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4 of 5 FACTS trial patients received their conditioning regimen and went home the same day, using a non-myeloablative, melphalan-based approach rather than bone-marrow-transplant-intensity chemotherapy.
For patients, that's materially less treatment burden. For the development program, it's also a differentiator from more intensive HSC-gene-therapy conditioning approaches used elsewhere in the field.
This is trial-level experience in 5 patients; the U.S. Phase 1/2 trial will be the next test of whether that holds at a larger scale under FDA-aligned protocols.
See the Sep 9 post above for the full efficacy data this conditioning approach was paired with.
Follow the whole series: https://bit.ly/Glafabra-TTW
@Brian Christie @Neva West @Brian Christie @Tetsu Yung
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