JPWO2020172490A5 - - Google Patents
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- Publication number
- JPWO2020172490A5 JPWO2020172490A5 JP2021549291A JP2021549291A JPWO2020172490A5 JP WO2020172490 A5 JPWO2020172490 A5 JP WO2020172490A5 JP 2021549291 A JP2021549291 A JP 2021549291A JP 2021549291 A JP2021549291 A JP 2021549291A JP WO2020172490 A5 JPWO2020172490 A5 JP WO2020172490A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- progranulin
- pharmaceutical composition
- composition
- vector genome
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 12
- 241000702421 Dependoparvovirus Species 0.000 claims 9
- 239000000203 mixture Substances 0.000 claims 9
- 101001027324 Homo sapiens Progranulin Proteins 0.000 claims 8
- 102000019204 Progranulins Human genes 0.000 claims 8
- 108010012809 Progranulins Proteins 0.000 claims 8
- 108091026890 Coding region Proteins 0.000 claims 7
- 238000000034 method Methods 0.000 claims 6
- 102000054121 human GRN Human genes 0.000 claims 5
- 230000001105 regulatory effect Effects 0.000 claims 5
- 210000000234 capsid Anatomy 0.000 claims 4
- 230000004770 neurodegeneration Effects 0.000 claims 4
- 102100037632 Progranulin Human genes 0.000 claims 3
- 210000004027 cell Anatomy 0.000 claims 3
- 210000003169 central nervous system Anatomy 0.000 claims 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 208000036632 Brain mass Diseases 0.000 claims 2
- 206010051290 Central nervous system lesion Diseases 0.000 claims 2
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 2
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 2
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 2
- 238000002591 computed tomography Methods 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 239000013612 plasmid Substances 0.000 claims 2
- 239000004094 surface-active agent Substances 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 108060003393 Granulin Proteins 0.000 claims 1
- 102100027377 HBS1-like protein Human genes 0.000 claims 1
- 101001009070 Homo sapiens HBS1-like protein Proteins 0.000 claims 1
- RVGRUAULSDPKGF-UHFFFAOYSA-N Poloxamer Chemical group C1CO1.CC1CO1 RVGRUAULSDPKGF-UHFFFAOYSA-N 0.000 claims 1
- 238000009825 accumulation Methods 0.000 claims 1
- 150000001413 amino acids Chemical class 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 210000003703 cisterna magna Anatomy 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 102000018146 globin Human genes 0.000 claims 1
- 108060003196 globin Proteins 0.000 claims 1
- 102000017941 granulin Human genes 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000007914 intraventricular administration Methods 0.000 claims 1
- 230000003902 lesion Effects 0.000 claims 1
- 239000007788 liquid Substances 0.000 claims 1
- 238000002595 magnetic resonance imaging Methods 0.000 claims 1
- YIEDSISPYKQADU-UHFFFAOYSA-N n-acetyl-n-[2-methyl-4-[(2-methylphenyl)diazenyl]phenyl]acetamide Chemical compound C1=C(C)C(N(C(C)=O)C(=O)C)=CC=C1N=NC1=CC=CC=C1C YIEDSISPYKQADU-UHFFFAOYSA-N 0.000 claims 1
- 229920001993 poloxamer 188 Polymers 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025022078A JP2025081439A (ja) | 2019-02-22 | 2025-02-14 | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962809329P | 2019-02-22 | 2019-02-22 | |
| US62/809,329 | 2019-02-22 | ||
| US201962923812P | 2019-10-21 | 2019-10-21 | |
| US62/923,812 | 2019-10-21 | ||
| US202062969108P | 2020-02-02 | 2020-02-02 | |
| US62/969,108 | 2020-02-02 | ||
| PCT/US2020/019149 WO2020172490A1 (en) | 2019-02-22 | 2020-02-21 | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025022078A Division JP2025081439A (ja) | 2019-02-22 | 2025-02-14 | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022523766A JP2022523766A (ja) | 2022-04-26 |
| JPWO2020172490A5 true JPWO2020172490A5 (enExample) | 2023-02-17 |
| JP7637058B2 JP7637058B2 (ja) | 2025-02-27 |
Family
ID=70058449
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549291A Active JP7637058B2 (ja) | 2019-02-22 | 2020-02-21 | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
| JP2025022078A Pending JP2025081439A (ja) | 2019-02-22 | 2025-02-14 | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025022078A Pending JP2025081439A (ja) | 2019-02-22 | 2025-02-14 | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12416017B2 (enExample) |
| EP (1) | EP3927381A1 (enExample) |
| JP (2) | JP7637058B2 (enExample) |
| KR (1) | KR20210131370A (enExample) |
| CN (1) | CN113710281A (enExample) |
| AU (1) | AU2020225472A1 (enExample) |
| BR (1) | BR112021015817A2 (enExample) |
| CA (1) | CA3129672A1 (enExample) |
| CL (1) | CL2021002172A1 (enExample) |
| CO (1) | CO2021011040A2 (enExample) |
| IL (1) | IL285654A (enExample) |
| MX (1) | MX2021010134A (enExample) |
| PE (1) | PE20211819A1 (enExample) |
| SG (1) | SG11202108504YA (enExample) |
| TW (1) | TW202045730A (enExample) |
| WO (1) | WO2020172490A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3128003A1 (en) * | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| KR20210131370A (ko) | 2019-02-22 | 2021-11-02 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스 |
