Is There a Cure for Fabry Disease?

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here is currently no approved cure for Fabry disease. Approved treatments, enzyme replacement therapy (an IV infusion about every two weeks) and, for patients with an amenable GLA variant, an oral chaperone (migalastat), manage the disease. Several gene therapy approaches aiming for durable enzyme production are in clinical development.

Interpretation: Live-cel (GT-GLA-S03) is one of those approaches. We describe it as designed to address the underlying enzyme deficiency by giving a patient's own stem cells a working GLA gene. Published FACTS data show durable enzyme expression and a 48% lyso-Gb3 reduction vs. no-ERT baseline at five years in five patients.

Uncertainty: Live-cel has not been tested in a U.S. trial or approved. Patients in any future trial would still need ongoing monitoring by their physicians, and long-term organ-level outcomes have not been established.

If you're a patient with questions about your own care, your Fabry specialist is the right first call. Questions about the program: post them below.

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@Brian Christie @Neva West @Chris Hopkins @Tetsu Yung

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