Vertex and CRISPR Therapeutics Present New Data of CTX001 from 22 Patients with TDT and SCD at EMA 2021

Shots:

• The ongoing two P-I/ II studies, CLIMB-Thal-111 & CLIMB-SCD-121 evaluating CTX001 in 45 patients with a follow-up of ~2yrs. after infusion aged 18-35yrs to treat transfusion-dependent beta-thalassemia & sickle cell disease
• The results from 22 patients with a follow-up of >3 mos., i.e 15 patients with TDT are transfusion-free after CTX001 infusion @ follow-up of 4 to 26 mos., improvement in total hemoglobin & fetal hemoglobin
• Remaining 7 patients with SCD showed free of VOC’s after CTX001 infusion @ follow-up of 5 to 22 mos., improvements in total hemoglobin & fetal hemoglobin levels. Additioanlly, 5 patients with TDT & 2 patients with SCD with follow-up of > 1yr. showed a stable & durable response

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