005-11-08 | 2007-05-09 | Actigenics | Methods for the identification of microRNA and their applications in research and human health |
| US20090018031A1 (en) | 2006-12-07 | 2009-01-15 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways tools, and methods |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| US20130129668A1 (en) * | 2011-09-01 | 2013-05-23 | The Regents Of The University Of California | Diagnosis and treatment of arthritis using epigenetics |
| JP6373763B2 (ja) | 2012-02-17 | 2018-08-22 | 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ベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法 |
| EP2909324B1 (en) | 2012-10-25 | 2020-02-26 | Tocagen Inc. | Retroviral vector with mini-promoter cassette |
| US11147887B2 (en) * | 2014-12-17 | 2021-10-19 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson disease |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| CN110944674B (zh) | 2017-05-19 | 2024-07-19 | 编码治疗公司 | 高活性调控元件 |
| BR112020010883A2 (pt) * | 2017-12-01 | 2020-11-10 | Encoded Therapeutics, Inc. | proteínas de ligação a dna engenheiradas |
| AU2020229382A1 (en) * | 2019-02-28 | 2021-09-16 | Encoded Therapeutics, Inc. | Compositions and methods for treating laminopathies |
-
2018
- 2018-05-18 CN CN201880048355.4A patent/CN110944674B/zh active Active
- 2018-05-18 AU AU2018269050A patent/AU2018269050A1/en not_active Abandoned
- 2018-05-18 CN CN202411101805.5A patent/CN118956965A/zh active Pending
- 2018-05-18 JP JP2019563548A patent/JP7315475B2/ja active Active
- 2018-05-18 CA CA3063464A patent/CA3063464A1/en active Pending
- 2018-05-18 WO PCT/US2018/033515 patent/WO2018213786A1/en not_active Ceased
- 2018-05-18 KR KR1020247004550A patent/KR20240024326A/ko not_active Ceased
- 2018-05-18 KR KR1020197037368A patent/KR102636351B1/ko active Active
- 2018-05-18 EP EP18803184.3A patent/EP3624858A4/en not_active Withdrawn
- 2018-05-18 BR BR112019024222-8A patent/BR112019024222A2/pt not_active Application Discontinuation
- 2018-05-18 MA MA049153A patent/MA49153A/fr unknown
- 2018-05-18 TW TW107117128A patent/TWI811219B/zh active
-
2019
- 2019-11-18 US US16/687,426 patent/US12104175B2/en active Active
-
2022
- 2022-09-26 JP JP2022152422A patent/JP2022173348A/ja active Pending
-
2023
- 2023-12-14 JP JP2023211093A patent/JP2024015408A/ja active Pending
-
2024
- 2024-08-01 US US18/792,310 patent/US20240376441A1/en not_active Abandoned
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