JP2020537543A5 - - Google Patents
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- JP2020537543A5 JP2020537543A5 JP2020540682A JP2020540682A JP2020537543A5 JP 2020537543 A5 JP2020537543 A5 JP 2020537543A5 JP 2020540682 A JP2020540682 A JP 2020540682A JP 2020540682 A JP2020540682 A JP 2020540682A JP 2020537543 A5 JP2020537543 A5 JP 2020537543A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- sequence
- seq
- itr
- 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.)
- Granted
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- 150000007523 nucleic acids Chemical class 0.000 claims 30
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 26
- 108020004707 nucleic acids Proteins 0.000 claims 26
- 102000039446 nucleic acids Human genes 0.000 claims 26
- 239000013608 rAAV vector Substances 0.000 claims 16
- 241000702421 Dependoparvovirus Species 0.000 claims 14
- 230000002401 inhibitory effect Effects 0.000 claims 12
- 239000013598 vector Substances 0.000 claims 12
- 210000004027 cell Anatomy 0.000 claims 11
- 108090000623 proteins and genes Proteins 0.000 claims 10
- 238000002347 injection Methods 0.000 claims 9
- 239000007924 injection Substances 0.000 claims 9
- 102000004169 proteins and genes Human genes 0.000 claims 9
- 108091026890 Coding region 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
- 230000008685 targeting Effects 0.000 claims 6
- 102000004547 Glucosylceramidase Human genes 0.000 claims 4
- 108010017544 Glucosylceramidase Proteins 0.000 claims 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 4
- 108700019146 Transgenes Proteins 0.000 claims 4
- 102000003802 alpha-Synuclein Human genes 0.000 claims 4
- 108090000185 alpha-Synuclein Proteins 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 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
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 102000019355 Synuclein Human genes 0.000 claims 2
- 108050006783 Synuclein 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
- 241000701447 unidentified baculovirus Species 0.000 claims 2
- 208000015872 Gaucher disease Diseases 0.000 claims 1
- 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
- 238000002955 isolation Methods 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 description 6
- 230000002068 genetic effect Effects 0.000 description 4
- 238000001415 gene therapy Methods 0.000 description 2
- 238000002560 therapeutic procedure Methods 0.000 description 2
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023052515A JP2023086740A (ja) | 2017-10-03 | 2023-03-29 | ライソゾーム病の遺伝子治療 |
| JP2024218686A JP2025063034A (ja) | 2017-10-03 | 2024-12-13 | ライソゾーム病の遺伝子治療 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762567296P | 2017-10-03 | 2017-10-03 | |
| US62/567,296 | 2017-10-03 | ||
| PCT/US2018/054225 WO2019070893A1 (en) | 2017-10-03 | 2018-10-03 | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023052515A Division JP2023086740A (ja) | 2017-10-03 | 2023-03-29 | ライソゾーム病の遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020537543A JP2020537543A (ja) | 2020-12-24 |
| JP2020537543A5 true JP2020537543A5 (enExample) | 2021-11-11 |
| JP7254815B2 JP7254815B2 (ja) | 2023-04-10 |
Family
ID=65994357
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020540682A Active JP7254815B2 (ja) | 2017-10-03 | 2018-10-03 | ライソゾーム病の遺伝子治療 |
| JP2023052515A Pending JP2023086740A (ja) | 2017-10-03 | 2023-03-29 | ライソゾーム病の遺伝子治療 |
| JP2024218686A Withdrawn JP2025063034A (ja) | 2017-10-03 | 2024-12-13 | ライソゾーム病の遺伝子治療 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023052515A Pending JP2023086740A (ja) | 2017-10-03 | 2023-03-29 | ライソゾーム病の遺伝子治療 |
| JP2024218686A Withdrawn JP2025063034A (ja) | 2017-10-03 | 2024-12-13 | ライソゾーム病の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US11802294B2 (enExample) |
| EP (1) | EP3692075A4 (enExample) |
| JP (3) | JP7254815B2 (enExample) |
| KR (1) | KR102693318B1 (enExample) |
| CN (1) | CN111542549A (enExample) |
