Drug development for cell and gene therapy is a tripod; its three legs are academia, business and the state. Pull one leg out and it falls. Without university laboratories, we would not have a single therapy to market. And without government support through institutions such as the Cell and Gene Therapy Catapult in the UK, cell and gene companies would not be doing their best. For the foreseeable future, we can expect these things to remain true.
However, I think that if the funding worked differently, the academic leg could stand on its own for longer. The problem is that academics simply cannot raise, say, £150m to fund the commercialization of a therapy. This is where the private sector comes in, turning pure academic science into viable intellectual property.
Conversely, I seriously doubt that the private sector’s leg could ever stand entirely on its own. Although certain large pharmaceutical companies have established cell therapy development teams, I expect that these companies are much more likely to release new iterations of existing products than truly new therapies. This is where business needs an academy.
I believe we could advance this field much more quickly if we could establish a way earlier to distribute industry financial resources to academic programs. If we could run a large farm to fund a bakery, rather than buying bread, we would shorten the years of process development.
Although I would not claim to have all the answers to what is certainly a very difficult and inflexible problem, I would insist that new and better bridges be built between pharmacy and academia. You don’t have to take it on faith. I am living proof.
If we could run a large farm to fund a bakery, rather than buying bread, we would shorten the years of process development.
In 1999, I started my academic career with a master’s degree at the University of Birmingham, Great Britain. I stayed to do my PhD on how chemokines cause inflammation and inflammatory liver disease. After I finished that, I stayed on again, this time in a postdoctoral position researching the myeloid cell biology of monocytes in order to develop dendritic cells as a primary therapy for liver cancer. This was my first time working on a cell therapy program. This eventually led to my first involvement in a cell therapy trial, treating end-stage liver cancer with a dendritic cell vaccine. That trial reached its goal and closed during the COVID-19 pandemic, ultimately yielding positive results.
Over the last half decade, I have taken on the leadership of GMP activities for the University of Birmingham as a whole. We have grown from a small, self-contained facility to a facility with various academic and commercial partners. Today we manufacture a wide range of cell types and offer a wide range of GMP services for the university.
Adding commercial viability to academic centers could transform the offering into early-stage startups. This is where academic CDMOs tend to falter; they are simply not designed with commercial issues such as speed and contracting in mind. Pairing with suitable commercial partners could smoothly accelerate the transition of academic programs into the world of privately funded cell therapy trials.
In my university role, I’m expected to break even on my current facility, but I’m not being made to pay off shareholders.
As a sector, academic CDMOs need to show a way out for people stuck in the rut of trying to build a therapy entirely on grants. After all, the moves that get you a grant are usually not the moves that will help you establish a robust, sustainable business. We need to spare these people the imperative to regularly reinvent the wheel just to keep moving forward.
In the event of my company moving to the market, I do not expect a huge change in our core function – a CDMO with a strong focus on development. We will continue to work with commercial partners and focus on how they can complement our academic program. There are partial precedents for this here in the UK, where we have seen people take academic programs into our government-funded cell and gene therapy catapult and go on to raise impressive capital investment. However, in many cases there is a lack of preparation and a lack of understanding of what is required for commercialization. Often the company’s processes require expensive development that comes too late, after the company has already moved into rented production space.
Sound commercial partnerships should help ease such transitions. We need to take advantage of the proximity of academic CDMO centers to patient care and their populations of key thought leaders in centers of clinical excellence. Our goal should be to work closely with early-stage therapy developers to get the product and process right the first time.
If we can create a network of academic centers with the right industrial partnerships, the initial costs of establishing this cooperative enterprise will pay for themselves.
Skeptics may ask: doesn’t teaming up with commercial partners bring new problems, replacing academic games with business games? These are valid concerns, but all I can say in response is that if we are careful to establish key partnerships, we can still make a difference for patients. In business, of course, we have to ensure a return on investment – but the right market exists and is receptive, as we can see from the ongoing acceleration of the sector. In my university role, I’m expected to break even on my current facility, but I’m not being made to pay off shareholders. Developing a commercial strategy would mark a change in my work, but I don’t see it as a big challenge.
One of the factors we need to consider is scale. Academic CDMOs must take advantage of economies of scale to become profitable as there are huge costs involved in running a GMP facility. If we can create a network of academic centers with the right industrial partnerships, the initial costs of establishing this cooperative enterprise will pay for themselves. For example, you can achieve a degree of remote leadership and quality control – so these elements can be dispersed across your network, rather than replicated at each node. Therefore, the larger your network, the more you can dilute these aspects of your running costs.
Dispersed mesh is also suitable for delivering autologous therapies to patients as it helps to avoid the current situation. Right now we are sending materials thousands of miles to factories in the middle of nowhere only to have them shipped back again. This is bad economic practice, bad environmental practice and adds unnecessary high risk to your process.
Companies like mine must play a significant role in ensuring GMP manufacturing for the clinical development of cell and gene therapy after grant funding. We want to provide a bridge into manufacturing for smaller institutions that want to develop cell and gene therapies, but have neither the resources nor the need to hire a large CDMO. This will allow more cell and gene therapies from a wider group of specialist organizations to progress to the clinic and potentially reach an even wider group of patients than could currently benefit from therapies in development.
Head of Business and Project Development – Advanced Therapies at the University of Birmingham, UK