Home > Haematology > EHA 2022 > Single-dosed exa-cel leads to early and durable increase of foetal haemoglobin

Single-dosed exa-cel leads to early and durable increase of foetal haemoglobin

Presented By
Prof. Franco Locatelli, University of Pavia, Italy
Conference
EHA 2022
Trial
Phase 2, CLIMB THAL-111, CLIMB SCD-121
Exa-cel, an ex vivo CRISPR/Cas9 gene-editing therapy, is associated with an early, consistent, and durable increase in foetal haemoglobin (Hb) and total Hb in patients with transfusion-dependent β-thalassemia or severe sickle cell disease, results from CLIMB THAL-111 and CLIMB SCD-121 show. Diminished production of adult HbA is a common feature in transfusion-dependent β-thalassemia and sickle cell disease. Elevated production of foetal Hb is associated with improved outcomes in these diseases. Naturally occurring genetic polymorphisms in BCL11A are associated with reduced expression of BCL11A resulting in elevated production of foetal Hb and decreased severity of transfusion-dependent β-thalassemia and sickle cell disease [1]. Exa-cel, also known as CTX001, is a cell therapy designed to reactivate foetal Hb production by ex vivo CRISPR/Cas9 gene-editing of BCL11A in autologous CD34-positive haematopoietic stem...


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