JP2020537542A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020537542A5 JP2020537542A5 JP2020540681A JP2020540681A JP2020537542A5 JP 2020537542 A5 JP2020537542 A5 JP 2020537542A5 JP 2020540681 A JP2020540681 A JP 2020540681A JP 2020540681 A JP2020540681 A JP 2020540681A JP 2020537542 A5 JP2020537542 A5 JP 2020537542A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- itr
- sequence
- raav
- 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.)
- Pending
Links
- 108090000623 proteins and genes Proteins 0.000 claims description 6
- 150000007523 nucleic acids Chemical class 0.000 claims 31
- 108020004707 nucleic acids Proteins 0.000 claims 29
- 102000039446 nucleic acids Human genes 0.000 claims 29
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 26
- 239000013608 rAAV vector Substances 0.000 claims 20
- 241000702421 Dependoparvovirus Species 0.000 claims 14
- 230000002401 inhibitory effect Effects 0.000 claims 14
- 239000013598 vector Substances 0.000 claims 12
- 210000004027 cell Anatomy 0.000 claims 11
- 238000002347 injection Methods 0.000 claims 9
- 239000007924 injection Substances 0.000 claims 9
- 239000002773 nucleotide Substances 0.000 claims 7
- 125000003729 nucleotide group Chemical group 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
- 108700019146 Transgenes Proteins 0.000 claims 5
- 108091070501 miRNA Proteins 0.000 claims 4
- 239000002679 microRNA Substances 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108091026890 Coding region Proteins 0.000 claims 3
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 claims 3
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 3
- 102000003802 alpha-Synuclein Human genes 0.000 claims 3
- 108090000185 alpha-Synuclein Proteins 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
- 230000008685 targeting Effects 0.000 claims 3
- 241000701447 unidentified baculovirus Species 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 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
- 241000238631 Hexapoda Species 0.000 claims 1
- 208000009829 Lewy Body Disease Diseases 0.000 claims 1
- 201000002832 Lewy body dementia Diseases 0.000 claims 1
- 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
- 230000002068 genetic effect Effects 0.000 description 3
- 238000001415 gene therapy Methods 0.000 description 2
- 238000002560 therapeutic procedure Methods 0.000 description 1
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022007759A JP2022060228A (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2023192753A JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
| JP2024001041A JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762567305P | 2017-10-03 | 2017-10-03 | |
| US201762567303P | 2017-10-03 | 2017-10-03 | |
| US62/567,305 | 2017-10-03 | ||
| US62/567,303 | 2017-10-03 | ||
| PCT/US2018/054223 WO2019070891A1 (en) | 2017-10-03 | 2018-10-03 | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022007759A Division JP2022060228A (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2023192753A Division JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020537542A JP2020537542A (ja) | 2020-12-24 |
| JP2020537542A5 true JP2020537542A5 (enExample) | 2021-11-11 |
Family
ID=65995011
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020540681A Pending JP2020537542A (ja) | 2017-10-03 | 2018-10-03 | ライソゾーム病の遺伝子治療 |
| JP2022007759A Pending JP2022060228A (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2023192753A Pending JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
| JP2024001041A Pending JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022007759A Pending JP2022060228A (ja) | 2017-10-03 | 2022-01-21 | ライソゾーム病の遺伝子治療 |
| JP2023192753A Pending JP2024014935A (ja) | 2017-10-03 | 2023-11-13 | ライソゾーム病の遺伝子治療 |
| JP2024001041A Pending JP2024037189A (ja) | 2017-10-03 | 2024-01-09 | ライソゾーム病の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11807849B2 (enExample) |
| EP (1) | EP3692151A4 (enExample) |
| JP (4) | JP2020537542A (enExample) |
| KR (2) | KR20220010062A (enExample) |
| CN (2) | CN112501208A (enExample) |
| AU (3) | AU2018346102B2 (enExample) |
| BR (1) | BR112020006671A2 (enExample) |
| CA (1) | CA3078501A1 (enExample) |
| IL (2) | IL273768A (enExample) |
| MX (2) | MX2020004005A (enExample) |
| WO (1) | WO2019070891A1 (enExample) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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. |
| 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 |
| WO2021030522A1 (en) * | 2019-08-13 | 2021-02-18 | Alnylam Pharmaceuticals, Inc. | SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| 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 |
| MX2023001562A (es) | 2020-08-10 | 2023-05-03 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
| 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 |
| TWI910261B (zh) * | 2020-11-06 | 2026-01-01 | 日商住友製藥股份有限公司 | Ｒｐｓ２５基因之表現及／或功能調節劑 |
| US20250382611A1 (en) * | 2021-06-21 | 2025-12-18 | Uniqure Biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| WO2023278305A1 (en) * | 2021-06-28 | 2023-01-05 | The Board Of Regents Of The University Of Texas System | Methods and compositions for tau reduction gene therapy |
| CN113846122B (zh) * | 2021-08-16 | 2023-08-25 | 中国医学科学院医学生物学研究所 | 一种过表达snca的腺相关病毒载体aav-snca、制备方法及其应用 |
| US20250195695A1 (en) * | 2022-03-18 | 2025-06-19 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating tmem43 related cardiomyopathy with a viral vector |
