JP2021087431A5 - - Google Patents
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- JP2021087431A5 JP2021087431A5 JP2021012811A JP2021012811A JP2021087431A5 JP 2021087431 A5 JP2021087431 A5 JP 2021087431A5 JP 2021012811 A JP2021012811 A JP 2021012811A JP 2021012811 A JP2021012811 A JP 2021012811A JP 2021087431 A5 JP2021087431 A5 JP 2021087431A5
- Authority
- JP
- Japan
- Prior art keywords
- raav vector
- seq
- raav
- vector according
- protein
- 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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- 239000013608 rAAV vector Substances 0.000 claims 37
- 102100022641 Coagulation factor IX Human genes 0.000 claims 16
- 108010076282 Factor IX Proteins 0.000 claims 15
- 238000000034 method Methods 0.000 claims 13
- 210000000234 capsid Anatomy 0.000 claims 9
- 239000003623 enhancer Substances 0.000 claims 8
- 239000002773 nucleotide Substances 0.000 claims 8
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 239000013598 vector Substances 0.000 claims 8
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 5
- 125000003275 alpha amino acid group Chemical group 0.000 claims 5
- 230000037396 body weight Effects 0.000 claims 4
- 229960004222 factor ix Drugs 0.000 claims 4
- 102000007592 Apolipoproteins Human genes 0.000 claims 3
- 108010071619 Apolipoproteins Proteins 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 101100172173 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) hcr-1 gene Proteins 0.000 claims 3
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 3
- 230000008488 polyadenylation Effects 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims 2
- 235000001014 amino acid Nutrition 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 239000004475 Arginine Substances 0.000 claims 1
- 206010008111 Cerebral haemorrhage Diseases 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 102000053602 DNA Human genes 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 208000026578 Severe hemophilia B Diseases 0.000 claims 1
- 108020004682 Single-Stranded DNA Proteins 0.000 claims 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 1
- 125000000637 arginyl group Chemical group N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 230000023597 hemostasis Effects 0.000 claims 1
- 229960000027 human factor ix Drugs 0.000 claims 1
- 210000005228 liver tissue Anatomy 0.000 claims 1
- 235000018977 lysine Nutrition 0.000 claims 1
- 125000003588 lysine group Chemical class [H]N([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 claims 1
- 150000007523 nucleic acids Chemical group 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 238000009256 replacement therapy Methods 0.000 claims 1
- 230000002269 spontaneous effect Effects 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023171763A JP2024009857A (ja) | 2015-06-23 | 2023-10-03 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562183599P | 2015-06-23 | 2015-06-23 | |
| US62/183,599 | 2015-06-23 | ||
| US201662315453P | 2016-03-30 | 2016-03-30 | |
| US62/315,453 | 2016-03-30 | ||
| US201662338315P | 2016-05-18 | 2016-05-18 | |
| US62/338,315 | 2016-05-18 | ||
| US201662348781P | 2016-06-10 | 2016-06-10 | |
| US62/348,781 | 2016-06-10 | ||
| US201662349572P | 2016-06-13 | 2016-06-13 | |
| US62/349,572 | 2016-06-13 | ||
| JP2017518931A JP6831779B2 (ja) | 2015-06-23 | 2016-06-23 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017518931A Division JP6831779B2 (ja) | 2015-06-23 | 2016-06-23 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023171763A Division JP2024009857A (ja) | 2015-06-23 | 2023-10-03 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021087431A JP2021087431A (ja) | 2021-06-10 |
| JP2021087431A5 true JP2021087431A5 (enExample) | 2021-11-04 |
| JP7361737B2 JP7361737B2 (ja) | 2023-10-16 |
Family
ID=57586246
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017518931A Active JP6831779B2 (ja) | 2015-06-23 | 2016-06-23 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
| JP2021012811A Active JP7361737B2 (ja) | 2015-06-23 | 2021-01-29 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
| JP2023171763A Pending JP2024009857A (ja) | 2015-06-23 | 2023-10-03 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017518931A Active JP6831779B2 (ja) | 2015-06-23 | 2016-06-23 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023171763A Pending JP2024009857A (ja) | 2015-06-23 | 2023-10-03 | 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
Country Status (25)
| Country | Link |
|---|---|
| US (6) | US10799566B2 (enExample) |
| EP (2) | EP4378487A3 (enExample) |
| JP (3) | JP6831779B2 (enExample) |
| KR (2) | KR102415896B1 (enExample) |
| CN (1) | CN108138159A (enExample) |
| AU (2) | AU2016282781A1 (enExample) |
| CA (1) | CA2990193A1 (enExample) |
| CO (1) | CO2018000134A2 (enExample) |
| DK (1) | DK3313991T3 (enExample) |
| ES (1) | ES2989774T3 (enExample) |
| FI (2) | FI3313991T3 (enExample) |
| FR (1) | FR25C1002I1 (enExample) |
| HR (1) | HRP20241180T1 (enExample) |
| HU (2) | HUE068603T2 (enExample) |
| IL (2) | IL256517B2 (enExample) |
| MX (2) | MX390848B (enExample) |
| NO (1) | NO2025003I1 (enExample) |
| PE (1) | PE20180675A1 (enExample) |
| PH (1) | PH12017502424A1 (enExample) |
| PL (1) | PL3313991T3 (enExample) |
| PT (1) | PT3313991T (enExample) |
| SA (1) | SA517390590B1 (enExample) |
| SG (1) | SG10202100131WA (enExample) |
| SI (1) | SI3313991T1 (enExample) |
| WO (1) | WO2016210170A1 (enExample) |
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| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
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| PT3313991T (pt) | 2015-06-23 | 2024-09-27 | Childrens Hospital Philadelphia | Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos |
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| EP4303225B1 (en) * | 2017-03-15 | 2025-11-26 | The University of North Carolina at Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
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