JP2020537542A5 - - Google Patents
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- JP2020537542A5 JP2020537542A5 JP2020540681A JP2020540681A JP2020537542A5 JP 2020537542 A5 JP2020537542 A5 JP 2020537542A5 JP 2020540681 A JP2020540681 A JP 2020540681A JP 2020540681 A JP2020540681 A JP 2020540681A JP 2020537542 A5 JP2020537542 A5 JP 2020537542A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- itr
- sequence
- raav
- 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.)
- Pending
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- 108090000623 proteins and genes Proteins 0.000 claims description 6
- 150000007523 nucleic acids Chemical class 0.000 claims 31
- 108020004707 nucleic acids Proteins 0.000 claims 29
- 102000039446 nucleic acids Human genes 0.000 claims 29
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 26
- 239000013608 rAAV vector Substances 0.000 claims 20
- 241000702421 Dependoparvovirus Species 0.000 claims 14
- 230000002401 inhibitory effect Effects 0.000 claims 14
- 239000013598 vector Substances 0.000 claims 12
- 210000004027 cell Anatomy 0.000 claims 11
- 238000002347 injection Methods 0.000 claims 9
- 239000007924 injection Substances 0.000 claims 9
- 239000002773 nucleotide Substances 0.000 claims 7
- 125000003729 nucleotide group Chemical group 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
- 108700019146 Transgenes Proteins 0.000 claims 5
- 108091070501 miRNA Proteins 0.000 claims 4
- 239000002679 microRNA Substances 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108091026890 Coding region Proteins 0.000 claims 3
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 claims 3
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 3
- 102000003802 alpha-Synuclein Human genes 0.000 claims 3
- 108090000185 alpha-Synuclein Proteins 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
- 230000008685 targeting Effects 0.000 claims 3
- 241000701447 unidentified baculovirus Species 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 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
- 241000238631 Hexapoda Species 0.000 claims 1
- 208000009829 Lewy Body Disease Diseases 0.000 claims 1
- 201000002832 Lewy body dementia Diseases 0.000 claims 1
- 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
- 230000002068 genetic effect Effects 0.000 description 3
- 238000001415 gene therapy Methods 0.000 description 2
- 238000002560 therapeutic procedure Methods 0.000 description 1
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022007759A JP2022060228A (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2023192753A JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
| JP2024001041A JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762567305P | 2017-10-03 | 2017-10-03 | |
| US201762567303P | 2017-10-03 | 2017-10-03 | |
| US62/567,305 | 2017-10-03 | ||
| US62/567,303 | 2017-10-03 | ||
| PCT/US2018/054223 WO2019070891A1 (en) | 2017-10-03 | 2018-10-03 | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022007759A Division JP2022060228A (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2023192753A Division JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020537542A JP2020537542A (ja) | 2020-12-24 |
| JP2020537542A5 true JP2020537542A5 (cg-RX-API-DMAC7.html) | 2021-11-11 |
Family
ID=65995011
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020540681A Pending JP2020537542A (ja) | 2017-10-03 | 2018-10-03 | ライソゾーム病の遺伝子治療 |
| JP2022007759A Pending JP2022060228A (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2023192753A Pending JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
| JP2024001041A Pending JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022007759A Pending JP2022060228A (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2023192753A Pending JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
| JP2024001041A Pending JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11807849B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3692151A4 (cg-RX-API-DMAC7.html) |
| JP (4) | JP2020537542A (cg-RX-API-DMAC7.html) |
| KR (2) | KR20220010062A (cg-RX-API-DMAC7.html) |
| CN (2) | CN111465691A (cg-RX-API-DMAC7.html) |
