JP2017521049A5 - - Google Patents
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- Publication number
- JP2017521049A5 JP2017521049A5 JP2016567082A JP2016567082A JP2017521049A5 JP 2017521049 A5 JP2017521049 A5 JP 2017521049A5 JP 2016567082 A JP2016567082 A JP 2016567082A JP 2016567082 A JP2016567082 A JP 2016567082A JP 2017521049 A5 JP2017521049 A5 JP 2017521049A5
- Authority
- JP
- Japan
- Prior art keywords
- lentiviral vector
- promoter
- vector according
- transgene
- hcef
- 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.)
- Granted
Links
- 239000013598 vector Substances 0.000 claims 25
- 108700019146 Transgenes Proteins 0.000 claims 10
- 102000015395 alpha 1-Antitrypsin Human genes 0.000 claims 6
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims 6
- 229940024142 alpha 1-antitrypsin Drugs 0.000 claims 6
- 238000000034 method Methods 0.000 claims 6
- 241000701022 Cytomegalovirus Species 0.000 claims 5
- 201000003883 Cystic fibrosis Diseases 0.000 claims 4
- 101001086862 Homo sapiens Pulmonary surfactant-associated protein B Proteins 0.000 claims 4
- 102100032617 Pulmonary surfactant-associated protein B Human genes 0.000 claims 4
- 239000003814 drug Substances 0.000 claims 4
- 229940124597 therapeutic agent Drugs 0.000 claims 4
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 claims 3
- 241000713311 Simian immunodeficiency virus Species 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 208000025678 Ciliary Motility disease Diseases 0.000 claims 2
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 2
- 241000713730 Equine infectious anemia virus Species 0.000 claims 2
- 108010054218 Factor VIII Proteins 0.000 claims 2
- 102000001690 Factor VIII Human genes 0.000 claims 2
- 241000713800 Feline immunodeficiency virus Species 0.000 claims 2
- 102000004457 Granulocyte-Macrophage Colony-Stimulating Factor Human genes 0.000 claims 2
- 108010017213 Granulocyte-Macrophage Colony-Stimulating Factor Proteins 0.000 claims 2
- 101710133291 Hemagglutinin-neuraminidase Proteins 0.000 claims 2
- 208000031220 Hemophilia Diseases 0.000 claims 2
- 208000009292 Hemophilia A Diseases 0.000 claims 2
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 2
- 241000725303 Human immunodeficiency virus Species 0.000 claims 2
- 241000713666 Lentivirus Species 0.000 claims 2
- 101710163270 Nuclease Proteins 0.000 claims 2
- 102000004142 Trypsin Human genes 0.000 claims 2
- 108090000631 Trypsin Proteins 0.000 claims 2
- 208000006682 alpha 1-Antitrypsin Deficiency Diseases 0.000 claims 2
- 230000007812 deficiency Effects 0.000 claims 2
- 239000003623 enhancer Substances 0.000 claims 2
- 229960000301 factor viii Drugs 0.000 claims 2
- 201000009266 primary ciliary dyskinesia Diseases 0.000 claims 2
- 238000000746 purification Methods 0.000 claims 2
- 230000000241 respiratory effect Effects 0.000 claims 2
- 239000012588 trypsin Substances 0.000 claims 2
- 206010001881 Alveolar proteinosis Diseases 0.000 claims 1
- 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 claims 1
- 102100032300 Dynein axonemal heavy chain 11 Human genes 0.000 claims 1
- 102100031648 Dynein axonemal heavy chain 5 Human genes 0.000 claims 1
- 102100033595 Dynein axonemal intermediate chain 1 Human genes 0.000 claims 1
- 102100033596 Dynein axonemal intermediate chain 2 Human genes 0.000 claims 1
- 101001016208 Homo sapiens Dynein axonemal heavy chain 11 Proteins 0.000 claims 1
