JP2019521696A5 - - Google Patents
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- JP2019521696A5 JP2019521696A5 JP2019504750A JP2019504750A JP2019521696A5 JP 2019521696 A5 JP2019521696 A5 JP 2019521696A5 JP 2019504750 A JP2019504750 A JP 2019504750A JP 2019504750 A JP2019504750 A JP 2019504750A JP 2019521696 A5 JP2019521696 A5 JP 2019521696A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- amino acids
- aav
- capsid protein
- heterologous
- 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
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- 150000001413 amino acids Chemical class 0.000 claims 19
- 210000002845 virion Anatomy 0.000 claims 15
- 108090000565 Capsid Proteins Proteins 0.000 claims 13
- 102100023321 Ceruloplasmin Human genes 0.000 claims 13
- 108090000765 processed proteins & peptides Proteins 0.000 claims 13
- 210000004027 cell Anatomy 0.000 claims 7
- 102000004196 processed proteins & peptides Human genes 0.000 claims 7
- 229920001184 polypeptide Polymers 0.000 claims 5
- 230000002207 retinal effect Effects 0.000 claims 5
- 239000012634 fragment Substances 0.000 claims 4
- 108020004707 nucleic acids Proteins 0.000 claims 4
- 102000039446 nucleic acids Human genes 0.000 claims 4
- 150000007523 nucleic acids Chemical class 0.000 claims 4
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 238000003780 insertion Methods 0.000 claims 3
- 230000037431 insertion Effects 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical compound O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 208000006992 Color Vision Defects Diseases 0.000 claims 2
- 102100031780 Endonuclease Human genes 0.000 claims 2
- 108010042407 Endonucleases Proteins 0.000 claims 2
- 208000003098 Ganglion Cysts Diseases 0.000 claims 2
- 101001104102 Homo sapiens X-linked retinitis pigmentosa GTPase regulator Proteins 0.000 claims 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 2
- 102000004330 Rhodopsin Human genes 0.000 claims 2
- 108090000820 Rhodopsin Proteins 0.000 claims 2
- 208000005400 Synovial Cyst Diseases 0.000 claims 2
- 102100040092 X-linked retinitis pigmentosa GTPase regulator Human genes 0.000 claims 2
- 210000000234 capsid Anatomy 0.000 claims 2
- 201000007254 color blindness Diseases 0.000 claims 2
- 208000030533 eye disease Diseases 0.000 claims 2
- 230000004807 localization Effects 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 108091008695 photoreceptors Proteins 0.000 claims 2
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 229940088872 Apoptosis inhibitor Drugs 0.000 claims 1
- 108091023037 Aptamer Proteins 0.000 claims 1
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 claims 1
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims 1
- 108010005939 Ciliary Neurotrophic Factor Proteins 0.000 claims 1
- 102100031614 Ciliary neurotrophic factor Human genes 0.000 claims 1
- 206010012689 Diabetic retinopathy Diseases 0.000 claims 1
- 101800003838 Epidermal growth factor Proteins 0.000 claims 1
- 102400001368 Epidermal growth factor Human genes 0.000 claims 1
- 108090000379 Fibroblast growth factor 2 Proteins 0.000 claims 1
- 102000003974 Fibroblast growth factor 2 Human genes 0.000 claims 1
- 102000013446 GTP Phosphohydrolases Human genes 0.000 claims 1
- 108091006109 GTPases Proteins 0.000 claims 1
- 208000010412 Glaucoma Diseases 0.000 claims 1
- 102000034615 Glial cell line-derived neurotrophic factor Human genes 0.000 claims 1
- 108091010837 Glial cell line-derived neurotrophic factor Proteins 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 claims 1
- 102000003693 Hedgehog Proteins Human genes 0.000 claims 1
- 108090000031 Hedgehog Proteins Proteins 0.000 claims 1
- 101000729271 Homo sapiens Retinoid isomerohydrolase Proteins 0.000 claims 1
- 101000668058 Infectious salmon anemia virus (isolate Atlantic salmon/Norway/810/9/99) RNA-directed RNA polymerase catalytic subunit Proteins 0.000 claims 1
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 1
- 101000597433 Mus musculus Nucleoredoxin-like protein 1 Proteins 0.000 claims 1
- 108010025020 Nerve Growth Factor Proteins 0.000 claims 1
- 102000015336 Nerve Growth Factor Human genes 0.000 claims 1
- 108010015406 Neurturin Proteins 0.000 claims 1
- 102100021584 Neurturin Human genes 0.000 claims 1
- 102100040375 Peripherin-2 Human genes 0.000 claims 1
- 101710135995 Peripherin-2 Proteins 0.000 claims 1
- 102000004590 Peripherins Human genes 0.000 claims 1
- 108010003081 Peripherins Proteins 0.000 claims 1
- 102100031176 Retinoid isomerohydrolase Human genes 0.000 claims 1
- 101710111169 Retinoschisin Proteins 0.000 claims 1
- 102100039507 Retinoschisin Human genes 0.000 claims 1
- 230000001772 anti-angiogenic effect Effects 0.000 claims 1
- 230000002424 anti-apoptotic effect Effects 0.000 claims 1
- 239000000158 apoptosis inhibitor Substances 0.000 claims 1
- 229940077737 brain-derived neurotrophic factor Drugs 0.000 claims 1
- 229940116977 epidermal growth factor Drugs 0.000 claims 1
- 230000001747 exhibiting effect Effects 0.000 claims 1
- 230000002452 interceptive effect Effects 0.000 claims 1
- 208000002780 macular degeneration Diseases 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 229940053128 nerve growth factor Drugs 0.000 claims 1
- 230000000324 neuroprotective effect Effects 0.000 claims 1
