Instead of jumping into building a big bioprocessing facility, leaders at Avrobio started small. This clinical-stage gene-therapy company, headquartered in Cambridge, MA, focused on automation and computation.
“We wanted to produce consistent, high-quality products, but do it in a way that was easy to scale,” says Kim Raineri, Avrobio’s chief manufacturing and technology officer. He and his colleagues turned off-the-shelf instruments into a system that accommodates the methods controlled by the company’s proprietary algorithm to make lentiviral-based gene therapy.
Raineri calls the resulting system “nearly fully closed and very nimble.” To switch the product being produced, “you just swap out the vector that provides the gene of interest,” Raineri adds.
This company’s pipeline includes six gene therapies for lysosomal-storage disorders. “Three of these are already in the clinic, and our platform can handle them all,” according to Raineri. Plus, this system provides fast bioprocessing—manufacturing a gene therapy in just three days and creating a ready-for-release gene product in about six weeks.
The 3-by-3-foot size system is also easy to scale out, notes Raineri. “Each unit handles one patient at a time,” he says. “With it taking only a few days to process a run, each unit can handle more than 100 patients a year.”
The platform also goes beyond manufacturing. “People often focus on manufacturing, but forget about QC and release,” points out Raineri. “We’re developing platform analytics that include purity assays and other safety features, as well as ones that measure vector copy number and potency of the gene therapy.”
As Raineri summarizes: “The key is analytics.”