- 101000866368 Homo sapiens Dynein axonemal heavy chain 5 Proteins 0.000 claims 1
- 101000872267 Homo sapiens Dynein axonemal intermediate chain 1 Proteins 0.000 claims 1
- 101000872272 Homo sapiens Dynein axonemal intermediate chain 2 Proteins 0.000 claims 1
- 208000019693 Lung disease Diseases 0.000 claims 1
- 241000711408 Murine respirovirus Species 0.000 claims 1
- 102000002508 Peptide Elongation Factors Human genes 0.000 claims 1
- 108010068204 Peptide Elongation Factors Proteins 0.000 claims 1
- 241000713325 Visna/maedi virus Species 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 238000011210 chromatographic step Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000004927 fusion Effects 0.000 claims 1
- 102000008371 intracellularly ATP-gated chloride channel activity proteins Human genes 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 239000000725 suspension Substances 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
- 108010047303 von Willebrand Factor Proteins 0.000 claims 1
- 102100036537 von Willebrand factor Human genes 0.000 claims 1
- 229960001134 von willebrand factor Drugs 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB1409089.8A GB2526339A (en) | 2014-05-21 | 2014-05-21 | Lentiviral vectors |
| GB1409089.8 | 2014-05-21 | ||
| PCT/GB2015/051201 WO2015177501A1 (en) | 2014-05-21 | 2015-04-23 | Lentiviral vectors |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017521049A JP2017521049A (ja) | 2017-08-03 |
| JP2017521049A5 true JP2017521049A5 (enExample) | 2018-06-07 |
| JP6637444B2 JP6637444B2 (ja) | 2020-01-29 |
Family
ID=51135270
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016567082A Active JP6637444B2 (ja) | 2014-05-21 | 2015-04-23 | レンチウイルスベクター |
Country Status (21)
| Country | Link |
|---|---|
| US (1) | US10704061B2 (enExample) |
| EP (1) | EP3145949B1 (enExample) |
| JP (1) | JP6637444B2 (enExample) |
| AU (1) | AU2015263150B2 (enExample) |
| CA (1) | CA2947125C (enExample) |
| CY (1) | CY1123413T1 (enExample) |
| DK (1) | DK3145949T3 (enExample) |
| EA (1) | EA038966B1 (enExample) |
| ES (1) | ES2805045T3 (enExample) |
| GB (1) | GB2526339A (enExample) |
| HR (1) | HRP20200930T1 (enExample) |
| HU (1) | HUE051269T2 (enExample) |
| LT (1) | LT3145949T (enExample) |
| ME (1) | ME03789B (enExample) |
| PL (1) | PL3145949T3 (enExample) |
| PT (1) | PT3145949T (enExample) |
| RS (1) | RS60586B1 (enExample) |
| SI (1) | SI3145949T1 (enExample) |
| SM (1) | SMT202000360T1 (enExample) |
| UA (1) | UA122127C2 (enExample) |
| WO (1) | WO2015177501A1 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RS61387B2 (sr) | 2013-02-15 | 2024-06-28 | Bioverativ Therapeutics Inc | Gen optimizovanog faktora viii |
| BR102015012334A2 (pt) | 2015-05-27 | 2016-11-29 | Fundação Hemoct De Ribeirão Preto Fundherp | processo de produção do fator vii de coagulação sanguínea e fator vii de coagulação sanguínea |
| ES2796488T3 (es) | 2016-04-08 | 2020-11-27 | Krystal Biotech Inc | Composiciones para el uso en métodos para el tratamiento de heridas, trastornos y enfermedades de la piel |
| JP2018174874A (ja) * | 2017-04-19 | 2018-11-15 | 国立大学法人 東京医科歯科大学 | 上皮細胞の水分泌機能測定方法 |
| US12134790B2 (en) | 2017-09-27 | 2024-11-05 | Inozyme Pharma, Inc. | Methods of improving cardiovascular function and treating cardiovascular disease using a recombinant ectonucleotide pyrophosphatase phosphodiesterase (NPP1) |
| GB201814590D0 (en) * | 2018-09-07 | 2018-10-24 | Oxford Biomedica Ltd | Viral vector production system |
