ST-400

ST-400 is an autologous cell therapy that uses gene editing to modify a patient's own CD34+ hematopoietic stem or progenitor cells. The goal is to increase fetal hemoglobin production in red blood cells by disrupting the BCL11A gene using zinc finger nuclease (ZFN) technology. This approach aims to treat hemoglobinopathies such as sickle cell disease and beta-thalassemia by enabling the body to produce functional red blood cells with higher levels of fetal hemoglobin. ST-400 was developed through a collaboration between Sangamo Therapeutics and Bioverativ (later Sanofi). It has received orphan drug designation for sickle cell anemia and beta-thalassemia but development for beta-thalassemia has been discontinued[1][2][5].

Other names

Autologous CD34+ hematopoietic stem cellsAutologous haematopoietic stem/progenitor cell therapy