Where Would Live-cel Fit Among Today's Fabry Treatments?

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Enzyme replacement therapy is the standard of care: an IV infusion about every two weeks, for life. Roughly 40% of ERT patients develop anti-drug antibodies over time, which can reduce its effectiveness, and ERT does not fully reach every tissue, notably the heart and nervous system. Oral chaperone therapy (migalastat) works only for the roughly 35-50% of patients with an amenable GLA variant. AAV-based gene therapies are in development; AAV typically can't be re-dosed because the immune system forms antibodies to the capsid.

Interpretation: Live-cel is designed for patients regardless of GLA variant, uses the patient's own cells (so there is no capsid barrier to re-dosing), and uses outpatient conditioning. Those are the gaps it aims to address.

Uncertainty: These are design features and early clinical observations in five patients, not comparative evidence. Head-to-head performance versus current treatments has not been established.

Next week: what Orphan Drug Designation and RMAT would, and would not, mean.

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