JPWO2020252136A5 - - Google Patents
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- Publication number
- JPWO2020252136A5 JPWO2020252136A5 JP2021573442A JP2021573442A JPWO2020252136A5 JP WO2020252136 A5 JPWO2020252136 A5 JP WO2020252136A5 JP 2021573442 A JP2021573442 A JP 2021573442A JP 2021573442 A JP2021573442 A JP 2021573442A JP WO2020252136 A5 JPWO2020252136 A5 JP WO2020252136A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- raav vector
- raav
- promoter
- plasma kallikrein
- 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.)
- Pending
Links
- 239000013608 rAAV vector Substances 0.000 claims description 107
- 102000001399 Kallikrein Human genes 0.000 claims description 48
- 108060005987 Kallikrein Proteins 0.000 claims description 48
- 239000013598 vector Substances 0.000 claims description 26
- 210000004185 liver Anatomy 0.000 claims description 24
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 14
- 108090000113 Plasma Kallikrein Proteins 0.000 claims description 12
- 230000035772 mutation Effects 0.000 claims description 12
- 206010019860 Hereditary angioedema Diseases 0.000 claims description 10
- 102000003827 Plasma Kallikrein Human genes 0.000 claims description 10
- 210000000234 capsid Anatomy 0.000 claims description 10
- 241000702421 Dependoparvovirus Species 0.000 claims description 8
- 101000772194 Homo sapiens Transthyretin Proteins 0.000 claims description 8
- 125000000174 L-prolyl group Chemical group [H]N1C([H])([H])C([H])([H])C([H])([H])[C@@]1([H])C(*)=O 0.000 claims description 8
- 208000035475 disorder Diseases 0.000 claims description 8
- 102000056556 human TTR Human genes 0.000 claims description 8
- 239000013607 AAV vector Substances 0.000 claims description 6
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 6
- 125000003275 alpha amino acid group Chemical class 0.000 claims description 6
- 230000007812 deficiency Effects 0.000 claims description 6
- 201000010099 disease Diseases 0.000 claims description 6
- 230000008482 dysregulation Effects 0.000 claims description 6
- 230000037361 pathway Effects 0.000 claims description 6
- 230000001105 regulatory effect Effects 0.000 claims description 6
- 108010088751 Albumins Proteins 0.000 claims description 4
- 102000009027 Albumins Human genes 0.000 claims description 4
- 206010048962 Brain oedema Diseases 0.000 claims description 4
- 102000004961 Furin Human genes 0.000 claims description 4
- 108090001126 Furin Proteins 0.000 claims description 4
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 4
- 208000005139 Hereditary Angioedema Types I and II Diseases 0.000 claims description 4
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 4
- 206010047115 Vasculitis Diseases 0.000 claims description 4
- 208000005707 acquired angioedema Diseases 0.000 claims description 4
- 208000006752 brain edema Diseases 0.000 claims description 4
- 238000011969 continuous reassessment method Methods 0.000 claims description 4
- 101150044687 crm gene Proteins 0.000 claims description 4
- 239000003623 enhancer Substances 0.000 claims description 4
- 229960000027 human factor ix Drugs 0.000 claims description 4
- 210000003657 middle cerebral artery Anatomy 0.000 claims description 4
- 230000028327 secretion Effects 0.000 claims description 4
- 238000013518 transcription Methods 0.000 claims description 4
- 230000035897 transcription Effects 0.000 claims description 4
- 239000002753 trypsin inhibitor Substances 0.000 claims description 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 2
- 208000028185 Angioedema Diseases 0.000 claims description 2
- 102000055157 Complement C1 Inhibitor Human genes 0.000 claims description 2
- 108700040183 Complement C1 Inhibitor Proteins 0.000 claims description 2
- 206010012688 Diabetic retinal oedema Diseases 0.000 claims description 2
- 201000005569 Gout Diseases 0.000 claims description 2
- 206010019196 Head injury Diseases 0.000 claims description 2
- 206010065390 Inflammatory pain Diseases 0.000 claims description 2
- 208000019695 Migraine disease Diseases 0.000 claims description 2
- 206010028980 Neoplasm Diseases 0.000 claims description 2
- 206010030113 Oedema Diseases 0.000 claims description 2
- 206010054048 Postoperative ileus Diseases 0.000 claims description 2
