TALGlobin01

TALGlobin01 is an **autologous hematopoietic stem and progenitor cell based gene therapy** being developed by **Cellectis** for **sickle cell disease**. The product uses the **TALEN-HBB01** gene-editing nuclease together with an **AAV-based donor template engineering process** to directly repair the mutant **beta-globin gene** in a patient's own HSPCs through **homology-directed repair**, with the goal of restoring **hemoglobin A** production and reducing red-cell sickling. Reported preclinical data showed high levels of HDR-mediated correction in patient-derived cells, increased HbA production, and reduced hypoxia-induced sickling, and the program was described as a **preclinical** candidate rather than a clinical-stage therapy.