Data collected from the clinical trial could provide proof of concept for the stem cell mobilization regimen for patients with sickle cell disease. The treatment has the potential to achieve safe, rapid, and reliable mobilization of sufficient quantities of high-quality stem cells to improve outcomes associated with stem cell transplantation. A simplified collection process can lead to an optimized patient experience, according to the companies.

Under the collaboration, stem cells will be fully characterized, and Magenta will undertake preclinical studies in mouse models. The companies will co-fund the clinical trial and Magenta will retain all rights to its product.

The collaboration will help bluebird advance its LentiGlobin ex vivo gene therapy for sickle cell disease. The companies hope to establish whether the combination can generate appropriate CD34+ stem cells with a single round of mobilization.

Currently available mobilization drugs, such as granulocyte-colony stimulating factor, an agent administered over the course of five to seven days in other transplant settings, is not used in sickle cell disease because it can trigger vaso-occlusive crises and even death in adults and adolescents.