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 |
|---|---|---|---|
| JP2022007752A JP7389828B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2022007736A JP7336730B2 (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 |
|---|---|---|---|
| US201762567310P | 2017-10-03 | 2017-10-03 | |
| US201762567319P | 2017-10-03 | 2017-10-03 | |
| US201762567296P | 2017-10-03 | 2017-10-03 | |
| US201762567301P | 2017-10-03 | 2017-10-03 | |
| US201762567311P | 2017-10-03 | 2017-10-03 | |
| US62/567,311 | 2017-10-03 | ||
| US62/567,296 | 2017-10-03 | ||
| US62/567,310 | 2017-10-03 | ||
| US62/567,319 | 2017-10-03 | ||
| US62/567,301 | 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 (enExample) | 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 |
| JP2022007736A Active JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | 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 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022007736A Active JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | 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 |
Country Status (11)
| Country | Link |
|---|---|
| US (6) | US11993790B2 (enExample) |
| EP (1) | EP3692158A4 (enExample) |
| JP (3) | JP7361037B2 (enExample) |
| KR (3) | KR102709597B1 (enExample) |
| CN (3) | CN112553210B (enExample) |
| AU (3) | AU2018346105C1 (enExample) |
| BR (1) | BR112020006661A2 (enExample) |
| CA (1) | CA3078464A1 (enExample) |
| IL (3) | IL273771B2 (enExample) |
| MX (3) | MX2020003965A (enExample) |
| WO (1) | WO2019070894A1 (enExample) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JOP20190248A1 (ar) | 2017-04-21 | 2019-10-20 | Amgen Inc | بروتينات ربط مولد ضد trem2 واستخداماته |
| HUE062436T2 (hu) | 2017-08-03 | 2023-11-28 | Alector Llc | Anti-trem2 antitestek és alkalmazási módszereik |
| EP3692158A4 (en) | 2017-10-03 | 2021-08-04 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| CN111542549A (zh) | 2017-10-03 | 2020-08-14 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| MX2020004005A (es) | 2017-10-03 | 2020-10-05 | Prevail Therapeutics Inc | Terapias genicas para los trastornos lisosomales. |
| CN113795575A (zh) | 2019-02-04 | 2021-12-14 | 自由行疗法有限公司 | 多核苷酸 |
| MX2021010134A (es) | 2019-02-22 | 2021-09-23 | Univ Pennsylvania | Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn. |
| EP3931337A1 (en) * | 2019-02-26 | 2022-01-05 | The Trustees of The University of Pennsylvania | Compositions useful in treatment of krabbe disease |
| JP7664848B2 (ja) | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス，インコーポレーテッド | リソソーム障害のための遺伝子療法 |
| HRP20251506T1 (hr) | 2019-04-10 | 2026-01-02 | Prevail Therapeutics, Inc. | Genska terapija za lizosomske poremećaje |
| GB201913974D0 (en) * | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| IL292382A (en) * | 2019-10-22 | 2022-06-01 | Applied Genetic Tech Corporation | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders |
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| AU2021271635A1 (en) * | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
| EP4700126A3 (en) * | 2020-07-23 | 2026-06-03 | The University of North Carolina at Chapel Hill | Optimized slc13a5 genes and expression cassettes and their use |
| BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
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| KR20230043181A (ko) * | 2020-08-03 | 2023-03-30 | 프리베일 테라퓨틱스, 인크. | 파킨을 코딩하는 aav 벡터 및 그의 용도 |
| CN116157527A (zh) | 2020-08-10 | 2023-05-23 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
| AU2021325891A1 (en) * | 2020-08-10 | 2023-04-06 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| EP4229419A2 (en) | 2020-10-14 | 2023-08-23 | Denali Therapeutics Inc. | Methods for treating and monitoring frontotemporal dementia |
| WO2022081985A1 (en) | 2020-10-15 | 2022-04-21 | Prevail Therapeutics, Inc. | Assay for measuring potency of gene therapy drug product |
| JP2023545830A (ja) * | 2020-10-15 | 2023-10-31 | プリベイル セラピューティクス，インコーポレーテッド | 組換えアデノ随伴ウイルス組成物及びその生産方法 |
| AU2022214529A1 (en) * | 2021-02-01 | 2023-08-03 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of niemann pick type a disease |
| WO2022271944A2 (en) * | 2021-06-23 | 2022-12-29 | Avrobio, Inc. | Compositions and methods for treatment of gaucher disease |
| US20250049955A1 (en) | 2021-11-17 | 2025-02-13 | Voyager Therapeutics, Inc. | Compositons 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 | |
| JP2025516136A (ja) * | 2022-04-19 | 2025-05-27 | シャンハイ ヴァイタルゲン バイオファーマ カンパニー リミテッド | 神経変性疾患の治療のための組み換えａａｖベクター |
| CN120077057A (zh) | 2022-10-04 | 2025-05-30 | 伊莱利利公司 | Trem2相关疾病和病症的基因治疗 |
| CN121406674A (zh) * | 2022-10-08 | 2026-01-27 | 凌意(杭州)生物科技有限公司 | 用于治疗GCase缺乏相关疾病的多核苷酸 |
| GB202216168D0 (en) | 2022-10-31 | 2022-12-14 | UCB Biopharma SRL | Route of administration |
| AU2023427408A1 (en) * | 2023-02-02 | 2025-09-04 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| JP2026509301A (ja) | 2023-03-14 | 2026-03-17 | ユーシービー バイオファルマ エスアールエル | 遺伝子治療 |
| 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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