JP7637058B2 - 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 - Google Patents

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 Download PDF

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JP7637058B2
JP7637058B2 JP2021549291A JP2021549291A JP7637058B2 JP 7637058 B2 JP7637058 B2 JP 7637058B2 JP 2021549291 A JP2021549291 A JP 2021549291A JP 2021549291 A JP2021549291 A JP 2021549291A JP 7637058 B2 JP7637058 B2 JP 7637058B2
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ヒンデラー，クリスチャン
ミラー，ニムロッド
ウイルソン，ジェームス・エム
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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
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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JP2021549291A 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 Active JP7637058B2 (ja)

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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

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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

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JP7637058B2 true JP7637058B2 (ja) 2025-02-27

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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

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US (2) US12416017B2 (https=)
EP (1) EP3927381A1 (https=)
JP (2) JP7637058B2 (https=)
KR (1) KR20210131370A (https=)
CN (1) CN113710281A (https=)
AU (1) AU2020225472A1 (https=)
BR (1) BR112021015817A2 (https=)
CA (1) CA3129672A1 (https=)
CL (1) CL2021002172A1 (https=)
CO (1) CO2021011040A2 (https=)
IL (1) IL285654A (https=)
MX (1) MX2021010134A (https=)
PE (1) PE20211819A1 (https=)
SG (1) SG11202108504YA (https=)
TW (1) TW202045730A (https=)
WO (1) WO2020172490A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023540054A (ja) * 2020-08-26 2023-09-21 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 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

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220111005A1 (en) * 2019-02-01 2022-04-14 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
BR112021015817A2 (pt) 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
IL292382A (en) * 2019-10-22 2022-06-01 Applied Genetic Tech Corporation Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders
US20240033332A1 (en) * 2020-12-17 2024-02-01 Kyoto University Agent for activating a neuron
EP4473123A4 (en) * 2022-02-01 2026-03-11 Shape Therapeutics Inc STABLE CELL LINES FOR INDUCIBLE VAAR VIRION PRODUCTION
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
TW202548022A (zh) * 2024-03-03 2025-12-16 美商帕西奇生物公司 用於治療神經退化性病症之重組腺相關病毒

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017151884A1 (en) 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia

Family Cites Families (45)

* Cited by examiner, † Cited by third party
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
WO1998046728A1 (en) 1997-04-14 1998-10-22 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant aav product
WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol 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
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
CA2653974A1 (en) 2006-05-30 2008-02-14 Mayo Foundation For Medical Education And Research Detecting and treating dementia
WO2007146046A2 (en) 2006-06-07 2007-12-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
WO2009089635A1 (en) 2008-01-16 2009-07-23 Neurodyn, Inc. Treating neurodegenerative diseases with progranulin (pgrn)
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
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
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US20150352185A1 (en) 2010-11-16 2015-12-10 Denis G. Kay Method for increasing neprilysin expression and activity
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
WO2015164723A1 (en) * 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
TN2017000353A1 (en) * 2015-02-10 2019-01-16 Genzyme Corp 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
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
EP3411488A1 (en) 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
US20210284701A1 (en) 2016-07-14 2021-09-16 Emory University Granulin Compositions and Uses Related Thereto
WO2018045347A1 (en) 2016-09-02 2018-03-08 Spark Therapeutics, Inc. Methods and vectors for treating cns disorders
JP7455579B2 (ja) 2017-02-28 2024-03-26 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 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
US20200283800A1 (en) 2017-10-23 2020-09-10 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
US20220111005A1 (en) 2019-02-01 2022-04-14 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
BR112021015817A2 (pt) 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn
EP4204014A1 (en) 2020-08-26 2023-07-05 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017151884A1 (en) 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
JP7436089B2 (ja) 2016-03-02 2024-02-21 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア 前頭側頭型認知症の治療

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Arrant, A. E. et al.，"Restoring neuronal progranulin reverses deficits in a mouse model of frontotemporal dementia"，Brain，2017年，Vol. 140，pp. 1447-1465
Yamazaki, Y. et al.，"Targeted gene transfer into ependymal cells through intraventricular injection of AAV1 vector and long-term enzyme replacement via the CSF"，Sci. Rep.，2014年，Vol. 4; 5506，pp. 1-7

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023540054A (ja) * 2020-08-26 2023-09-21 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 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

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EP3927381A1 (en) 2021-12-29
BR112021015817A2 (pt) 2021-10-13
AU2020225472A1 (en) 2021-08-26
PE20211819A1 (es) 2021-09-14
US12416017B2 (en) 2025-09-16
JP2022523766A (ja) 2022-04-26
MX2021010134A (es) 2021-09-23
CL2021002172A1 (es) 2022-02-11
CA3129672A1 (en) 2020-08-27
CN113710281A (zh) 2021-11-26
US20220136008A1 (en) 2022-05-05
IL285654A (en) 2021-09-30
CO2021011040A2 (es) 2021-09-09
JP2025081439A (ja) 2025-05-27
TW202045730A (zh) 2020-12-16
KR20210131370A (ko) 2021-11-02
WO2020172490A1 (en) 2020-08-27
SG11202108504YA (en) 2021-09-29
US20260049335A1 (en) 2026-02-19

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