exagamglogene autotemcel
CRISPR,Vertex
['pivotal trial', '$vrtx', 'vertex']
vertex $vrtx positive results from pivotal trials of exa-cel for transfusion-dpdt beta thal &amp; severe scd presented at #eha2023 *n=48 tdt bt
ti for &gt;=12 consecutive mo in 24/27 or 88.9%
mean ti duration 20.5mo/max 40.7mo 
Biotellytics / https://t.co/37itIfRolp = business intel service on listed European Bios / Views=mine only, RT-like=/=endorse, no advice. 16y in Tech (chip design)