Making facilities commercialization-ready is next big challenge for CGT sector

Bulging late-stage R&D pipelines suggest many in the cell and gene therapy sector are preparing to go market. For such firms, having the right manufacturing infrastructure will be vital for commercial success, says Ken Green from Vertex.

Commercializing a cell or gene therapy is a complex process. Once a therapy is approved, the developer has to manufacture it and bring it to market in a stable and sustainable way.

“The big question for us as an industry is how are going to commercialize the cell and gene therapies we have in our clinical portfolios?” Green told delegates at the Cell and Gene Therapy Manufacturing and Commercialization conference.

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“I think we’re expecting something like 50 approvals of cell and gene therapies over the next couple of years. And to date, obviously, we only have a handful which are commercialized. So how can we get our manufacturing facilities up to up to speed?”

And getting a commercial cell and gene therapy plant “up to speed” is a significant, multi-step challenge, according to Green.

“There is a lot of specialized equipment involved in making cell and gene therapy [….] there’s a small number of manufacturers and the equipment is not as robust as we would like it.”

Sterility

One major hurdle for firms looking towards commercial launch is ensuring products are sterile particularly, as Green points out, because many cell and gene therapies are made using semi-open processing steps.

“I think many of us deal with a number of open manipulations we have to do in grade A environments particularly in biosafety cabinets. So it’s a big concern for us. How do we ensure sterility through the manufacturing process?”

Particulates are another challenge. Green told delegates that because cell and gene therapy manufacturers cannot use sterilizing grade filtration steps like those used in traditional biopharma, the presence of some particles in products is inevitable.

“So what is acceptable? What types of particles whatever sources? How do we control those all kinds of really important questions?”

Scale-out

In traditional biopharmaceutical manufacturing, efforts to increase output have focused on using larger reactors and adding clean room capacity to meet increased demand.

For cell and gene therapies, such methods are not applicable according to Green, who said that instead a “scale-out” approach is needed.

“For patient specific therapies the traditional approach is not sustainable when you start thinking about how you have to add additional clean rooms or suites as your patient demand increases. So there is a big challenge for the industry around how we scale up in a sustainable manner. We can’t keep adding manufacturing suites as demand goes up!”

Again, the use of non-closed steps during cell and gene therapy production is the crux of the issue and will need to be kept in mind by facility design engineers.

“We have some steps which are not closed for autologous cell therapies. A lot of manual manipulation of product in biosafety cabinets, which introduces aseptic risk. So, instead of having a ballroom design, you have to have grade C and B and A suites and managing the flow of personnel and materials is incredibly complex.”

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