Shots:

• The P-I/II (FORTIS) trial evaluates AT845 (AAV gene replacement therapy) in adults with LOPD
• The 1EPs are safety & tolerability along with efficacy measures, including change in muscle GAA protein expression & enzyme activity from baseline. The 2EPs includes improvements in respiratory, endurance & QoL measures
• As of Dec 3, 2021 data cut-off, 4 patients have been enrolled in which 2 were dosed at 3 x 1013vg/kg (Cohort 1) & the remaining 2 at 6 x 1013vg/kg (Cohort 2). The results include interim safety & tolerability assessments along with ~24wks of follow-up for the 2 patients in Cohort 1 & preliminary data from 2 patients in Cohort 2, no SAEs were reported in any patients

Ref: PR Newswire | Image: Astellas