bluebird bio, a Cambridge-Mass.-based privately-held biotechnology company focused on gene therapy, has formed a broad, global strategic collaboration with Celgene, a Summit, N.J.-based integrated global biopharmaceutical company focused on cancer and inflammatory diseases, to discover, develop and commercialize novel disease-altering gene therapies in oncology.

The collaboration will focus on applying gene therapy technology to genetically modify a patient's own T-cells, known as chimeric antigen receptor (CAR) T-cells, to target and destroy cancer cells. The multi-year R&D collaboration has the potential to lead to the development and commercialization of multiple CAR T-cell products. Celgene has an option to license any products resulting from the collaboration after the completion of a phase I clinical study for each such product. bluebird bio will be responsible for R&D activity through phase I studies.

Financial terms of the agreement include an upfront payment and up to $225 million per product in potential option fees and clinical and regulatory milestones. bluebird bio also has the right to participate in the development and commercialization of any licensed products resulting from the collaboration through a 50/50 co-development and profit share in the U.S. in exchange for a reduction of milestones. Royalties would also be paid in regions where there is no profit share including in the U.S. if bluebird bio declines to exercise their co-development and profit sharing rights.

Additionally, Celgene has also entered into a separate strategic collaboration in the CAR T-cell field with the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital and The Methodist Hospital, Houston, led by Malcolm Brenner, M.D., Ph.D. bluebird bio, Celgene and Brenner's team will work collaboratively to advance and develop existing and new products and programs in the CAR T-cell field.  

"The genetic manipulation of autologous T-cells is a new frontier in oncology, one that shows early promise in emerging clinical trials," said Tom Daniel, president, research and early development, Celgene. "We see strong prospects for this collaboration between Celgene, bluebird bio and Baylor College of Medicine's experienced leaders in this emerging field, led by Dr. Brenner, to advance this innovative approach to intractable problems in oncology."

Nick Leschly, CEO of bluebird bio, said, "We believe that our recent advances in the industrialization of our gene therapy platform will drive improvements in the potency, purity, efficiency and scalability of our lentiviral gene therapy programs. These advances provide us with an opportunity to apply our platform, intellectual property and know-how to the development of additional product candidates in indications such as CAR T-cells for cancer. Celgene is a global leader in oncology and, combined with Baylor's expertise in the CAR T-cell field, we have created a great opportunity to drive innovation in a new and exciting area."

The gene therapy products currently in clinical development at bluebird bio for the treatment of childhood cerebral adrenoleukodystrophy, beta-thalassemia and sickle cell disease are independent of this collaboration.