SRT8

SRT8 (Thalagen) is an investigational autologous gene therapy developed by Errant Gene Therapeutics (now San Rocco Therapeutics) in collaboration with Memorial Sloan Kettering Cancer Center for the treatment of beta-thalassemia major. The therapy utilizes the TNS9.3.55 lentiviral vector to transduce a patient's own hematopoietic stem cells with a functional human β-globin gene. Once re-infused, these modified cells aim to restore the production of normal hemoglobin, potentially eliminating the need for chronic blood transfusions. It has received Orphan Drug Designation in the US and Europe for chronic beta-thalassemias. Clinical results have demonstrated sustained reductions in transfusion requirements in treated patients.

Brand names

Thalagen

Other names

TNS9.3.55 lentiviral vectorTNS-9.3.55 lentiviral vectorTNS 9.3.55 lentiviral vectorErrant Gene Therapeutics beta-globin gene therapy