JPWO2020223322A5 - - Google Patents

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Publication number
JPWO2020223322A5
JPWO2020223322A5 JP2021564486A JP2021564486A JPWO2020223322A5 JP WO2020223322 A5 JPWO2020223322 A5 JP WO2020223322A5 JP 2021564486 A JP2021564486 A JP 2021564486A JP 2021564486 A JP2021564486 A JP 2021564486A JP WO2020223322 A5 JPWO2020223322 A5 JP WO2020223322A5
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JP
Japan
Prior art keywords
aav
polynucleotide
viral particle
particle
viral
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.)
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Application number
JP2021564486A
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English (en)
Japanese (ja)
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JP2022530264A5 (https=
JP2022530264A (ja
JP7788594B2 (ja
Publication date
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Priority claimed from PCT/US2020/030427 external-priority patent/WO2020223322A1/en
Publication of JP2022530264A publication Critical patent/JP2022530264A/ja
Publication of JP2022530264A5 publication Critical patent/JP2022530264A5/ja
Publication of JPWO2020223322A5 publication Critical patent/JPWO2020223322A5/ja
Application granted granted Critical
Publication of JP7788594B2 publication Critical patent/JP7788594B2/ja
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JP2021564486A 2019-04-29 2020-04-29 乳児バッテン病の治療のための髄腔内および静脈内組合せ遺伝子療法 Active JP7788594B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962840360P 2019-04-29 2019-04-29
US62/840,360 2019-04-29
PCT/US2020/030427 WO2020223322A1 (en) 2019-04-29 2020-04-29 Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease

Publications (4)

Publication Number Publication Date
JP2022530264A JP2022530264A (ja) 2022-06-28
JP2022530264A5 JP2022530264A5 (https=) 2023-05-12
JPWO2020223322A5 true JPWO2020223322A5 (https=) 2023-05-12
JP7788594B2 JP7788594B2 (ja) 2025-12-19

Family

ID=73029200

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021564486A Active JP7788594B2 (ja) 2019-04-29 2020-04-29 乳児バッテン病の治療のための髄腔内および静脈内組合せ遺伝子療法

Country Status (12)

Country Link
US (1) US20220193268A1 (https=)
EP (1) EP3963081A4 (https=)
JP (1) JP7788594B2 (https=)
KR (1) KR20220046513A (https=)
CN (1) CN114269935A (https=)
AU (1) AU2020264438A1 (https=)
BR (1) BR112021021632A8 (https=)
CA (1) CA3138274A1 (https=)
IL (1) IL287608A (https=)
MX (1) MX2021013275A (https=)
SG (1) SG11202111908XA (https=)
WO (1) WO2020223322A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023303015A1 (en) * 2022-07-06 2025-01-23 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery of cln1 polynucleotide

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2794196A1 (en) * 2010-03-23 2011-09-29 Intrexon Corporation Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof
CN107249646B (zh) * 2014-12-16 2021-06-29 内布拉斯加大学董事会 用于青少年巴滕病的基因疗法
RU2764919C2 (ru) * 2016-06-13 2022-01-24 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Оптимизированные гены и экспрессионные кассеты cln1, и их применение
US20200370069A1 (en) * 2017-07-08 2020-11-26 Genethon Treatment of spinal muscular atrophy
WO2019046815A1 (en) * 2017-08-31 2019-03-07 Poseida Therapeutics, Inc. TRANSPOSON SYSTEM AND METHODS OF USE

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