JP2020537544A5 - - Google Patents
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- JP2020537544A5 JP2020537544A5 JP2020540683A JP2020540683A JP2020537544A5 JP 2020537544 A5 JP2020537544 A5 JP 2020537544A5 JP 2020540683 A JP2020540683 A JP 2020540683A JP 2020540683 A JP2020540683 A JP 2020540683A JP 2020537544 A5 JP2020537544 A5 JP 2020537544A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- itr
- raav
- expression construct
- nucleic acid
- 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.)
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- 150000007523 nucleic acids Chemical class 0.000 claims description 20
- 108020004707 nucleic acids Proteins 0.000 claims description 17
- 102000039446 nucleic acids Human genes 0.000 claims description 17
- 108090000623 proteins and genes Proteins 0.000 claims description 12
- 102000019204 Progranulins Human genes 0.000 claims description 8
- 108010012809 Progranulins Proteins 0.000 claims description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 8
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 26
- 239000013608 rAAV vector Substances 0.000 claims 16
- 241000702421 Dependoparvovirus Species 0.000 claims 13
- 239000013598 vector Substances 0.000 claims 12
- 239000002773 nucleotide Substances 0.000 claims 10
- 125000003729 nucleotide group Chemical group 0.000 claims 10
- 238000002347 injection Methods 0.000 claims 9
- 239000007924 injection Substances 0.000 claims 9
- 210000004027 cell Anatomy 0.000 claims 8
- 108700019146 Transgenes Proteins 0.000 claims 7
- 108090000565 Capsid Proteins Proteins 0.000 claims 6
- 102100023321 Ceruloplasmin Human genes 0.000 claims 6
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 6
- 210000003169 central nervous system Anatomy 0.000 claims 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 claims 3
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 3
- 108010006025 bovine growth hormone Proteins 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- 229940079593 drug Drugs 0.000 claims 3
- 239000003623 enhancer Substances 0.000 claims 3
- 239000013612 plasmid Substances 0.000 claims 3
- 230000001124 posttranscriptional effect Effects 0.000 claims 3
- 230000001105 regulatory effect Effects 0.000 claims 3
- 241000701447 unidentified baculovirus Species 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 238000007913 intrathecal administration Methods 0.000 claims 2
- 210000004962 mammalian cell Anatomy 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 230000002093 peripheral effect Effects 0.000 claims 2
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 239000001963 growth medium Substances 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 description 7
- 101710114165 Progranulin Proteins 0.000 description 4
- 230000002068 genetic effect Effects 0.000 description 3
- 102100037632 Progranulin Human genes 0.000 description 2
- 239000003795 chemical substances by application Substances 0.000 description 2
- 238000001415 gene therapy Methods 0.000 description 2
- 108091026890 Coding region Proteins 0.000 description 1
- 206010061218 Inflammation Diseases 0.000 description 1
- 230000004663 cell proliferation Effects 0.000 description 1
- 210000000349 chromosome Anatomy 0.000 description 1
- 230000018109 developmental process Effects 0.000 description 1
- 230000004054 inflammatory process Effects 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 230000007111 proteostasis Effects 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022007752A JP7389828B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2022007736A JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762567296P | 2017-10-03 | 2017-10-03 | |
| US201762567311P | 2017-10-03 | 2017-10-03 | |
| US201762567301P | 2017-10-03 | 2017-10-03 | |
| US201762567319P | 2017-10-03 | 2017-10-03 | |
| US201762567310P | 2017-10-03 | 2017-10-03 | |
