Wrapping up a Fantastic Program on Cell & Gene Therapy

Cell & Gene therapies are becoming more common as many candidates hike their way through the development and regulatory approval process.  However, the science and manufacturing techniques are vastly different than conventional cell culture techniques that have dominated biopharmaceutical production over the past several decades.

The Educational Program Committee’s November 14^th program entitled “Cell & Gene Therapy: Distinct Challenges in Commercializing an Autologous Therapy” provided a window into the world of autologous C&G therapies.  This program was featured at Northeastern University’s spectacular Innovation Campus in Burlington, MA (ICBM)[1].

The afternoon consisted of a 1-hour introductory presentation, a networking session with tours, and the main session.  The two featured speakers hailed from bluebird bio: Paul O’Sullivan, Director of QA, and Kelly Kral, VP of CMC Strategy and Operations.

For the intro session, Kelly and Paul provided an overview of the complex process of manufacturing and delivering a genetic therapy.  The process begins with harvesting hematopoietic stem cells from the patient, then transporting them to the production site.  There the target genes are transduced into the cells using a lentiviral vector.  Now considered a “drug product”, the cells are transported back to the therapy site and introduced back into the patient.  Kelly and Paul deftly led even us “newbies” to gain a basic knowledge of the therapy, including all the interim steps to assure safety and quality.

Between the presentations, attendees enjoyed a scrumptious spread of appetizers by New England Culinary Arts Training.  (Check out this terrific non-profit at NE-CAT.org!)  Attendees were also treated to tours featuring presentations on various labs and activities at the ICBM campus.

The main program walked us through the simultaneous developmental and regulatory challenges leading to the approval of bluebird bio’s groundbreaking treatment for sickle cell disease, LYFGENIA^TM.   For context, the audience was introduced to Kendric Cromer, a 12-year-old boy who was among the first patients to be treated with gene therapy for sickle cell disease after approval.  Kendric is among the first to be cured of this painful and debilitating disease thanks to LYFGENIA^TM.

An important theme emphasized by Kelly and Paul was the super-high level of trust and collaboration established among the stakeholders.  These included bluebird’s developmental team, QA, and regulatory/CMC groups, as well as the CDMO, the treatment centers, and, of course, the patient.

During Q&A, Kelly mentioned that a key goal moving forward is reduction in the cost of the overall process.  This will make LYFGENIA^TM acceptable to more payers around the world and, hence, accessible to many more sickle cell patients.

In closing, Paul emphasized that bluebird bio’s current process is “one way” to produce a gene therapy product.  No doubt there will be both innovation and standardization as more and more therapies come to market in the decades ahead.

The meeting managers Audrey Berner of Azzur Group and Jack Campion of Hart Engineering, would like to thank the speakers Kelly Kral and Paul O’Sullivan as well as their colleague Marc d’Anjou for their dedication and generosity in presenting to ISPE Boston. Also, to Northeastern University’s Keyara Lomax, Vice Provost Jared Auclair, Associate V.P. Rachel Spiller, and Randa Smith for their outstanding hospitality, the many arrangements, tour guidance, and use of their excellent facility.

Handouts for this program (and others) can be found here: https://www.ispeboston.org/resources/resource-library/

[1] The acronym is not by accident.  The campus is the former site of an actual Inter-Continental Ballistic Missile installation!!