JP2023076606A5 - - Google Patents
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- JP2023076606A5 JP2023076606A5 JP2023060936A JP2023060936A JP2023076606A5 JP 2023076606 A5 JP2023076606 A5 JP 2023076606A5 JP 2023060936 A JP2023060936 A JP 2023060936A JP 2023060936 A JP2023060936 A JP 2023060936A JP 2023076606 A5 JP2023076606 A5 JP 2023076606A5
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- Prior art keywords
- acid sequence
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- region
- aav
- 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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- 150000001413 amino acids Chemical group 0.000 claims 63
- 108090000565 Capsid Proteins Proteins 0.000 claims 21
- 102100023321 Ceruloplasmin Human genes 0.000 claims 21
- 239000002245 particle Substances 0.000 claims 18
- 108700019146 Transgenes Proteins 0.000 claims 17
- 108090000623 proteins and genes Proteins 0.000 claims 14
- 210000004027 cell Anatomy 0.000 claims 12
- 238000000034 method Methods 0.000 claims 11
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 10
- 241000700605 Viruses Species 0.000 claims 9
- 230000003612 virological effect Effects 0.000 claims 9
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 7
- 150000007523 nucleic acids Chemical group 0.000 claims 7
- 241000702421 Dependoparvovirus Species 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 5
- 208000035475 disorder Diseases 0.000 claims 5
- 102000004169 proteins and genes Human genes 0.000 claims 5
- 230000001105 regulatory effect Effects 0.000 claims 5
- 108010069013 Phenylalanine Hydroxylase Proteins 0.000 claims 4
- 102100038223 Phenylalanine-4-hydroxylase Human genes 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 230000001225 therapeutic effect Effects 0.000 claims 3
- 102000001039 Dystrophin Human genes 0.000 claims 2
- 108010069091 Dystrophin Proteins 0.000 claims 2
- 108010054218 Factor VIII Proteins 0.000 claims 2
- 102000001690 Factor VIII Human genes 0.000 claims 2
- 238000004113 cell culture Methods 0.000 claims 2
- 229960000301 factor viii Drugs 0.000 claims 2
- 210000000663 muscle cell Anatomy 0.000 claims 2
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 241000238631 Hexapoda Species 0.000 claims 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 1
- 201000011252 Phenylketonuria Diseases 0.000 claims 1
- 230000002159 abnormal effect Effects 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 210000003205 muscle Anatomy 0.000 claims 1
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662366838P | 2016-07-26 | 2016-07-26 | |
| US62/366,838 | 2016-07-26 | ||
| JP2019503445A JP6994018B2 (ja) | 2016-07-26 | 2017-07-25 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| JP2021165270A JP7395550B2 (ja) | 2016-07-26 | 2021-10-07 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021165270A Division JP7395550B2 (ja) | 2016-07-26 | 2021-10-07 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023076606A JP2023076606A (ja) | 2023-06-01 |
| JP2023076606A5 true JP2023076606A5 (https=) | 2023-07-07 |
Family
ID=59558468
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019503445A Expired - Fee Related JP6994018B2 (ja) | 2016-07-26 | 2017-07-25 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| JP2021165270A Active JP7395550B2 (ja) | 2016-07-26 | 2021-10-07 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| JP2021215368A Active JP7303287B2 (ja) | 2016-07-26 | 2021-12-29 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| JP2023060936A Pending JP2023076606A (ja) | 2016-07-26 | 2023-04-04 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
Family Applications Before (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019503445A Expired - Fee Related JP6994018B2 (ja) | 2016-07-26 | 2017-07-25 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| JP2021165270A Active JP7395550B2 (ja) | 2016-07-26 | 2021-10-07 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| JP2021215368A Active JP7303287B2 (ja) | 2016-07-26 | 2021-12-29 | 新規アデノ随伴ウイルスキャプシドタンパク質 |
Country Status (15)
| Country | Link |
|---|---|
| US (3) | US11584780B2 (https=) |
| EP (1) | EP3491008A2 (https=) |
| JP (4) | JP6994018B2 (https=) |
| KR (2) | KR102604510B1 (https=) |
| CN (1) | CN109715650B (https=) |
| AU (2) | AU2017301600B2 (https=) |
| BR (1) | BR112019001532A2 (https=) |
| CA (1) | CA3031026A1 (https=) |
| CL (6) | CL2019000167A1 (https=) |
| IL (2) | IL305149A (https=) |
| MX (1) | MX2019000962A (https=) |
| PE (1) | PE20190401A1 (https=) |
| PH (1) | PH12019500068A1 (https=) |
| SG (1) | SG11201811603WA (https=) |
| WO (1) | WO2018022608A2 (https=) |
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| MX2020008932A (es) * | 2018-02-27 | 2020-10-01 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
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| JP7807380B2 (ja) | 2020-02-21 | 2026-01-27 | アコーオス インコーポレイテッド | ヒト対象において非加齢関連聴力障害を処置するための組成物および方法 |
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| IL297391A (en) | 2020-04-20 | 2022-12-01 | Tenaya Therapeutics Inc | An adeno-associated virus with an engineered capsid |
| AU2021265103A1 (en) | 2020-04-29 | 2023-01-19 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
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| CN116209768A (zh) * | 2020-07-03 | 2023-06-02 | 吉尼松公司 | 用于通过高变区交换工程化新杂合aav衣壳的方法 |
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| PL4211231T3 (pl) * | 2020-09-10 | 2024-11-04 | Genethon | Kapsyd AAV modyfikowany peptydem |
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| IL302127A (en) | 2020-10-18 | 2023-06-01 | Univ Pennsylvania | An improved adeno-associated virus (AAV) vector and its uses |
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| CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| WO2023183623A1 (en) * | 2022-03-25 | 2023-09-28 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
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2017
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- 2017-07-25 WO PCT/US2017/043703 patent/WO2018022608A2/en not_active Ceased
- 2017-07-25 KR KR1020237039466A patent/KR20230161535A/ko not_active Abandoned
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- 2017-07-25 JP JP2019503445A patent/JP6994018B2/ja not_active Expired - Fee Related
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2019
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