“Regenerative medicine products have considerable therapeutic potential for treating a wide range of serious diseases and injuries, but are not currently thought of as viable front-line therapies, largely due to a combination of manufacturing complexity, product variability, and high cost,” explained Brian Hawkins, Chief Technology Officer, adding, “As a result, these products are often relegated to rare or orphan diseases where alternative therapies are unavailable.”
The widespread adoption of regenerative medicine products as a viable front-line therapy for chronic ailments in the general population, such as heart disease or diabetes, requires overcoming these challenges and manufacturing a large number of cells at reasonable cost. These cells also need to be manufactured reproducibly under Good Manufacturing Practices (GMP) and tested to pre-determined specifications as required by regulatory agencies.
Pluristyx was established in 2018 with the goal of solving these challenges by manufacturing GMP grade pluripotent stem cells at large scale for use as a universal starting material for the further manufacturing of therapeutic regenerative medicine products. Pluristyx also provides contract development and manufacturing services along with Chemistry, Manufacturing, and Controls (CMC) consulting to companies in the field of drug development, regenerative medicine, and cellular and gene-modified cellular therapies.
“At Pluristyx we believe that allogeneic regenerative medicine therapies derived from pluripotent stem cells can be manufactured at appropriate scale and cost to speed development and enable viable off-the-shelf products that can compete with, or even outperform, current therapies. We believe that cell-based therapies have the capacity to not only manage disease symptoms, but effectively cure patients with chronic, age-related, and debilitating diseases,” Dr. Hawkins continued.
Pluristyx first engaged with ARMI|BioFabUSA prior to the organization’s formal incorporation in early 2018. The company’s co-founder, Dr. Ben Fryer, formerly Head of Stem Cell Manufacturing at the University of Washington, lead the push to commercialize pluripotent stem cells for clinical use. Dr. Fryer identified early on the need for platform technologies that can drive down costs and reduce the variability inherent in current cell manufacturing methods, and joined ARMI at that time to leverage the combined knowledge of its members to aid his efforts. Dr. Fryer transferred his enthusiasm and talents to ARMI as soon as Pluristyx was founded, and the Pluristyx team has since become highly involved in advancing the ARMI mission for the ‘practical large-scale manufacturing of engineered tissue and tissue-related technologies.’
“With the potential to differentiate into all cell types, pluripotent cells are an ideal starting material for regenerative medicine applications,” Hawkins said, adding, “With the proper controls and manufacturing practices, pluripotent cells can be developed into master cells banks that can serve as an essential building block for clinical products throughout their lifetime.”
Cells manufactured using small-scale production models are labor-intensive and suffer from inherent and unacceptable product variability according to Pluristyx, so the company developed a platform technology which incorporates in-process monitoring and controls to minimize both comparability issues and process changes inherent in traditional cell therapy manufacturing models. “The final pluripotent stem cell product is fully-characterized, documented to GMP requirements, and tracked in-process, to generate a large lot release that can be immediately differentiated into any tissue type in either 2D or 3D format without further expansion,” Hawkins explained.
The Pluristyx Ready-To-Differentiate™ (RTD™-PSC) product will enable a more consistent process and product development workflow that can be scaled throughout the pre-clinical/ clinical development continuum. The large lot release afforded by Pluristyx’s manufacturing design permits the development of applications that require large numbers of cells, including but not limited to organoids and 3D bioprinting.
“We know that the mission of ARMI is to make practical the large-scale manufacturing of engineered tissue and tissue-related technologies and, from our perspective, the key words in that mission are ‘practical’ and ‘large-scale’ as approved cell therapies to date are autologous products that are manufactured as individual batches for a single patient. While highly successful in selected patient populations, the autologous manufacturing model is neither scalable nor cost-effective for large cohorts. In order to truly bring regenerative medicine to the masses, practical and scalable manufacturing technologies are needed to standardize product development and commercialization,” Dr. Hawkins concluded.
Pluristyx hopes to provide both CMC knowledge support and contract development services to ARMI Members to bridge the gap between preclinical/proof-of-concept studies and a commercialized clinical product. The company aims to deliver ARMI Members fully-characterized, documented, and ready-to-differentiate pluripotent stem cells that can be used as a universal starting material for downstream product development.