Trial of Gene Therapy for Cardiomyopathy in Patients With Fabry Disease Can Resume

In a second-quarter earnings report, 4D Molecular Therapeutics announced that the US Food and Drug Administration has lifted a clinical hold on the phase 1/2 INGLAXA clinical trial of the experimental gene therapy 4D-310 for Fabry disease cardiomyopathy.

INGLAXA was halted in early 2023 after cases of atypical hemolytic uremic syndrome among participants were observed. Now that the hold has been lifted, enrollment for INGLAXA is expected to resume this year.

Cardiomyopathy is the leading cause of death among patients with Fabry disease. The 4D-310 therapy uses the cardiac-targeted and evolved C102 vector to deliver a copy of the galactosidase alpha gene, which encodes for the alpha-galactosidase A (AGA) enzyme, to the heart after a single, low-dose, intravenous administration. The therapy is designed to generate high local levels of AGA directly within heart tissue and other affected organs, with the goal of reversing cardiomyopathy in patients with Fabry disease.