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 |
|---|---|---|---|
| US201762567311P | 2017-10-03 | 2017-10-03 | |
| US201762567296P | 2017-10-03 | 2017-10-03 | |
| US201762567310P | 2017-10-03 | 2017-10-03 | |
| US201762567319P | 2017-10-03 | 2017-10-03 | |
| US201762567301P | 2017-10-03 | 2017-10-03 | |
| US62/567,310 | 2017-10-03 | ||
| US62/567,301 | 2017-10-03 | ||
| US62/567,319 | 2017-10-03 | ||
| US62/567,296 | 2017-10-03 | ||
| US62/567,311 | 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 |
| 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 (enExample) |
| EP (1) | EP3692158A4 (enExample) |
| JP (3) | JP7361037B2 (enExample) |
| KR (3) | KR102700963B1 (enExample) |
| CN (3) | CN111492061A (enExample) |
| AU (3) | AU2018346105C1 (enExample) |
| BR (1) | BR112020006661A2 (enExample) |
| CA (1) | CA3078464A1 (enExample) |
| IL (3) | IL273771B1 (enExample) |
| MX (3) | MX2020003965A (enExample) |
| WO (1) | WO2019070894A1 (enExample) |
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| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| US11802294B2 (en) | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| FI3850089T3 (fi) | 2019-02-04 | 2024-02-14 | Freeline Therapeutics Ltd | Polynukleotidejä |
| EP3927381A1 (en) | 2019-02-22 | 2021-12-29 | The Trustees of The University of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| MX2021010266A (es) * | 2019-02-26 | 2021-09-23 | Univ Pennsylvania | Composiciones utiles en el tratamiento de la enfermedad de krabbe. |
| WO2020210713A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| DK3953377T3 (da) * | 2019-04-10 | 2025-12-01 | Prevail Therapeutics Inc | Genterapier til lysosomale lidelser |
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| JP2023545462A (ja) | 2020-10-14 | 2023-10-30 | デナリ セラピューティクス インコーポレイテッド | 前頭側頭型認知症を治療及びモニタリングするための方法 |
| KR20230088393A (ko) | 2020-10-15 | 2023-06-19 | 프리베일 테라퓨틱스, 인크. | 재조합 아데노 연관 바이러스 조성물 및 이의 제조 방법 |
| JP2023545835A (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 | 遺伝子療法製剤の効力を測定するためのアッセイ |
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| 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 |
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| CN119487188A (zh) * | 2022-04-19 | 2025-02-18 | 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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