JPWO2020172490A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020172490A5 JPWO2020172490A5 JP2021549291A JP2021549291A JPWO2020172490A5 JP WO2020172490 A5 JPWO2020172490 A5 JP WO2020172490A5 JP 2021549291 A JP2021549291 A JP 2021549291A JP 2021549291 A JP2021549291 A JP 2021549291A JP WO2020172490 A5 JPWO2020172490 A5 JP WO2020172490A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- progranulin
- pharmaceutical composition
- composition
- vector genome
- 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
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025022078A JP2025081439A (ja) | 2019-02-22 | 2025-02-14 | 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 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962809329P | 2019-02-22 | 2019-02-22 | |
| US62/809,329 | 2019-02-22 | ||
| US201962923812P | 2019-10-21 | 2019-10-21 | |
| US62/923,812 | 2019-10-21 | ||
| US202062969108P | 2020-02-02 | 2020-02-02 | |
| US62/969,108 | 2020-02-02 | ||
| PCT/US2020/019149 WO2020172490A1 (en) | 2019-02-22 | 2020-02-21 | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025022078A Division JP2025081439A (ja) | 2019-02-22 | 2025-02-14 | 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 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022523766A JP2022523766A (ja) | 2022-04-26 |
| JP2022523766A5 JP2022523766A5 (https=) | 2023-02-17 |
| JPWO2020172490A5 true JPWO2020172490A5 (https=) | 2023-02-17 |
| JP7637058B2 JP7637058B2 (ja) | 2025-02-27 |
Family
ID=70058449
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549291A Active JP7637058B2 (ja) | 2019-02-22 | 2020-02-21 | 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 |
| JP2025022078A Pending JP2025081439A (ja) | 2019-02-22 | 2025-02-14 | 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025022078A Pending JP2025081439A (ja) | 2019-02-22 | 2025-02-14 | 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 |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US12416017B2 (https=) |
| EP (1) | EP3927381A1 (https=) |
| JP (2) | JP7637058B2 (https=) |
| KR (1) | KR20210131370A (https=) |
| CN (1) | CN113710281A (https=) |
| AU (1) | AU2020225472A1 (https=) |
| BR (1) | BR112021015817A2 (https=) |
| CA (1) | CA3129672A1 (https=) |
| CL (1) | CL2021002172A1 (https=) |
| CO (1) | CO2021011040A2 (https=) |
| IL (1) | IL285654A (https=) |
| MX (1) | MX2021010134A (https=) |
| PE (1) | PE20211819A1 (https=) |
| SG (1) | SG11202108504YA (https=) |
| TW (1) | TW202045730A (https=) |
| WO (1) | WO2020172490A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220111005A1 (en) * | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| 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 |
| GB201913974D0 (en) * | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| IL292382A (en) * | 2019-10-22 | 2022-06-01 | Applied Genetic Tech Corporation | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders |
| EP4204014A1 (en) * | 2020-08-26 | 2023-07-05 | The Trustees of The University of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| US20240033332A1 (en) * | 2020-12-17 | 2024-02-01 | Kyoto University | Agent for activating a neuron |
| EP4473123A4 (en) * | 2022-02-01 | 2026-03-11 | Shape Therapeutics Inc | STABLE CELL LINES FOR INDUCIBLE VAAR VIRION PRODUCTION |
| WO2024245120A1 (en) * | 2023-05-26 | 2024-12-05 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating proteinopathies in central nervous system |
| WO2024263567A2 (en) * | 2023-06-21 | 2024-12-26 | Eli Lilly And Company | Compounds for the delivery of granulin across the blood brain barrier |
| TW202548022A (zh) * | 2024-03-03 | 2025-12-16 | 美商帕西奇生物公司 | 用於治療神經退化性病症之重組腺相關病毒 |
Family Cites Families (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| WO1998046728A1 (en) | 1997-04-14 | 1998-10-22 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| CA2349838C (en) | 1998-11-05 | 2011-06-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| CA2653974A1 (en) | 2006-05-30 | 2008-02-14 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| WO2007146046A2 (en) | 2006-06-07 | 2007-12-21 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| WO2009089635A1 (en) | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| US20110203007A1 (en) | 2010-02-16 | 2011-08-18 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Assays of neurodegenerative disorders, including frontotemporal