Equipment suppliers are vying to fill gaps in processing say cell and gene therapy experts, but understanding what end-users need will be central for success.

In 2017, the US FDA approved two chimeric antigen receptor T (CAR-T) cell therapies. Since then there have been several other cell and gene therapies approved across several markets, including Europe’s first allogeneic stem cell in the form of Takeda’s Alofisel (darvadstrocel).

And with several hundred cell and gene candidates moving through the clinical stages of development, it is clear the industry is beginning to blossom. However, being in its infancy comes with several issues, one of which is in the manufacture of these new therapies and something Novartis has experienced firsthand.

This roundtable took place on Tuesday, 5 June 2018, in the BioProcess Theater at the BIO International Convention in Boston, MA. (Left to Right) Moderator: Dan Stanton, editor, BioProcess Insider. Panelists: Ohad Karniel (CEO and cofounder of Atvio Biotech, ISCT process and product development co-chair), Alan Moore (vice president and commercial chief for advanced therapies, WuXi AppTec); and Dr. David Brindley (University of Oxford and Harvard University)

Supplier innovation

One of the keys to improving systems is the relationship between manufacturers and vendors, experts said at the BIO International Convention in Boston in June.

“Suppliers are in the space and willing to innovate. They want to push forward and get a good position,” Ohad Karniel, CEO and cofounder of Atvio Biotech and process and product development co-chair at advocacy group ISCT, told an audience at the Bioprocess International Theater.

However, “the gap is in understanding what we need: That is, we don’t know what we need, and they don’t know what we need,” he continued, adding there is a need for innovation from the bottom up. “My interest is in developing technologies for this industry, so being with a CMO allows me to gain that hands-on experience, to understand GMP issues, and to talk with engineers.”

Alan Moore, vice president and commercial chief for advanced therapies at WuXi AppTec, said progress so far is encouraging. Moore cited how the Corning HYPERstack platform allows his contract manufacturing organization (CMO) to produce viral vectors in a more flexible manner, yielding up to 250 L from an inherent system.

“Bioreactor companies have awakened. They recognize the potential here. They’ve been listening to the expectations that the biomanufacturers have and the requirements for efficient production. Single-use systems are amenable or are morphing to accommodate the needs of cell therapies.”

He added equipment supplier are also continuing to innovate and are spurred by recent commercial successes.

Shortages

But demand for such technology is driving shortages, no more so than with Corning’s HYPERstacks, which have waiting lists as long as six months due to the surge in gene therapy development.

David Brindley from the University of Oxford and Harvard University referred to this as an obstacle in progressing cell and gene therapies. He advises companies to repurpose tools and technologies wherever they can to prioritize efforts toward codevelopment of what is truly not adaptable.

“We don’t appreciate the complexity of establishing a new production line for bags and what kind of investment that takes,” he told the Theater. “We’re seeing a lag, certainly, but what’s encouraging is that the dialogue is started, and we’re starting to talk about the numbers of units we expect to need in the future.”

You can watch the full interview online here.