JP2023513487A5 - - Google Patents

Info

Publication number
JP2023513487A5
JP2023513487A5 JP2022547042A JP2022547042A JP2023513487A5 JP 2023513487 A5 JP2023513487 A5 JP 2023513487A5 JP 2022547042 A JP2022547042 A JP 2022547042A JP 2022547042 A JP2022547042 A JP 2022547042A JP 2023513487 A5 JP2023513487 A5 JP 2023513487A5
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JP
Japan
Prior art keywords
patient
seq
raav
human
sequence
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
JP2022547042A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023513487A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/015988 external-priority patent/WO2021155337A1/en
Publication of JP2023513487A publication Critical patent/JP2023513487A/ja
Publication of JP2023513487A5 publication Critical patent/JP2023513487A5/ja
Pending legal-status Critical Current

Links

JP2022547042A 2020-02-02 2021-02-01 Gm1ガングリオシドーシスを治療するのに有用な組成物 Pending JP2023513487A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202062969142P 2020-02-02 2020-02-02
US62/969,142 2020-02-02
US202063007297P 2020-04-08 2020-04-08
US63/007,297 2020-04-08
US202063063119P 2020-08-07 2020-08-07
US63/063,119 2020-08-07
PCT/US2021/015988 WO2021155337A1 (en) 2020-02-02 2021-02-01 Compositions useful for treating gm1 gangliosidosis

Publications (2)

Publication Number Publication Date
JP2023513487A JP2023513487A (ja) 2023-03-31
JP2023513487A5 true JP2023513487A5 (OSRAM) 2024-02-08

Family

ID=74867612

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022547042A Pending JP2023513487A (ja) 2020-02-02 2021-02-01 Gm1ガングリオシドーシスを治療するのに有用な組成物

Country Status (12)

Country Link
US (1) US20230190966A1 (OSRAM)
EP (1) EP4096721A1 (OSRAM)
JP (1) JP2023513487A (OSRAM)
KR (1) KR20220145838A (OSRAM)
CN (1) CN115867323A (OSRAM)
AU (1) AU2021213826A1 (OSRAM)
BR (1) BR112022013914A2 (OSRAM)
CA (1) CA3165057A1 (OSRAM)
IL (1) IL294924A (OSRAM)
MX (1) MX2022009462A (OSRAM)
TW (1) TWI905145B (OSRAM)
WO (1) WO2021155337A1 (OSRAM)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112021006060A2 (pt) 2018-10-01 2021-07-20 The Trustees Of The University Of Pennsylvania composições úteis para tratamento de gangliosidose gm1
WO2023147351A1 (en) * 2022-01-25 2023-08-03 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
WO2005013090A2 (en) 2003-08-01 2005-02-10 Dna Twopointo Inc. Systems and methods for biopolymer engineering
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
EP3085389A1 (en) 2005-04-07 2016-10-26 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
KR20130040844A (ko) 2010-03-29 2013-04-24 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 약학적으로 유발된 전이유전자 제거 시스템
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
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
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
AU2017215211C1 (en) * 2016-02-03 2023-11-16 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
MX2019010275A (es) * 2017-02-28 2019-10-15 Univ Pennsylvania Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este.
EP3794131A4 (en) * 2018-05-15 2022-01-26 University of Massachusetts RAAV VECTORS ENCODING FOR LYSOSOMAL BETA-GALACTOSIDASE (GLB1) AND CATHEPSIN A
BR112021006060A2 (pt) * 2018-10-01 2021-07-20 The Trustees Of The University Of Pennsylvania composições úteis para tratamento de gangliosidose gm1

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