TALGlobin01
/ Cellectis
- LARVOL DELTA
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November 05, 2021
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
August 05, 2021
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
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