Last month I took part in a workshop on gene and cell therapy manufacturing at the Alliance for Regenerative Medicine’s Cell and Gene Meeting on the Mesa. It was a pleasure to participate with very distinguished colleagues: Matthew Caple, Vice President of Site Operations at Brammer Bio; Mike Kelly, Director, Asset Leadership and Portfolio Management, Gene Therapy, Biogen; and Michael Roberts, Ph.D., Chief Scientific Officer, Synpromics.
The workshop was moderated by Jessica Carmen, Ph.D., Director of Business Development, Cellular Therapy Partnerships, MaxCyte and sponsored by MaxCyte and Brammer Bio. Dr. Carmen did a great job taking us on a journey through manufacturing from construct design through raw material manufacturing, viral vector manufacturing, and ultimately commercialization.
Controlling genes to enhance expression
Dr. Roberts’ talk was a compelling presentation of the issues one must consider when designing plasmids for viral vector production and bioprocessing. Synpromics has developed methods to control genes in very specific circumstances, which can greatly enhance expression. Their technology can also provide precise control of expression, for example, allowing different components of viral vectors to be “turned on” at different times.
Importance of ancillary materials
In my talk I discussed the importance of ancillary and raw materials in gene and cell therapy production. These materials must be of high quality and well characterized; however, the quality level at which they are produced can be customized to reduce cost and timeline. The stage of development and proximity to the patient are critical factors when considering the requirements for ancillary materials. I discussed Aldevron's philosophy and how we deliver the best service for each clients application.
New methods for improving patient health
Matthew Caple described the many changes within Brammer Bio, including their merger with Florida Biologics. He described their Manufacturing Personalized™ philosophy as a means toward their ultimate mission of improving patients’ health. Brammer Bio has extensive experience from process development to commercial manufacturing, including 100 projects resulting in >200 clinical lots released.
Applying gene therapy to disease treatment
Mike Kelly discussed Biogen’s approach to which diseases they believe are amenable to gene therapy. Ophthalmic and central nervous diseases are areas of interest to Biogen. He described the importance of manufacturing platforms that can span multiple products and analytics. Biogen takes a best-in-class approach, realizing that they will not have all the answers, and seeks partnerships with leading organizations in manufacturing and process development.
The question and answer session went for over 45 minutes and ranged from regulatory matters to raw material management. The group discussed challenges presented by the heterogeneity of the treatments in the field as well as in patient responses. Given the time it takes to develop therapies, companies must think very early in the process about issues such as construct design and process characterization.
Overall, there was a dynamic interaction between presenters and attendees that resulted in a very informative and productive workshop. We are all looking forward to next year’s meeting!
The workshop video is available below. Let us know what you think in the comments below or contact us today to learn more.