| US62/567,310 | 2017-10-03 | ||
| US62/567,311 | 2017-10-03 | ||
| US62/567,319 | 2017-10-03 | ||
| US62/567,296 | 2017-10-03 | ||
| US62/567,301 | 2017-10-03 | ||
| PCT/US2018/054227 WO2019070894A1 (en) | 2017-10-03 | 2018-10-03 | GENE THERAPIES FOR LYSOSOMIAL DISORDERS |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022007752A Division JP7389828B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2022007736A Division JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020537544A JP2020537544A (ja) | 2020-12-24 |
| JP2020537544A5 true JP2020537544A5 (enExample) | 2021-11-11 |
| JP7361037B2 JP7361037B2 (ja) | 2023-10-13 |
Family
ID=65994362
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020540683A Active JP7361037B2 (ja) | 2017-10-03 | 2018-10-03 | ライソゾーム病の遺伝子治療 |
| JP2022007736A Active JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2022007752A Active JP7389828B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022007736A Active JP7336730B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2022007752A Active JP7389828B2 (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (5) | US11993790B2 (enExample) |
| EP (1) | EP3692158A4 (enExample) |
| JP (3) | JP7361037B2 (enExample) |
| KR (3) | KR102700963B1 (enExample) |
| CN (3) | CN112553210B (enExample) |
| AU (3) | AU2018346105C1 (enExample) |
| BR (1) | BR112020006661A2 (enExample) |
| CA (1) | CA3078464A1 (enExample) |
| IL (3) | IL273771B2 (enExample) |
| MX (3) | MX2020003965A (enExample) |
| WO (1) | WO2019070894A1 (enExample) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JOP20190248A1 (ar) | 2017-04-21 | 2019-10-20 | Amgen Inc | بروتينات ربط مولد ضد trem2 واستخداماته |
| TWI868807B (zh) | 2017-08-03 | 2025-01-01 | 美商阿列克特有限責任公司 | 抗trem2抗體及其使用方法 |
| CN112553210B (zh) | 2017-10-03 | 2024-09-24 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| BR112020006633A2 (pt) | 2017-10-03 | 2020-10-06 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| PL3850089T3 (pl) | 2019-02-04 | 2024-05-06 | Freeline Therapeutics Limited | Polinukleotydy |
| MX2021010134A (es) | 2019-02-22 | 2021-09-23 | Univ Pennsylvania | Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn. |
| SG11202108480UA (en) * | 2019-02-26 | 2021-09-29 | Univ Pennsylvania | Compositions useful in treatment of krabbe disease |
| KR20220006527A (ko) | 2019-04-10 | 2022-01-17 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
| US11999974B2 (en) * | 2019-04-10 | 2024-06-04 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| 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. |
| US20230021959A1 (en) * | 2019-12-05 | 2023-01-26 | The Trustees Of Columbia University In The City Of New York | Stabilization of Retromer for the Treatment of Alzheimer's Disease and Other Neurodegenerative Disorders |
| WO2021163681A2 (en) * | 2020-02-14 | 2021-08-19 | The Trustees Of Columbia University In The City Of New York | Combination of retromer pharmacological chaperones and exogenous retromer for the treatment of alzheimer's disease and other neurodegenerative diseases and disorders |
| US20230210941A1 (en) * | 2020-05-12 | 2023-07-06 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
| ES3057565T3 (en) * | 2020-07-23 | 2026-03-03 | Univ North Carolina Chapel Hill | Optimized slc13a5 genes and expression cassettes and their use |
| BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
| IL299811A (en) | 2020-07-29 | 2023-03-01 | Freeline Therapeutics Ltd | polypeptide |
| CN116113701A (zh) * | 2020-08-03 | 2023-05-12 | 普利维尔治疗公司 | 编码parkin的aav载体及其用途 |
| MX2023001562A (es) | 2020-08-10 | 2023-05-03 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
| CN116437968A (zh) * | 2020-08-10 | 2023-07-14 | 普利维尔治疗公司 | 用于神经退行性病症的基因疗法 |
| JP2023545462A (ja) | 2020-10-14 | 2023-10-30 | デナリ セラピューティクス インコーポレイテッド | 前頭側頭型認知症を治療及びモニタリングするための方法 |
