Cell therapy is complex with a lot of moving parts, said Phil Vanek by way of introducing the panel he brought together for a session dedicated to one of the hottest segments in the biotech industry.
“There is a lot of interest in this space, and I don’t want to use the H-word, but we did a study and calculated that the top seven companies in this space have a market cap in excess of USD 12 billion without a single product on the market,” he said.
The General Manager for Cell Therapy technologies at GE Healthcare, Vanek moved the panelist through clinical, manufacturing, and regulatory processes, persistently teasing out both the promise and the pitfalls in this emerging field.
And yes, it is complex, and yes it is hard to get a grasp on where we are with all the moving pieces of the puzzle.
The clearest line-of-sight came at the end of the session when Vanek asked a simple, almost silly question, that provoked unexpected clarity: If you had a magic wand, how would you use it?
Here are the answers.
Georges Rawadi, Celyad
From Georges Rawadi, VP for Business Development at Celyad: “We are at the beginning of something we don’t yet control. If I was to dream, I would want clinical success in many indications, to show we can cure patients, that we can change the development of disease, and not just give patients an incremental therapy, but really curing them.
I think there are many technologies that will develop and we will need these developments for technologies not as expensive as they are today, and for miniaturization. And we need a market, which is not there.”
From Thomas Liénard, Chief Business Officer at Bone Therapeutics: “Thinking of allogeneics, how can we improve our yield so we rely less on less-healthy donors? How do we extend the shelf life to reduce the logistical issues? How do we make the back-and-forth between the patient function better? These are things we are currently working on improving and we need to solve if we want this to become a great commercial success. Clinical stage is one thing, the commercial stage is many other things.”
From Miguel Mulet, Director of Strategy and New Projects with TiGenix: “There are issues we all face in trying to make a pharma-like product. Besides the ones Thomas mentioned, there is an issue working with the raw materials within the regulatory constraints. So if I had a magic wand? I would use it to understand very clearly what regulators will allow me to do to change the ways I produce the product yet still give a product that will do exactly the same thing.”
From Jeff Till the Director, External Innovation for Merck: “A black box that would do it all.
“But for now, key is basic science and understanding how these therapies work and why they work, and mitigating the safety risks. The field is moving very quickly. We see strong efficacy. It is upon us to develop the fundamental biology, the science, so we can understand what is happening in here, understand what it is capable of, the range of possibilities before us.
“The other area would be seeing the infrastructure for cell therapy grow. With a magic wand we would have experts we can hand off our process and they could manufacture as we need. Infrastructure will be a tipping point for cell therapy. when there is enough manufacturing, logistics and things as simple as defined media available. These are some of critical milestones that could change the tempo of development in cell therapy space. If I could wave that magic wand and have all that infrastructure and expertise in place, the science. That would be great.”