Overseas cell processing may take a hit as FDA reviews trials

A Food and Drug Administration crackdown on new clinical trials that send U.S. patients’ live cells overseas to China for gene editing and return infusion could negatively impact smaller cell and gene therapy companies that rely on foreign manufacturing, according to data and analytics firm GlobalData.

In June, the FDA announced an immediate review of new clinical trials that export Americans’ cells to foreign labs in China and “other hostile countries” for genetic engineering and subsequent infusion back into U.S. patients — sometimes without their knowledge or consent.

“While the scope and impact are unclear, smaller cell and gene therapy companies that rely on foreign manufacturing may be hardest hit,” GlobalData said in a Sept. 15 report. “Regulators haven’t specified how many trials are affected, though therapies like chimeric antigen receptor T-cell (CAR-T), which sometimes use overseas manufacturing steps, could fall under the policy.”

A spokesperson for the Alliance for Regenerative Medicine, which represents the cell and gene therapy industry and has more than 400 members across 25 countries, told Pharma Manufacturing that they are “not aware of any examples of this occurring” so far with any clinical trials and “can’t determine the impact of this policy” at this stage of implementation by the FDA.

“As the FDA’s recent stance continues to evolve, the full impact remains to be seen,” Abigail Beaney, editor of Clinical Trials Arena at GlobalData, said in a statement. “However, the decisions cell and gene therapy (CGT) companies make under the Trump administration may indicate a trend toward more cost-effective development strategies.”

Fabian Gerlinghaus, co-founder and CEO of integrated development and manufacturing organization Cellares, said he is not surprised by the FDA’s recent action. 

“Governments want tighter control over patient-derived materials and genomic data,” Gerlinghaus said. “Shipping American patients’ cells overseas for processing is not a viable path, especially for smaller cell and gene therapy companies. The bigger picture is that long-haul, cross-border logistics were never a sustainable foundation for commercial cell therapy.”

Centralized, domestic manufacturing “shortens vein-to-vein times, reduces risk, and increases confidence that therapies are being produced under the same rigorous, auditable standards,” according to Gerlinghaus. “The challenge is that the industry has to move faster to put the right infrastructure in place.”

Cellares is deploying its Cell Shuttle, which automates cell therapy manufacturing, and Cell Q, which automates quality control, at the company’s “smart” factories. The first commercial scale smart factory is operational in Bridgewater, New Jersey, with other facilities under construction in Europe and Japan.

“Sponsors can manufacture domestically in the United States, scale on demand, and expand globally by replicating a validated template in Cellares’ Smart Factories in Europe and Japan,” Gerlinghaus contends. “It’s how smaller biotechs get enterprise-grade capacity close to home, without the CAPEX [capital expenditure] burden or the operational complexity of running many small sites.”

Jason Foster, CEO of CGT technology company Ori Biotech, says the FDA policy is a reminder that advanced therapy manufacturers need flexible, multi-site manufacturing capability in all the major geographies where they are shipping approved products to patients.

“Right now, if a site ex-U.S. couldn’t supply the U.S. any longer, it would take one to two years to get another qualified site up and running from scratch in the U.S. due to the highly manual nature of processes, tech transfer, and analytical methods,” according to Foster.

Advanced automation platforms like Ori Biotech’s IRO system, which automates, digitizes, and standardizes labor-intensive steps of CGT manufacturing, could potentially shorten the time to get new capacity up and running, Foster contends.