| AU (3) | AU2018346102B2 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112020006671A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3078501A1 (cg-RX-API-DMAC7.html) |
| IL (2) | IL273768A (cg-RX-API-DMAC7.html) |
| MX (2) | MX2020004005A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2019070891A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR102697811B1 (ko) | 2017-10-03 | 2024-08-22 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| 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 |
| 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 |
| AU2020270984B2 (en) | 2019-04-10 | 2025-11-20 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| PT3953377T (pt) | 2019-04-10 | 2025-11-27 | Prevail Therapeutics Inc | Terapias génicas para distúrbios lisossomais |
| WO2021030522A1 (en) * | 2019-08-13 | 2021-02-18 | Alnylam Pharmaceuticals, Inc. | SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| 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 |
| CN116710567A (zh) | 2020-10-15 | 2023-09-05 | 普利维尔治疗公司 | 重组腺相关病毒组合物及其产生方法 |
| MX2023004419A (es) | 2020-10-15 | 2023-07-05 | Prevail Therapeutics Inc | Ensayo para medir la potencia de un producto farmacológico para genoterapia. |
| WO2022097727A1 (ja) * | 2020-11-06 | 2022-05-12 | 住友ファーマ株式会社 | Ｒｐｓ２５遺伝子の発現及び／又は機能調節剤 |
| JP2024523445A (ja) * | 2021-06-21 | 2024-06-28 | ユニキュアー バイオファーマ ビー．ブイ． | α－シヌクレインをサイレンシングするための遺伝子構築物及びその使用 |
| JP2024527313A (ja) * | 2021-06-28 | 2024-07-24 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | タウ低減遺伝子療法のための方法および組成物 |
| CN113846122B (zh) * | 2021-08-16 | 2023-08-25 | 中国医学科学院医学生物学研究所 | 一种过表达snca的腺相关病毒载体aav-snca、制备方法及其应用 |
| WO2023178338A2 (en) * | 2022-03-18 | 2023-09-21 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating tmem43 related cardiomyopathy with a viral vector |
| WO2025077806A1 (en) * | 2023-10-11 | 2025-04-17 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of microtubule associated protein tau (mapt) |
| WO2025160429A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
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| AU2020270984B2 (en) | 2019-04-10 | 2025-11-20 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US20220211871A1 (en) | 2019-04-10 | 2022-07-07 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| PT3953377T (pt) | 2019-04-10 | 2025-11-27 | Prevail Therapeutics Inc | Terapias génicas para distúrbios lisossomais |
| WO2020210615A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
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2018
- 2018-10-03 CA CA3078501A patent/CA3078501A1/en active Pending
- 2018-10-03 MX MX2020004005A patent/MX2020004005A/es unknown
- 2018-10-03 EP EP18864256.5A patent/EP3692151A4/en active Pending
- 2018-10-03 CN CN201880075728.7A patent/CN111465691A/zh active Pending
- 2018-10-03 CN CN202011187145.9A patent/CN112501208A/zh active Pending
- 2018-10-03 KR KR1020227000760A patent/KR20220010062A/ko not_active Ceased
- 2018-10-03 JP JP2020540681A patent/JP2020537542A/ja active Pending
- 2018-10-03 BR BR112020006671-0A patent/BR112020006671A2/pt unknown
- 2018-10-03 US US16/753,018 patent/US11807849B2/en active Active
- 2018-10-03 KR KR1020207012192A patent/KR20200078513A/ko not_active Ceased
- 2018-10-03 WO PCT/US2018/054223 patent/WO2019070891A1/en not_active Ceased
- 2018-10-03 AU AU2018346102A patent/AU2018346102B2/en active Active
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2020
- 2020-04-02 IL IL273768A patent/IL273768A/en unknown
- 2020-04-02 US US16/838,993 patent/US12049626B2/en active Active
- 2020-07-13 MX MX2020011748A patent/MX2020011748A/es unknown
- 2020-10-29 AU AU2020260476A patent/AU2020260476B2/en active Active
- 2020-12-22 IL IL279669A patent/IL279669A/en unknown
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2022
- 2022-01-21 JP JP2022007759A patent/JP2022060228A/ja active Pending
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2023
- 2023-08-11 AU AU2023214366A patent/AU2023214366B2/en active Active
- 2023-11-13 JP JP2023192753A patent/JP2024014935A/ja active Pending
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2024
- 2024-01-09 JP JP2024001041A patent/JP2024037189A/ja active Pending
- 2024-06-14 US US18/743,624 patent/US20250011783A1/en active Pending
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