CO2020004738A2 - Variantes de cápsides de virus adenoasociados y métodos de uso de estas - Google Patents

Variantes de cápsides de virus adenoasociados y métodos de uso de estas

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Publication number
CO2020004738A2
CO2020004738A2 CONC2020/0004738A CO2020004738A CO2020004738A2 CO 2020004738 A2 CO2020004738 A2 CO 2020004738A2 CO 2020004738 A CO2020004738 A CO 2020004738A CO 2020004738 A2 CO2020004738 A2 CO 2020004738A2
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Colombia
Prior art keywords
aav
methods
capsid
virions
adeno
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CONC2020/0004738A
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English (en)
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David H Kirn
Melissa Kotterman
David Schaffer
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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 CO2020004738A2 publication Critical patent/CO2020004738A2/es

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Abstract

En la presente se proporcionan proteínas de cápsides variantes de virus adenoasociados (AAV) que tienen una o más modificaciones en la secuencia de aminoácidos en relación con una proteína de la cápside de AAV parental, que, cuando están presentes en un virión de AAV, confieren un aumento en la infectividad de uno o más tipos de células musculares en comparación con la infectividad de las células musculares por un virión de AAV que comprende la proteína de la cápside de AAV parental no modificada. También se proporcionan viriones de AAV recombinantes y composiciones farmacéuticas de estos que comprenden una proteína de variante de cápside de AAV como se describe en la presente, métodos para producir estos viriones y proteínas de la cápside de rAAV, y métodos para usar estos viriones y proteínas de la cápside de rAAV en la investigación y en la práctica clínica, por ejemplo, en la administración de secuencias de ácidos nucleicos a una o más células musculares para el tratamiento de trastornos y enfermedades musculares.
CONC2020/0004738A 2017-09-20 2020-04-17 Variantes de cápsides de virus adenoasociados y métodos de uso de estas CO2020004738A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762560901P 2017-09-20 2017-09-20
PCT/US2018/051812 WO2019060454A2 (en) 2017-09-20 2018-09-19 CAPSID VARIANT ADENO-ASSOCIATED VIRUSES AND METHODS OF USE

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CO2020004738A2 true CO2020004738A2 (es) 2020-05-05

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US (4) US11118192B2 (es)
EP (2) EP4218828A3 (es)
JP (1) JP6825167B2 (es)
KR (2) KR20220070075A (es)
CN (1) CN111836649A (es)
AU (1) AU2018337833B2 (es)
BR (1) BR112020005436B1 (es)
CA (1) CA3076348A1 (es)
CL (1) CL2020000728A1 (es)
CO (1) CO2020004738A2 (es)
CR (1) CR20200165A (es)
DK (1) DK3684423T5 (es)
ES (1) ES2948902T3 (es)
FI (1) FI3684423T3 (es)
IL (1) IL273261A (es)
MX (1) MX2020003176A (es)
NZ (1) NZ763018A (es)
PE (1) PE20201264A1 (es)
PH (1) PH12020550094A1 (es)
PT (1) PT3684423T (es)
RU (1) RU2770922C2 (es)
SG (1) SG11202002276VA (es)
UA (1) UA127455C2 (es)
WO (1) WO2019060454A2 (es)
ZA (1) ZA202001679B (es)

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