Abstract
Objectives: We have developed a lentiviral vector platform pseudotyped with the Sendai virus F and HN envelope proteins (rSIV.F/HN), including the clinical candidate BI 3720931 for cystic fibrosis (CF) gene therapy. We have previously demonstrated restoration of CFTR function in CF-patient bronchial epithelial cell air–liquid interface cultures and intestinal organoids, as well as efficient and persistent in vivo transduction of murine airways. We have now assessed transduction efficiency and acute toxicology in non-human primates (NHPs).
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