Vinmec HBB CRISPR gene therapy

Vinmec HBB CRISPR gene therapy is an experimental ex vivo gene-edited cell therapy developed by the Vinmec Center for Stem Cells and Gene Technology for the treatment of beta-thalassemia. The therapy utilizes the CRISPR/Cas9 system to target and correct mutations in the HBB (hemoglobin subunit beta) gene within a patient's own hematopoietic stem cells. By restoring the functional sequence of the HBB gene, the therapy aims to enable the production of functional adult hemoglobin (HbA), thereby addressing the underlying genetic cause of beta-thalassemia and potentially eliminating the need for chronic blood transfusions. The edited stem cells are re-infused into the patient following a conditioning regimen. The program is currently in the preclinical stage of development.

Other names

Vinmec Center for Stem Cells and Gene Technology-HBB-gene therapy-CRISPR/Cas9-ThalassemiaCRISPR/Cas9-based HBB gene editing therapy