| IL301858A (en) | 2020-10-15 | 2023-06-01 | Prevail Therapeutics Inc | Assay for measuring potency of gene therapy drug product |
| JP2023545830A (ja) * | 2020-10-15 | 2023-10-31 | プリベイル セラピューティクス，インコーポレーテッド | 組換えアデノ随伴ウイルス組成物及びその生産方法 |
| EP4284442A4 (en) * | 2021-02-01 | 2025-04-23 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR TREATING NIEMANN PICK DISEASE TYPE A |
| WO2022271944A2 (en) * | 2021-06-23 | 2022-12-29 | Avrobio, Inc. | Compositions and methods for treatment of gaucher disease |
| US20250049955A1 (en) | 2021-11-17 | 2025-02-13 | Voyager Therapeutics, Inc. | Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| JP1727698S (ja) * | 2021-12-20 | 2022-10-19 | ゴルフボールケース | |
| JP2025516136A (ja) | 2022-04-19 | 2025-05-27 | シャンハイ ヴァイタルゲン バイオファーマ カンパニー リミテッド | 神経変性疾患の治療のための組み換えａａｖベクター |
| EP4598940A1 (en) | 2022-10-04 | 2025-08-13 | Eli Lilly and Company | Gene therapy for trem2-associated diseases and disorders |
| JP2025534899A (ja) * | 2022-10-08 | 2025-10-21 | リンイ バイオテック カンパニー リミテッド | ＧＣａｓｅ欠乏関連疾患を治療するためのポリヌクレオチド |
| GB202216168D0 (en) | 2022-10-31 | 2022-12-14 | UCB Biopharma SRL | Route of administration |
| AU2023427408A1 (en) * | 2023-02-02 | 2025-09-04 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| JP2026509301A (ja) | 2023-03-14 | 2026-03-17 | ユーシービー バイオファルマ エスアールエル | 遺伝子治療 |
| WO2025039622A1 (zh) * | 2023-08-23 | 2025-02-27 | 上海金珂博生物技术有限公司 | 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途 |
| CN118086341B (zh) * | 2024-04-25 | 2024-08-02 | 上海凌医生物科技有限公司 | 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框 |
Family Cites Families (89)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS6182035A (ja) * | 1984-09-27 | 1986-04-25 | Toshiba Corp | 制振装置 |
| US5879680A (en) | 1987-12-23 | 1999-03-09 | The United States Of America As Represented By The Department Of Health And Human Services | Cloned DNA for synthesizing unique glucocerebrosidase |
| US5490559A (en) * | 1994-07-20 | 1996-02-13 | Dinulescu; Horia A. | Heat exchanger with finned partition walls |
| US20020068285A1 (en) * | 1996-01-11 | 2002-06-06 | Frudakis Tony N. | Compositions and methods for the therapy and diagnosis of breast cancer |
| US6521225B1 (en) | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| US6538119B2 (en) | 1997-11-18 | 2003-03-25 | Abbott Laboratories | Reagents and methods useful for detecting diseases of the breast |
| EP1112090A2 (en) | 1998-09-09 | 2001-07-04 | Myelos Corporation | Method of stimulating prosaposin receptor activity |
| AU6234099A (en) | 1998-10-16 | 2000-05-08 | Introgene B.V. | Gene therapy of alzheimer's disease by delivery of an encoded apoliprotein |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| US6696272B1 (en) | 1999-06-02 | 2004-02-24 | Hsc Research & Development Limited Partnership | Products and methods for gaucher disease therapy |
| DE19937800B4 (de) * | 1999-08-10 | 2005-06-16 | Gea Energietechnik Gmbh | Anlage zur Kondensation von Dampf |
| JP2003530835A (ja) | 2000-04-06 | 2003-10-21 | ケイオウエス ファーマシューティカルズ インコーポレイテッド | 高トリグリセリド血症を誘発することなくコレステロールレベルを低下するための化合物と方法 |
| GB0009887D0 (en) | 2000-04-20 | 2000-06-07 | Btg Int Ltd | Cytotoxic agents |
| AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | 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 |
| US7232670B2 (en) | 2001-09-28 | 2007-06-19 | St. Jude Children's Research Hospital | Targeting proteins to cells expressing mannose receptors via expression in insect cells |
| US20030133924A1 (en) | 2001-12-21 | 2003-07-17 | Novazyme Pharmaceuticals, Inc. | Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease |
| US20060292117A1 (en) | 2002-04-17 | 2006-12-28 | Loiler Scott A | Improved rAAv vectors |
| US6691742B1 (en) * | 2003-04-25 | 2004-02-17 | Thomas L. Cooper | Method and apparatus for supporting a pipe |
| ES2562490T3 (es) | 2003-05-01 | 2016-03-04 | Genzyme Corporation | Terapia génica para trastornos neurometabólicos |