- 101800001494 Protease 2A Proteins 0.000 claims description 2
- 101800001066 Protein 2A Proteins 0.000 claims description 2
- 208000010378 Pulmonary Embolism Diseases 0.000 claims description 2
- 206010040047 Sepsis Diseases 0.000 claims description 2
- 208000020307 Spinal disease Diseases 0.000 claims description 2
- 208000006011 Stroke Diseases 0.000 claims description 2
- 102000040945 Transcription factor Human genes 0.000 claims description 2
- 108091023040 Transcription factor Proteins 0.000 claims description 2
- 206010047249 Venous thrombosis Diseases 0.000 claims description 2
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims description 2
- 208000027418 Wounds and injury Diseases 0.000 claims description 2
- 230000001154 acute effect Effects 0.000 claims description 2
- 208000007474 aortic aneurysm Diseases 0.000 claims description 2
- 210000001367 artery Anatomy 0.000 claims description 2
- 230000015271 coagulation Effects 0.000 claims description 2
- 238000005345 coagulation Methods 0.000 claims description 2
- 230000006378 damage Effects 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 201000011190 diabetic macular edema Diseases 0.000 claims description 2
- 239000012636 effector Substances 0.000 claims description 2
- 230000000694 effects Effects 0.000 claims description 2
- 230000001939 inductive effect Effects 0.000 claims description 2
- 208000014674 injury Diseases 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims description 2
- 230000000302 ischemic effect Effects 0.000 claims description 2
- 206010027599 migraine Diseases 0.000 claims description 2
- 238000012986 modification Methods 0.000 claims description 2
- 230000004048 modification Effects 0.000 claims description 2
- 201000008383 nephritis Diseases 0.000 claims description 2
- 208000004296 neuralgia Diseases 0.000 claims description 2
- 230000004770 neurodegeneration Effects 0.000 claims description 2
- 208000015122 neurodegenerative disease Diseases 0.000 claims description 2
- 208000021722 neuropathic pain Diseases 0.000 claims description 2
- 201000008482 osteoarthritis Diseases 0.000 claims description 2
- 230000001124 posttranscriptional effect Effects 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 230000002797 proteolythic effect Effects 0.000 claims description 2
- 208000037803 restenosis Diseases 0.000 claims description 2
- 206010039073 rheumatoid arthritis Diseases 0.000 claims description 2
- 230000003248 secreting effect Effects 0.000 claims description 2
- 208000005198 spinal stenosis Diseases 0.000 claims description 2
- 208000003265 stomatitis Diseases 0.000 claims description 2
- 238000007920 subcutaneous administration Methods 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 201000000596 systemic lupus erythematosus Diseases 0.000 claims description 2
- 238000011144 upstream manufacturing Methods 0.000 claims description 2
- 230000002861 ventricular Effects 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 claims description 2
- 108091036407 Polyadenylation Proteins 0.000 claims 1
- 238000000034 method Methods 0.000 description 13
- 206010011878 Deafness Diseases 0.000 description 1
- 108091034057 RNA (poly(A)) Proteins 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962860101P | 2019-06-11 | 2019-06-11 | |
| US62/860,101 | 2019-06-11 | ||
| PCT/US2020/037189 WO2020252136A1 (en) | 2019-06-11 | 2020-06-11 | Adeno associated viral vector delivery of antibodies for the treatment of disease mediated dysregulated plasma kallikrein |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022536692A JP2022536692A (ja) | 2022-08-18 |
| JPWO2020252136A5 true JPWO2020252136A5 (enExample) | 2023-06-20 |
Family
ID=71995023
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021573442A Pending JP2022536692A (ja) | 2019-06-11 | 2020-06-11 | 調節不全の血漿カリクレインによって媒介される疾患の治療のための抗体のアデノ随伴ウイルスベクター送達 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20230038502A1 (enExample) |
| EP (1) | EP3983449A1 (enExample) |
| JP (1) | JP2022536692A (enExample) |
| CN (1) | CN114207135A (enExample) |
| AR (1) | AR119271A1 (enExample) |
| TW (1) | TW202115126A (enExample) |
