Nov. 16 at 4:01 AM
Comparison of the meta-analysis of enzyme replacement therapies with
$SGMO gene therapy ST-920:
The confidence intervals for ST-920 show some uncertainty, but the mean value is positive and the upper CI value is well above “no effect.” The lower limit of the CI is slightly negative (–0.153), which statistically shows that the positive effect is not completely certain, but close to zero. Overall, the interval is wide, which is typical for smaller studies (n = 32 and 19, respectively), but the mean value is positive, which is clinically very relevant. Compared to historical/meta-analyzed eGFR slopes from existing Fabry therapies, the ST-920 effect is potentially beneficial: the positive mean value of ST-920 exceeds typical negative or slightly negative rates for ERT or chaperone, at least at the upper end of the uncertainty ranges. However, the comparison is not perfect because the underlying data (population structure, study conditions) differ.
