JP2021500049A5 - - Google Patents
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- Publication number
- JP2021500049A5 JP2021500049A5 JP2020523011A JP2020523011A JP2021500049A5 JP 2021500049 A5 JP2021500049 A5 JP 2021500049A5 JP 2020523011 A JP2020523011 A JP 2020523011A JP 2020523011 A JP2020523011 A JP 2020523011A JP 2021500049 A5 JP2021500049 A5 JP 2021500049A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- isolated nucleic
- vector
- set forth
- 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 |
|---|---|---|---|
| JP2023220260A JP2024045131A (ja) | 2017-10-23 | 2023-12-27 | 神経変性疾患の遺伝子治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762575795P | 2017-10-23 | 2017-10-23 | |
| US62/575,795 | 2017-10-23 | ||
| US201862742723P | 2018-10-08 | 2018-10-08 | |
| US62/742,723 | 2018-10-08 | ||
| PCT/US2018/057187 WO2019084068A1 (en) | 2017-10-23 | 2018-10-23 | GENE THERAPIES FOR NEURODEGENERATIVE DISEASE |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023220260A Division JP2024045131A (ja) | 2017-10-23 | 2023-12-27 | 神経変性疾患の遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021500049A JP2021500049A (ja) | 2021-01-07 |
| JP2021500049A5 true JP2021500049A5 (https=) | 2021-11-25 |
| JP7413256B2 JP7413256B2 (ja) | 2024-01-15 |
Family
ID=66247008
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020523011A Active JP7413256B2 (ja) | 2017-10-23 | 2018-10-23 | 神経変性疾患の遺伝子治療 |
| JP2023220260A Pending JP2024045131A (ja) | 2017-10-23 | 2023-12-27 | 神経変性疾患の遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023220260A Pending JP2024045131A (ja) | 2017-10-23 | 2023-12-27 | 神経変性疾患の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20210261981A1 (https=) |
| EP (1) | EP3701030A4 (https=) |
| JP (2) | JP7413256B2 (https=) |
| KR (1) | KR20200075865A (https=) |
| CN (2) | CN113005123A (https=) |
| AU (1) | AU2018354195A1 (https=) |
| BR (1) | BR112020008033A2 (https=) |
| CA (2) | CA3177979A1 (https=) |
| IL (2) | IL274129A (https=) |
| MX (1) | MX2020004207A (https=) |
| WO (1) | WO2019084068A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| MA54792A (fr) * | 2018-03-09 | 2021-12-01 | Avrobio Inc | Compositions et méthodes pour le traitement de la maladie de parkinson |
| PL3953377T3 (pl) | 2019-04-10 | 2026-02-16 | Prevail Therapeutics, Inc. | Terapie genowe zaburzeń lizosomalnych |
| CA3136117A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| MX2022004771A (es) * | 2019-10-22 | 2022-10-07 | Applied Genetic Tech Corporation | Vectores de virus adeno-asociados (aav) de triple funcion para el tratamiento de enfermedades asociadas a c9orf72. |
| US20240294906A1 (en) * | 2020-07-29 | 2024-09-05 | Alnylam Pharmaceuticals, Inc. | Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases |
| KR20230088393A (ko) | 2020-10-15 | 2023-06-19 | 프리베일 테라퓨틱스, 인크. | 재조합 아데노 연관 바이러스 조성물 및 이의 제조 방법 |
| US20240044869A1 (en) | 2020-10-15 | 2024-02-08 | Prevail Therapeutics, Inc. | Assay for measuring potency of gene therapy drug product |
| EP4252846A4 (en) | 2020-11-06 | 2025-12-03 | Sumitomo Pharma Co Ltd | REGULATOR FOR THE EXPRESSION AND/OR FUNCTION OF THE RPS25 GENE |
| WO2022164260A1 (ko) | 2021-01-28 | 2022-08-04 | 주식회사 에이브레인 | 신경 퇴행성 질환의 치료를 위한 유전자 요법 |
| CN117043179A (zh) | 2021-01-28 | 2023-11-10 | 爱博瑞株式会社 | 用于治疗神经退行性疾病的基因治疗 |
| KR102707250B1 (ko) | 2021-10-28 | 2024-09-13 | 한국해양과학기술원 | 신규 화합물 및 이를 유효성분으로 포함하는 신경염증성 뇌질환 예방 또는 치료용 조성물 |
| AR128239A1 (es) * | 2022-01-10 | 2024-04-10 | Univ Pennsylvania | Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72 |
