JP2018522529A5 - - Google Patents
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- JP2018522529A5 JP2018522529A5 JP2017518931A JP2017518931A JP2018522529A5 JP 2018522529 A5 JP2018522529 A5 JP 2018522529A5 JP 2017518931 A JP2017518931 A JP 2017518931A JP 2017518931 A JP2017518931 A JP 2017518931A JP 2018522529 A5 JP2018522529 A5 JP 2018522529A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- recombinant aav
- aav vector
- sequence
- 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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- 150000007523 nucleic acids Chemical class 0.000 claims 46
- 239000013607 AAV vector Substances 0.000 claims 37
- 108020004707 nucleic acids Proteins 0.000 claims 28
- 102000039446 nucleic acids Human genes 0.000 claims 28
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 17
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 11
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 11
- 241000702421 Dependoparvovirus Species 0.000 claims 7
- 239000003623 enhancer Substances 0.000 claims 7
- 229960000027 human factor ix Drugs 0.000 claims 7
- 239000002773 nucleotide Substances 0.000 claims 7
- 125000003729 nucleotide group Chemical group 0.000 claims 7
- 102000004169 proteins and genes Human genes 0.000 claims 7
- 108090000623 proteins and genes Proteins 0.000 claims 7
- 210000000234 capsid Anatomy 0.000 claims 6
- 238000000034 method Methods 0.000 claims 5
- 101710095339 Apolipoprotein E Proteins 0.000 claims 4
- 102100029470 Apolipoprotein E Human genes 0.000 claims 4
- 238000004806 packaging method and process Methods 0.000 claims 4
- 102100022641 Coagulation factor IX Human genes 0.000 claims 3
- 108091092195 Intron Proteins 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
- 208000009429 hemophilia B Diseases 0.000 claims 3
- 239000013612 plasmid Substances 0.000 claims 3
- 108091033319 polynucleotide Proteins 0.000 claims 3
- 102000040430 polynucleotide Human genes 0.000 claims 3
- 239000002157 polynucleotide Substances 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 108010013935 factor IX-Padua Proteins 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 239000002245 particle Substances 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 238000013518 transcription Methods 0.000 claims 2
- 230000035897 transcription Effects 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
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 102000053602 DNA Human genes 0.000 claims 1
- 101000823116 Homo sapiens Alpha-1-antitrypsin Proteins 0.000 claims 1
- 208000026578 Severe hemophilia B Diseases 0.000 claims 1
- 108020004682 Single-Stranded DNA Proteins 0.000 claims 1
- 102000051631 human SERPINA1 Human genes 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021012811A 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 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 | ||
| PCT/US2016/039075 WO2016210170A1 (en) | 2015-06-23 | 2016-06-23 | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021012811A Division 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018522529A JP2018522529A (ja) | 2018-08-16 |
| JP2018522529A5 true JP2018522529A5 (OSRAM) | 2019-07-25 |
| JP6831779B2 JP6831779B2 (ja) | 2021-02-17 |
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 After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (25)
| Country | Link |
|---|---|
| US (6) | US10799566B2 (OSRAM) |
| EP (2) | EP4378487A3 (OSRAM) |
| JP (3) | JP6831779B2 (OSRAM) |
| KR (2) | KR102178322B1 (OSRAM) |
| CN (1) | CN108138159A (OSRAM) |
| AU (2) | AU2016282781A1 (OSRAM) |
| CA (1) | CA2990193A1 (OSRAM) |
| CO (1) | CO2018000134A2 (OSRAM) |
| DK (1) | DK3313991T3 (OSRAM) |
| ES (1) | ES2989774T3 (OSRAM) |
| FI (2) | FI3313991T3 (OSRAM) |
| FR (1) | FR25C1002I1 (OSRAM) |
| HR (1) | HRP20241180T1 (OSRAM) |
| HU (2) | HUE068603T2 (OSRAM) |
| IL (2) | IL256517B2 (OSRAM) |
| MX (2) | MX390848B (OSRAM) |
| NO (1) | NO2025003I1 (OSRAM) |
| PE (1) | PE20180675A1 (OSRAM) |
| PH (1) | PH12017502424A1 (OSRAM) |
| PL (1) | PL3313991T3 (OSRAM) |
| PT (1) | PT3313991T (OSRAM) |
| SA (1) | SA517390590B1 (OSRAM) |
| SG (1) | SG10202100131WA (OSRAM) |
| SI (1) | SI3313991T1 (OSRAM) |
| WO (1) | WO2016210170A1 (OSRAM) |
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| MX2019004487A (es) | 2016-10-20 | 2020-02-07 | Sangamo Therapeutics Inc | Metodos y composiciones para el tratamiento de la enfermedad de fabry. |
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| GB201213117D0 (en) * | 2012-07-24 | 2012-09-05 | Ucl Business Plc | Transgene expression |
| EP3513802B1 (en) | 2012-10-26 | 2023-11-29 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| US20140271550A1 (en) | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| US9700486B2 (en) | 2013-04-24 | 2017-07-11 | Corning Incorporated | Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients |
| EP3019620A4 (en) | 2013-07-12 | 2017-01-18 | The Children's Hospital of Philadelphia | Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies |
| IL297919A (en) * | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| NL2012981B1 (en) * | 2014-06-11 | 2017-01-17 | Heineken Supply Chain Bv | Beverage dispensing system, beverage container and pressurizing system for use in a beverage dispensing system or container. |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| EP3270944B1 (en) | 2015-03-17 | 2019-10-23 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
| IL256517B2 (en) | 2015-06-23 | 2024-06-01 | Childrens Hospital Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| US11191847B2 (en) | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
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