| GB201913974D0 (en) * | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| KR20230019402A (ko) * | 2019-10-22 | 2023-02-08 | 어플라이드 제네틱스 테크놀로지스 코퍼레이션 | 프로그래뉼린 연관 신경변성 질환 또는 장애의 치료를 위한 아데노-연관 바이러스 (aav) 시스템 |
| US20230364264A1 (en) * | 2020-08-26 | 2023-11-16 | The Trustees Of The University Of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| US20240033332A1 (en) * | 2020-12-17 | 2024-02-01 | Kyoto University | Agent for activating a neuron |
| US20250109410A1 (en) * | 2022-02-01 | 2025-04-03 | Shape Therapeutics Inc. | Stable Cell Lines for Inducible Production of rAAV Virions |
| WO2024245120A1 (en) * | 2023-05-26 | 2024-12-05 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating proteinopathies in central nervous system |
| WO2024263567A2 (en) * | 2023-06-21 | 2024-12-26 | Eli Lilly And Company | Compounds for the delivery of granulin across the blood brain barrier |
| WO2025188625A1 (en) * | 2024-03-03 | 2025-09-12 | Passage Bio, Inc. | Recombinant adeno-associated virus for treatment of neurodegenerative disorders |
Family Cites Families (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| DE69824859T2 (de) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| DE69941905D1 (de) | 1998-11-10 | 2010-02-25 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| EP1286703B1 (en) | 2000-06-01 | 2009-08-05 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| NZ618298A (en) | 2001-11-13 | 2015-04-24 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP2292780B1 (en) | 2003-09-30 | 2017-08-23 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2359865B1 (en) | 2005-04-07 | 2013-10-02 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| JP5123936B2 (ja) | 2006-05-30 | 2013-01-23 | メイヨ・ファウンデーション・フォー・メディカル・エデュケーション・アンド・リサーチ | 認知症の検出および治療 |
| PL2489733T3 (pl) | 2006-06-07 | 2019-08-30 | Genzyme Corporation | Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego |
| CN102006882B (zh) | 2008-01-16 | 2017-06-06 | 神经动力公司 | 利用颗粒体蛋白前体(pgrn)治疗神经变性疾病 |
| DK3421603T3 (da) | 2009-05-02 | 2022-01-10 | Genzyme Corp | Genterapi for neurodegenerative forstyrrelser |
| US20110203007A1 (en) | 2010-02-16 | 2011-08-18 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Assays of neurodegenerative disorders, including frontotemporal dementia and amyotrophic lateral sclerosis |
| SG183929A1 (en) | 2010-03-29 | 2012-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2640407A4 (en) | 2010-11-16 | 2014-07-09 | Denis G Kay | PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| JP6983511B2 (ja) * | 2014-04-25 | 2021-12-17 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 脳中の転移乳癌および他の癌を処置するための方法および組成物 |
| CA2976082A1 (en) * | 2015-02-10 | 2016-08-18 | Genzyme Corporation | Enhanced delivery of viral particles to the striatum and cortex |
| WO2017075338A2 (en) * | 2015-10-29 | 2017-05-04 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| US20190060359A1 (en) | 2015-11-02 | 2019-02-28 | Neuraltus Pharmaceuticals, Inc. | Treatment of neurodegenerative disease with sodium chlorite |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| ES2918998T3 (es) | 2015-12-11 | 2022-07-21 | Univ Pennsylvania | Método de purificación escalable para AAVrh10 |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| WO2017151884A1 (en) * | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| WO2018013775A2 (en) | 2016-07-14 | 2018-01-18 | Emory University | Granulin compositions and uses related thereto |
| MX2019002518A (es) | 2016-09-02 | 2019-07-18 | Spark Therapeutics Inc | Metodos y vectores para el tratamiento de trastornos del snc. |
| WO2018160582A1 (en) | 2017-02-28 | 2018-09-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
| CN111465691A (zh) | 2017-10-03 | 2020-07-28 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| CA3128003A1 (en) | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| KR20210131370A (ko) | 2019-02-22 | 2021-11-02 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스 |
| US20230364264A1 (en) | 2020-08-26 | 2023-11-16 | The Trustees Of The University Of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
-
2020
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/ko active Pending
- 2020-02-21 WO PCT/US2020/019149 patent/WO2020172490A1/en not_active Ceased
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/es unknown
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/es unknown
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/zh active Pending
- 2020-02-21 AU AU2020225472A patent/AU2020225472A1/en active Pending
- 2020-02-21 SG SG11202108504YA patent/SG11202108504YA/en unknown
- 2020-02-21 TW TW109105680A patent/TW202045730A/zh unknown
- 2020-02-21 CA CA3129672A patent/CA3129672A1/en active Pending
- 2020-02-21 EP EP20715985.6A patent/EP3927381A1/en active Pending
- 2020-02-21 BR BR112021015817-0A patent/BR112021015817A2/pt unknown
- 2020-02-21 JP JP2021549291A patent/JP7637058B2/ja active Active
- 2020-02-21 US US17/431,937 patent/US12416017B2/en active Active
-
2021
- 2021-08-16 IL IL285654A patent/IL285654A/en unknown
- 2021-08-16 CL CL2021002172A patent/CL2021002172A1/es unknown
- 2021-08-23 CO CONC2021/0011040A patent/CO2021011040A2/es unknown
-
2025
- 2025-02-14 JP JP2025022078A patent/JP2025081439A/ja active Pending
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