JP2020537544A5 - - Google Patents
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- JP2020537544A5 JP2020537544A5 JP2020540683A JP2020540683A JP2020537544A5 JP 2020537544 A5 JP2020537544 A5 JP 2020537544A5 JP 2020540683 A JP2020540683 A JP 2020540683A JP 2020540683 A JP2020540683 A JP 2020540683A JP 2020537544 A5 JP2020537544 A5 JP 2020537544A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- itr
- raav
- expression construct
- nucleic acid
- 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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- 150000007523 nucleic acids Chemical class 0.000 claims description 20
- 108020004707 nucleic acids Proteins 0.000 claims description 17
- 102000039446 nucleic acids Human genes 0.000 claims description 17
- 108090000623 proteins and genes Proteins 0.000 claims description 12
- 102000019204 Progranulins Human genes 0.000 claims description 8
- 108010012809 Progranulins Proteins 0.000 claims description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 8
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 26
- 239000013608 rAAV vector Substances 0.000 claims 16
- 241000702421 Dependoparvovirus Species 0.000 claims 13
- 239000013598 vector Substances 0.000 claims 12
- 239000002773 nucleotide Substances 0.000 claims 10
- 125000003729 nucleotide group Chemical group 0.000 claims 10
- 238000002347 injection Methods 0.000 claims 9
- 239000007924 injection Substances 0.000 claims 9
- 210000004027 cell Anatomy 0.000 claims 8
- 108700019146 Transgenes Proteins 0.000 claims 7
- 108090000565 Capsid Proteins Proteins 0.000 claims 6
- 102100023321 Ceruloplasmin Human genes 0.000 claims 6
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 6
- 210000003169 central nervous system Anatomy 0.000 claims 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 claims 3
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 3
- 108010006025 bovine growth hormone Proteins 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- 229940079593 drug Drugs 0.000 claims 3
- 239000003623 enhancer Substances 0.000 claims 3
- 239000013612 plasmid Substances 0.000 claims 3
- 230000001124 posttranscriptional effect Effects 0.000 claims 3
- 230000001105 regulatory effect Effects 0.000 claims 3
- 241000701447 unidentified baculovirus Species 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 238000007913 intrathecal administration Methods 0.000 claims 2
- 210000004962 mammalian cell Anatomy 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 230000002093 peripheral effect Effects 0.000 claims 2
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 239000001963 growth medium Substances 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 description 7
- 101710114165 Progranulin Proteins 0.000 description 4
- 230000002068 genetic effect Effects 0.000 description 3
- 102100037632 Progranulin Human genes 0.000 description 2
- 239000003795 chemical substances by application Substances 0.000 description 2
- 238000001415 gene therapy Methods 0.000 description 2
- 108091026890 Coding region Proteins 0.000 description 1
- 206010061218 Inflammation Diseases 0.000 description 1
- 230000004663 cell proliferation Effects 0.000 description 1
- 210000000349 chromosome Anatomy 0.000 description 1
- 230000018109 developmental process Effects 0.000 description 1
- 230000004054 inflammatory process Effects 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 230000007111 proteostasis Effects 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022007736A JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2022007752A JP7389828B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762567296P | 2017-10-03 | 2017-10-03 | |
| US201762567301P | 2017-10-03 | 2017-10-03 | |
| US201762567319P | 2017-10-03 | 2017-10-03 | |
| US201762567310P | 2017-10-03 | 2017-10-03 | |
| US201762567311P | 2017-10-03 | 2017-10-03 | |
| US62/567,319 | 2017-10-03 | ||
| US62/567,310 | 2017-10-03 | ||
| US62/567,311 | 2017-10-03 | ||
| US62/567,301 | 2017-10-03 | ||
| US62/567,296 | 2017-10-03 | ||
| PCT/US2018/054227 WO2019070894A1 (en) | 2017-10-03 | 2018-10-03 | GENE THERAPIES FOR LYSOSOMIAL DISORDERS |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022007736A Division JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2022007752A Division JP7389828B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020537544A JP2020537544A (ja) | 2020-12-24 |
| JP2020537544A5 true JP2020537544A5 (cg-RX-API-DMAC7.html) | 2021-11-11 |
| JP7361037B2 JP7361037B2 (ja) | 2023-10-13 |
Family
ID=65994362
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020540683A Active JP7361037B2 (ja) | 2017-10-03 | 2018-10-03 | ライソゾーム病の遺伝子治療 |
| JP2022007752A Active JP7389828B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2022007736A Active JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022007752A Active JP7389828B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2022007736A Active JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (5) | US11993790B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3692158A4 (cg-RX-API-DMAC7.html) |
| JP (3) | JP7361037B2 (cg-RX-API-DMAC7.html) |
| KR (3) | KR102697811B1 (cg-RX-API-DMAC7.html) |
| CN (3) | CN112553210B (cg-RX-API-DMAC7.html) |
| AU (3) | AU2018346105C1 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112020006661A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3078464A1 (cg-RX-API-DMAC7.html) |
| IL (3) | IL273771B1 (cg-RX-API-DMAC7.html) |
| MX (3) | MX2020003965A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2019070894A1 (cg-RX-API-DMAC7.html) |
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| JOP20190248A1 (ar) | 2017-04-21 | 2019-10-20 | Amgen Inc | بروتينات ربط مولد ضد trem2 واستخداماته |
| TWI811229B (zh) | 2017-08-03 | 2023-08-11 | 美商阿列克特有限責任公司 | 抗trem2抗體及其使用方法 |
| JP7254815B2 (ja) | 2017-10-03 | 2023-04-10 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| KR102697811B1 (ko) | 2017-10-03 | 2024-08-22 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| HRP20240184T1 (hr) | 2019-02-04 | 2024-04-26 | Freeline Therapeutics Limited | Polinukleotidi |
| JP7637058B2 (ja) | 2019-02-22 | 2025-02-27 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
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| GB201913974D0 (en) * | 2019-09-27 | 2019-11-13 | King S College London | Vector |
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| US20230210941A1 (en) * | 2020-05-12 | 2023-07-06 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
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| WO2022035903A2 (en) | 2020-08-10 | 2022-02-17 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| IL300409A (en) * | 2020-08-10 | 2023-04-01 | Prevail Therapeutics Inc | Gene therapies for neurodegenerative disorders |
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| MX2023004419A (es) | 2020-10-15 | 2023-07-05 | Prevail Therapeutics Inc | Ensayo para medir la potencia de un producto farmacológico para genoterapia. |
| JP2024506860A (ja) * | 2021-02-01 | 2024-02-15 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ニーマンピック病ａ型を治療するための組成物及び方法 |
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| WO2023091949A2 (en) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| JP1727698S (ja) * | 2021-12-20 | 2022-10-19 | ゴルフボールケース | |
| CN120866357A (zh) * | 2022-04-19 | 2025-10-31 | 上海天泽云泰生物医药有限公司 | 用于治疗神经退行性障碍的重组aav载体 |
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| EP4599052A1 (en) * | 2022-10-08 | 2025-08-13 | Lingyi Biotech Co., Ltd. | Polynucleotides for the treatment of disease associated with gcase deficiency |
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| WO2025039622A1 (zh) * | 2023-08-23 | 2025-02-27 | 上海金珂博生物技术有限公司 | 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途 |
| CN118086341B (zh) * | 2024-04-25 | 2024-08-02 | 上海凌医生物科技有限公司 | 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框 |
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