| WO2005049676A1 (de) | 2003-11-21 | 2005-06-02 | Basf Aktiengesellschaft | Polyalkylenoxidgruppen und quartäre stickstoffatome enthaltende copolymere |
| CN101443046A (zh) * | 2004-06-23 | 2009-05-27 | 建新公司 | 治疗多囊病的方法和组合物 |
| WO2006036465A2 (en) | 2004-09-03 | 2006-04-06 | University Of Florida | Compositions and methods for treating cystic fibrosis |
| EP1799826B1 (en) | 2004-09-29 | 2009-08-12 | Children's Memorial Hospital | siRNA-MEDIATED GENE SILENCING OF ALPHA SYNUCLEIN |
| MX2007015602A (es) | 2005-06-08 | 2008-02-21 | Amicus Therapeutics Inc | Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas. |
| GB0605337D0 (en) | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
| EP2687223B2 (en) | 2006-05-30 | 2024-11-20 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| WO2008019187A2 (en) * | 2006-05-30 | 2008-02-14 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| CN101460621B (zh) * | 2006-06-07 | 2016-01-27 | 建新公司 | 肌萎缩性侧索硬化和其他脊髓失调症的基因治疗 |
| DE102006029773B3 (de) * | 2006-06-27 | 2007-07-12 | Gea Energietechnik Gmbh | Verfahren zur Errichtung einer Kondensationsanlage |
| CN101583621A (zh) * | 2006-07-20 | 2009-11-18 | 诺华有限公司 | 治疗、诊断或检测癌症的amigo-2抑制剂 |
| US20080249058A1 (en) | 2007-04-05 | 2008-10-09 | Erik Roberson | Agents that reduce neuronal overexcitation |
| US8962273B2 (en) | 2007-05-11 | 2015-02-24 | Genzyme Corporation | Methods of producing a secreted protein |
| US8454954B2 (en) | 2007-05-16 | 2013-06-04 | The Brigham And Women's Hospital, Inc. | Treatment of synucleinopathies |
| EP3127549B1 (en) * | 2007-06-22 | 2019-12-18 | Children's Medical Center Corporation | Methods and uses thereof of a fragment of saposin a |
| WO2009079399A2 (en) | 2007-12-14 | 2009-06-25 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| EP2249861B1 (en) | 2008-01-16 | 2016-07-06 | Neurodyn Life Sciences Inc. | Progranulin for use in treating parkinson's disease or alzheimer's disease |
| 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 |
| 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 |
| US9255266B2 (en) | 2009-05-06 | 2016-02-09 | Rutgers, The State University Of New Jersey | RNA targeting in alpha-synucleinopathies |
| CN102858985A (zh) * | 2009-07-24 | 2013-01-02 | 西格马-奥尔德里奇有限责任公司 | 基因组编辑方法 |
| EP2513137B1 (en) * | 2009-12-17 | 2018-02-28 | Children's Medical Center Corporation | Saposin-a derived peptides and uses thereof |
| EP3567106A1 (en) | 2010-04-23 | 2019-11-13 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| US9290759B2 (en) | 2010-08-25 | 2016-03-22 | The Trustees Of Columbia University In The City Of New York | Optimized miRNA constructs |
| WO2012027713A2 (en) | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
| CN107828820B (zh) | 2010-10-27 | 2022-06-07 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
| WO2012065248A1 (en) | 2010-11-16 | 2012-05-24 | Kay Denis G | Method for increasing neprilysin expression and activity |
| CN103842362B (zh) | 2011-05-09 | 2017-05-24 | 爱普制药有限责任公司 | 用于治疗阿尔茨海默氏病的组合物和方法 |
| WO2013151663A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| EP2850195B1 (en) | 2012-05-18 | 2020-01-22 | University of Iowa Research Foundation | Methods and compositions for treating amyloid deposits |
| CA2875618C (en) | 2012-07-11 | 2021-04-27 | Sangamo Biosciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| BR112015009746A2 (pt) * | 2012-11-05 | 2017-08-15 | Genzyme Corp | Agente que aumenta atividade de glicocerebrosidase em mamíferos |
| US9828587B2 (en) | 2013-04-08 | 2017-11-28 | University Of Iowa Research Foundation | Chimeric adeno-associated virus/ bocavirus parvovirus vector |
| KR20160010526A (ko) | 2013-05-15 | 2016-01-27 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
| US9347107B2 (en) | 2013-05-23 | 2016-05-24 | Norman Z Lai | Vector containing multiple nucleotide sequences for the expression of enzymes |
