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 Ceased 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 Ceased 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) | IL278868B2 (enExample) |
| MX (2) | MX2020003557A (enExample) |
| WO (1) | WO2019070893A1 (enExample) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN112553210B (zh) | 2017-10-03 | 2024-09-24 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| BR112020006633A2 (pt) | 2017-10-03 | 2020-10-06 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| PL3850089T3 (pl) | 2019-02-04 | 2024-05-06 | Freeline Therapeutics Limited | Polinukleotydy |
| US11999974B2 (en) | 2019-04-10 | 2024-06-04 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20220006527A (ko) | 2019-04-10 | 2022-01-17 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
| TW202120687A (zh) * | 2019-08-12 | 2021-06-01 | 應用醫學研究基金會 | 用於藉由基因療法治療諸如帕金森氏症之突觸核蛋白病變的病毒顆粒 |
| MX2022004812A (es) * | 2019-10-22 | 2023-02-23 | Applied Genetic Tech Corporation | Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina. |
| JP2021083987A (ja) * | 2019-11-29 | 2021-06-03 | 株式会社三洋物産 | 遊技機 |
| JP2023530454A (ja) * | 2020-06-18 | 2023-07-18 | ルンドクヴィスト，ヨーアン | アルファ－シヌクレインターゲティング抗体、プログラニュリン及びプロサポシン並びにそれらの複合体、並びにｇｄｎｆを分泌する細胞株 |
| 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 |
| IL299811A (en) | 2020-07-29 | 2023-03-01 | Freeline Therapeutics Ltd | polypeptide |
| CN116437968A (zh) | 2020-08-10 | 2023-07-14 | 普利维尔治疗公司 | 用于神经退行性病症的基因疗法 |
| MX2023001562A (es) * | 2020-08-10 | 2023-05-03 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
| IL301858A (en) * | 2020-10-15 | 2023-06-01 | 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 |
| 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 |
| WO2023139496A1 (en) * | 2022-01-21 | 2023-07-27 | Pfizer Inc. | 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 |
| AU2024265526A1 (en) | 2023-05-03 | 2025-12-11 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency |
| 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 (48)
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| 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 |
| US6521225B1 (en) | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| 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 |
| AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| JP2004516016A (ja) | 2000-09-18 | 2004-06-03 | ジェンザイム・コーポレイション | ハイブリッドユビキチンプロモーターを含む発現ベクター |
| 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 |
| ES2562490T3 (es) | 2003-05-01 | 2016-03-04 | Genzyme Corporation | Terapia génica para trastornos neurometabólicos |
| 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 |
| CN101460621B (zh) | 2006-06-07 | 2016-01-27 | 建新公司 | 肌萎缩性侧索硬化和其他脊髓失调症的基因治疗 |
| US8962273B2 (en) | 2007-05-11 | 2015-02-24 | Genzyme Corporation | Methods of producing a secreted protein |
| US8454954B2 (en) | 2007-05-16 | 2013-06-04 | The Brigham And Women's Hospital, Inc. | Treatment of synucleinopathies |
| WO2009079399A2 (en) * | 2007-12-14 | 2009-06-25 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| CA2753362C (en) | 2009-02-24 | 2018-06-19 | The Scripps Research Institute | Reengineering mrna primary structure for enhanced protein production |
| US9255266B2 (en) | 2009-05-06 | 2016-02-09 | Rutgers, The State University Of New Jersey | RNA targeting in alpha-synucleinopathies |
| EP3567106A1 (en) | 2010-04-23 | 2019-11-13 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| 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 |
| WO2012065248A1 (en) | 2010-11-16 | 2012-05-24 | Kay Denis G | Method for increasing neprilysin expression and activity |
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| CA2875618C (en) | 2012-07-11 | 2021-04-27 | Sangamo Biosciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| BR112015009746A2 (pt) * | 2012-11-05 | 2017-08-15 | Genzyme Corp | Agente que aumenta atividade de glicocerebrosidase em mamíferos |
| KR20160010526A (ko) | 2013-05-15 | 2016-01-27 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
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| US20170035860A1 (en) | 2015-04-02 | 2017-02-09 | Alexander C. Flynn | Compositions and methods for treatment of neurogenerative diseases |
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| 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 |
| CA2986252A1 (en) | 2015-05-15 | 2016-11-24 | Regents Of The University Of Minnesota | Adeno-associated virus vector for delivery of iduronate-2-sulfatase to the central nervous system |
| WO2017075338A2 (en) | 2015-10-29 | 2017-05-04 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| PE20181156A1 (es) | 2015-11-05 | 2018-07-19 | Bamboo Therapeutics Inc | Genes de ataxia de friedreich modificados y vectores para terapia genica |
| WO2017136536A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| US12070510B2 (en) | 2016-02-05 | 2024-08-27 | Emory University | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid |
| US20190328906A1 (en) | 2016-03-02 | 2019-10-31 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| CN112553210B (zh) | 2017-10-03 | 2024-09-24 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| BR112020006633A2 (pt) | 2017-10-03 | 2020-10-06 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN111819281B (zh) * | 2017-10-23 | 2024-12-31 | 普利维尔治疗公司 | 用于神经变性疾病的基因疗法 |
| CA3134841A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20220006527A (ko) | 2019-04-10 | 2022-01-17 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
| US20220211871A1 (en) | 2019-04-10 | 2022-07-07 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| MX2023001562A (es) | 2020-08-10 | 2023-05-03 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
-
2018
- 2018-10-03 BR BR112020006633-8A patent/BR112020006633A2/pt unknown
- 2018-10-03 CA CA3078371A patent/CA3078371A1/en active Pending
- 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 IL IL278868A patent/IL278868B2/en unknown
- 2018-10-03 AU AU2018346104A patent/AU2018346104B2/en active Active
- 2018-10-03 KR KR1020207012188A patent/KR102693318B1/ko active Active
- 2018-10-03 WO PCT/US2018/054225 patent/WO2019070893A1/en not_active Ceased
- 2018-10-03 MX MX2020003557A patent/MX2020003557A/es unknown
- 2018-10-03 IL IL273776A patent/IL273776B2/en unknown
- 2018-10-03 JP JP2020540682A patent/JP7254815B2/ja active Active
- 2018-10-03 EP EP18865080.8A patent/EP3692075A4/en active Pending
-
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/US12571002B2/en active Active
-
2023
- 2023-03-29 JP JP2023052515A patent/JP2023086740A/ja not_active Ceased
-
2024
- 2024-12-13 JP JP2024218686A patent/JP2025063034A/ja not_active Withdrawn
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