The P-I/II (STAAR) study evaluates the safety & tolerability of ST-920 (0.5e13/1e13/3e13 vg/kg) in 3 groups of patients (n=6) aged 24-48yrs with Fabry disease that are on ERT, are ERT pseudo-naïve/ERT-naïve. After the cutoff date, 5 more patients have been dosed under the same study
The results showed that the α-Gal A activity measured at ERT trough for patients 1 & 2 from group 1 was 12.4 & 2.7-fold @15mos., from group 2 was 3.5 & 10.3-fold @48 & 36wks. & from group 3 was 16.7-fold @16wks & increased within normal range @2wks
The 1st patient from group 2 showed a 40% reduction in lyso-Gb3 plasma levels @10wks, whereas the 2nd patient showed a moderate increase post-ERT withdrawal & the other 4 patients sustained steady levels
Ref: Businesswire | Image: Sangamo
