Bluebird Bio Scientists Shed Light on Cell and Gene Therapy

The Chapter’s educational program on March 21st featured a fascinating insight into the current state of gene and cell therapy. Hosted and presented by Bluebird Bio at their facility in Kendall Square, the talented team of scientists and engineers from Bluebird outlined how their process works from the preparation of the Lentivirus Vector and extraction of patient cells, through transduction of the cells in their facility and finally to re-introduction of the transformed cells back to the patient.

Bluebird, like several others in the industry, has made significant advances using this ex-vivo approach to train the patients’ own cells to either correct a genetic deficiency or to attack cancer cells.  It was clear from the enthusiastic presentation by Kendrick Goss and his team that the technology works, as a proof of concept, and it offers great hope and inspiration for those afflicted by these devastating diseases.

It was also clear just how complex a process it is. The Bluebird team acknowledged that many parts of the process would need to be streamlined in order to make the system work robustly at a realistic scale. Every aspect of the sequence is currently skilled-labor intensive, difficult and time critical. The documentation and quality control testing alone is an enormous burden and the stakes for each batch are high.

The Bluebird team, while not allowed to know the identities of their trial patients by name, admitted to knowing each and every one of them by number – and cheering for them when their cases showed improvement as if they were old and cherished friends.

The presentation did not just discuss science. Marc d’Anjou and Anne Kantardjieff described the practical aspects of dealing with individual patients, managing their cells as a single batch, and how (and where) to build a facility to make all this happen. The closing Q&A session was lively, with more questions than answers really available at this stage of play for the entire concept. It will be fascinating to revisit this session in a year or two’s time to see how it all pans out…

Thank you to our host, Bluebird Bio, and our sponsors ICQ Consultants, PM Group and NECI for their contributions to a fascinating and informative introduction to an exciting new  technology on the cutting edge of patient care.