A new generation of automated technologies in cell and gene therapy manufacturing systems will help more people benefit according to research identifying high production cost as the main impediment to access.
The authors of a new study reached this conclusion after looking at factors determining the success—or failure—of cell and gene therapies developed for children.
Lead author Crystal Mackall, MD, founding director of the Stanford Center for Cancer Cell Therapy, told GEN: “Autologous cell therapies are more expensive to produce than small molecules or biologics because each product is individualized and requires a dedicated workflow.
“Advances in automation are poised to rapidly reduce costs in the near term. Automation is also driving increased opportunity for distributed and point-of-care manufacturing which can reduce logistics costs,” she added.
To illustrate the benefits of automated production the authors cite the CliniMACS Prodigy— developed by German technology supplier Miltenyi Biotec—as an example, explaining, “By enabling distributed and point-of-care manufacturing, automation can also reduce logistics costs.”
The CliniMACS Prodigy system is an automated platform used to engineer T cells according to current good manufacturing practice (cGMP) specifications for both early-phase clinical trials and commercial supply.
The authors added: “Several additional automated platforms are emerging that show promise for dramatic scaling, integration of automated quality-control monitoring, and the potential to modify manufacturing conditions in real-time to deliver more consistent products.
“Increasing availability and sophistication of automated platforms are predicted to substantially reduce costs of manufacturing and improve product quality and consistency in the near term,” they said.
Standardization
Cell and gene therapy efforts to standardize common processes are also advancing and will, according to the authors, help to further reduce manufacturing costs.
“There is great interest in developing more standardized platforms for delivering gene therapies, to reduce both the cost and the regulatory burden. This approach is most advanced for AAV-based therapies, for which efforts are underway to use one common vector ‘backbone’ across indications—into which a specific gene of interest can be inserted, depending upon the disease targeted.”
They cited the work of the Bespoke Gene Therapy Consortium—a public-private partnership tasked with providing common platforms—and the Platform Vector Gene Therapy Pilot Project, launched by the National Center for Advancing Translational Sciences as examples.
The latter project is working on a manufacturing process for an AAV9 vector, being used to make therapies for four different monogenic diseases.
According to the authors, “There is hope that a standardized AAV platform that enables a ‘plug-and-play’ model to treat many diseases could dramatically lower costs, shorten timelines from bench to clinic, and diminish regulatory risk, thereby accelerating development of gene replacement therapies for pediatric disease.”
Manufacturing support
The authors also suggested a dedicated manufacturing partner could help researchers bring promising cell and gene therapies to market. The proposed organization—named the Pediatric Advanced Medicine Biotech—would help developers move from the lab to the manufacturing plant, according to Mackall.
”The Pediatric Advanced Medicine Biotech will leverage an extensive, existing GMP infrastructure and deep expertise in cell manufacturing that already exists within non-profit academic medical centers and research institutes in the United States.”
She added that, “Leveraging existing infrastructure and knowledge alongside emerging automation efficiencies provides a unique opportunity to dramatically reduce costs for producing autologous cell products for small markets such as pediatric disease and other rare diseases.”
