JP2022526823A5 - - Google Patents
Info
- Publication number
- JP2022526823A5 JP2022526823A5 JP2021559727A JP2021559727A JP2022526823A5 JP 2022526823 A5 JP2022526823 A5 JP 2022526823A5 JP 2021559727 A JP2021559727 A JP 2021559727A JP 2021559727 A JP2021559727 A JP 2021559727A JP 2022526823 A5 JP2022526823 A5 JP 2022526823A5
- Authority
- JP
- Japan
- Prior art keywords
- approximately
- pharmaceutical composition
- raav
- composition according
- itr
- 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 |
|---|---|---|---|
| JP2024176952A JP7855045B2 (ja) | 2019-04-10 | 2024-10-09 | リソソーム障害のための遺伝子療法 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962831846P | 2019-04-10 | 2019-04-10 | |
| US62/831,846 | 2019-04-10 | ||
| US201962934450P | 2019-11-12 | 2019-11-12 | |
| US62/934,450 | 2019-11-12 | ||
| US201962954089P | 2019-12-27 | 2019-12-27 | |
| US62/954,089 | 2019-12-27 | ||
| US202062960471P | 2020-01-13 | 2020-01-13 | |
| US62/960,471 | 2020-01-13 | ||
| US202062988665P | 2020-03-12 | 2020-03-12 | |
| US62/988,665 | 2020-03-12 | ||
| PCT/US2020/027764 WO2020210698A1 (en) | 2019-04-10 | 2020-04-10 | Gene therapies for lysosomal disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024176952A Division JP7855045B2 (ja) | 2019-04-10 | 2024-10-09 | リソソーム障害のための遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022526823A JP2022526823A (ja) | 2022-05-26 |
| JP2022526823A5 true JP2022526823A5 (https=) | 2024-01-19 |
| JP7571044B2 JP7571044B2 (ja) | 2024-10-22 |
Family
ID=70554180
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021559727A Active JP7571044B2 (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 (20)
| Country | Link |
|---|---|
| US (2) | US11999974B2 (https=) |
| EP (2) | EP3953377B1 (https=) |
| JP (1) | JP7571044B2 (https=) |
| KR (1) | KR20210150486A (https=) |
| CN (1) | CN114174324A (https=) |
| AU (2) | AU2020273182B2 (https=) |
| BR (1) | BR112021020183A2 (https=) |
| CA (1) | CA3136004A1 (https=) |
| DK (1) | DK3953377T3 (https=) |
| ES (1) | ES3055985T3 (https=) |
| FI (1) | FI3953377T3 (https=) |
| HR (1) | HRP20251506T1 (https=) |
| IL (1) | IL286903A (https=) |
| LT (1) | LT3953377T (https=) |
| MX (1) | MX2021011958A (https=) |
| PL (1) | PL3953377T3 (https=) |
| PT (1) | PT3953377T (https=) |
| RS (1) | RS67476B1 (https=) |
| SI (1) | SI3953377T1 (https=) |
| WO (1) | WO2020210698A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3051839A1 (en) | 2017-02-17 | 2018-08-23 | Bristol-Myers Squibb Company | Antibodies to alpha-synuclein and uses thereof |
| 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 |
| CA3136004A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| AU2021325891A1 (en) * | 2020-08-10 | 2023-04-06 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| EP4511482A4 (en) * | 2022-04-19 | 2026-03-18 | Shanghai Vitalgen Biopharma Co Ltd | Recombinant AAV vectors for the treatment of neurodegenerative disorders |
| AU2023378899A1 (en) * | 2022-11-13 | 2025-06-26 | Alexion Pharma International Operations Limited | Gene therapy for frontotemporal dementia |
| WO2024178352A2 (en) * | 2023-02-24 | 2024-08-29 | The Board Of Trustees Of The Leland Stanford Junior University | Bmp synthase activators and inhibitors |
| WO2025251041A1 (en) * | 2024-05-31 | 2025-12-04 | Bluerock Therapeutics Lp | METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6538119B2 (en) | 1997-11-18 | 2003-03-25 | Abbott Laboratories | Reagents and methods useful for detecting diseases of the breast |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | 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 |
| AU2003221733A1 (en) | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
| WO2006036465A2 (en) | 2004-09-03 | 2006-04-06 | University Of Florida | Compositions and methods for treating cystic fibrosis |
