JPWO2020223232A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2020223232A5
JPWO2020223232A5 JP2021564269A JP2021564269A JPWO2020223232A5 JP WO2020223232 A5 JPWO2020223232 A5 JP WO2020223232A5 JP 2021564269 A JP2021564269 A JP 2021564269A JP 2021564269 A JP2021564269 A JP 2021564269A JP WO2020223232 A5 JPWO2020223232 A5 JP WO2020223232A5
Authority
JP
Japan
Prior art keywords
seq
acid sequence
aav
nucleic acid
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
JP2021564269A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022530633A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/030273 external-priority patent/WO2020223232A1/en
Publication of JP2022530633A publication Critical patent/JP2022530633A/ja
Publication of JPWO2020223232A5 publication Critical patent/JPWO2020223232A5/ja
Pending legal-status Critical Current

Links

JP2021564269A 2019-04-29 2020-04-28 新規ａａｖカプシドおよびそれを含む組成物 Pending JP2022530633A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962840184P 2019-04-29 2019-04-29
US62/840,184 2019-04-29
US201962913314P 2019-10-10 2019-10-10
US62/913,314 2019-10-10
US201962924095P 2019-10-21 2019-10-21
US62/924,095 2019-10-21
PCT/US2020/030273 WO2020223232A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (2)

Publication Number Publication Date
JP2022530633A JP2022530633A (ja) 2022-06-30
JPWO2020223232A5 true JPWO2020223232A5 (es) 2023-04-18

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2021564542A Pending JP2022530544A (ja) 2019-04-29 2020-04-28 新規ａａｖカプシドおよびそれを含む組成物
JP2021564543A Pending JP2022530656A (ja) 2019-04-29 2020-04-28 新規ａａｖカプシドおよびそれを含む組成物
JP2021564269A Pending JP2022530633A (ja) 2019-04-29 2020-04-28 新規ａａｖカプシドおよびそれを含む組成物

Family Applications Before (2)

Application Number Title Priority Date Filing Date
JP2021564542A Pending JP2022530544A (ja) 2019-04-29 2020-04-28 新規ａａｖカプシドおよびそれを含む組成物
JP2021564543A Pending JP2022530656A (ja) 2019-04-29 2020-04-28 新規ａａｖカプシドおよびそれを含む組成物

Country Status (15)

Country Link
US (3) US20220249705A1 (es)
EP (3) EP3976078A4 (es)
JP (3) JP2022530544A (es)
KR (3) KR20220004695A (es)
CN (3) CN113853208A (es)
AU (3) AU2020264975A1 (es)
BR (3) BR112021021502A2 (es)
CA (3) CA3134379A1 (es)
CL (3) CL2021002768A1 (es)
CO (3) CO2021015928A2 (es)
IL (3) IL287492A (es)
MX (3) MX2021013266A (es)
PE (3) PE20212267A1 (es)
SG (3) SG11202111102SA (es)
WO (3) WO2020223231A1 (es)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2021292200A1 (en) 2020-06-17 2023-02-02 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
EP4200429A1 (en) 2020-08-24 2023-06-28 The Trustees of The University of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
MX2023005446A (es) 2020-11-11 2023-07-18 European Molecular Biology Laboratory Particulas virales modificadas para terapia genica.
US20240024507A1 (en) 2020-12-01 2024-01-25 The Trustees Of The University Of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
JP2023551911A (ja) 2020-12-01 2023-12-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アンジェルマン症候群の治療のための組成物及びその使用
MX2023012513A (es) 2021-04-23 2023-12-15 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023168293A2 (en) * 2022-03-01 2023-09-07 The Trustees Of The University Of Pennsylvania Viral vector genome encoding an insulin fusion protein
WO2023168403A2 (en) * 2022-03-03 2023-09-07 The Trustees Of The University Of Pennsylvania Viral vectors encoding parathyroid hormone fusions and uses thereof in treating hypoparathyroidism
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2024015966A2 (en) * 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE520707T1 (de) * 2001-11-13 2011-09-15 Univ Pennsylvania Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
EP2298926A1 (en) * 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
EP3409296A1 (en) * 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
US20090317417A1 (en) * 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
CA3066596A1 (en) * 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
EA201691809A1 (ru) * 2014-03-10 2017-01-30 ЮНИКЬЮРЕ АйПи Б.В. Дополнительно усовершенствованные векторы aav, продуцируемые клетками насекомого
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3632923A1 (en) * 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
EP3872085B1 (en) * 2015-07-30 2023-03-01 Massachusetts Eye & Ear Infirmary Ancestral aav sequences and uses thereof
EP3368054A4 (en) * 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
CA3002406A1 (en) * 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
WO2017189959A1 (en) * 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
IL302748A (en) * 2016-05-18 2023-07-01 Voyager Therapeutics Inc modulatory polynucleotides
WO2018075798A1 (en) * 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
WO2018200419A1 (en) * 2017-04-23 2018-11-01 The Trustees Of The University Of Pennsylvania Viral vectors comprising engineered aav capsids and compositions containing the same
JOP20190269A1 (ar) * 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3662060A2 (en) * 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav

Similar Documents

Publication Publication Date Title
JP2018108113A5 (es)
AU2019200949B2 (en) High-transduction-efficiency raav vectors, compositions, and methods of use
US20230129762A1 (en) High-transduction-efficiency raav vectors, compositions, and methods of use
JP2020510429A5 (es)
CA3054131C (en) Modified aav capsid proteins and uses thereof
FI3684423T3 (fi) Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä
JP2023123765A5 (es)
US9611302B2 (en) High-transduction-efficiency RAAV vectors, compositions, and methods of use
JPWO2020223232A5 (es)
EP3633041A2 (en) Synthetic combinatorial aav capsid library for targeted gene therapy
JP2011101646A5 (es)
KR20180091863A (ko) 임상적 사용에 적합한 무혈청 현탁 세포 배양 시스템에서 재조합 아데노-관련 바이러스(aav) 벡터를 생성하기 위한 확장가능한 방법
JPWO2020223236A5 (es)
JP2017536116A5 (es)
JPWO2020223231A5 (es)
JPWO2019169004A5 (es)
JPWO2020068990A5 (es)
WO2020142714A1 (en) Aav expression cassette and aav vectors comprising the same
JP2022545121A (ja) Ａａｖ５の単離修飾ｖｐ１カプシドタンパク質
JP2024514956A (ja) 組織標的化された改変ａａｖカプシドおよびその使用方法
JP2023513892A (ja) Ｄｎａ増幅方法
JPWO2021080991A5 (es)
RU2820088C1 (ru) Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
EP4389759A1 (en) Isolated modified aav5 capsid protein vp1
JPWO2020219766A5 (es)