JPWO2020154535A5 - - Google Patents
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- Publication number
- JPWO2020154535A5 JPWO2020154535A5 JP2021543230A JP2021543230A JPWO2020154535A5 JP WO2020154535 A5 JPWO2020154535 A5 JP WO2020154535A5 JP 2021543230 A JP2021543230 A JP 2021543230A JP 2021543230 A JP2021543230 A JP 2021543230A JP WO2020154535 A5 JPWO2020154535 A5 JP WO2020154535A5
- Authority
- JP
- Japan
- Prior art keywords
- acid sequence
- nucleic acid
- raav
- mammal
- therapeutic agent
- 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
- 239000002245 particle Substances 0.000 claims 30
- 150000007523 nucleic acids Chemical group 0.000 claims 19
- 241000124008 Mammalia Species 0.000 claims 17
- 239000003814 drug Substances 0.000 claims 16
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 15
- 210000004027 cell Anatomy 0.000 claims 12
- 229940124597 therapeutic agent Drugs 0.000 claims 12
- 125000003275 alpha amino acid group Chemical group 0.000 claims 7
- 210000000608 photoreceptor cell Anatomy 0.000 claims 7
- 238000004519 manufacturing process Methods 0.000 claims 6
- 108091033319 polynucleotide Proteins 0.000 claims 6
- 102000040430 polynucleotide Human genes 0.000 claims 6
- 239000002157 polynucleotide Substances 0.000 claims 6
- 108090000565 Capsid Proteins Proteins 0.000 claims 5
- 102100023321 Ceruloplasmin Human genes 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 208000017442 Retinal disease Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 208000014674 injury Diseases 0.000 claims 4
- 108020004707 nucleic acids Proteins 0.000 claims 4
- 102000039446 nucleic acids Human genes 0.000 claims 4
- 108091008695 photoreceptors Proteins 0.000 claims 4
- 101710197658 Capsid protein VP1 Proteins 0.000 claims 3
- 208000036443 AIPL1-related retinopathy Diseases 0.000 claims 2
- 201000001320 Atherosclerosis Diseases 0.000 claims 2
- 101000805768 Banna virus (strain Indonesia/JKT-6423/1980) mRNA (guanine-N(7))-methyltransferase Proteins 0.000 claims 2
- 101710132601 Capsid protein Proteins 0.000 claims 2
- 101000686790 Chaetoceros protobacilladnavirus 2 Replication-associated protein Proteins 0.000 claims 2
- 101000864475 Chlamydia phage 1 Internal scaffolding protein VP3 Proteins 0.000 claims 2
- 101000803553 Eumenes pomiformis Venom peptide 3 Proteins 0.000 claims 2
- 101000583961 Halorubrum pleomorphic virus 1 Matrix protein Proteins 0.000 claims 2
- 101000899806 Homo sapiens Retinal guanylyl cyclase 1 Proteins 0.000 claims 2
- 208000032578 Inherited retinal disease Diseases 0.000 claims 2
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 2
- 208000035719 Maculopathy Diseases 0.000 claims 2
- 101710081079 Minor spike protein H Proteins 0.000 claims 2
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 2
- 208000034461 Progressive cone dystrophy Diseases 0.000 claims 2
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 claims 2
- 208000032430 Retinal dystrophy Diseases 0.000 claims 2
- 102100022663 Retinal guanylyl cyclase 1 Human genes 0.000 claims 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 2
- 101710108545 Viral protein 1 Proteins 0.000 claims 2
- 208000027418 Wounds and injury Diseases 0.000 claims 2
- 210000000234 capsid Anatomy 0.000 claims 2
- 201000008615 cone dystrophy Diseases 0.000 claims 2
- 201000006754 cone-rod dystrophy Diseases 0.000 claims 2
- 230000006378 damage Effects 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000035475 disorder Diseases 0.000 claims 2
- 230000004064 dysfunction Effects 0.000 claims 2
- 201000006321 fundus dystrophy Diseases 0.000 claims 2
- 208000017532 inherited retinal dystrophy Diseases 0.000 claims 2
- 208000002780 macular degeneration Diseases 0.000 claims 2
- 230000001404 mediated effect Effects 0.000 claims 2
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- 239000013608 rAAV vector Substances 0.000 claims 2
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 claims 2
- 238000006467 substitution reaction Methods 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- 230000002463 transducing effect Effects 0.000 claims 2
- 230000008733 trauma Effects 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 201000001321 Bardet-Biedl syndrome Diseases 0.000 claims 1
- 208000009278 Blue cone monochromatism Diseases 0.000 claims 1
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 208000016615 Central areolar choroidal dystrophy Diseases 0.000 claims 1
- 208000033810 Choroidal dystrophy Diseases 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- 208000006992 Color Vision Defects Diseases 0.000 claims 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
- 208000024412 Friedreich ataxia Diseases 0.000 claims 1
