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Astellas Presents Results of AT845 in P-I/II (FORTIS) Trial for Late-Onset Pompe Disease (LOPD) at WORLD Symposium 2022

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Astellas Presents Results of AT845 in P-I/II (FORTIS) Trial for Late-Onset Pompe Disease (LOPD) at WORLD Symposium 2022

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

Click here to­ read the full press release 

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