UPDATE: LentiGlobin Clinical Trials

In September 2020 approval was granted to BluebirdBio to undertake trials in gene therapy in sickle cell disease. The gene therapy, LentiGlobin is designed to deliver a modified copy of the beta haemoglobin gene in order to lower the proportion of affected haemoglobin in sickle cell disease.

The trials were suspended in February 2021 after learning that a patient developed a form of blood cancer known as acute myeloid leukaemia (AML)  5 years after receiving treatment.

In March 2021 it was found following numerous independent analyses that the patient has significant chromosomal abnormalities (unrelated to sickle cell disease) which typically can be associated with the development of AML. In addition, the insertion of the vector took place in a particular gene, VAMP4, which has no known role in the development of AML or any cellular process related to cancer.

The trials remain paused but we will update you if they are resumed in the future.