Cartesian Therapeutics focuses on RNA-based cell therapies. In August, for example, the company announced successful results from a Phase I trial of an RNA-based treatment called Descartes-08 for the autoimmune disorder myasthenia gravis. Murat Kalayoglu, MD, PhD, president and CEO describes this treatment as “an autologous T-cell product engineered with RNA to express a chimeric antigen receptor, or CAR.” He adds, “The CAR binds to a target on pathogenic long-lived plasma cells called B-cell maturation antigen.”
To make this therapy, Kalayoglu explains that the company’s “cGMP manufacturing facility was designed from the ground-up for RNA cell therapies.” The bioprocessing technology called the RNA Armory is “a cell-based combination therapy platform that allows us to generate large quantities of effector cells armed with RNA therapeutics,” Kalayoglu says. “We use the engineered cell as both a factory for producing—as well as a target for delivering—a combination of RNA therapies directly to the site of disease.”
When asked about the biggest bioprocessing challenges faced in developing Descartes-08, Kalayoglu says, “Ensuring quality, consistency and scalability for a cell therapy product can be difficult. “We’ve designed our procedures to extract as much information as possible from each run so that we can optimize our process iteratively over time.”
So optimizing the bioprocess for an RNA-based therapy depends on designing for purpose, but also improving the process as needed. That approach could be used to make powerful RNA-based treatments for a wide range of diseases.
