The experimental gene therapy LentiGlobin for the treatment of severe sickle cell disease is being investigated in an ongoing Phase 1 clinical trial. Bluebird Bio, the company developing the therapy, has released interim results from the study. You can read the full article here, at Business Wire.
Sickle cell disease (SCD) is a group of genetic conditions that affect red blood cells. It is a lifelong condition characterised by the production of abnormally shaped red blood cells that are then unable to function normally; often they have shorter lives and can become stuck in blood vessels. People who have sickle cell disease often experience symptoms such as frequent infections, sickle cell episodes (periods of excruciating pain), and anaemia, which is caused by a lack of oxygen in the blood and can lead to tiredness. SCD varies in severity, but for many people, it is a severe and life-shortening condition. It is estimated that about 100,000 people in the US have SCD, and some groups, such as people of African, Caribbean, and Asian descent, are more frequently affected than others.
Bluebird Bio’s gene therapy, LentiGlobin, is designed to offer patients with SCD a new treatment option that addresses the cause of the condition. LentiGlobin works by putting a functioning copy of the human beta-globin gene into stem cells in a lab and then transplanting the cells into a patient through an infusion. The new gene is expected to reduce the sickling (abnormal shape) of red blood cells.
The new interim data from the Phase 1 study of LentiGlobin has shown promising results. The on-going study, which is taking place across several centres, is an open-label study. It contains three cohorts of patients; group A was treated using the original study protocol, while groups B and C were treated using an amended protocol. Group C also had stem cells collected from a different area.