| AU (2) | AU2018346104B2 (enExample) |
| BR (1) | BR112020006633A2 (enExample) |
| CA (1) | CA3078371A1 (enExample) |
| IL (2) | IL273776B2 (enExample) |
| MX (2) | MX2020003557A (enExample) |
| WO (1) | WO2019070893A1 (enExample) |
Families Citing this family (22)
| 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 |
| 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 |
| DK3953377T3 (da) | 2019-04-10 | 2025-12-01 | Prevail Therapeutics Inc | Genterapier til lysosomale lidelser |
| WO2020210713A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP2023500011A (ja) * | 2019-08-12 | 2023-01-04 | ファンダシオン パラ ラ インベスティガシオン メディカ アプリカダ | 遺伝子療法によるパーキンソン病などのシヌクレイノパチーの治療に使用するためのウイルス粒子 |
| CN115715327A (zh) * | 2019-10-22 | 2023-02-24 | 应用遗传科技公司 | 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统 |
| JP2021083987A (ja) * | 2019-11-29 | 2021-06-03 | 株式会社三洋物産 | 遊技機 |
| WO2021258074A2 (en) * | 2020-06-18 | 2021-12-23 | Lundkvist Johan | Cell lines secreting alpha-synuclein targeting antibodies, progranulin and prosaposin and a complex of both, and gdnf |
| CA3190309A1 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| CA3189801A1 (en) | 2020-07-29 | 2022-02-03 | Freeline Therapeutics Limited | Polypeptide |
| 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 |
| KR20230066360A (ko) | 2020-08-10 | 2023-05-15 | 프리베일 테라퓨틱스, 인크. | 신경퇴행성 장애를 위한 유전자 요법 |
| 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 |
| 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 |
| EP4465998A1 (en) * | 2022-01-21 | 2024-11-27 | Astrazeneca Ireland Limited | Gene therapy for gaucher disease |
| 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 |
| CN121127597A (zh) | 2023-05-03 | 2025-12-12 | 沃雅戈治疗公司 | 用于治疗与葡糖苷酰鞘氨醇酶β1缺陷相关的病症的组合物及方法 |
| WO2025038805A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency |
| KR20250046126A (ko) * | 2023-09-22 | 2025-04-02 | 주식회사 스탠드업테라퓨티스 | 신규한 파킨슨 병 치료제 |
Family Cites Families (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5879680A (en) | 1987-12-23 | 1999-03-09 | The United States Of America As Represented By The Department Of Health And Human Services | Cloned DNA for synthesizing unique glucocerebrosidase |
| EP0933997A4 (en) | 1996-09-06 | 2000-08-16 | Chiron Corp | METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS |
| 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 |
| GB0009887D0 (en) | 2000-04-20 | 2000-06-07 | Btg Int Ltd | Cytotoxic agents |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| EP1319082B1 (en) | 2000-09-18 | 2005-11-16 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
| US20030133924A1 (en) * | 2001-12-21 | 2003-07-17 | Novazyme Pharmaceuticals, Inc. | Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease |
| US20060292117A1 (en) | 2002-04-17 | 2006-12-28 | Loiler Scott A | Improved rAAv vectors |
| PL1620133T3 (pl) | 2003-05-01 | 2016-05-31 | Genzyme Corp | Terapia genowa dla zaburzeń neurometabolicznych |
| WO2006036465A2 (en) | 2004-09-03 | 2006-04-06 | University Of Florida | Compositions and methods for treating cystic fibrosis |
| EP1799826B1 (en) | 2004-09-29 | 2009-08-12 | Children's Memorial Hospital | siRNA-MEDIATED GENE SILENCING OF ALPHA SYNUCLEIN |
| ES2596885T3 (es) | 2006-06-07 | 2017-01-12 | Genzyme Corporation | Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la medula espinal |
| WO2008143797A1 (en) | 2007-05-11 | 2008-11-27 | Genzyme Corporation | Methods of producing a secreted protein |
| HUE027278T2 (en) | 2007-05-16 | 2016-11-28 | Brigham & Womens Hospital Inc | Treatment of synucleinopathies |
| WO2009079399A2 (en) | 2007-12-14 | 2009-06-25 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| US9255266B2 (en) | 2009-05-06 | 2016-02-09 | Rutgers, The State University Of New Jersey | RNA targeting in alpha-synucleinopathies |
| US9290759B2 (en) | 2010-08-25 | 2016-03-22 | The Trustees Of Columbia University In The City Of New York | Optimized miRNA constructs |
