JP2020510648A5 - - Google Patents

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Publication number
JP2020510648A5
JP2020510648A5 JP2019545363A JP2019545363A JP2020510648A5 JP 2020510648 A5 JP2020510648 A5 JP 2020510648A5 JP 2019545363 A JP2019545363 A JP 2019545363A JP 2019545363 A JP2019545363 A JP 2019545363A JP 2020510648 A5 JP2020510648 A5 JP 2020510648A5
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JP
Japan
Prior art keywords
pharmaceutical composition
suspension
raav
patient
genome
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
JP2019545363A
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English (en)
Japanese (ja)
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JP2020510648A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/018678 external-priority patent/WO2018152485A1/en
Publication of JP2020510648A publication Critical patent/JP2020510648A/ja
Publication of JP2020510648A5 publication Critical patent/JP2020510648A5/ja
Priority to JP2023030651A priority Critical patent/JP2023071829A/ja
Pending legal-status Critical Current

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JP2019545363A 2017-02-20 2018-02-20 家族性高コレステロール血症を処置するための遺伝子治療 Pending JP2020510648A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023030651A JP2023071829A (ja) 2017-02-20 2023-03-01 家族性高コレステロール血症を処置するための遺伝子治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762461015P 2017-02-20 2017-02-20
US62/461,015 2017-02-20
PCT/US2018/018678 WO2018152485A1 (en) 2017-02-20 2018-02-20 Gene therapy for treating familial hypercholesterolemia

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023030651A Division JP2023071829A (ja) 2017-02-20 2023-03-01 家族性高コレステロール血症を処置するための遺伝子治療

Publications (2)

Publication Number Publication Date
JP2020510648A JP2020510648A (ja) 2020-04-09
JP2020510648A5 true JP2020510648A5 (https=) 2021-03-25

Family

ID=63170484

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019545363A Pending JP2020510648A (ja) 2017-02-20 2018-02-20 家族性高コレステロール血症を処置するための遺伝子治療
JP2023030651A Pending JP2023071829A (ja) 2017-02-20 2023-03-01 家族性高コレステロール血症を処置するための遺伝子治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023030651A Pending JP2023071829A (ja) 2017-02-20 2023-03-01 家族性高コレステロール血症を処置するための遺伝子治療

Country Status (8)

Country Link
US (2) US11554147B2 (https=)
EP (1) EP3595688A4 (https=)
JP (2) JP2020510648A (https=)
KR (1) KR20190118163A (https=)
AU (1) AU2018220212A1 (https=)
CA (1) CA3051011A1 (https=)
IL (1) IL268276A (https=)
WO (1) WO2018152485A1 (https=)

Families Citing this family (6)

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ES2876409T3 (es) 2014-04-25 2021-11-12 Univ Pennsylvania Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
IL303850A (en) 2015-12-11 2023-08-01 Univ Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
WO2021080975A1 (en) * 2019-10-21 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing cholesterol levels
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2023081807A1 (en) * 2021-11-04 2023-05-11 The Trustees of the University of Pennsylvania Penn Center for Innovation Compositions and methods for reducing pcsk9 levels in a subject

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WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
IL303850A (en) 2015-12-11 2023-08-01 Univ Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11554147B2 (en) * 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia

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