| AU2024359425A1 (en) * | 2023-10-11 | 2026-05-07 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of microtubule associated protein tau (mapt) |
| TW202546230A (zh) * | 2024-01-26 | 2025-12-01 | 美商健臻公司 | 靶向ｓｎｃａ之人工ｍｉｃｒｏｒｎａ |
Family Cites Families (74)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US6521225B1 (en) * | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| 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 |
| 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 |
| SG10202108118RA (en) * | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| 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 |
| ES2562490T3 (es) | 2003-05-01 | 2016-03-04 | Genzyme Corporation | Terapia génica para trastornos neurometabólicos |
| 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 |
| WO2008019187A2 (en) | 2006-05-30 | 2008-02-14 | 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| 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 |
| 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 |
| WO2015060722A1 (en) | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| GB201403260D0 (en) * | 2014-02-25 | 2014-04-09 | Univ Manchester | Treatment of retinal degeneration using gene therapy |
| WO2016025523A1 (en) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| CN107106689A (zh) * | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| WO2016081927A2 (en) | 2014-11-21 | 2016-05-26 | University Of Florida Research Foundation, Inc. | Genome-modified recombinant adeno-associated virus vectors |
| 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 |
| 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 |
| 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 |
| 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 |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| BR112021010234A2 (pt) | 2018-11-28 | 2021-08-24 | Prevail Therapeutics, Inc. | Terapias de gene para doença neurodegenerativa |
| CA3134841A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US20220211871A1 (en) | 2019-04-10 | 2022-07-07 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US11999974B2 (en) | 2019-04-10 | 2024-06-04 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20220006527A (ko) | 2019-04-10 | 2022-01-17 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
-
2018
- 2018-10-03 CA CA3078501A patent/CA3078501A1/en active Pending
- 2018-10-03 JP JP2020540681A patent/JP2020537542A/ja active Pending
- 2018-10-03 EP EP18864256.5A patent/EP3692151A4/en active Pending
- 2018-10-03 BR BR112020006671-0A patent/BR112020006671A2/pt not_active Application Discontinuation
- 2018-10-03 MX MX2020004005A patent/MX2020004005A/es unknown
- 2018-10-03 WO PCT/US2018/054223 patent/WO2019070891A1/en not_active Ceased
- 2018-10-03 US US16/753,018 patent/US11807849B2/en active Active
- 2018-10-03 CN CN202011187145.9A patent/CN112501208A/zh active Pending
- 2018-10-03 KR KR1020227000760A patent/KR20220010062A/ko not_active Ceased
- 2018-10-03 CN CN201880075728.7A patent/CN111465691A/zh active Pending
- 2018-10-03 KR KR1020207012192A patent/KR20200078513A/ko not_active Ceased
- 2018-10-03 AU AU2018346102A patent/AU2018346102B2/en active Active
-
2020
- 2020-04-02 IL IL273768A patent/IL273768A/en unknown
- 2020-04-02 US US16/838,993 patent/US12049626B2/en active Active
- 2020-07-13 MX MX2020011748A patent/MX2020011748A/es unknown
- 2020-10-29 AU AU2020260476A patent/AU2020260476B2/en active Active
- 2020-12-22 IL IL279669A patent/IL279669A/en unknown
-
2022
- 2022-01-21 JP JP2022007759A patent/JP2022060228A/ja active Pending
-
2023
- 2023-08-11 AU AU2023214366A patent/AU2023214366B2/en active Active
- 2023-11-13 JP JP2023192753A patent/JP2024014935A/ja active Pending
-
2024
- 2024-01-09 JP JP2024001041A patent/JP2024037189A/ja active Pending
- 2024-06-14 US US18/743,624 patent/US20250011783A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020537543A5 (enExample) | ||
| JP2020537544A5 (enExample) | ||
| JP2022060228A5 (enExample) | ||
| US20220331451A1 (en) | Adeno-associated virus virions with variant capsid and methods of use thereof | |
| US11938197B2 (en) | Polynucleotides and vectors for the expression of transgenes | |
| CN109715650B (zh) | 新颖腺相关病毒衣壳蛋白 | |
| FI3684423T3 (fi) | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä | |
| JP2022508182A5 (enExample) | ||
| RU2021102893A (ru) | Модифицированные гены атаксии фридрейха и векторы для генной терапии | |
| CN101511373A (zh) | 用于基因治疗的修饰的因子viii和因子ix基因和载体 | |
| US20230049066A1 (en) | Novel aav3b variants that target human hepatocytes in the liver of humanized mice | |
| WO2022126189A1 (en) | Adeno-associated virus capsids and vectors | |
| KR20230019402A (ko) | 프로그래뉼린 연관 신경변성 질환 또는 장애의 치료를 위한 아데노-연관 바이러스 (aav) 시스템 | |
| TW202229315A (zh) | 改良的腺相關病毒(aav)載體及其用途 | |
| CN116096904A (zh) | 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途 | |
| JP2023539247A (ja) | Ｇｌｐ－１受容体アゴニスト融合物をコードするウイルスベクター及びネコの代謝性疾患の治療におけるその使用 | |
| CN120548368A (zh) | 用于正确包装重复元件的腺相关病毒载体 | |
| JP2021520811A5 (enExample) | ||
| CN119487183A (zh) | 用于生产重组细小病毒的组合物和方法 | |
| CN118414348A (zh) | 经修饰的aav衣壳和载体 | |
| CN119306807A (zh) | 一种变体AAV衣壳蛋白、重组腺相关病毒rAAV | |
| EP4582546A1 (en) | Adeno-associated virus structural plasmid capable of improving adeno-associated virus titer | |
| CN117836312A (zh) | 分离的修饰的aav9衣壳蛋白vp1 | |
| KR102874560B1 (ko) | 아데노연관바이러스 변이체 | |
| US20240425879A1 (en) | Compositions and methods for adeno-associated (aav) virus dnase expression |