JP2023540054A5 - - Google Patents

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Publication number
JP2023540054A5
JP2023540054A5 JP2023513708A JP2023513708A JP2023540054A5 JP 2023540054 A5 JP2023540054 A5 JP 2023540054A5 JP 2023513708 A JP2023513708 A JP 2023513708A JP 2023513708 A JP2023513708 A JP 2023513708A JP 2023540054 A5 JP2023540054 A5 JP 2023540054A5
Authority
JP
Japan
Prior art keywords
approximately
pharmaceutical composition
grams
progranulin
brain mass
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
Application number
JP2023513708A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023540054A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/047686 external-priority patent/WO2022046988A1/en
Publication of JP2023540054A publication Critical patent/JP2023540054A/ja
Publication of JP2023540054A5 publication Critical patent/JP2023540054A5/ja
Pending legal-status Critical Current

Links

JP2023513708A 2020-08-26 2021-08-26 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 Pending JP2023540054A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063070639P 2020-08-26 2020-08-26
US63/070,639 2020-08-26
PCT/US2021/047686 WO2022046988A1 (en) 2020-08-26 2021-08-26 Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration

Publications (2)

Publication Number Publication Date
JP2023540054A JP2023540054A (ja) 2023-09-21
JP2023540054A5 true JP2023540054A5 (https=) 2024-08-30

Family

ID=78032503

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023513708A Pending JP2023540054A (ja) 2020-08-26 2021-08-26 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 (13)

Country Link
US (1) US20230364264A1 (https=)
EP (1) EP4204014A1 (https=)
JP (1) JP2023540054A (https=)
KR (1) KR20230058102A (https=)
CN (1) CN116887867A (https=)
AR (1) AR123358A1 (https=)
AU (1) AU2021332331A1 (https=)
BR (1) BR112023002904A2 (https=)
CA (1) CA3189107A1 (https=)
IL (1) IL300622A (https=)
MX (1) MX2023002364A (https=)
TW (1) TW202227633A (https=)
WO (1) WO2022046988A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20250295807A1 (en) * 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2024035649A1 (en) * 2022-08-08 2024-02-15 Shape Therapeutics Inc. Compositions for progranulin expression and methods of use thereof
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration
WO2024191873A1 (en) * 2023-03-10 2024-09-19 Biogen Ma Inc. Packaging plasmids for aav production
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
WO2025231406A1 (en) * 2024-05-02 2025-11-06 The Children's Hospital Of Philadelphia Methods to increase transduction of ependyma cells in brain

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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
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
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
US6548286B1 (en) 1997-04-14 2003-04-15 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
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
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
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
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
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US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
CA3012195A1 (en) * 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
US20190328906A1 (en) * 2016-03-02 2019-10-31 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
PT3589730T (pt) 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações
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

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