TALGlobin01 / Cellectis - LARVOL DELTA

Pre-Clinical Development of a Highly Efficient TALEN®-Based Correction of the β-Globin Gene in Patient-Derived Hematopoietic Stem and Progenitor Cells (HSPCs) to Treat Sickle Cell Disease (ASH 2021) - "These results are the first demonstration that a TALEN-based engineering process could be used to efficiently correct the SCD mutated HBB gene in HbSS patient-derived CD34+ HSPCs. Taken together, the high level of HbA expression and reversion of sickling phenotype, the efficient in vivo long-term engraftment potential of TALGlobin01 edited cells and the low levels of HBB KO or off-target cleavage generated by our gene correction process, warrant the clinical evaluation of TALGlobin01 to treat SCD patients."

Preclinical • Bone Marrow Transplantation • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation • CD34

Cellectis Provides Business Update and Reports Financial Results for Second Quarter and First Six Months 2021 (GlobeNewswire) - "Preclinical pipeline disclosed at Innovation Days....TALGlobin01 (Sickle Cell Disease)...HEAL’s lead product candidate is TALGlobin01, an autologous ex vivo TALEN®-edited CD34+ HSC therapy for the treatment of SCD....Cellectis plans to file an IND for TALGlobin01 in 2022."

IND • Hematological Disorders • Sickle Cell Disease