JPWO2020210713A5 - - Google Patents
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- JPWO2020210713A5 JPWO2020210713A5 JP2021559877A JP2021559877A JPWO2020210713A5 JP WO2020210713 A5 JPWO2020210713 A5 JP WO2020210713A5 JP 2021559877 A JP2021559877 A JP 2021559877A JP 2021559877 A JP2021559877 A JP 2021559877A JP WO2020210713 A5 JPWO2020210713 A5 JP WO2020210713A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- raav
- itr
- seq
- 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.)
- Granted
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962831840P | 2019-04-10 | 2019-04-10 | |
| US62/831,840 | 2019-04-10 | ||
| US202062990246P | 2020-03-16 | 2020-03-16 | |
| US62/990,246 | 2020-03-16 | ||
| PCT/US2020/027788 WO2020210713A1 (en) | 2019-04-10 | 2020-04-10 | Gene therapies for lysosomal disorders |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022526021A JP2022526021A (ja) | 2022-05-20 |
| JPWO2020210713A5 true JPWO2020210713A5 (https=) | 2024-01-19 |
| JP2022526021A5 JP2022526021A5 (https=) | 2024-01-19 |
| JP7664848B2 JP7664848B2 (ja) | 2025-04-18 |
Family
ID=70554182
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021559877A Active JP7664848B2 (ja) | 2019-04-10 | 2020-04-10 | リソソーム障害のための遺伝子療法 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11903985B2 (https=) |
| EP (1) | EP3953378A1 (https=) |
| JP (1) | JP7664848B2 (https=) |
| KR (1) | KR20220006527A (https=) |
| CN (1) | CN114026115A (https=) |
| AU (1) | AU2020270984B2 (https=) |
| BR (1) | BR112021019880A2 (https=) |
| CA (1) | CA3136117A1 (https=) |
| IL (1) | IL286901A (https=) |
| MX (1) | MX2021012184A (https=) |
| WO (1) | WO2020210713A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| EP3692075A4 (en) | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| BR112021015312A2 (pt) | 2019-02-04 | 2021-11-09 | Freeline Therapeutics Ltd | Polinucleotídeos, partícula viral e composição |
| PL3953377T3 (pl) | 2019-04-10 | 2026-02-16 | Prevail Therapeutics, Inc. | Terapie genowe zaburzeń lizosomalnych |
| IL300407A (en) | 2020-08-10 | 2023-04-01 | Prevail Therapeutics Inc | Gene therapies for lysosomal disorders |
| US20240044869A1 (en) * | 2020-10-15 | 2024-02-08 | Prevail Therapeutics, Inc. | Assay for measuring potency of gene therapy drug product |
| US20250082686A1 (en) * | 2021-07-21 | 2025-03-13 | Aspen Neuroscience, Inc. | Aav-based modulation of gba1 and related compositions and uses thereof |
| EP4377471A4 (en) * | 2021-07-28 | 2025-10-15 | Atsena Therapeutics Inc | AAV TRANSFER PLASMIDS |
| WO2023018674A1 (en) * | 2021-08-09 | 2023-02-16 | Amicus Therapeutics, Inc. | Determination of gene transduction potency in neuron-like cells |
| AR126839A1 (es) * | 2021-08-20 | 2023-11-22 | Llc «Anabion» | Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta |
| MX2024009003A (es) * | 2022-01-21 | 2024-09-17 | Astrazeneca Ireland Ltd | Terapia genica para la enfermedad de gaucher. |
| WO2023202637A1 (en) | 2022-04-19 | 2023-10-26 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating neurodegenerative disorders |
| CN117018231B (zh) * | 2023-08-16 | 2024-05-10 | 科辉智药(深圳)新药研究中心有限公司 | 用于治疗神经病变的基因疗法及其应用 |
| CN121752725A (zh) * | 2023-08-23 | 2026-03-27 | 上海金珂博生物技术有限公司 | 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途 |
| WO2025166048A1 (en) * | 2024-01-31 | 2025-08-07 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Synthetic gba1 genes |
| WO2025229340A1 (en) | 2024-05-01 | 2025-11-06 | Spur Therapeutics Limited | Methods and compositions for treatment of a synucleinopathy and/or a disease associated with a gcase deficiency |
Family Cites Families (48)
| 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 |
| US6521225B1 (en) | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| 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 |
| AU2001290984A1 (en) | 2000-09-18 | 2002-04-02 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
| US7232670B2 (en) | 2001-09-28 | 2007-06-19 | St. Jude Children's Research Hospital | Targeting proteins to cells expressing mannose receptors via expression in insect cells |
