Unlocking the Potential of Cell and Gene Therapies

In a video interview with Pharma Commerce, Jason C. Foster, CEO of Ori Biotech, describes how the current state of cell and gene therapies (CGTs) shows promising potential, particularly in curing cancer and rare diseases. These therapies are safe and effective, with evidence demonstrating their curative capability. However, their widespread adoption is hindered by challenges in manufacturing at large scales and at affordable costs, limiting patient access. While three CGT products have advanced from last-line to second-line therapies, the number of patients receiving treatment has remained relatively unchanged, signaling that access, not prescription willingness, is the primary issue.

Only about 10,000 patients were treated with CGTs in 2024, out of a much larger global pool of patients who could benefit, with access in the United States at just 20%, and far lower in other regions. The real obstacle is the inability to produce these living, often personalized therapies at the scale needed for broader distribution. Many existing production processes rely on outdated technology that cannot meet the demand or provide the level of access needed.

This situation creates a vicious cycle: limited access leads to doubts about the commercial viability of these therapies, affecting investment in future innovations. CGTs cost billions to develop, and investors and companies require financial returns to continue funding new therapies. If the therapies cannot reach the market or gain sufficient coverage, the commercial future of CGTs is uncertain, risking investment in this area and potentially diverting capital to other treatment modalities. Overcoming these manufacturing and access barriers is crucial for unlocking the full potential of CGTs and improving patient outcomes globally.

Foster also comments on the manufacturing bottlenecks; regulatory hurdles; role of automation; and economic and healthcare system challenges pertaining to CGTs.

A transcript of his conversation with PC can be found below.

PC: Can you provide your perspective on the current state of CGTs, along with their potential for transforming medicine?

Foster: I think we've demonstrated beyond a doubt, the curative potential. These are safe products, these are effective products, and they offer the opportunity to cure cancer and rare diseases, and that's not really in dispute. I think everyone sort of agrees that that's the case. What's really holding the industry back today—and ultimately impacting access for patients—is that these products just can't be made in large enough quantities and at a cost that can make them widely available. Clinically, we have three products I believe that have moved from last-line therapy, where they started, to second-line therapy, so they moved up several rows or several lines of therapy, showing that they're clinically effective after those earlier patients. But still, the number of patients getting access has basically stayed the same, and that shows us that the constraint isn't actually necessarily prescribing or the willingness of physicians to use the products.

It's more about, how do we make the products available? To your point, in almost all the products that are available only in the US or in some small parts of Europe, all together, there are eight or nine approved cell therapies. I'm going to focus most of my comments on cell therapy—gene’s a little bit different—but both the comments apply to both. Across all of the approved cell therapies, we treated about 10,000 patients last year in 2024 and that is great for those 10,000 patients and their families, but unfortunately, that leaves hundreds of thousands of patients that didn't get treated. The numbers, the statistics you often see include that about 20% of patients in the US are able to get access to these therapies, which isn't a great number; one in five. Ultimately, most of these patients are very sick, and this is their last hope.

If only one in five can get access, clearly those other four out of five are having the worst outcome, which isn't what any of us want. In the rest of the world, it's far fewer than that. The global average is about less than 5% of patients get treated. That’s the current state of affairs. I think the challenge that we face is we're trying to apply lessons from small molecules and biologics to a totally new modality. These are living medicines. They're often personalized for that individual patient. This brings with it a whole host of unique challenges, and the platforms that were developed for regenerative medicine or other types of manufacturing just aren't good enough.

They haven't been able to solve the problem. In a lot of our processes found in currently available products, we're using 10-year-old or 15-year-old technology, which has proven it can't scale and proven it can't actually create the kind of access that we would want for patients. That's the big issue facing patient access today. Because of these challenges, there's kind of this, I don't know what the negative of a positive cycle is. It's the opposite of that, which is unfortunately because these products haven't been able to reach patients in large numbers, there's a question about their commercial viability.

You know very well that it costs $1 billion or $2 billion to develop a cell therapy or a gene therapy. There's a couple billion dollars potentially at risk, so the investors that put that capital up and the companies that spend 10 or more years developing it need to be able to make that money back and make a profit to invest then in the next wave of therapies. Unfortunately, the commercial viability of these products is in question. Can they actually reach the market? Can they actually get market access and get coverage? Will they get prescribed for patients, because if the answer is no to those questions, those early-stage venture capitalists might look elsewhere, in other modalities, for other types of therapies that could be developed, because ultimately, it's a business and it needs to deliver an ROI.