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
Links
- 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 |
|---|---|---|---|
| US201762567303P | 2017-10-03 | 2017-10-03 | |
| US201762567305P | 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 (https=) | 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 (https=) |
| EP (1) | EP3692151A4 (https=) |
| JP (4) | JP2020537542A (https=) |
| KR (2) | KR20200078513A (https=) |
| CN (2) | CN112501208A (https=) |
| AU (3) | AU2018346102B2 (https=) |
| BR (1) | BR112020006671A2 (https=) |
| CA (1) | CA3078501A1 (https=) |
| IL (2) | IL273768A (https=) |
| MX (2) | MX2020004005A (https=) |
| WO (1) | WO2019070891A1 (https=) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| 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 |
| KR20210148101A (ko) | 2019-02-04 | 2021-12-07 | 프리라인 테라퓨틱스 리미티드 | 폴리뉴클레오티드 |
| BR112021015817A2 (pt) | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
| CA3136004A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| 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 |
| EP4013870A1 (en) * | 2019-08-13 | 2022-06-22 | Alnylam Pharmaceuticals, Inc. | Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof |
| AU2021325891A1 (en) | 2020-08-10 | 2023-04-06 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| JP2023537903A (ja) | 2020-08-10 | 2023-09-06 | プリベイル セラピューティクス，インコーポレーテッド | リソソーム障害に対する遺伝子療法 |
| EP4229213A1 (en) | 2020-10-15 | 2023-08-23 | Prevail Therapeutics, Inc. | Assay for measuring potency of gene therapy drug product |
| MX2023004420A (es) | 2020-10-15 | 2023-07-05 | Prevail Therapeutics Inc | Composiciones de virus adenoasociados recombinantes y métodos para su producción. |
| CN116507727A (zh) * | 2020-11-06 | 2023-07-28 | 住友制药株式会社 | Rps25基因的表达和/或功能调节剂 |
| WO2022268835A1 (en) * | 2021-06-21 | 2022-12-29 | Uniqure Biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| 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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| WO1998009524A1 (en) * | 1996-09-06 | 1998-03-12 | Chiron Corporation | Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors |
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| EP1121429A2 (en) | 1998-10-16 | 2001-08-08 | Introgene B.V. | Gene therapy of alzheimer's disease by delivery of an encoded apolipoprotein e |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US6696272B1 (en) | 1999-06-02 | 2004-02-24 | Hsc Research & Development Limited Partnership | Products and methods for gaucher disease therapy |
| AU5323301A (en) | 2000-04-06 | 2001-10-23 | Kos Pharma Inc | Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia |
| GB0009887D0 (en) * | 2000-04-20 | 2000-06-07 | Btg Int Ltd | Cytotoxic agents |
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| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| BR112020008033A2 (pt) | 2017-10-23 | 2020-10-27 | Prevail Therapeutics, Inc. | terapias gênicas para doença neurodegenerativa |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| JP7616995B2 (ja) | 2018-11-28 | 2025-01-17 | プリベイル セラピューティクス，インコーポレーテッド | 神経変性疾患のための遺伝子治療 |
| WO2020210615A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US20220211871A1 (en) | 2019-04-10 | 2022-07-07 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CA3136004A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| 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 |
-
2018
- 2018-10-03 CN CN202011187145.9A patent/CN112501208A/zh active Pending
- 2018-10-03 KR KR1020207012192A patent/KR20200078513A/ko not_active Ceased
- 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 BR BR112020006671-0A patent/BR112020006671A2/pt not_active Application Discontinuation
- 2018-10-03 WO PCT/US2018/054223 patent/WO2019070891A1/en not_active Ceased
- 2018-10-03 JP JP2020540681A patent/JP2020537542A/ja active Pending
- 2018-10-03 KR KR1020227000760A patent/KR20220010062A/ko not_active Ceased
- 2018-10-03 AU AU2018346102A patent/AU2018346102B2/en active Active
- 2018-10-03 US US16/753,018 patent/US11807849B2/en active Active
- 2018-10-03 CN CN201880075728.7A patent/CN111465691A/zh active Pending
-
2020
- 2020-04-02 US US16/838,993 patent/US12049626B2/en active Active
- 2020-04-02 IL IL273768A patent/IL273768A/en unknown
- 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
-
2022
- 2022-01-21 JP JP2022007759A patent/JP2022060228A/ja active Pending
-
2023
- 2023-08-11 AU AU2023214366A patent/AU2023214366B2/en active Active
- 2023-11-13 JP JP2023192753A patent/JP2024014935A/ja active Pending
-
2024
- 2024-01-09 JP JP2024001041A patent/JP2024037189A/ja active Pending
- 2024-06-14 US US18/743,624 patent/US20250011783A1/en not_active Abandoned
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