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The planned U.S. Phase 1/2 trial is designed to measure alpha-Gal A enzyme activity, plasma lyso-Gb3, and eGFR slope, the same readouts FDA emphasized in its INTERACT feedback. University of Utah Health is planned to be a clinical site. Current planning targets an IND in mid-2027 and first patient enrolled in H1 2028, with a 12-month data readout in 2029.
Interpretation: The core question is whether the FACTS results (durable enzyme expression, lyso-Gb3 reduction, a clean safety profile) hold up in a larger, FDA-aligned study.
Uncertainty: Trial design isn't final until after the Pre-IND meeting, and sites, enrollment criteria, and timelines may change.
Clinicians interested in future trial participation or site involvement: reach out.
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@Brian Christie @Neva West @Chris Hopkins @Tetsu Yung
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