JP7644026B2 - 新規ａａｖカプシドおよびそれを含む組成物 - Google Patents

新規ａａｖカプシドおよびそれを含む組成物 Download PDF

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JP7644026B2
JP7644026B2 JP2021564542A JP2021564542A JP7644026B2 JP 7644026 B2 JP7644026 B2 JP 7644026B2 JP 2021564542 A JP2021564542 A JP 2021564542A JP 2021564542 A JP2021564542 A JP 2021564542A JP 7644026 B2 JP7644026 B2 JP 7644026B2
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nucleic acid
seq
acid sequence
aav
aavrh
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JPWO2020223231A5 (enExample
JP2022530544A (ja
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ナンビアー，カリヤニ
ウイルソン，ジェームス・エム
ワン，チャン
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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
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
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JP2021564542A 2019-04-29 2020-04-28 新規ａａｖカプシドおよびそれを含む組成物 Active JP7644026B2 (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/US2020/030266 WO2020223231A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (3)

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JP2022530544A JP2022530544A (ja) 2022-06-29
JPWO2020223231A5 JPWO2020223231A5 (enExample) 2023-05-08
JP7644026B2 true JP7644026B2 (ja) 2025-03-11

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JP2021564269A Active JP7690401B2 (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
JP2021564542A Active JP7644026B2 (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

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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

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US (3) US20220249705A1 (enExample)
EP (3) EP3962507A4 (enExample)
JP (3) JP7690401B2 (enExample)
KR (3) KR20220004681A (enExample)
CN (3) CN113853207A (enExample)
AU (3) AU2020264975A1 (enExample)
BR (3) BR112021020054A2 (enExample)
CA (3) CA3134468A1 (enExample)
CL (3) CL2021002768A1 (enExample)
CO (3) CO2021015916A2 (enExample)
IL (3) IL287492A (enExample)
MX (3) MX2021013266A (enExample)
PE (3) PE20220014A1 (enExample)
PH (3) PH12021552608A1 (enExample)
SG (3) SG11202111102SA (enExample)
WO (3) WO2020223236A1 (enExample)

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WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
CN116648448A (zh) 2020-11-11 2023-08-25 欧洲分子生物学实验室 用于基因治疗的修饰病毒粒子
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
MX2023006444A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen.
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
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KR20250007064A (ko) 2022-04-06 2025-01-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Her2 양성 전이성 유방암 및 기타 암을 치료하기 위한 조성물 및 방법
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
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
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WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
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WO2016115503A1 (en) 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
WO2017075338A2 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES

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US20220220453A1 (en) 2022-07-14
CN113853207A (zh) 2021-12-28
CA3134507A1 (en) 2020-11-05
WO2020223236A1 (en) 2020-11-05
KR20220004681A (ko) 2022-01-11
BR112021020054A2 (pt) 2021-12-07
CA3134379A1 (en) 2020-11-05
JP7690401B2 (ja) 2025-06-10
BR112021020545A2 (pt) 2022-01-04
SG11202111373YA (en) 2021-11-29
CA3134468A1 (en) 2020-11-05
MX2021013268A (es) 2021-11-17
AU2020266523A1 (en) 2021-11-04
AU2020264976A1 (en) 2021-11-11
MX2021013267A (es) 2021-11-17
SG11202111102SA (en) 2021-11-29
EP3976077A1 (en) 2022-04-06
PE20212267A1 (es) 2021-11-30
CL2021002824A1 (es) 2022-05-27
EP3976078A4 (en) 2023-08-30
PE20212334A1 (es) 2021-12-14
PH12021552540A1 (en) 2022-06-20
IL287498A (en) 2021-12-01
PE20220014A1 (es) 2022-01-11
KR20220004695A (ko) 2022-01-11
EP3976077A4 (en) 2023-08-30
US20220249705A1 (en) 2022-08-11
CO2021015916A2 (es) 2021-11-30
MX2021013266A (es) 2021-11-17
CN113853209A (zh) 2021-12-28
EP3962507A1 (en) 2022-03-09
JP2022530633A (ja) 2022-06-30
EP3976078A1 (en) 2022-04-06
IL287491A (en) 2021-12-01
IL287492A (en) 2021-12-01
SG11202111375RA (en) 2021-11-29
WO2020223231A1 (en) 2020-11-05
CN113853208A (zh) 2021-12-28
JP2022530656A (ja) 2022-06-30
JP2022530544A (ja) 2022-06-29
PH12021552607A1 (en) 2022-06-27
EP3962507A4 (en) 2023-10-11
BR112021021502A2 (pt) 2022-07-19
CN113853209B (zh) 2024-12-13
CO2021015924A2 (es) 2021-11-30
AU2020264975A1 (en) 2021-11-11
WO2020223232A1 (en) 2020-11-05
KR20220004680A (ko) 2022-01-11
US20220204990A1 (en) 2022-06-30
CL2021002811A1 (es) 2022-05-27
PH12021552608A1 (en) 2022-06-20
CO2021015928A2 (es) 2021-11-30

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