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Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, has been hailed as curative for sickle cell disease (SCD) patients

If no improvement occurs after 3-4 weeks at appropriate doses, product quality may be an issue, or the condition may not respond to this approach

We are wasting the most critical biological window for repair while patients sit in dark rooms waiting for a consultation

Thus, removal of the C-terminal L585 reduces the ability of the albumin fusions to engage human FcRn, and thereby compromises their rescue from intracellular lysosomal degradation