New research on the preclinical product TALGlobin01 reveals the potential impact that gene therapy can have on the future of sickle cell disease treatment.
The American Society of Hematology (ASH) 2021 Annual Meeting and Exposition included exciting new data regarding gene therapy and what developments could mean for sickle cell disease (SCD) treatment.
In an interview with HCPLive®, Julien Valton, PhD, Vice President of Gene Therapy at Cellectis, discussed new research on TALGlobin01, a preclinical product candidate that's not yet in the clinic.
The data showed that when TALGlobin01 was used, it resulted in up to 70% of homology directed repair-mediated HBB gene correction in homozygous sickle patient HSPCs. Only 20% of non-homologous end joining-dependent insertion/deletion (indels) events were detected.
According to Valton, who is spearheading the development of TALGlobin, the autologous cell-based gene therapy is designed to repair the mutated hemoglobin subunit beta (HBB) gene and restore the production of hemoglobin A (HbA) in homozygous (HbSS) sickle cell disease patients.
"So, the motivation was obviously to put together a product that will be able to cure sickle cell disease patients," Valton explained, "because as you know, today, most of the treatments are symptomatic.
Stem cell transplants are an effective, curative treatment, but the procedure is only available to about 20% of patients with sickle cell disease, therefore leaving 80% of patients without any curative options.
"We believe that developing an autologous cell-based therapy to cure sickle cell disease and sickle cell anemia is of great impact for sickle cell disease patients," Valton said, "and this is why we embarked into the development of this new product named TALGlobin01."