dementia and amyotrophic lateral sclerosis |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US20150352185A1 (en) | 2010-11-16 | 2015-12-10 | Denis G. Kay | Method for increasing neprilysin expression and activity |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| WO2015164723A1 (en) * | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| TN2017000353A1 (en) * | 2015-02-10 | 2019-01-16 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| WO2017075338A2 (en) * | 2015-10-29 | 2017-05-04 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| US20190060359A1 (en) | 2015-11-02 | 2019-02-28 | Neuraltus Pharmaceuticals, Inc. | Treatment of neurodegenerative disease with sodium chlorite |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| WO2017151884A1 (en) * | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| US20210284701A1 (en) | 2016-07-14 | 2021-09-16 | Emory University | Granulin Compositions and Uses Related Thereto |
| WO2018045347A1 (en) | 2016-09-02 | 2018-03-08 | Spark Therapeutics, Inc. | Methods and vectors for treating cns disorders |
| JP7455579B2 (ja) | 2017-02-28 | 2024-03-26 | 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 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| US20220111005A1 (en) | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| 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 |
| EP4204014A1 (en) | 2020-08-26 | 2023-07-05 | The Trustees of The University of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
-
2020
- 2020-02-21 BR BR112021015817-0A patent/BR112021015817A2/pt unknown
- 2020-02-21 TW TW109105680A patent/TW202045730A/zh unknown
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/ko active Pending
- 2020-02-21 CA CA3129672A patent/CA3129672A1/en active Pending
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/es unknown
- 2020-02-21 AU AU2020225472A patent/AU2020225472A1/en active Pending
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/zh active Pending
- 2020-02-21 US US17/431,937 patent/US12416017B2/en active Active
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/es unknown
- 2020-02-21 SG SG11202108504YA patent/SG11202108504YA/en unknown
- 2020-02-21 EP EP20715985.6A patent/EP3927381A1/en active Pending
- 2020-02-21 JP JP2021549291A patent/JP7637058B2/ja active Active
- 2020-02-21 WO PCT/US2020/019149 patent/WO2020172490A1/en not_active Ceased
-
2021
- 2021-08-16 IL IL285654A patent/IL285654A/en unknown
- 2021-08-16 CL CL2021002172A patent/CL2021002172A1/es unknown
- 2021-08-23 CO CONC2021/0011040A patent/CO2021011040A2/es unknown
-
2025
- 2025-02-14 JP JP2025022078A patent/JP2025081439A/ja active Pending
- 2025-08-22 US US19/307,613 patent/US20260049335A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| TWI878204B (zh) | 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症 | |
| JP2023144087A (ja) | 組換えＧｌｕｔ１アデノ随伴ウイルスベクターコンストラクトおよびＧｌｕｔ１発現を回復させる方法 | |
| JP2025063034A (ja) | ライソゾーム病の遺伝子治療 | |
| AU2023202598B2 (en) | Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2 | |
| US20250041453A1 (en) | Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector | |
| US20260049336A1 (en) | Bicistronic aav vectors encoding hexosaminidase alpha and beta-subunits and uses thereof | |
| US20250041452A1 (en) | Methods and compositions for treating mybpc3 related hypertrophic cardiomyopathy with a viral vector | |
| JP2025138648A (ja) | Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達 | |
| JPWO2020172490A5 (https=) | ||
| CA3178591A1 (en) | Immunosuppressive agents and viral delivery re-dosing methods for gene therapy | |
| CA3240197A1 (en) | Compositions comprising kozak sequences selected for enhanced expression | |
| JP2024528997A (ja) | 放射線誘発性唾液機能低下を予防するためのａｑｐ１遺伝子療法 | |
| US20220136005A1 (en) | One-step gene therapy for duchenne muscular dystrophy via gene replacement and anti-inflammation | |
| JP7534961B2 (ja) | ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 | |
| CA2978917C (en) | Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression | |
| WO2024094009A1 (zh) | 用于目的基因的表达盒及其应用 | |
| Gregorevic et al. | Functional enhancement of skeletal muscle by gene transfer | |
| HK40063258A (zh) | 腺相关病毒对cln6多核苷酸的递送 | |
| Transgene | 290. A Rationally Engineered Novel Capsid Variant of AAV9 for Peripheral Tissue-Detargeted and CNS-Directed Systemic Gene Delivery | |
| JPWO2022221276A5 (https=) |