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
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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 (enExample) | 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 (enExample) |
| EP (1) | EP3491008A2 (enExample) |
| JP (4) | JP6994018B2 (enExample) |
| KR (2) | KR20230161535A (enExample) |
| CN (1) | CN109715650B (enExample) |
| AU (2) | AU2017301600B2 (enExample) |
| BR (1) | BR112019001532A2 (enExample) |
| CA (1) | CA3031026A1 (enExample) |
| CL (6) | CL2019000167A1 (enExample) |
| IL (2) | IL263801B2 (enExample) |
| MX (1) | MX2019000962A (enExample) |
| PE (1) | PE20190401A1 (enExample) |
| PH (1) | PH12019500068A1 (enExample) |
| SG (1) | SG11201811603WA (enExample) |
| WO (1) | WO2018022608A2 (enExample) |
Families Citing this family (50)
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| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| EP3510161A4 (en) | 2016-08-23 | 2020-04-22 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING DEFICIENCY IN A HUMAN SUBJECT |
| 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. |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
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| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| WO2019193119A1 (en) | 2018-04-05 | 2019-10-10 | Genethon | HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM |
| US12054724B2 (en) | 2018-04-10 | 2024-08-06 | President And Fellows Of Harvard College | AAV vectors encoding clarin-1 or GJB2 and uses thereof |
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| CN112368391A (zh) * | 2018-05-11 | 2021-02-12 | 马萨诸塞眼科耳科诊所 | 腺相关病毒的肝脏特异性嗜性 |
| TW202005978A (zh) * | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
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| JP7590968B2 (ja) * | 2018-12-05 | 2024-11-27 | アビオナ・セラピュ―ティクス・インコーポレイテッド | 遺伝子送達のための組み換えアデノ随伴ウイルスベクター |
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| KR20220098210A (ko) | 2019-11-14 | 2022-07-11 | 바이오마린 파머수티컬 인크. | 간-특이적 유전자 요법 벡터를 이용한 유전성 혈관 부종의 치료 |
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| CA3169063A1 (en) * | 2020-02-25 | 2021-09-02 | Leszek Lisowski | Adeno-associated virus capsid polypeptides and vectors |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| TW202144575A (zh) | 2020-04-03 | 2021-12-01 | 美商拜奧馬林製藥公司 | 使用ａａｖ及治療調配物之苯酮尿症治療 |
| MX2022013477A (es) | 2020-04-29 | 2022-11-30 | Bristol Myers Squibb Co | Distrofinas miniaturizadas con dominios de fusion de espectrina y usos de las mismas. |
| WO2021236908A2 (en) | 2020-05-20 | 2021-11-25 | Biomarin Pharmaceutical Inc. | Use of regulatory proteins for the production of adeno-associated virus |
| CA3187635A1 (en) * | 2020-07-03 | 2022-01-06 | Genethon | Method for engineering novel hybrid aav capsids through hypervariable regions swapping |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| EP4211231B1 (en) * | 2020-09-10 | 2024-06-26 | Genethon | Peptide-modified aav capsid |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| TW202229315A (zh) * | 2020-10-18 | 2022-08-01 | 賓州大學委員會 | 改良的腺相關病毒(aav)載體及其用途 |
| WO2022094461A1 (en) | 2020-11-02 | 2022-05-05 | Biomarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
| WO2023034980A1 (en) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| US20240376495A1 (en) | 2021-09-03 | 2024-11-14 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| JP2024534897A (ja) | 2021-09-03 | 2024-09-26 | ビオマリン プハルマセウトイカル インコーポレイテッド | Ａａｖカプシド組成物及び送達方法 |
| JP2024533174A (ja) | 2021-09-03 | 2024-09-12 | ビオマリン プハルマセウトイカル インコーポレイテッド | Ａａｖカプシド組成物及び送達方法 |
| WO2023034990A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| EP4396202A1 (en) | 2021-09-03 | 2024-07-10 | BioMarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| EP4408481A2 (en) | 2021-10-01 | 2024-08-07 | BioMarin Pharmaceutical Inc. | Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations |
| 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 |
| EP4499154A1 (en) * | 2022-03-25 | 2025-02-05 | REGENXBIO Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
| WO2024064863A2 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors |
| UY40443A (es) | 2022-09-22 | 2024-02-15 | Biomarin Pharm Inc | Tratamiento de la miocardiopatía con vectores de genoterapia con aav |
| CN115925819B (zh) * | 2022-12-30 | 2023-10-13 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2025051970A2 (en) | 2023-09-06 | 2025-03-13 | Dinaqor Ag | Methods of treatment of hypertrophic cardiomyopathy with aav gene therapy vectors and therapeutic formulations |
| WO2025054465A1 (en) * | 2023-09-08 | 2025-03-13 | California Institute Of Technology | Compositions for transport of therapeutic cargos using miniprotein binders targeting ca-iv |
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