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
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Application number
JP2021534702A
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English (en)
Japanese (ja)
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JP7486274B2 (ja
JP2022514271A (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
Anticipated expiration legal-status Critical

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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 (OSRAM) 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 (14)

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

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
CA2713338C (en) 2008-01-29 2021-10-26 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
ES2680915T3 (es) 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
AU2014216160B2 (en) * 2013-02-15 2017-03-30 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services AAV8 retinoschisin expression vector for treating X-linked retinoschisis
US9644216B2 (en) * 2013-11-26 2017-05-09 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
ES2824829T3 (es) * 2014-12-23 2021-05-13 Us Health Vectores de virus adenoasociado que codifican G6PC modificada y usos de estos
LT3436593T (lt) 2016-03-28 2023-03-10 Ultragenyx Pharmaceutical Inc. Adenoviruso šiluminio inaktyvavimo būdai
JP2020506695A (ja) * 2017-01-30 2020-03-05 ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド バイ ザ セクレタリー, デパートメント オブ ヘルス アンド ヒューマン サービシーズ 糖原病を処置するための組換えウイルスベクター

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