| WO2023202637A1 (en) | 2022-04-19 | 2023-10-26 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating neurodegenerative disorders |
| TW202511485A (zh) | 2023-06-16 | 2025-03-16 | 日商住友製藥股份有限公司 | 調節ｒｐｓ２５基因之表現及／或其功能的反義寡核苷酸 |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6204059B1 (en) * | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US20040053876A1 (en) * | 2002-03-26 | 2004-03-18 | The Regents Of The University Of Michigan | siRNAs and uses therof |
| US7829694B2 (en) * | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| WO2004069173A2 (en) * | 2003-01-31 | 2004-08-19 | The Trustees Of The University Of Pennsylvania | Methods for modulating an inflammatory response |
| EP1844148A2 (en) * | 2005-01-31 | 2007-10-17 | University of Iowa Research Foundation | Nucleic acid silencing of huntington's disease gene |
| JP5559185B2 (ja) * | 2008-11-11 | 2014-07-23 | オックスフォード バイオメディカ（ユーケー）リミテッド | 方法 |
| US20110142789A1 (en) * | 2009-12-10 | 2011-06-16 | The Trustees Of The University Of Pennsylvania | Compositions and Methods for the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| SMT201700339T1 (it) * | 2012-04-23 | 2017-09-07 | Biomarin Tech Bv | Oligonucleotidi di modulazione dell'rna con caratteristiche migliorate per il trattamento dei disturbi neuromuscolari |
| 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 |
| US20140271550A1 (en) * | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| EP4166667A3 (en) * | 2013-10-11 | 2023-08-02 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| EP4410805A3 (en) * | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| CN106103717A (zh) * | 2014-03-19 | 2016-11-09 | Ionis制药公司 | 用于调节共济失调蛋白2表达的组合物 |
| WO2015153760A2 (en) * | 2014-04-01 | 2015-10-08 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a nervous system disorder |
| WO2016024205A1 (en) * | 2014-08-15 | 2016-02-18 | Pfizer Inc. | Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene |
| CA3193811A1 (en) * | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CA3019315A1 (en) * | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| EP3292206B8 (en) * | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| WO2017151884A1 (en) * | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| JP7026678B2 (ja) * | 2016-09-30 | 2022-02-28 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Ｃ９ｏｒｆ７２座位中にヘキサヌクレオチドリピート伸長を有する非ヒト動物 |
| EP3850098A1 (en) * | 2018-09-12 | 2021-07-21 | uniQure IP B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
-
2018
- 2018-10-23 AU AU2018354195A patent/AU2018354195A1/en not_active Abandoned
- 2018-10-23 WO PCT/US2018/057187 patent/WO2019084068A1/en not_active Ceased
- 2018-10-23 US US16/758,077 patent/US20210261981A1/en not_active Abandoned
- 2018-10-23 EP EP18870021.5A patent/EP3701030A4/en not_active Withdrawn
- 2018-10-23 CA CA3177979A patent/CA3177979A1/en active Pending
- 2018-10-23 BR BR112020008033-0A patent/BR112020008033A2/pt not_active Application Discontinuation
- 2018-10-23 JP JP2020523011A patent/JP7413256B2/ja active Active
- 2018-10-23 CN CN202110196181.XA patent/CN113005123A/zh active Pending
- 2018-10-23 CA CA3083582A patent/CA3083582A1/en active Pending
- 2018-10-23 KR KR1020207014683A patent/KR20200075865A/ko active Pending
- 2018-10-23 MX MX2020004207A patent/MX2020004207A/es unknown
- 2018-10-23 CN CN201880081355.4A patent/CN111819281B/zh active Active
-
2020
- 2020-04-22 IL IL274129A patent/IL274129A/en unknown
-
2021
- 2021-02-23 IL IL281057A patent/IL281057A/en unknown
-
2023
- 2023-12-27 JP JP2023220260A patent/JP2024045131A/ja active Pending
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