| EP3775236A1 (en) | 2018-04-12 | 2021-02-17 | Krystal Biotech, LLC | Compositions and methods for the treatment of autosomal recessive congenital ichthyosis |
| MX2020011257A (es) | 2018-04-27 | 2021-01-15 | Krystal Biotech Inc | Ácidos nucleicos recombinantes que codifican una o más proteínas cosméticas para usos estéticos. |
| WO2020009444A1 (ko) * | 2018-07-02 | 2020-01-09 | 서울대학교산학협력단 | 세포융합 기술을 이용한 유전자 및 세포 치료제 및 이의 용도 |
| CN112384247B (zh) * | 2018-07-02 | 2024-09-27 | 库拉米斯公司 | 利用细胞融合技术的基因及细胞治疗剂以及其的用途 |
| AU2019346549B2 (en) | 2018-09-24 | 2024-09-12 | Krystal Biotech, Inc. | Compositions and methods for the treatment of Netherton Syndrome |
| EP3856914A1 (en) | 2018-09-26 | 2021-08-04 | Krystal Biotech, Inc. | Compositions and methods for the treatment of skin diseases |
| CN113454105A (zh) | 2019-02-08 | 2021-09-28 | 克里斯托生物技术股份有限公司 | 用于递送cftr多肽的组合物和方法 |
| KR20210151785A (ko) * | 2019-03-13 | 2021-12-14 | 제너레이션 바이오 컴퍼니 | 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 |
| CN112575034B (zh) * | 2019-09-29 | 2023-04-25 | 济南赛尔生物科技股份有限公司 | 一种治疗a型血友病的产品及应用 |
| EP4077688A1 (en) * | 2019-12-20 | 2022-10-26 | Krystal Biotech, Inc. | Compositions and methods for gene delivery to the airways and/or lungs |
| EP4143326A1 (en) * | 2020-04-27 | 2023-03-08 | University of Iowa Research Foundation | Compositions and methods for the treatment of cystic fibrosis |
| US20230190871A1 (en) | 2020-05-20 | 2023-06-22 | Sana Biotechnology, Inc. | Methods and compositions for treatment of viral infections |
| EP4162036A1 (en) | 2020-06-09 | 2023-04-12 | Inozyme Pharma, Inc. | Soluble enpp1 or enpp3 proteins and uses thereof |
| JP2023537565A (ja) * | 2020-06-24 | 2023-09-04 | バイオベラティブ セラピューティクス インコーポレイテッド | タンパク質を発現するように改変されたレンチウイルスベクターの調製物から遊離第ｖｉｉｉ因子を除去する方法 |
| WO2022047316A1 (en) | 2020-08-28 | 2022-03-03 | Sana Biotechnology, Inc. | Modified anti-viral binding agents |
| CA3191743A1 (en) * | 2020-09-16 | 2022-03-24 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
| GB202100688D0 (en) * | 2021-01-19 | 2021-03-03 | Autolus Ltd | Process |
| GB202102832D0 (en) * | 2021-02-26 | 2021-04-14 | Ip2Ipo Innovations Ltd | Retroviral vectors |
| IL307350A (en) | 2021-04-02 | 2023-11-01 | Krystal Biotech Inc | Viral vectors for cancer treatment |
| GB202105278D0 (en) | 2021-04-13 | 2021-05-26 | Imperial College Innovations Ltd | Cell therapy |
| GB202105276D0 (en) * | 2021-04-13 | 2021-05-26 | Imperial College Innovations Ltd | Delivery of gene therapy vectors |
| GB202105277D0 (en) * | 2021-04-13 | 2021-05-26 | Imperial College Innovations Ltd | Signal peptides |
| EP4373951A1 (en) * | 2021-07-19 | 2024-05-29 | 2seventy bio, Inc. | Vector manufacturing processes |
| WO2023114949A1 (en) | 2021-12-16 | 2023-06-22 | Sana Biotechnology, Inc. | Methods and systems of particle production |
| US20250082777A1 (en) | 2022-01-10 | 2025-03-13 | Sana Biotechnology, Inc. | Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses |
| WO2023150647A1 (en) | 2022-02-02 | 2023-08-10 | Sana Biotechnology, Inc. | Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses |
| AU2023253826A1 (en) | 2022-04-11 | 2024-11-21 | Boehringer Ingelheim International Gmbh | Combination treatment |
| US20260027145A1 (en) | 2022-07-27 | 2026-01-29 | Sana Biotechnology, Inc. | Methods of transduction using a viral vector and inhibitors of antiviral restriction factors |
