JP2022514271A5 - - Google Patents

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Publication number
JP2022514271A5
JP2022514271A5 JP2021534702A JP2021534702A JP2022514271A5 JP 2022514271 A5 JP2022514271 A5 JP 2022514271A5 JP 2021534702 A JP2021534702 A JP 2021534702A JP 2021534702 A JP2021534702 A JP 2021534702A JP 2022514271 A5 JP2022514271 A5 JP 2022514271A5
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JP
Japan
Prior art keywords
sequence
raav
nucleic acid
composition according
acid molecule
Prior art date
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Application number
JP2021534702A
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English (en)
Japanese (ja)
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JP2022514271A (ja
JP7486274B2 (ja
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Priority claimed from PCT/US2019/067247 external-priority patent/WO2020132115A1/en
Publication of JP2022514271A publication Critical patent/JP2022514271A/ja
Publication of JP2022514271A5 publication Critical patent/JP2022514271A5/ja
Application granted granted Critical
Publication of JP7486274B2 publication Critical patent/JP7486274B2/ja
Active legal-status Critical Current
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JP2021534702A 2018-12-18 2019-12-18 糖原病を処置するための方法および組成物 Active JP7486274B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862781380P 2018-12-18 2018-12-18
US62/781,380 2018-12-18
PCT/US2019/067247 WO2020132115A1 (en) 2018-12-18 2019-12-18 Methods and compositions for treating glycogen storage diseases

Publications (3)

Publication Number Publication Date
JP2022514271A JP2022514271A (ja) 2022-02-10
JP2022514271A5 true JP2022514271A5 (enExample) 2022-05-20
JP7486274B2 JP7486274B2 (ja) 2024-05-17

Family

ID=69400618

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021534702A Active JP7486274B2 (ja) 2018-12-18 2019-12-18 糖原病を処置するための方法および組成物

Country Status (15)

Country Link
US (1) US20220017922A1 (enExample)
EP (1) EP3898981B1 (enExample)
JP (1) JP7486274B2 (enExample)
KR (1) KR20210105390A (enExample)
CN (1) CN113454226B (enExample)
AU (1) AU2019405758B2 (enExample)
BR (1) BR112021012104A2 (enExample)
CA (1) CA3123841A1 (enExample)
DK (1) DK3898981T5 (enExample)
ES (1) ES2969222T3 (enExample)
IL (1) IL283956A (enExample)
MX (1) MX2021007379A (enExample)
PL (1) PL3898981T3 (enExample)
PT (1) PT3898981T (enExample)
WO (1) WO2020132115A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023069891A1 (en) * 2021-10-19 2023-04-27 University Of Connecticut Compositions and methods for the treatment of glycogen storage disorders

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
JP2002191363A (ja) * 1999-07-29 2002-07-09 Herikkusu Kenkyusho:Kk 全長cDNA合成用プライマー、およびその用途
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
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
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
WO2014127196A1 (en) * 2013-02-15 2014-08-21 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Aav8 retinoschisin expression vector for treating x-linked retinoschisis
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
FI3436593T3 (fi) 2016-03-28 2023-03-16 Ultragenyx Pharmaceutical Inc Adenoviruksen lämpöinaktivointimenetelmiä
CA3050917A1 (en) * 2017-01-30 2018-08-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Recombinant virus vectors for the treatment of glycogen storage disease

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