BACH2 NRF2 Pathway Offers New Route to Boost Fetal Hemoglobin
A massive genomic study has found the BACH2 NRF2 axis is a strong controller of fetal hemoglobin, opening new options for sickle cell and thalassemia therapies.
Casgevy 2 articles
The therapeutic identity of Casgevy is defined by exagamglogene autotemcel, with CRISPR-Cas9 edited cell therapy providing its principal modality. Mechanistically, the product connects autologous CD34-positive hematopoietic stem cells with BCL11A erythroid enhancer editing and fetal hemoglobin induction, and its clinical or biological context includes sickle cell disease therapy, transfusion-dependent beta thalassemia therapy and ex vivo genome editing. The named therapy remains distinct from other products that may share fetal hemoglobin induction but use a different intervention design.
Patients reclaim their future as gene and cell therapies rewrite cancer treatment
At The University of Kansas Cancer Center, patients with sickle cell disease and multiple myeloma are experiencing a new era of medicine—where their own cells become the treatment.