Opportunities and Challenges in Cell and Gene Therapy Development

One of the most revolutionary trends driving the biopharmaceutical sector is cell and gene therapy. According to the Journal of Gene Medicine, between 1989 and February 2016, over 2,300 clinical trials were conducted with more than half of them in phase one.

In a recent interview, Ger Brophy gave his perspective on this growing segment of the bioprocessing industry as he discussed several key trends and challenges. 

Is the excitement about cell and gene therapy justified—or is it hype?

I think it’s a very exciting space, and we’re seeing the number of trials grow. It’s probably most exciting because of the technology’s ability to impact patients’ lives. Yes, the numbers of patients are relatively small at this point in time. This is partly because biopharma researchers and manufacturers wanted to deal with small populations that they understood well and, in many cases, that didn’t really have many other options for treatments.
We’re seeing these companies moving on now to larger populations — starting with leukemia, now lymphomas. From our perspective, the Holy Grail is multiple myeloma. If those patients begin to see benefits from cell and gene therapies, I think the excitement will feel justified.

Why is cell and gene therapy considered such a game-changer for the biopharma industry?
The game-changer here is that you are using the body’s own systems, either from a cellular immune system or from the ability to repair and replace defective or missing genes. We’re seeing the patient’s own immune system used to fight cancers. CAR-T cell therapy is arguably among the most personalized medicine one can consider. The patient’s own T cells are extracted, modified, activated, expanded, purified and returned to the patient.
Among the first patients treated for acute lymphoblastic leukemia, several are alive and thriving — four, five, six years later. The promise of personalized medicine has been held out for a long time. We’re actually beginning to see real, tangible effects from the molecular knowledge now that we have an understanding of how the disease develops and how the patient responds to it.

Tell us about the industry—what kind of companies are working today in cell and gene therapy and what are some of their major challenges?
Many of the early movers in cell and gene therapy were small biotech startups. Increasingly, we’ve all seen a greater interest by the major biopharma industry. Novartis was probably the biggest; it started the earliest, and was successful in getting approval for Kymriah. As companies of this size get involved, we expect they will leverage their increased breadth and depth to develop new labels, develop new trials, and find ways to manufacture these therapies at scale.

And two of the most critical challenges are scalability and manufacturability. If we can manufacture these treatments at scale, then can we do so safely? Can we do so at a reasonable cost so the populations that are affected by these diseases can access treatments?

Variables and failure modes have to be taken out of the process. If we can improve technologies like sterile fluid transfer, use excipient technology to further stabilize these technologies and use analytical technology to understand what will make a successful or less successful therapy, we can increase the efficacy, decrease the risk and decrease the cost.

This is where Avantor can help, since we supply cell-culture components, production chemicals and single-use technologies that aid in these processes. I think our knowledge – of cell culture, of technology development, sterile fluid transfer, fill and finish and excipients and the technology surrounding those – will be valuable and applicable to helping make these technologies available at scale.