JP2022525848A5 - - Google Patents

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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
CA3177924A1 (en) * 2020-05-12 2021-11-18 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)
WO2024215493A2 (en) * 2023-04-10 2024-10-17 M6P Therapeutics, Inc. Compositions comprising modified human galactocerebrosidase

Family Cites Families (35)

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MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
EP2434420A3 (en) 2003-08-01 2012-07-25 Dna Twopointo Inc. Systems and methods for biopolymer engineering
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EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
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US9986722B2 (en) * 2012-08-07 2018-06-05 Chiesi Farmaceutici S.P.A. Animal model of Krabbe's disease
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
JP6678648B2 (ja) 2014-09-26 2020-04-08 テラダイン、 インコーポレイテッド グリッパと自動試験装置
HK1256341A1 (zh) * 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
CN108779167A (zh) * 2015-10-28 2018-11-09 宾夕法尼亚州大学信托人 鞘内施用腺伴随病毒载体用于基因治疗
CN118078860A (zh) * 2017-01-20 2024-05-28 联邦高等教育系统匹兹堡大学 用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病
PT3589730T (pt) * 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações
US20180250496A1 (en) 2017-03-03 2018-09-06 Cook Medical Technologies Llc Rapid exchange multiple catheter system
BR112020006661A2 (pt) * 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais

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