Figure 1.

Schematic representation of AAV gene therapy. The wild-type AAV genome contains inverted terminal repeats (ITRs) and rep (replication) and cap (capsid) genes. To create a rAAV vector genome, rep and cap are replaced by a promoter, intron, and polyA genes, and the transgene of interest. In the case of the AAV5 vector for AMT-060, the transgene is human wild-type FIX (codon optimized) and the promoter is liver-specific promotor 1 (LP1) and SV40 intron. AMT-061 was created by replacing the hFIX transgene with a hyperactive FIX Padua.

Source: Adapted from Doshi et al. ¹²