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Every Live-cel dose starts with the patient's own stem cells, so each batch is made for one person. FDA's INTERACT feedback identified finalizing potency assays and release specifications, and running a GMP manufacturing campaign under a closed process, as remaining work before an IND. FDA also confirmed CMC knowledge (vector backbone, packaging system, process steps) can be leveraged across our Fabry, Pompe, and Gaucher programs with appropriate justification.
Interpretation: A closed, automated process reduces manual handling and variability, which matters for patient safety and for scaling beyond a small trial. Because the process is shared across programs, manufacturing work for Fabry has read-through to Pompe and Gaucher.
Uncertainty: The manufacturing partnership and GMP campaign timelines are not finalized.
Manufacturing or analytical development partners with autologous lentiviral experience: reach out.
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@Brian Christie @Neva West @Chris Hopkins @Tetsu Yung
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