Evaluated across ages and genotypes¹

41 patients aged 4 to 34 years were part of the safety and efficacy assessment of ZYNTEGLO^®

Both phase 3 studies were open-label, single-arm, 24-month studies
After completing the parent study, patients were invited to enroll in Study 3 (LTF-303), a 13-year follow-up study in safety and efficacy
Patients enrolled in all studies had diagnosis of beta-thalassemia and received regular RBC transfusions (a history of at least 100 mL/kg/year of RBCs or ≥8 transfusions of RBCs per year for the prior 2 years)
All patients were administered ZYNTEGLO with a median (min, max) dose of 9.4 (5.0, 42.1) x 10⁶ CD34+ cells/kg as an intravenous infusion (N=41)

The primary outcome for Study 1 and Study 2 was transfusion independence.

Transfusion independence: a weighted average Hb ≥9 g/dL without any packed red blood cell transfusions for a continuous period of ≥12 months at any time during the study after ZYNTEGLO infusion.¹

The two phase 3 studies are ongoing. All data is as of March 2021.^1,2

	STUDY 1: HGB-207
PATIENTS ENROLLED IN PARENT STUDY	23
PATIENTS ENROLLED IN LTF-303 STUDY	19
MEDIAN DURATION^t OF FOLLOW-UP MONTHS (MIN, MAX)	29.5 (13.0, 48.2)
GENOTYPE	23 non-β⁰/β⁰
MEDIAN AGE (YEARS) (MIN, MAX)	13 (4, 34) 9 adult, 14 pediatric*
PRE-ENROLLMENT^t TRANSFUSION VOLUME (mL/kg/YEAR) MEDIAN (MIN, MAX)	208 (142, 274)
PRE-ENROLLMENT^t NUMBER OF TRANSFUSIONS PER YEAR MEDIAN (MIN, MAX)	16 (12, 37)

	STUDY 2: HGB-212
PATIENTS ENROLLED IN PARENT STUDY	18
PATIENTS ENROLLED IN LTF-303 STUDY	10
MEDIAN DURATION^t OF FOLLOW-UP MONTHS (MIN, MAX)	24.6 (4.1, 35.5)
GENOTYPE (Non-β⁰/β⁰ patients were IVS-I-110 homozygous or β⁰/IVS-I-110)	12 β⁰/β^{0^‡}
	6 non-β⁰/β⁰
MEDIAN AGE (YEARS) (MIN, MAX)	13 (4, 33) 5 adult, 13 pediatric*
PRE-ENROLLMENT^t TRANSFUSION VOLUME (mL/kg/YEAR) MEDIAN (MIN, MAX)	194 (75, 289)
PRE-ENROLLMENT^t NUMBER OF TRANSFUSIONS PER YEAR MEDIAN (MIN, MAX)	17 (11, 40)

*The phase 3 studies included 16 children (<12 years) and 11 adolescents (12 years to <18 years).
^†Data beyond 24 months are from the long-term follow-up study, Study 3 (LTF-303).¹

As of March 2021, all patients who completed a study enrolled in a long-term study 3 (LTF-303).