PHCbi-Panasonic eBook - 25

Cell Therapy Today * New Developments in Immunotherapy * Cell Therapy Production Moves to Factory Floor

required an entirely new approach to both the
supply chain and manufacturing."

Novartis declares that its Kymriah
suspension for intravenous transfusion is
the first FDA-approved chimeric antigen
receptor T-cell (CAR-T) therapy. Kymriah,
which was granted priority review
for treatment of adult patients with
relapsed or refractory diffuse large B-cell
lymphoma, is manufactured at Novartis'
Morris Plains, NJ, facility. Each dose uses
the patient's own cells.

25

| GENengnews.com

Regenerative medicine has shifted the healthcare paradigm away from alleviating symptoms
and toward potentially curative approaches that
address the underlying disease, but the continued
success of these revolutionary therapies relies
on establishing new manufacturing paradigms.
Manufacturing cell therapies safely, reliably, and
affordably at a commercial scale will require ingenuity in the design of custom equipment, facilities,
and workflows.

Scalable Solutions
"When stem cells were discovered, there was a surge
of hope," said Nina Bauer, Ph.D., associate director,
commercial development, Lonza, as she chronicled
the evolution of cell therapies, which began as
an endeavor to cure diseases like Alzheimer's and
Parkinson's. While the industry has made little
headway against these giants, it has demonstrated
the potential for allogeneic stem cell therapies to
treat, and perhaps cure, a variety of other diseases

like macular degeneration and type 1 diabetes.
Although allogeneic stem cell therapies, which
use the same product batch to treat multiple
patients, allow a traditional scaleup manufacturing model, expanding these sensitive cells to
create a sufficient quantity for therapeutic applications while maintaining viability, pluripotency,
and functionality presents a major challenge.
"The cells are very sensitive," explained Antoine
Heron, Ph.D., head of cell and gene therapy
commercial development, MilliporeSigma. "Very
limited changes in your process could lead to
differentiation of the cells, and then you will lose
your product."
Over the past two years, Dr. Heron and Frank
Edenhofer, Ph.D., a professor of anatomy and
cell biology at the University of Würzburg, have
collaborated on a scalable solution for manufacturing induced pluripotent stem cells (iPSCs). After
culturing iPSCs for two weeks in a 3-L bioreactor,
they achieved a 125-fold increase in cell number-
essentially doubling the process yields reported in
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