A	B	C	D	E	F	G	H	I
1	Main
2		Brand	CTX001
3		Generic	exa-cel, exagamglogene autotemcel
4		Indication	beta-thalassemia, sickle cell disease
5		Economics	CRSP
6		MOA	non-viral ex vivo transfection
7		Administration	myeloablative conditioning followed by autologous SCT
8		Regulatory	plans submissions in late 2022
9		Clinical Trials
10			Phase I/III "CLIMB-111"
11			EHA2022 update n=44 TDT, n=31 SCD #LB2367						https://library.ehaweb.org/eha/#!*menu=16*browseby=9*sortby=1*trend=4016
12			42/44 TDT transfusion-free, 1.2-37.2mo follow-up
13			2/44 had -75% and -98% reductions in transfusion volume
14			Increases in HbF, mean Hb >11g/dL by month 3
15			31/31 SCD free from VOCs from 2.0-32.3mo follow-up
16			two unrelated SAEs
17			Phase III component fully enrolled
18
19			Phase I/III "CLIMB-121"
20			Phase III component fully enrolled
21
22			Phase I "CLIMB-131" long-term follow-up
23
24			Phase III "CLIMB-141" n=12 pediatric TDT
25
26			Phase III "CLIMB-151" n=12 pediatric SCD