| WO2024044655A1 (en) | 2022-08-24 | 2024-02-29 | Sana Biotechnology, Inc. | Delivery of heterologous proteins |
| GB202212472D0 (en) * | 2022-08-26 | 2022-10-12 | Ip2Ipo Innovations Ltd | Retroviral vectors |
| WO2024057315A1 (en) | 2022-09-15 | 2024-03-21 | Yeda Research And Development Co. Ltd. | Compositions and methods for virotherapy |
| WO2024119157A1 (en) | 2022-12-02 | 2024-06-06 | Sana Biotechnology, Inc. | Lipid particles with cofusogens and methods of producing and using the same |
| WO2024220597A2 (en) | 2023-04-18 | 2024-10-24 | Sana Biotechnology, Inc. | Digital droplet based assay for detecting replication competent lentiviral vector |
| AU2024274792A1 (en) | 2023-05-23 | 2025-10-23 | Sana Biotechnology, Inc. | Tandem fusogens and related lipid particles |
| WO2025184529A1 (en) | 2024-03-01 | 2025-09-04 | Sana Biotechnology, Inc. | Viral particles with fusogen display and related compositions and methods |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2221600A (en) * | 1998-12-31 | 2000-07-31 | Chiron Corporation | Improved expression of hiv polypeptides and production of virus-like particles |
| CA2413995C (en) * | 2000-06-01 | 2013-08-13 | Dnavec Research Inc. | Pseudotype retroviral vectors containing membrane proteins having hemagglutinin activity |
| US20050048030A1 (en) * | 2001-09-28 | 2005-03-03 | Raymond Pickles | Paramyxoviruses as gene transfer vectors to lung cells |
| CA2462628C (en) * | 2001-10-02 | 2019-08-20 | Institut Clayton De La Recherche | Restricted expression lentiviral vectors |
| EP1950307B8 (en) * | 2005-10-28 | 2016-02-24 | IP Pharma Co., Ltd. | Gene transfer into airway epithelial stem cell by using lentiviral vector pseudotyped with rna virus spike protein |
| GB0606190D0 (en) * | 2006-03-28 | 2006-05-10 | Isis Innovation | Construct |
| EP1950303A1 (de) | 2007-01-26 | 2008-07-30 | Bayer CropScience AG | Genetisch modifizierte Pflanzen, die eine Stärke mit geringem Amylosegehalt und erhöhtem Quellvermögen synthetisieren |
| DK2623605T3 (en) * | 2007-04-09 | 2019-03-25 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
-
2014
- 2014-05-21 GB GB1409089.8A patent/GB2526339A/en not_active Withdrawn
-
2015
- 2015-04-23 JP JP2016567082A patent/JP6637444B2/ja active Active
- 2015-04-23 HR HRP20200930TT patent/HRP20200930T1/hr unknown
- 2015-04-23 HU HUE15720762A patent/HUE051269T2/hu unknown
- 2015-04-23 PL PL15720762T patent/PL3145949T3/pl unknown
- 2015-04-23 SI SI201531275T patent/SI3145949T1/sl unknown
- 2015-04-23 US US15/312,026 patent/US10704061B2/en active Active
- 2015-04-23 WO PCT/GB2015/051201 patent/WO2015177501A1/en not_active Ceased
- 2015-04-23 AU AU2015263150A patent/AU2015263150B2/en active Active
- 2015-04-23 ES ES15720762T patent/ES2805045T3/es active Active
- 2015-04-23 RS RS20200724A patent/RS60586B1/sr unknown
- 2015-04-23 CA CA2947125A patent/CA2947125C/en active Active
- 2015-04-23 SM SM20200360T patent/SMT202000360T1/it unknown
- 2015-04-23 PT PT157207622T patent/PT3145949T/pt unknown
- 2015-04-23 UA UAA201612986A patent/UA122127C2/uk unknown
- 2015-04-23 DK DK15720762.2T patent/DK3145949T3/da active
- 2015-04-23 EP EP15720762.2A patent/EP3145949B1/en active Active
- 2015-04-23 EA EA201692361A patent/EA038966B1/ru unknown
- 2015-04-23 ME MEP-2020-114A patent/ME03789B/me unknown
- 2015-04-23 LT LTEP15720762.2T patent/LT3145949T/lt unknown
-
2020
- 2020-07-03 CY CY20201100617T patent/CY1123413T1/el unknown
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