| US9951330B2 (en) | 2013-07-11 | 2018-04-24 | The Trustees Of Columbia University In The City Of New York | Micrornas that silence tau expression |
| US9574184B2 (en) | 2013-09-25 | 2017-02-21 | Children's Hospital Medical Center | Lysosomal protein targeting sequence and therapeutic applications of same |
| SG10201913611QA (en) * | 2014-08-08 | 2020-03-30 | Alector Llc | Anti-trem2 antibodies and methods of use thereof |
| WO2016025523A1 (en) * | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| WO2016036403A1 (en) * | 2014-09-05 | 2016-03-10 | Population Diagnostics Inc. | Methods and compositions for inhibiting and treating neurological conditions |
| WO2016081927A2 (en) * | 2014-11-21 | 2016-05-26 | University Of Florida Research Foundation, Inc. | Genome-modified recombinant adeno-associated virus vectors |
| ES2714130T3 (es) * | 2015-02-02 | 2019-05-27 | Procter & Gamble | Composición detergente |
| US10513701B2 (en) | 2015-03-25 | 2019-12-24 | Università Degli Studi Di Trento | RNA interference mediated therapy for neurodegenerative diseases |
| US20170035860A1 (en) | 2015-04-02 | 2017-02-09 | Alexander C. Flynn | Compositions and methods for treatment of neurogenerative diseases |
| EP4491733A3 (en) * | 2015-04-23 | 2025-03-26 | University of Massachusetts | Modulation of aav vector transgene expression |
| WO2016179497A1 (en) | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
| EP3091087A1 (en) | 2015-05-08 | 2016-11-09 | Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol | Method for in vitro diagnosis of synucleinopathies using alpha-synuclein gene transcripts |
| CA2986252A1 (en) | 2015-05-15 | 2016-11-24 | Regents Of The University Of Minnesota | Adeno-associated virus vector for delivery of iduronate-2-sulfatase to the central nervous system |
| EP3356507A1 (en) * | 2015-10-02 | 2018-08-08 | The Procter and Gamble Company | Cleaning pad, cleaning implement, and a method of improving shine of a hard surface using the cleaning pad or cleaning implement with a cleaning composition |
| WO2017075338A2 (en) | 2015-10-29 | 2017-05-04 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| PE20181156A1 (es) | 2015-11-05 | 2018-07-19 | Bamboo Therapeutics Inc | Genes de ataxia de friedreich modificados y vectores para terapia genica |
| WO2017136536A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| US12070510B2 (en) | 2016-02-05 | 2024-08-27 | Emory University | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid |
| US11066456B2 (en) | 2016-02-25 | 2021-07-20 | Washington University | Compositions comprising TREM2 and methods of use thereof |
| US20190328906A1 (en) | 2016-03-02 | 2019-10-31 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| ES2802454T3 (es) * | 2016-04-08 | 2021-01-19 | Procter & Gamble | Composición de limpieza para lavavajillas |
| TWI832036B (zh) | 2017-08-03 | 2024-02-11 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| BR112020006633A2 (pt) | 2017-10-03 | 2020-10-06 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN112553210B (zh) | 2017-10-03 | 2024-09-24 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| CN111819281B (zh) | 2017-10-23 | 2024-12-31 | 普利维尔治疗公司 | 用于神经变性疾病的基因疗法 |
| BR112021010234A2 (pt) | 2018-11-28 | 2021-08-24 | Prevail Therapeutics, Inc. | Terapias de gene para doença neurodegenerativa |
| US11999974B2 (en) | 2019-04-10 | 2024-06-04 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US20220211871A1 (en) | 2019-04-10 | 2022-07-07 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20220006527A (ko) | 2019-04-10 | 2022-01-17 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
| CA3134841A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN116437968A (zh) | 2020-08-10 | 2023-07-14 | 普利维尔治疗公司 | 用于神经退行性病症的基因疗法 |
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