MX2020005451A - Capsides variantes de virus adenoasociados y uso para inhibir la angiogenesis. - Google Patents

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

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Publication number
MX2020005451A
MX2020005451A MX2020005451A MX2020005451A MX2020005451A MX 2020005451 A MX2020005451 A MX 2020005451A MX 2020005451 A MX2020005451 A MX 2020005451A MX 2020005451 A MX2020005451 A MX 2020005451A MX 2020005451 A MX2020005451 A MX 2020005451A
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Mexico
Prior art keywords
aav
variant
virions
capsides
capsid protein
Prior art date
Application number
MX2020005451A
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English (en)
Inventor
Melissa Kotterman
David Schaffer
David Kirn
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 MX2020005451A publication Critical patent/MX2020005451A/es

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    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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Abstract

Se proveen proteínas variantes de la cápside de un virus adenoasociado (AAV) que tienen una o más modificaciones en la secuencia de aminoácidos en relación a una proteína de la cápside de AAV parental, que, cuando están presentes en un virión de AAV, confieren infectividad aumentada de uno o más tipos de células de la retina en comparación con la infectividad de las células de la retina por un virión de AAV que comprende la proteína no modificada de la cápside del AAV parental. También se proveen viriones de AAV recombinantes y composiciones farmacéuticas de los mismos que comprende una proteína variante de la cápside de AAV como se describe en la presente, métodos para elaborar estas proteínas de la cápside y viriones de rAAV, y métodos para usar estas proteínas de la cápside y viriones de rAAV en investigación y en la práctica clínica, por ejemplo en, por ejemplo, la administración de secuencias de ácido nucleico a una o más células de la retina para el tratamiento de trastornos y enfermedades de la retina.
MX2020005451A 2017-11-27 2018-11-26 Capsides variantes de virus adenoasociados y uso para inhibir la angiogenesis. MX2020005451A (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)

Publication Number Publication Date
MX2020005451A true MX2020005451A (es) 2020-08-27

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MX2020005451A MX2020005451A (es) 2017-11-27 2018-11-26 Capsides variantes de virus adenoasociados y uso para inhibir la angiogenesis.

Country Status (21)

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US (1) US11766489B2 (es)
EP (3) EP4219695A3 (es)
JP (4) JP7184894B2 (es)
KR (2) KR20250052478A (es)
CN (1) CN111770999A (es)
AU (3) AU2018372235B9 (es)
BR (1) BR112020010674A2 (es)
CA (1) CA3083472A1 (es)
CL (1) CL2020001360A1 (es)
CR (1) CR20200282A (es)
DK (1) DK3717636T5 (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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