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In this interview, Valton details the editing function of TALGlobin01 and how it stands out among traditional gene therapy approaches to sickle cell disease.
New data from Cellectis showed that when TALGlobin01 was used in patients with sickle cell disease (SCD), it resulted in up to 70% of homology directed repair-mediated hemoglobin subunit beta (HBB) gene correction in homozygous (HbSS) sickle patient HSPCs.
Only 20% of non-homologous end joining-dependent insertion/deletion (indels) events were detected.
Julien Valton, PhD, Vice President of Gene Therapy at Cellectis, is spearheading the development of TALGlobin01, a preclinical product candidate that's not yet in the clinic.
In an interview with HCPLive®, Valton explained the function of TALGlobin01 by comparing a genome to a book.
“So as you know, in a genome, you have chapters,” Valton said, “you have paragraphs, you have like, sentences, words, and letters. In the context of sickle cell disease, or sickle cell anemia, you have one letter that is dysfunctional.”
What makes TALGlobin01 different from traditional treatments is that it’s an autologous cell-based gene therapy that’s designed to repair the mutated HBB gene and restore the production of hemoglobin A (HbA) in homozygous sickle cell disease patients.
“So, you don't fix the typo, but you promote other hemoglobin paragraphs in the genome,” Valton said.
This exciting new gene therapy data from Cellectis was presented at the American Society of Hematology (ASH) 2021 Annual Meeting and Exposition.