CTX001 a thread 1/ #ASH20

. @DrAlexisThompsn Intro:
- BCL11A is a key regulator of the fetal to adult hemoglobin switch
- CRISPR Nobel prize in chemistry to @doudna_lab
- can gene editing of BCL11A modify HbF expression well?

#HaydarFrangoul #ASH20 /3

Thalassemia: 7 patients
Neutrophil engraftment median 32 days
Platelet engraftment 37 days
AEs consistent with auto-transplant #ASH20 /4

Wowza. #ASH20 #HaydarFrangoul

Circulating red blood cells expressing fetal hemoglobin was at 99%. PANCELLULAR!!!
All patients are transfusion INDEPNDENT! #ASH20 /7

#SICKLECELL
3 patients
No serious adverse events #ASH20 /8

No VOCs after infusion
PANCELLULAR fetal hemoglobin
No hemolysis

#ASH20 /9

CURE. A POTENTIAL CURE. #SickleCell #ASH20 @VertexPharma

Full text article here. #ASH20

https://www.nejm.org/doi/full/10.1056/NEJMoa2031054

Cautiously optimistic, but hope is important to hold on to. #ASH20