JP2020506695A5 - - Google Patents

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Publication number
JP2020506695A5
JP2020506695A5 JP2019540667A JP2019540667A JP2020506695A5 JP 2020506695 A5 JP2020506695 A5 JP 2020506695A5 JP 2019540667 A JP2019540667 A JP 2019540667A JP 2019540667 A JP2019540667 A JP 2019540667A JP 2020506695 A5 JP2020506695 A5 JP 2020506695A5
Authority
JP
Japan
Prior art keywords
composition
vector
raav
recombinant
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.)
Pending
Application number
JP2019540667A
Other languages
English (en)
Japanese (ja)
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JP2020506695A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/015957 external-priority patent/WO2018140946A1/en
Publication of JP2020506695A publication Critical patent/JP2020506695A/ja
Publication of JP2020506695A5 publication Critical patent/JP2020506695A5/ja
Priority to JP2022169032A priority Critical patent/JP2023002715A/ja
Pending legal-status Critical Current

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JP2019540667A 2017-01-30 2018-01-30 糖原病を処置するための組換えウイルスベクター Pending JP2020506695A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022169032A JP2023002715A (ja) 2017-01-30 2022-10-21 糖原病を処置するための組換えウイルスベクター

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762451963P 2017-01-30 2017-01-30
US62/451,963 2017-01-30
PCT/US2018/015957 WO2018140946A1 (en) 2017-01-30 2018-01-30 Recombinant virus vectors for the treatment of glycogen storage disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022169032A Division JP2023002715A (ja) 2017-01-30 2022-10-21 糖原病を処置するための組換えウイルスベクター

Publications (2)

Publication Number Publication Date
JP2020506695A JP2020506695A (ja) 2020-03-05
JP2020506695A5 true JP2020506695A5 (https=) 2021-02-25

Family

ID=61274327

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019540667A Pending JP2020506695A (ja) 2017-01-30 2018-01-30 糖原病を処置するための組換えウイルスベクター
JP2022169032A Pending JP2023002715A (ja) 2017-01-30 2022-10-21 糖原病を処置するための組換えウイルスベクター

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022169032A Pending JP2023002715A (ja) 2017-01-30 2022-10-21 糖原病を処置するための組換えウイルスベクター

Country Status (6)

Country Link
US (1) US20190367944A1 (https=)
EP (1) EP3574104A1 (https=)
JP (2) JP2020506695A (https=)
AU (1) AU2018212002A1 (https=)
CA (1) CA3050917A1 (https=)
WO (1) WO2018140946A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112021012104A2 (pt) * 2018-12-18 2021-09-08 Ultragenyx Pharmaceutical Inc. Métodos e composições para o tratamento de doenças de armazenamento de glicogênio
WO2023069891A1 (en) * 2021-10-19 2023-04-27 University Of Connecticut Compositions and methods for the treatment of glycogen storage disorders

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6207455B1 (en) 1997-05-01 2001-03-27 Lung-Ji Chang Lentiviral vectors
IL132463A0 (en) 1997-05-13 2001-03-19 Univ North Carolina Lentivirus - based gene transfer vectors
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6218181B1 (en) 1998-03-18 2001-04-17 The Salk Institute For Biological Studies Retroviral packaging cell line
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6218186B1 (en) 1999-11-12 2001-04-17 Trustees Of The University Of Pennsylvania HIV-MSCV hybrid viral vector for gene transfer
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
RU2305708C2 (ru) 2001-10-02 2007-09-10 Энститю Клейтон Де Ля Решерш Рекомбинантный лентивирусный вектор, клетка-хозяин, трансдуцированная лентивирусным вектором, способ ее трансдукции и применение
DE60333487D1 (de) 2002-12-13 2010-09-02 Genetix Pharmaceuticals Inc Therapeutische retrovirus-vektoren für gentherapie
US20050261233A1 (en) * 2004-04-21 2005-11-24 Sanjay Bhanot Modulation of glucose-6-phosphatase translocase expression
DK2185192T3 (en) 2007-08-03 2019-02-18 Pasteur Institut LENTIVIRAL TRANSFER VECTORS AND THEIR MEDICAL USES
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
WO2011000373A1 (en) 2009-06-30 2011-01-06 Asp Technology Aps Pause adviser system and use thereof
WO2011094198A1 (en) 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
AU2014354839B2 (en) * 2013-11-26 2020-06-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Adeno-associated virus vectors for treatment of glycogen storage disease
WO2016106303A1 (en) * 2014-12-23 2016-06-30 THE UNITED STATES OF AMERICA, as representd by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Adeno-associated virus vectors encoding modified g6pc and uses thereof

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