By Erin Harris, Editor-In-Chief, Cell & Gene
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Orgenesis is a global biotech company, which is unlocking the full potential of personalized therapies and closed processing systems through its Cell & Gene Therapy Biotech Platform. I had the opportunity to talk to Orgenesis’ CEO, Vered Caplan about why the company sold its CDMO subsidiary, Masthercell, the importance of partnerships in driving down therapy costs, and more. Here’s what she had to say.
Orgenesis recently sold its CGT CDMO subsidiary, Masthercell, and is now fully focused on providing cell and gene therapies that may be manufactured at the point of care, working with hospitals and healthcare providers to enable them to participate in cell and gene therapy development on their sites and providing these therapies in house by adapting them to closed system manufacturing rather than relying on the typical biotech model. What does that mean for hospitals and healthcare providers focused on cell and gene therapy?
Caplan: Orgenesis until recently had two lines of business — its operations in the point of care business where we focus on providing CGTs working in partnership with hospitals. The other was a CDMO, Masthercell.
This was centralized manufacturing, and we were providing services for some of the leading biotech pharmaceutical companies and providing this as a third-party service. We felt they would need much wider services and that it made more sense for this activity to be integrated with other services that a large CMO like Catalent can provide.
We mainly wanted to focus on our point of care program. The service business was important, but we came to a point where we felt this business must be separated, because going from phase one, phase two, phase three to market is a big jump. That was a centralized service, and we would like to focus on the point of care for the supply of our own therapeutic pipeline.
How is the closed system different and advantageous?
Caplan: Let's think of it this way. What we’ve been seeing for instance, in hospitals, in Germany, even in the U.S. in some cases, where a hospital is saying “we want our own “homegrown” therapy. We want to provide our own therapy. We want to make sure our patients are doing well, and we’ll be able to cut costs in a massive way — in some cases they are claiming up to a tenth of the cost for the patient. What we need in order to do this is to have our own GMP quality supply capability.
For a hospital to go and set up a whole new clean manufacturing GMP facility is very challenging. Some hospitals have managed to do this, but it is challenging, and it also limits the amount of hospitals that can supply this kind of treatment.
On the other hand, if you have, I would call it a clean box, or closed system where you don’t need four people working on a product in a traditional GMP setting, and you can actually get these therapies made in a lower grade clean room with a much more automated system in a closed setting, then you'll make these therapies available to a many more hospitals.
In order to get that done, you must consider technologies needed for this, for example closed fluid systems, sensor systems, cells sorting. There’s a whole range of technologies. That’s the challenge - to bring them all together, to combine them with a therapy at an early stage and get this done in the hospital setting so it’s validated and can then be rolled out to every location.
What does this mean for the patient?
Caplan: We all know the science beneath the surface — it’s what’s leading this new era in healthcare. We have seen this in cancer treatment, and now it’s slowly moving into other indications. Both autologous therapies and allogeneic therapies have great potential. Autologous therapies are on the market, but the cost is so prohibitive, and that’s just painful to see for someone who comes from a biotech background. Biotech wants to bring these therapies to patients globally at a tremendous cost saving. The way to do this, we believe, is to link hospitals, therapies, and technologies, and to provide the therapies locally.
What other partnerships do you think are necessary to make this all possible?
Caplan: We try to be a hub. We need to try not to be one thing or the other. We try to bring in the therapeutic capabilities, bring in the technology capabilities, and bring in the hospital settings. On one side you have the partnership with the hospital, but no less important is having the technology to manufacture these drugs at the point of care and the license to the therapy. Developing these technologies in a way that they can be made available at an affordable cost is challenging.
It's not so simple to make these therapies in a closed system and at a high quality. In order to get that done, you must begin very early in the development process. This is not something you can start in a clean room and then go back and develop all the necessary technologies to then make it in a closed system.
From Day 1, you need to be thinking about developing this in a way that demands quality control; You need technology that can be validated during clinical development so you can actually get these therapies to market all in a closed system. Put another way, you need to develop the technology together with the biology from day one, and that’s usually done well under partnership models.
How concerned are you with partnership between hospitals and biotechs?
Caplan: We are trying to set an example; it’s important for us to partner with the hospitals and involve them in the therapeutic developments; to make them as motivated as a biotech company to get therapies out to patients instead of today’s situation, which is where a therapy is licensed to a biotech from a hospital and then the biotech pays tremendous amounts for clinical development and then eventually charges tremendous amount back to the hospital to cover the expenses.
But if you can work in a partnership model, the cost of development goes down, timelines drastically decrease, and you can utilize a wide network of clinical facilities. Working together on the development of therapies means they will ultimately be much more widely available for patients.
When we talk about collaborating, we mean collaboration and partnership. Our platform is based on this, not the typical model of hospitals on one side, biotech and technology companies on the other. Rather, networked into one system that benefit patients’ health.
We are in unprecedented times with COVID-19. How is Orgenesis handling COVID-19?
Caplan: This is a challenging time for everyone including biotech, and internally our development employees have received a special permit so they may continue to work on our programs and continue the work they’re doing. I really appreciate the risks they are taking to get this done.
Our scientists have been investing a tremendous amount of effort, researching the tools we have and their relevance for fighting this virus. We have about many different programs – technologies and therapies that are in various development stages, they are determining how existing programs may be utilized for the COVID-19 challenge as well as how they can combine these programs as quickly as possible with any available external technology, and are exploring if they can be of benefit in dealing with the COVID-19 situation. As an example, using different cells or different cell components, which enable the efficient transport of anti-viral components to the patient.