JP2022523766A5 - - Google Patents
Info
- Publication number
- JP2022523766A5 JP2022523766A5 JP2021549291A JP2021549291A JP2022523766A5 JP 2022523766 A5 JP2022523766 A5 JP 2022523766A5 JP 2021549291 A JP2021549291 A JP 2021549291A JP 2021549291 A JP2021549291 A JP 2021549291A JP 2022523766 A5 JP2022523766 A5 JP 2022523766A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- pharmaceutical composition
- composition according
- coding sequence
- human
- 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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2022523766A JP2022523766A (ja) | 2022-04-26 |
| JP2022523766A5 true JP2022523766A5 (enExample) | 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 (enExample) |
| EP (1) | EP3927381A1 (enExample) |
| JP (2) | JP7637058B2 (enExample) |
| KR (1) | KR20210131370A (enExample) |
| CN (1) | CN113710281A (enExample) |
| AU (1) | AU2020225472A1 (enExample) |
| BR (1) | BR112021015817A2 (enExample) |
| CA (1) | CA3129672A1 (enExample) |
| CL (1) | CL2021002172A1 (enExample) |
| CO (1) | CO2021011040A2 (enExample) |
| IL (1) | IL285654A (enExample) |
| MX (1) | MX2021010134A (enExample) |
| PE (1) | PE20211819A1 (enExample) |
| SG (1) | SG11202108504YA (enExample) |
| TW (1) | TW202045730A (enExample) |
| WO (1) | WO2020172490A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020160458A1 (en) * | 2019-02-01 | 2020-08-06 | 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 |
| 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. |
| 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 |
| EP4720313A1 (en) * | 2023-05-26 | 2026-04-08 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating proteinopathies in central nervous system |
| US20250000992A1 (en) * | 2023-06-21 | 2025-01-02 | 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| 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 |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| 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 |
| 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 |
| 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 |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US8486635B2 (en) | 2006-05-30 | 2013-07-16 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| HUE031156T2 (en) | 2006-06-07 | 2017-06-28 | Genzyme Corp | 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) |
| HRP20250897T1 (hr) | 2009-05-02 | 2025-09-26 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| 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 |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| JP6312436B2 (ja) | 2010-11-16 | 2018-04-18 | ニューロダイン ライフ サイエンシズ インコーポレイテッドＮｅｕｒｏｄｙｎ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｉｎｃ． | ネプリライシンの発現および活性を増大させるための方法および医薬組成物 |
| 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 |
| CN107530447A (zh) * | 2015-02-10 | 2018-01-02 | 建新公司 | 病毒颗粒至纹状体和皮质的增强递送 |
| MX2018004755A (es) * | 2015-10-29 | 2018-12-19 | Voyager Therapeutics Inc | Entrega de polinucleotidos dirigidos al sistema nervioso central. |
| US20190060359A1 (en) | 2015-11-02 | 2019-02-28 | Neuraltus Pharmaceuticals, Inc. | Treatment of neurodegenerative disease with sodium chlorite |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| US20190328906A1 (en) | 2016-03-02 | 2019-10-31 | 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 |
| JP7610923B2 (ja) | 2016-09-02 | 2025-01-09 | スパーク セラピューティクス インコーポレイテッド | Ｃｎｓ障害を治療するための方法及びベクター |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| 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 |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| WO2020160458A1 (en) | 2019-02-01 | 2020-08-06 | 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 WO PCT/US2020/019149 patent/WO2020172490A1/en not_active Ceased
- 2020-02-21 EP EP20715985.6A patent/EP3927381A1/en active Pending
- 2020-02-21 TW TW109105680A patent/TW202045730A/zh unknown
- 2020-02-21 JP JP2021549291A patent/JP7637058B2/ja active Active
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/ko active Pending
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/zh active Pending
- 2020-02-21 AU AU2020225472A patent/AU2020225472A1/en active Pending
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/es unknown
- 2020-02-21 CA CA3129672A patent/CA3129672A1/en active Pending
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/es unknown
- 2020-02-21 US US17/431,937 patent/US12416017B2/en active Active
- 2020-02-21 SG SG11202108504YA patent/SG11202108504YA/en unknown
-
2021
- 2021-08-16 CL CL2021002172A patent/CL2021002172A1/es unknown
- 2021-08-16 IL IL285654A patent/IL285654A/en 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
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