| WO2012027713A2 (en) | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
| EP2640407A4 (en) | 2010-11-16 | 2014-07-09 | Denis G Kay | PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN |
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| RU2692251C2 (ru) | 2013-05-15 | 2019-06-24 | Риджентс Оф Зэ Юниверсити Оф Миннесота | Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему |
| US9347107B2 (en) | 2013-05-23 | 2016-05-24 | Norman Z Lai | Vector containing multiple nucleotide sequences for the expression of enzymes |
| US9574184B2 (en) * | 2013-09-25 | 2017-02-21 | Children's Hospital Medical Center | Lysosomal protein targeting sequence and therapeutic applications of same |
| US10900053B2 (en) * | 2014-11-21 | 2021-01-26 | University Of Florida Research Foundation, Incorporated | Genome-modified recombinant adeno-associated virus vectors |
| US20170035860A1 (en) | 2015-04-02 | 2017-02-09 | Alexander C. Flynn | Compositions and methods for treatment of neurogenerative diseases |
| JP2015180203A (ja) * | 2015-04-17 | 2015-10-15 | ザ スクリプス リサーチ インスティテュート | タンパク質生産の増強のためのｍＲＮＡの一次構造の再操作 |
| AU2016256895B2 (en) | 2015-05-07 | 2022-05-26 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| EP3091087A1 (en) | 2015-05-08 | 2016-11-09 | Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol | Method for in vitro diagnosis of synucleinopathies using alpha-synuclein gene transcripts |
| WO2016187017A1 (en) | 2015-05-15 | 2016-11-24 | Mcivor R Scott | Adeno-associated for therapeutic delivery to central nervous system |
| WO2017075338A2 (en) | 2015-10-29 | 2017-05-04 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| CN108348621A (zh) * | 2015-11-05 | 2018-07-31 | 竹治疗股份有限公司 | 用于基因治疗的经修饰的弗里德赖希共济失调基因及载体 |
| US11826433B2 (en) | 2016-02-02 | 2023-11-28 | University Of Massachusetts | Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system |
| IL300254B2 (en) | 2016-02-05 | 2025-06-01 | Univ Emory | Administration of single stranded or self-complementary adeno-associated virus 9 by injection into the cerebrospinal fluid to target gene therapy to the central nervous system |
| CA3016314A1 (en) | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
| US11802294B2 (en) | 2017-10-03 | 2023-10-31 | 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 |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| BR112020008033A2 (pt) | 2017-10-23 | 2020-10-27 | Prevail Therapeutics, Inc. | terapias gênicas para doença neurodegenerativa |
| CA3134841A1 (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 |
| WO2020210713A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
-
2018
- 2018-10-03 US US16/753,016 patent/US11802294B2/en active Active
- 2018-10-03 CN CN201880075674.4A patent/CN111542549A/zh active Pending
- 2018-10-03 EP EP18865080.8A patent/EP3692075A4/en active Pending
- 2018-10-03 AU AU2018346104A patent/AU2018346104B2/en active Active
- 2018-10-03 IL IL273776A patent/IL273776B2/en unknown
- 2018-10-03 WO PCT/US2018/054225 patent/WO2019070893A1/en not_active Ceased
- 2018-10-03 IL IL278868A patent/IL278868B2/en unknown
- 2018-10-03 KR KR1020207012188A patent/KR102693318B1/ko active Active
- 2018-10-03 JP JP2020540682A patent/JP7254815B2/ja active Active
- 2018-10-03 BR BR112020006633-8A patent/BR112020006633A2/pt unknown
- 2018-10-03 CA CA3078371A patent/CA3078371A1/en active Pending
- 2018-10-03 MX MX2020003557A patent/MX2020003557A/es unknown
-
2019
- 2019-11-20 US US16/689,865 patent/US10837028B2/en active Active
-
2020
- 2020-07-13 MX MX2020011745A patent/MX2020011745A/es unknown
- 2020-07-14 AU AU2020205228A patent/AU2020205228B2/en active Active
- 2020-09-17 US US17/024,117 patent/US11060113B2/en active Active
-
2021
- 2021-07-07 US US17/369,168 patent/US20210332385A1/en active Pending
-
2023
- 2023-03-29 JP JP2023052515A patent/JP2023086740A/ja active Pending
-
2024
- 2024-12-13 JP JP2024218686A patent/JP2025063034A/ja not_active Withdrawn
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