BR112020008033A2 - terapias gênicas para doença neurodegenerativa - Google Patents
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Applications Claiming Priority (5)
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| 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 |
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| BR112020008033A2 true BR112020008033A2 (pt) | 2020-10-27 |
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| EP (1) | EP3701030A4 (https=) |
| JP (2) | JP7413256B2 (https=) |
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| CA (2) | CA3177979A1 (https=) |
| IL (2) | IL274129A (https=) |
| MX (1) | MX2020004207A (https=) |
| WO (1) | WO2019084068A1 (https=) |
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| 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 |
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| US6204059B1 (en) * | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
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| 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 |
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2018
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- 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
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2020
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2021
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2023
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| CA3083582A1 (en) | 2019-05-02 |
| EP3701030A4 (en) | 2022-04-20 |
| JP2021500049A (ja) | 2021-01-07 |
| US20210261981A1 (en) | 2021-08-26 |
| CN111819281B (zh) | 2024-12-31 |
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| CN111819281A (zh) | 2020-10-23 |
| CN113005123A (zh) | 2021-06-22 |
| MX2020004207A (es) | 2020-11-11 |
| JP7413256B2 (ja) | 2024-01-15 |
| WO2019084068A1 (en) | 2019-05-02 |
| KR20200075865A (ko) | 2020-06-26 |
| JP2024045131A (ja) | 2024-04-02 |
| IL274129A (en) | 2020-06-30 |
| AU2018354195A1 (en) | 2020-06-04 |
| CA3177979A1 (en) | 2019-05-02 |
| EP3701030A1 (en) | 2020-09-02 |
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