Manufacturing cell and gene therapies at hospitals in dedicated, GMP-accredited facilities would overcome many of the technical and logistical challenges associated with decentralized production. So says Na Kyung Lee, PhD, from Sungkyunkwan University in Seoul, Korea, in new research looking at factors limiting access to cell and gene therapies.
“In-house manufacturing in a GMP facility of a hospital represents a centralized approach to manufacturing that secures patient accessibility,” she wrote, citing reduced production timelines as the critical advantage. “The quality of the tissue source—the starting material—can be ensured through rapid delivery from the operation room to the GMP facility, which would reduce the time delay between manufacturing and clinical application and enable parallel production and patient monitoring at a single location.”
Lee and co-author, Jong Wook Chang, PhD, contrasted this with outsourced production models, arguing that simplified logistics are the key benefit of the in-house approach. They used CAR-T therapies as an example.
“If a hospital has no GMP facility, CAR-T cell production must be outsourced, involving local cell collection, cryopreservation, and shipping to the manufacturing site. Outsourcing of the manufacturing process, followed by the shipment of the final product back to the point-of-care location, increases the vein-to-vein time,” they say.
According to Lee and Chang, the minimum vein-to-vein time for a CAR-T cell therapy made using outsourced production is 28 days. In contrast, a closed, automated, single-use GMP-compliant system at a hospital could produce a therapy within two weeks.
“This advancement will provide a standardized method for producing CAR-T cells and eliminate the influence of manual handling or manipulations on the overall quality of the manufactured CAR-T cell product,” write the scientists.
Spin-off idea
So, in principle, on-site GMP production is the ideal approach in terms of patient access. The difficulty for hospitals is that setting up such a production operation can be costly and complex.
“The high cost and [achieving] GMP regulatory compliance are just a few of the multiple challenges hospitals face in building a GMP facility. Hospitals must be able to pay for the costs of building and maintaining a manufacturing facility, and the facility must abide by GMP laws and regulations,” continue the investigators
An alternative option, for hospitals that lack the requisite funding, would be to establish spin-off manufacturing operations in partnership with neighboring hospitals, according to the authors.
“These spin-off firms could maintain a strong network with local hospitals and specialize in meeting their manufacturing needs. Ideally, the spin-off companies would provide one-stop services where the production and manufacturing of cell, gene, and cell-based gene therapies occur at a single location near the point-of-care and, most importantly, the patient.”
