Cell and gene therapy has evolved into one of the biopharma industry's hottest markets, with a major splash of investment and a run of approvals anticipated in the coming years. To meet what's likely to be massive demand for manufacturing capacity, industry experts are calling for "forward-looking" investments—but as one pointed out, those checks aren't easy to write.
During a virtual roundtable Monday hosted by Fierce Pharma, manufacturing experts from Novartis, Gilead's Kite unit, BioMarin and Astellas' Audentes said building capacity and figuring out how to scale production would make the difference in whether the industry will be ready to handle a slate of expected approvals.
According to former FDA Commissioner Scott Gottlieb, the agency could approve between 10 and 20 cell and gene therapies a year through 2025, with as many as 800 such therapies moving through drugmakers' pipelines.
Chuck Calderaro, Kite's global head of technical operations, said Kite's primary concern was how to scale production of its cell therapy offerings, including Yescarta, the company's only approved therapy in the space.
VIRTUAL ROUNDTABLE: Up to the Challenge: Manufacturing’s Central Role in the Cell- and Gene-Therapy Revolution (reg. req'd)
Calderaro noted that Kite has a turnaround time of 16 days from patients having their blood drawn to infusion—and the drugmaker is hoping to keep that figure stable as its manufacturing chain grows.
"Access to capacity is always a challenge in a growing area, and especially in cell therapy, which is personalized to order," Calderaro said. "The challenge for us is to be able to scale that excellence as we begin to globalize our cell therapy treatments."
The first challenge for Gilead will come from bringing its newest facility in Amsterdam online after the EU gave it the go-ahead earlier this month.
Gilead's 117,000-square-foot CAR-T facility at SEGRO Park Amsterdam Airport won a green light after the European Medicines Agency (EMA) approved the plant's end-to-end manufacturing process. The site will house European production fo Yescarta, which won an EMA approval back in August 2018 to treat relapsed or refractory diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma.
Calderaro highlighted the Amsterdam facility as the "next step" in Gilead's global manufacturing ramp-up for Yescarta, as the site will be able to churn out enough of the pricey therapy for 4,000 patients each year.
Gilead also plans to build a 67,000-square-foot facility at its Oceanside, California, biologics site just for developing viral vectors, the tools needed to deliver genetic material into cells. Calderaro said that facility would give Gilead "a little more control" of its supply chain as it looks to scale up.
Meanwhile, Steffen Lang, Novartis' head of technical operations, pointed out that building capacity for the future will go hand-in-hand with building an experienced team to lead.
"It’s about the people and capabilities," Lang said. "You have to have the right team in place to build capacity at the appropriate scale across the globe."
Novartis' gene therapy Kymriah was the first FDA-approved therapy of its kind back in August 2017. Since then, the Swiss drugmaker has expanded Kymriah's reach globally, including opening a new facility in Stein, Switzerland, that cleared up a transatlantic bottleneck for shipments to Novartis' facility in Morris Plains, New Jersey.
Meanwhile, BioMarin and Audentes are both pursuing first FDA approvals for their cell and gene therapy candidates and are looking to scale up manufacturing to make the leap into commercial.
In February, Audentes announced it would invest $109 million into a 135,000-square-foot facility in Sanford, North Carolina to flesh out its cell and gene therapy manufacturing needs. The first phase is slated to take 18 months to build and will be operational by 2021. The rest of the investment will play out over two more years.
Donald Wuchterl, Audentes' SVP of technical operations, said building capacity would require a "forward-looking" approach to investing—but the lengthy timelines for these therapies make that foresight difficult.
"These are tough checks to write," Wuchterl said. "We’re looking at potentially three years out in a field that’s growing rapidly—it takes some constitution, I would say."
BioMarin, which is in the homestretch for an FDA approval for its hemophilia A gene therapy candidate valoctocogene roxaparvovec, or valrox, is in a similar boat as it works to reach the commercial space. For the Robert Baffi, special advisor to the company's CEO, scaling up manufacturing is a big task––but he hopes that a "biology revolution" could provide a big breakthrough for production in the coming years.
"While I think there's improvements to be had on the manufacturing side today, I think there's a biology revolution still to come in terms of making the vectors more specific, more targeted, more precise—and that would be a big boon for the industry," he said.