PE20210915A1 - Capsides variantes de virus adenoasociados y su uso para inhibir la angiogenesis - Google Patents

Capsides variantes de virus adenoasociados y su uso para inhibir la angiogenesis

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Publication number
PE20210915A1
PE20210915A1 PE2020000575A PE2020000575A PE20210915A1 PE 20210915 A1 PE20210915 A1 PE 20210915A1 PE 2020000575 A PE2020000575 A PE 2020000575A PE 2020000575 A PE2020000575 A PE 2020000575A PE 20210915 A1 PE20210915 A1 PE 20210915A1
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PE
Peru
Prior art keywords
aav
capsid
adeno
proteins
capsids
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PE2020000575A
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English (en)
Inventor
David Kirn
Melissa Kotterman
David Schaffer
Paul Szymanski
Peter Francis
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4D Molecular Therapeutics Inc
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Publication date
Application filed by 4D Molecular Therapeutics Inc filed Critical 4D Molecular Therapeutics Inc
Publication of PE20210915A1 publication Critical patent/PE20210915A1/es

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Peptides Or Proteins (AREA)
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Abstract

La invencion se refiere a proteinas variantes de la capside de un virus adenoasociado (AAV) que tienen una o mas modificaciones en la secuencia de aminoacidos en relacion a una proteina de la capside de AAV parental, que, cuando estan presentes en un virion de AAV, confieren infectividad aumentada de uno o mas tipos de celulas de la retina en comparacion con la infectividad de las celulas de la retina por un virion de AAV que comprende la proteina no modificada de la capside del AAV parental. Tambien se refiere a viriones de AAV recombinantes (rAAV) y composiciones farmaceuticas de los mismos que comprende una proteina variante de la capside de AAV como se describe en la presente, metodos para elaborar estas proteinas de la capside y viriones de rAAV, y metodos para usar estas proteinas de la capside y viriones de rAAV en investigacion y en la practica clinica, por ejemplo en la administracion de secuencias de acido nucleico a una o mas celulas de la retina para el tratamiento de trastornos y enfermedades de la retina
PE2020000575A 2017-11-27 2018-11-26 Capsides variantes de virus adenoasociados y su uso para inhibir la angiogenesis PE20210915A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762590976P 2017-11-27 2017-11-27
US201862664726P 2018-04-30 2018-04-30
PCT/US2018/062478 WO2019104279A1 (en) 2017-11-27 2018-11-26 Adeno-associated virus variant capsids and use for inhibiting angiogenesis

Publications (1)

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PE20210915A1 true PE20210915A1 (es) 2021-05-19

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PE2020000575A PE20210915A1 (es) 2017-11-27 2018-11-26 Capsides variantes de virus adenoasociados y su uso para inhibir la angiogenesis

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Country Link
US (1) US11766489B2 (es)
EP (3) EP3717636B1 (es)
JP (3) JP7184894B2 (es)
KR (1) KR20200088853A (es)
CN (1) CN111770999A (es)
AU (2) AU2018372235B9 (es)
BR (1) BR112020010674A2 (es)
CA (1) CA3083472A1 (es)
CL (1) CL2020001360A1 (es)
CR (1) CR20200282A (es)
DK (1) DK3717636T3 (es)
ES (1) ES2946747T3 (es)
FI (1) FI3717636T3 (es)
IL (1) IL274712A (es)
MX (1) MX2020005451A (es)
PE (1) PE20210915A1 (es)
PH (1) PH12020550706A1 (es)
PT (1) PT3717636T (es)
SG (1) SG11202004545XA (es)
UA (1) UA127831C2 (es)
WO (1) WO2019104279A1 (es)

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