JPWO2020047472A5 - - Google Patents

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Publication number
JPWO2020047472A5
JPWO2020047472A5 JP2021510129A JP2021510129A JPWO2020047472A5 JP WO2020047472 A5 JPWO2020047472 A5 JP WO2020047472A5 JP 2021510129 A JP2021510129 A JP 2021510129A JP 2021510129 A JP2021510129 A JP 2021510129A JP WO2020047472 A5 JPWO2020047472 A5 JP WO2020047472A5
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JP
Japan
Prior art keywords
recombinant aav
capsid protein
promoter
item
composition
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.)
Granted
Application number
JP2021510129A
Other languages
English (en)
Japanese (ja)
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JP2021534766A5 (https=
JP2021534766A (ja
JP7688572B2 (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/049157 external-priority patent/WO2020047472A1/en
Publication of JP2021534766A publication Critical patent/JP2021534766A/ja
Publication of JPWO2020047472A5 publication Critical patent/JPWO2020047472A5/ja
Publication of JP2021534766A5 publication Critical patent/JP2021534766A5/ja
Priority to JP2024174226A priority Critical patent/JP2025004107A/ja
Application granted granted Critical
Publication of JP7688572B2 publication Critical patent/JP7688572B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2021510129A 2018-08-30 2019-08-30 ガラクトース血症の処置のための遺伝子治療 Active JP7688572B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024174226A JP2025004107A (ja) 2018-08-30 2024-10-03 ガラクトース血症の処置のための遺伝子治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862725225P 2018-08-30 2018-08-30
US62/725,225 2018-08-30
PCT/US2019/049157 WO2020047472A1 (en) 2018-08-30 2019-08-30 Gene therapy for the treatment of galactosemia

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024174226A Division JP2025004107A (ja) 2018-08-30 2024-10-03 ガラクトース血症の処置のための遺伝子治療

Publications (4)

Publication Number Publication Date
JP2021534766A JP2021534766A (ja) 2021-12-16
JPWO2020047472A5 true JPWO2020047472A5 (https=) 2022-09-06
JP2021534766A5 JP2021534766A5 (https=) 2022-09-06
JP7688572B2 JP7688572B2 (ja) 2025-06-04

Family

ID=69643062

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021510129A Active JP7688572B2 (ja) 2018-08-30 2019-08-30 ガラクトース血症の処置のための遺伝子治療
JP2024174226A Withdrawn JP2025004107A (ja) 2018-08-30 2024-10-03 ガラクトース血症の処置のための遺伝子治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024174226A Withdrawn JP2025004107A (ja) 2018-08-30 2024-10-03 ガラクトース血症の処置のための遺伝子治療

Country Status (11)

Country Link
US (1) US20210299275A1 (https=)
EP (1) EP3844294A4 (https=)
JP (2) JP7688572B2 (https=)
KR (1) KR20210052469A (https=)
CN (2) CN120843445A (https=)
AU (2) AU2019328573A1 (https=)
BR (1) BR112021003174A2 (https=)
CA (1) CA3110290A1 (https=)
MX (1) MX2021002041A (https=)
SG (1) SG11202101819QA (https=)
WO (1) WO2020047472A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118373887A (zh) 2018-03-16 2024-07-23 国家儿童医院研究所 通过衣壳修饰增加组织特异性的基因递送
US20250001008A1 (en) * 2021-07-01 2025-01-02 Bridgebio Gene Therapy Research, Inc. Gene therapy for galactosemia
WO2023034940A2 (en) * 2021-09-01 2023-03-09 Jaguar Gene Therapy, Llc Adeno-associated vectors and virions to treat galactosemia and methods of use and manufacture
PE20241891A1 (es) * 2021-09-01 2024-09-19 Jaguar Gene Therapy Llc Vectores y viriones adenoasociados para tratar la galactosemia y metodos de uso y fabricacion

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020106381A1 (en) * 2000-06-13 2002-08-08 High Katherine A. Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
US7795002B2 (en) * 2000-06-28 2010-09-14 Glycofi, Inc. Production of galactosylated glycoproteins in lower eukaryotes
WO2002088319A2 (en) * 2001-05-01 2002-11-07 Genstar Therapeutics Corp. Mini-adenoviral vector and methods of using same
AU2003253595A1 (en) * 2002-04-05 2003-11-03 The Children's Hospital Of Philadelphia Methods for the production of chimeric adeno-associated virus (aav) vectors, compositions of chimeric aav vectors, and methods of use thereof
SI3235827T1 (sl) * 2003-06-19 2021-05-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
WO2005117576A2 (en) * 2004-05-28 2005-12-15 University Of Pittsburgh Of The Commonwealth System Of Higher Education Modification of sugar metabolic process in transgenic cells, tissues and animals
US8663981B2 (en) * 2008-08-07 2014-03-04 Her Majesty The Queen In Right Of Canada As Represented By The Minister Of Health Optimized promoter sequence
US20110223635A1 (en) * 2008-08-11 2011-09-15 Karl Deisseroth Method and Composition for Controlling Gene Expression
CA2863964C (en) * 2012-02-07 2021-10-26 Global Bio Therapeutics Usa, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
US10130649B2 (en) * 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
EP3452101A2 (en) * 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
WO2017201348A1 (en) * 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
JP7632835B2 (ja) * 2016-05-20 2025-02-19 プレジデント アンド フェローズ オブ ハーバード カレッジ 加齢関連疾患及び症状の遺伝子治療法

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