- 210000005047 peripherin Anatomy 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 201000007714 retinoschisis Diseases 0.000 claims 1
- VBEQCZHXXJYVRD-GACYYNSASA-N uroanthelone Chemical compound C([C@@H](C(=O)N[C@H](C(=O)N[C@@H](CS)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CS)C(=O)N[C@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)NCC(=O)N[C@@H](CC=1C=CC(O)=CC=1)C(=O)N[C@@H](CO)C(=O)NCC(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CS)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(O)=O)C(C)C)[C@@H](C)O)NC(=O)[C@H](CO)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CO)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@@H](NC(=O)[C@H](CC=1NC=NC=1)NC(=O)[C@H](CCSC)NC(=O)[C@H](CS)NC(=O)[C@@H](NC(=O)CNC(=O)CNC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CS)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)CNC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CO)NC(=O)[C@H](CO)NC(=O)[C@H]1N(CCC1)C(=O)[C@H](CS)NC(=O)CNC(=O)[C@H]1N(CCC1)C(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CO)NC(=O)[C@@H](N)CC(N)=O)C(C)C)[C@@H](C)CC)C1=CC=C(O)C=C1 VBEQCZHXXJYVRD-GACYYNSASA-N 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022076635A JP7423682B2 (ja) | 2016-07-29 | 2022-05-06 | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
| JP2024005406A JP7706582B2 (ja) | 2016-07-29 | 2024-01-17 | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662368929P | 2016-07-29 | 2016-07-29 | |
| US62/368,929 | 2016-07-29 | ||
| PCT/US2017/044206 WO2018022905A2 (en) | 2016-07-29 | 2017-07-27 | Adeno-associated virus virions with variant capsid and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022076635A Division JP7423682B2 (ja) | 2016-07-29 | 2022-05-06 | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019521696A JP2019521696A (ja) | 2019-08-08 |
| JP2019521696A5 true JP2019521696A5 (enExample) | 2020-08-27 |
| JP7071332B2 JP7071332B2 (ja) | 2022-05-18 |
Family
ID=61016812
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019504750A Active JP7071332B2 (ja) | 2016-07-29 | 2017-07-27 | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
| JP2022076635A Active JP7423682B2 (ja) | 2016-07-29 | 2022-05-06 | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
| JP2024005406A Active JP7706582B2 (ja) | 2016-07-29 | 2024-01-17 | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022076635A Active JP7423682B2 (ja) | 2016-07-29 | 2022-05-06 | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
| JP2024005406A Active JP7706582B2 (ja) | 2016-07-29 | 2024-01-17 | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (5) | US11554180B2 (enExample) |
| EP (2) | EP3490531A4 (enExample) |
| JP (3) | JP7071332B2 (enExample) |
| KR (3) | KR20230039779A (enExample) |
| CN (3) | CN116286986A (enExample) |
| AU (3) | AU2017302013B2 (enExample) |
| CA (1) | CA3029833A1 (enExample) |
| MX (3) | MX2019001276A (enExample) |
| WO (1) | WO2018022905A2 (enExample) |
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| CN109476707B (zh) | 2016-05-13 | 2022-12-02 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和其使用方法 |
| MA44546B1 (fr) * | 2016-06-15 | 2021-03-31 | Univ California | Virus adéno-associés variants et procédés d'utilisation |
| JP7071332B2 (ja) | 2016-07-29 | 2022-05-18 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
| RU2019109021A (ru) | 2016-08-29 | 2020-09-29 | Уэйн Стейт Юниверсити | Идентификация мутаций в вариантах каналопсина, имеющих улучшенную чувствительность к свету, и способы их применения |
| US11192925B2 (en) | 2016-10-19 | 2021-12-07 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| US20200121746A1 (en) | 2017-06-30 | 2020-04-23 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
| FI3684423T3 (fi) * | 2017-09-20 | 2023-06-15 | 4D Molecular Therapeutics Inc | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä |
| CA3095660A1 (en) * | 2018-02-15 | 2019-08-22 | Tomas Bjorklund | Modified viral capsids |
| KR20210084459A (ko) | 2018-09-26 | 2021-07-07 | 캘리포니아 인스티튜트 오브 테크놀로지 | 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물 |
| CN112955557A (zh) | 2018-10-01 | 2021-06-11 | 奥特吉尼克斯制药公司 | 用于治疗丙酸血症的基因疗法 |
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| TW202102526A (zh) * | 2019-04-04 | 2021-01-16 | 美商銳進科斯生物股份有限公司 | 重組腺相關病毒及其用途 |
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| CN113518824B (zh) * | 2019-10-16 | 2024-02-23 | 上海药明康德新药开发有限公司 | 新的aav变体 |
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| US12031147B2 (en) | 2020-05-29 | 2024-07-09 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
| JP2023529503A (ja) * | 2020-06-13 | 2023-07-10 | オクロジェネクス インコーポレイテッド | 網膜症のためのａａｖ媒介性遺伝子導入 |
| WO2022011390A1 (en) | 2020-07-08 | 2022-01-13 | Baylor College Of Medicine | Gene therapy for stxbp1 encephalopathy |
| CN113952472A (zh) * | 2020-07-21 | 2022-01-21 | 英斯培瑞有限公司 | 用于治疗眼部疾病的组合物和方法 |
| JP2023538129A (ja) * | 2020-08-21 | 2023-09-06 | カプシダ， インコーポレイテッド | 好ましい発現レベルを有するアデノ随伴ウイルス組成物 |
| CN112194706B (zh) * | 2020-09-30 | 2022-03-08 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
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| KR20240113624A (ko) | 2021-11-02 | 2024-07-22 | 보이저 테라퓨틱스, 인크. | Aav 캡시드 변이체 및 이의 용도 |
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