JP2022525848A5 - - Google Patents

Info

Publication number
JP2022525848A5
JP2022525848A5 JP2021549986A JP2021549986A JP2022525848A5 JP 2022525848 A5 JP2022525848 A5 JP 2022525848A5 JP 2021549986 A JP2021549986 A JP 2021549986A JP 2021549986 A JP2021549986 A JP 2021549986A JP 2022525848 A5 JP2022525848 A5 JP 2022525848A5
Authority
JP
Japan
Prior art keywords
raav
seq
sequence
aavhu68
galactosylceramidase
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
JP2021549986A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022525848A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/019794 external-priority patent/WO2020176562A1/en
Publication of JP2022525848A publication Critical patent/JP2022525848A/ja
Publication of JP2022525848A5 publication Critical patent/JP2022525848A5/ja
Priority to JP2024208739A priority Critical patent/JP2025032175A/ja
Pending legal-status Critical Current

Links

JP2021549986A 2019-02-26 2020-02-26 クラッベ病の治療に有用な組成物 Pending JP2022525848A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024208739A JP2025032175A (ja) 2019-02-26 2024-11-29 クラッベ病の治療に有用な組成物

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962810708P 2019-02-26 2019-02-26
US62/810,708 2019-02-26
US201962817482P 2019-03-12 2019-03-12
US62/817,482 2019-03-12
US201962877707P 2019-07-23 2019-07-23
US62/877,707 2019-07-23
US201962916652P 2019-10-17 2019-10-17
US62/916,652 2019-10-17
PCT/US2020/019794 WO2020176562A1 (en) 2019-02-26 2020-02-26 Compositions useful in treatment of krabbe disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024208739A Division JP2025032175A (ja) 2019-02-26 2024-11-29 クラッベ病の治療に有用な組成物

Publications (2)

Publication Number Publication Date
JP2022525848A JP2022525848A (ja) 2022-05-20
JP2022525848A5 true JP2022525848A5 (enExample) 2023-02-22

Family

ID=69941503

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021549986A Pending JP2022525848A (ja) 2019-02-26 2020-02-26 クラッベ病の治療に有用な組成物
JP2024208739A Pending JP2025032175A (ja) 2019-02-26 2024-11-29 クラッベ病の治療に有用な組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024208739A Pending JP2025032175A (ja) 2019-02-26 2024-11-29 クラッベ病の治療に有用な組成物

Country Status (16)

Country Link
US (1) US20220118108A1 (enExample)
EP (1) EP3931337A1 (enExample)
JP (2) JP2022525848A (enExample)
KR (1) KR20210132095A (enExample)
CN (1) CN114040980A (enExample)
AU (1) AU2020229772A1 (enExample)
BR (1) BR112021016566A2 (enExample)
CA (1) CA3130055A1 (enExample)
CL (1) CL2021002248A1 (enExample)
CO (1) CO2021011090A2 (enExample)
IL (1) IL285776A (enExample)
MX (1) MX2021010266A (enExample)
PE (1) PE20212072A1 (enExample)
SG (1) SG11202108480UA (enExample)
TW (1) TW202045728A (enExample)
WO (1) WO2020176562A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2021271635A1 (en) * 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
IL307633A (en) * 2021-04-12 2023-12-01 Univ Pennsylvania Useful preparations in the treatment of spinal muscular atrophy (SBMA)
CN120917141A (zh) * 2023-04-10 2025-11-07 M6P医疗公司 包含修饰的人半乳糖脑苷脂酶的组合物

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AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
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EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
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US9986722B2 (en) * 2012-08-07 2018-06-05 Chiesi Farmaceutici S.P.A. Animal model of Krabbe's disease
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JP2020505317A (ja) * 2017-01-20 2020-02-20 ユニバーシティ オブ ピッツバーグ − オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション 臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置
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