| US20030133924A1 (en) | 2001-12-21 | 2003-07-17 | Novazyme Pharmaceuticals, Inc. | Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease |
| AU2003221733A1 (en) | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | 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 |
| WO2006039253A2 (en) | 2004-09-29 | 2006-04-13 | Children's Memorial Hospital | Sirna-mediated gene silencing of alpha synuclein |
| CN101541172A (zh) * | 2005-06-08 | 2009-09-23 | 阿米库斯治疗学公司 | 溶酶体酶编码基因突变相关的cns紊乱的治疗 |
| ES2385679T3 (es) | 2006-08-24 | 2012-07-30 | Virovek, Inc. | Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos |
| US8962273B2 (en) | 2007-05-11 | 2015-02-24 | Genzyme Corporation | Methods of producing a secreted protein |
| EP2154969B1 (en) | 2007-05-16 | 2015-11-18 | 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 |
| WO2010129791A1 (en) | 2009-05-06 | 2010-11-11 | University Of Medicine And Dentistry Of New Jersey | Rna targeting in alpha-synucleinopathies |
| EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| WO2012027558A2 (en) * | 2010-08-25 | 2012-03-01 | 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 |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| US20150352185A1 (en) | 2010-11-16 | 2015-12-10 | Denis G. Kay | Method for increasing neprilysin expression and activity |
| CA2835707C (en) | 2011-05-09 | 2019-02-26 | Eip Pharma, Llc | Compositions and methods for treating alzheimer's disease |
| JP2016503405A (ja) * | 2012-11-05 | 2016-02-04 | ジェンザイム・コーポレーション | タンパク質症を処置するための組成物および方法 |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| US9347107B2 (en) | 2013-05-23 | 2016-05-24 | Norman Z Lai | Vector containing multiple nucleotide sequences for the expression of enzymes |
| CN120174012A (zh) | 2013-10-24 | 2025-06-20 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| WO2016081927A2 (en) | 2014-11-21 | 2016-05-26 | University Of Florida Research Foundation, Inc. | 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 |
| EP3292206B8 (en) | 2015-05-07 | 2022-02-09 | 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 |
| HK1256341A1 (zh) | 2015-05-15 | 2019-09-20 | 明尼苏达大学董事会 | 用於治疗性递送到中枢神经系统的腺相关物 |
| WO2017075338A2 (en) | 2015-10-29 | 2017-05-04 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| RU2021102893A (ru) | 2015-11-05 | 2021-03-03 | Бамбу Терапьютикс, Инк. | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
| 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 |
| PL3411484T3 (pl) | 2016-02-05 | 2024-02-19 | Emory University | Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego |
| WO2017151884A1 (en) | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| KR20230079511A (ko) | 2016-04-21 | 2023-06-07 | 바이로베크 인코포레이티드 | 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물 |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| EP3692075A4 (en) | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| AU2018354195A1 (en) | 2017-10-23 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| 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 |
-
2020
- 2020-04-10 CA CA3136117A patent/CA3136117A1/en active Pending
- 2020-04-10 US US16/846,210 patent/US11903985B2/en active Active
- 2020-04-10 AU AU2020270984A patent/AU2020270984B2/en active Active
- 2020-04-10 CN CN202080041473.XA patent/CN114026115A/zh active Pending
- 2020-04-10 MX MX2021012184A patent/MX2021012184A/es unknown
- 2020-04-10 KR KR1020217036235A patent/KR20220006527A/ko active Pending
- 2020-04-10 BR BR112021019880A patent/BR112021019880A2/pt unknown
- 2020-04-10 WO PCT/US2020/027788 patent/WO2020210713A1/en not_active Ceased
- 2020-04-10 EP EP20724264.5A patent/EP3953378A1/en active Pending
- 2020-04-10 JP JP2021559877A patent/JP7664848B2/ja active Active
-
2021
- 2021-10-03 IL IL286901A patent/IL286901A/en unknown
-
2023
- 2023-12-21 US US18/392,002 patent/US20240123003A1/en active Pending
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