Publication in Nature Medicine of the long-term results of the HGB-205 gene therapy trial for beta-hemoglobinopathies, conducted at the Necker Hospital and for which the analyses of patients with sickle cell disease were carried out by the France Pirenne team (N. Hebert, L. Kiger, P. Bartolucci).

In the HGB-205 phase I/II clinical trial, four patients with transfusion-dependent beta thalassemia and three patients with sickle cell disease, aged 13 to 21 years, were treated with lentiviral gene therapy. They were followed for a median of 4.5 years.

The patients with beta thalassemia all became “transfusion independent” within the first month after treatment, with a marked improvement in iron overload and correction of biological parameters related to chronic anemia. Remission of all clinical symptomatology and correction of biological parameters sustained over time were achieved in two of the three treated sickle cell patients. A reduction of the transfusion rate was obtained in the third sickle cell patient.

All these results were maintained over time with more than 4.5 years of follow-up for three patients. No adverse effects related to the use of the therapeutic lentiviral vector were observed. In the case of sickle cell disease, the correction of biological parameters related to chronic anemia in two out of three patients provides proof of principle of its efficacy and opens the way to the introduction of further improvements with the aim of obtaining the same result in all treated sickle cell patients.

More information about the French Pirenne team

Access to the abstract of the article published in Nature Medicine on January 24, 2022