| US8486635B2 (en) | 2006-05-30 | 2013-07-16 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| EP2687223B2 (en) | 2006-05-30 | 2024-11-20 | 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 |
| WO2008024998A2 (en) | 2006-08-24 | 2008-02-28 | Virovek, Inc. | Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor |
| US20090176729A1 (en) | 2007-12-14 | 2009-07-09 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| WO2009089635A1 (en) | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| US9265843B2 (en) | 2008-03-27 | 2016-02-23 | The Ohio State University | Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor |
| HRP20250897T1 (hr) | 2009-05-02 | 2025-09-26 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| 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 |
| AU2013337354A1 (en) | 2012-11-05 | 2015-05-21 | Genzyme Corporation | Compositions and methods for treating proteinopathies |
| CN115120746A (zh) | 2013-05-15 | 2022-09-30 | 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 |
| MA40460A (fr) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisation correspondante |
| US10900053B2 (en) | 2014-11-21 | 2021-01-26 | University Of Florida Research Foundation, Incorporated | Genome-modified recombinant adeno-associated virus vectors |
| CA2985235A1 (en) | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
| EP3411484B1 (en) | 2016-02-05 | 2023-10-04 | 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 |
| JP7496667B2 (ja) | 2016-04-21 | 2024-06-07 | ビロベク，インコーポレイテッド | 昆虫細胞中でのａａｖ生成、方法およびその組成物 |
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | 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 |
| WO2019094253A1 (en) * | 2017-11-08 | 2019-05-16 | Avexis Inc. | Means and method for preparing viral vectors and uses of same |
| CA3136004A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| WO2020210615A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP7664848B2 (ja) | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス，インコーポレーテッド | リソソーム障害のための遺伝子療法 |
| AU2021325891A1 (en) | 2020-08-10 | 2023-04-06 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
-
2020
- 2020-04-10 CA CA3136004A patent/CA3136004A1/en active Pending
- 2020-04-10 EP EP20724262.9A patent/EP3953377B1/en active Active
- 2020-04-10 PL PL20724262.9T patent/PL3953377T3/pl unknown
- 2020-04-10 LT LTEPPCT/US2020/027764T patent/LT3953377T/lt unknown
- 2020-04-10 CN CN202080041413.8A patent/CN114174324A/zh active Pending
- 2020-04-10 WO PCT/US2020/027764 patent/WO2020210698A1/en not_active Ceased
- 2020-04-10 PT PT207242629T patent/PT3953377T/pt unknown
- 2020-04-10 AU AU2020273182A patent/AU2020273182B2/en active Active
- 2020-04-10 ES ES20724262T patent/ES3055985T3/es active Active
- 2020-04-10 BR BR112021020183A patent/BR112021020183A2/pt unknown
- 2020-04-10 DK DK20724262.9T patent/DK3953377T3/da active
- 2020-04-10 RS RS20251192A patent/RS67476B1/sr unknown
- 2020-04-10 HR HRP20251506TT patent/HRP20251506T1/hr unknown
- 2020-04-10 US US16/846,065 patent/US11999974B2/en active Active
- 2020-04-10 EP EP25202531.7A patent/EP4707400A3/en active Pending
- 2020-04-10 MX MX2021011958A patent/MX2021011958A/es unknown
- 2020-04-10 SI SI202030683T patent/SI3953377T1/sl unknown
- 2020-04-10 FI FIEP20724262.9T patent/FI3953377T3/fi active
- 2020-04-10 JP JP2021559727A patent/JP7571044B2/ja active Active
- 2020-04-10 KR KR1020217036234A patent/KR20210150486A/ko active Pending
-
2021
- 2021-10-03 IL IL286903A patent/IL286903A/en unknown
-
2024
- 2024-04-26 US US18/646,957 patent/US12516298B2/en active Active
-
2026
- 2026-02-17 AU AU2026201184A patent/AU2026201184A1/en active Pending
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