exa-cel
Vertex pharmaceuticals,CRISPR therapeutics AG
['atara']
auto ex vivo crispr gene editing w up to 3y follow-up. 42/44 transfusion-dependent beta-thal now w/o transfusions. 31/31 sickle cell w recurrent vasoocclusive crises now w/o crises. promising! #eha2022 
Dir of Prev Cardiology @MassGeneralNews | Assoc Prof @harvardmed | Assoc Mem @broadinstitute | Assoc Ed @JAMACardio @ScienceAdvances | Co-Founder @tensixteenbio