iECURE’s early gene-therapy success in a baby fails to replicate in larger trial

Early last year iECURE reported that its experimental gene therapy appeared to cure an infant suffering from a severe genetic disorder.

The company has now released data from a follow-up study that included several additional infants, and the results did not show the same durable correction.

Researchers said the variability suggests the approach may not be as reliably effective as initially hoped, raising questions about the therapy’s scalability.

iECURE plans to analyze the new data and adjust its protocol, while investors and the broader gene-editing community watch closely for further developments.