- 102000004437 G-Protein-Coupled Receptor Kinase 1 Human genes 0.000 claims 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 241000238631 Hexapoda Species 0.000 claims 1
- 101000611338 Homo sapiens Rhodopsin Proteins 0.000 claims 1
- 206010056715 Laurence-Moon-Bardet-Biedl syndrome Diseases 0.000 claims 1
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 1
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 claims 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 208000034247 Pattern dystrophy Diseases 0.000 claims 1
- 108091093037 Peptide nucleic acid Proteins 0.000 claims 1
- 108091030071 RNAI Proteins 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 208000027073 Stargardt disease Diseases 0.000 claims 1
- 208000014769 Usher Syndromes Diseases 0.000 claims 1
- 201000001408 X-linked juvenile retinoschisis 1 Diseases 0.000 claims 1
- 208000017441 X-linked retinoschisis Diseases 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- -1 antibody Proteins 0.000 claims 1
- 239000000427 antigen Substances 0.000 claims 1
- 102000036639 antigens Human genes 0.000 claims 1
- 108091007433 antigens Proteins 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 201000007728 blue cone monochromacy Diseases 0.000 claims 1
- 239000000872 buffer Substances 0.000 claims 1
- 201000007254 color blindness Diseases 0.000 claims 1
- 239000000032 diagnostic agent Substances 0.000 claims 1
- 229940039227 diagnostic agent Drugs 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000013600 plasmid vector Substances 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 239000000790 retinal pigment Substances 0.000 claims 1
- 201000007714 retinoschisis Diseases 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 1
- 238000003892 spreading Methods 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962795695P | 2019-01-23 | 2019-01-23 | |
| US62/795,695 | 2019-01-23 | ||
| PCT/US2020/014838 WO2020154535A1 (en) | 2019-01-23 | 2020-01-23 | Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022523050A JP2022523050A (ja) | 2022-04-21 |
| JP2022523050A5 JP2022523050A5 (https=) | 2023-02-06 |
| JPWO2020154535A5 true JPWO2020154535A5 (https=) | 2023-02-06 |
| JP7624727B2 JP7624727B2 (ja) | 2025-01-31 |
Family
ID=71737005
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021543230A Active JP7624727B2 (ja) | 2019-01-23 | 2020-01-23 | 合理的設計により増強された新規なａａｖウイルスによる網膜における高効率の形質導入および側方への広がり |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20220133909A1 (https=) |
| EP (1) | EP3914229A4 (https=) |
| JP (1) | JP7624727B2 (https=) |
| CN (1) | CN113347962B (https=) |
| AU (1) | AU2020212026B2 (https=) |
| CA (1) | CA3125294A1 (https=) |
| WO (1) | WO2020154535A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024223696A1 (en) * | 2023-04-24 | 2024-10-31 | Universiteit Gent | Compounds to treat inherited retinal disease |
| CN121046460A (zh) * | 2024-05-31 | 2025-12-02 | 上海朗昇生物科技有限公司 | 用于治疗Prom1相关视网膜疾病的新型治疗药物 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2287495A1 (en) * | 1997-04-21 | 1998-10-29 | University Of Florida | Materials and methods for treatment of retinal diseases |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| WO2006110689A2 (en) * | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| HRP20190144T1 (hr) * | 2010-04-23 | 2019-03-22 | University Of Florida Research Foundation, Inc. | Pripravci raav-gvanilat ciklaze i postupci za liječenje leberove urođene amauroze-1 (lca1) |
| ES2768763T3 (es) * | 2014-03-04 | 2020-06-23 | Univ Florida | Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| WO2016149664A1 (en) | 2015-03-18 | 2016-09-22 | University Of Florida Research Foundation, Inc. | Methods and compositions for restoration of cone function in bcm |
| JP6805174B2 (ja) * | 2015-05-12 | 2020-12-23 | アメリカ合衆国 | 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質 |
| US20170348387A1 (en) * | 2016-02-29 | 2017-12-07 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for nphp5 lca-ciliopathy |
| PE20190401A1 (es) * | 2016-07-26 | 2019-03-13 | Biomarin Pharm Inc | Novedosas proteinas de la capside del virus adenoasociado |
| AU2018224044B2 (en) | 2017-02-21 | 2024-01-25 | The Uab Research Foundation | Modified AAV capsid proteins and uses thereof |
-
2020
- 2020-01-23 US US17/425,292 patent/US20220133909A1/en active Pending
- 2020-01-23 JP JP2021543230A patent/JP7624727B2/ja active Active
- 2020-01-23 AU AU2020212026A patent/AU2020212026B2/en active Active
- 2020-01-23 CN CN202080010452.1A patent/CN113347962B/zh active Active
- 2020-01-23 EP EP20744697.2A patent/EP3914229A4/en active Pending
- 2020-01-23 WO PCT/US2020/014838 patent/WO2020154535A1/en not_active Ceased
- 2020-01-23 CA CA3125294A patent/CA3125294A1/en active Pending
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