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[VIRTUAL] Identification and Characterization of a Novel AAV Capsid and Product for the Treatment of Cystic Fibrosis () - Mar 14, 2021 - Abstract #ATS2021ATS_3078; We showed that our gene replacement therapy A101-CFTR∆R (4D-710) is capable of safe, robust, and widespread transduction and transgene expression throughout the primate lung with minimal systemic exposure. The tropism profile after aerosol delivery in NHPs, coupled with its resistance to human Abs and transduction efficiency in human lung cells, represents a significant advance over existing AAV serotypes for the development of gene therapies for cystic fibrosis and other lung diseases.
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[VIRTUAL] 4D-A101 IDENTIFIED BY DIRECTED EVOLUTION IN NON-HUMAN PRIMATES DEMONSTRATES ROBUST GENE DELIVERY BOTH IN VITRO AND IN VIVO () - Aug 26, 2020 - Abstract #NACFC2020NACFC_281; We have identified a novel AAV vector, 4D-A101, that when delivered by aerosolization is capable of safe, robust and widespread transduction and transgene expression throughout the primate lung with minimal systemic exposure. The tropism profile of this novel AAV vector after aerosol delivery in NHPs, coupled with its resistance to human Abs and transduction efficiency in human lung cells, represents a significant advance over existing AAV serotypes for the development of gene therapies for CF.
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