| WO (1) | WO2020252136A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2021120890A1 (en) * | 2019-12-20 | 2021-06-24 | Novartis Ag | Pyrazolyl derivatives useful as anti-cancer agents |
| WO2022060915A1 (en) * | 2020-09-15 | 2022-03-24 | Regenxbio Inc. | Vectorized lanadelumab and administration thereof |
| CA3094859A1 (en) * | 2020-10-01 | 2022-04-01 | Entos Pharmaceuticals Inc. | Proteolipid vesicles formulated with fusion associated small transmembrane proteins |
| WO2022156798A1 (zh) * | 2021-01-25 | 2022-07-28 | 成都康弘生物科技有限公司 | 一种抗体及其用途 |
| JP7675193B2 (ja) * | 2021-01-28 | 2025-05-12 | アステリア セラピューティクス，インコーポレイテッド | 血漿カリクレイン抗体及びその使用 |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6136597A (en) | 1997-09-18 | 2000-10-24 | The Salk Institute For Biological Studies | RNA export element |
| US7419829B2 (en) | 2000-10-06 | 2008-09-02 | Oxford Biomedica (Uk) Limited | Vector system |
| US7575924B2 (en) | 2000-11-13 | 2009-08-18 | Research Development Foundation | Methods and compositions relating to improved lentiviral vectors and their applications |
| AU2002343458B2 (en) | 2001-10-02 | 2008-03-20 | Institut Clayton De La Recherche | Methods and compositions relating to restricted expression lentiviral vectors and their applications |
| ATE557094T1 (de) | 2004-04-02 | 2012-05-15 | Univ Texas | Krebsspezifische promotoren |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| EP2678433B1 (en) | 2011-02-22 | 2017-05-03 | California Institute of Technology | Delivery of proteins using adeno-associated virus (aav) vectors |
| GB201206455D0 (en) | 2012-04-12 | 2012-05-30 | Royal Holloway & Bedford New College | Gene expression |
| CN104540848B (zh) * | 2012-08-08 | 2019-05-31 | 罗切格利卡特公司 | 白介素-10融合蛋白及其用途 |
| WO2014144756A1 (en) | 2013-03-15 | 2014-09-18 | Vlp Biotech, Inc. | Palivizumab epitope-based virus-like particles |
| CA2906624A1 (en) | 2013-03-15 | 2014-09-25 | Dyax Corp. | Anti-plasma kallikrein antibodies |
| KR20230109785A (ko) * | 2014-03-27 | 2023-07-20 | 다케다 파머수티컬 컴패니 리미티드 | 당뇨병성 황반 부종의 치료를 위한 조성물 및 방법 |
| US20160130324A1 (en) * | 2014-10-31 | 2016-05-12 | Shire Human Genetic Therapies, Inc. | C1 Inhibitor Fusion Proteins and Uses Thereof |
| CA2967468A1 (en) | 2014-12-16 | 2016-06-23 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
| CL2014003590A1 (es) | 2014-12-30 | 2015-07-10 | Univ Chile | Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimizacion y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje. |
| MA41346A (fr) | 2015-01-12 | 2017-11-21 | Juno Therapeutics Inc | Eléments régulateurs post-transcriptionnels d'hépatite modifiée |
| CA2979495A1 (en) * | 2015-03-17 | 2016-09-22 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
| US10179918B2 (en) | 2015-05-07 | 2019-01-15 | Sangamo Therapeutics, Inc. | Methods and compositions for increasing transgene activity |
| GB2547179A (en) | 2015-10-26 | 2017-08-16 | Quethera Ltd | Genetic construct |
| US10190135B2 (en) | 2015-10-27 | 2019-01-29 | Celltheon Corporation | Chimeric post-transcriptional regulatory element |
| JP2019510503A (ja) | 2016-04-07 | 2019-04-18 | ブルーバード バイオ， インコーポレイテッド | キメラ抗原受容体ｔ細胞組成物 |
| CA3025020A1 (en) | 2016-05-20 | 2017-11-23 | President And Fellows Of Harvard College | Gene therapy methods for age-related diseases and conditions |
| ES3024357T3 (en) | 2017-02-17 | 2025-06-04 | Purdue Research Foundation | Targeted ligand-payload based drug delivery for cell therapy |
| AR112057A1 (es) * | 2017-05-22 | 2019-09-18 | Baxalta Inc | Vectores virales que codifican variantes de fix recombinantes con mayor expresión para la terapia génica de hemofilia b |
| CA3079568A1 (en) * | 2017-10-18 | 2019-04-25 | Regenxbio Inc. | Fully-human post-translationally modified antibody therapeutics |
-
2020
- 2020-06-11 AR ARP200101656A patent/AR119271A1/es unknown
- 2020-06-11 US US17/618,193 patent/US20230038502A1/en not_active Abandoned
- 2020-06-11 TW TW109119759A patent/TW202115126A/zh unknown
- 2020-06-11 EP EP20753482.7A patent/EP3983449A1/en active Pending
- 2020-06-11 CN CN202080055596.9A patent/CN114207135A/zh active Pending
- 2020-06-11 WO PCT/US2020/037189 patent/WO2020252136A1/en not_active Ceased
- 2020-06-11 JP JP2021573442A patent/JP2022536692A/ja active Pending
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