BPR: This is the fourth Horizons report on the Life Sciences industry produced by CRB. What do you think is the most surprising finding this time around?
The report goes deep on the various modalities that make up our industry, such as cell therapy, gene therapy, RNA technologies, and even how AI can remake manufacturing and make everything we do more efficient. The most surprising trend – and maybe the most encouraging –is the heightened focus on raising patient access to the incredible work our industry does.
There’s a fun but also serious analogy in the report made by my colleague Noel Maestre, CRB’s vice president of life sciences. Without spoiling the details, his point is that until researchers, manufacturers, regulators and insurers coordinate to move scientific breakthroughs into the broad marketplace, the advances we’re seeing – including cell therapies, gene therapies, and antibody drug conjugates – will never reach the patients who need them the most but may struggle to afford them. I think a growing number of our industry leaders understand this, and it gives me great hope.
BPR: The report explores the role of data in manufacturing. With so much talk about AI, what can we make about how the life sciences might be shaped by AI?
We had more than 500 respondents to this year’s survey, and we were surprised by how many – more than 90 percent – actively collect and act on data. More than 75% have long-range AI plans, so it’s clear that leaders are making their bets on digital transformation.
When you consider how AI can influence and speed many of the things we do every day, it’s natural to think about how AI might, for instance, design molecules to cut safety risks and reduce regulatory complexity. But we have to act with extreme care, given what’s at stake with patient health.
BPR: There’s a line in the report that suggests innovation and discovery have tapered off in the life science industry? What do you think is behind that fallback?
Some of the industry’s leading companies are pulling back from certain manufacturing programs to focus on what they believe will be competitive. We’ve seen some big players discontinue their gene therapy programs, for instance, to focus on core therapeutics. Overcrowding has something to do with this, but AI and automation changes are driving companies to prioritize R&D. In this case it isn’t a pullback from innovation, but a change in how companies are approaching it. Intelligent tools show us how discoveries that would’ve taken months to identify and document could be possible in just weeks or days.
BPR: The report found multiple differences in the way North American and European companies are approaching the market. Can you describe one of those?
The data shows that while nearly all surveyed mRNA experts pointed to the potential of mRNA for therapeutic vaccines, vaccine developers continue to embrace coding RNA to prevent infectious diseases. One of our European subject matter experts, David Estape, found that despite universally high expectations for the development and production of therapeutic vaccines, significant differences exist between U.S. and European producers in their approaches.
Clearly, there’s a higher focus in Europe on prophylactic vaccines. I think where North Americans value fundamental characteristics of mRNA technology, Europeans are focused more on the advantages in the development of mRNA products. Another interesting data point: While Europeans had positive opinions about most factors related to prophylactic vaccines, only about a quarter of them said their company was active in this modality. That suggests decisions about what modalities they pursue are influenced by economic and business factors rather than R&D advantages.
BPR: In your piece on gene therapy, you noted a split when it comes to gene therapy. Some companies are pulling back or investing in stable producer lines, while another group is focused on emerging non-viral manufacturing methods to reach patients. Can you explain?
We’re seeing that after years spent developing viral-based platforms, the gene therapy submarket is moving toward non-viral methods. The promise of CRISPR technologies and other factors are likely driving this; we saw about a quarter of our respondents say they plan to develop vectors using lipids, peptides or polymers as opposed to viruses.
We also found some differences in the responses based on company size. Small-company respondents said they were less likely to pursue non-viral means, maybe because they’ve been focused on leveraging platform-wise what is already out there. Medium-sized companies are eager to pursue these emerging platforms. As for large companies, they appear to be holding steady with viral-based platforms after the 2021 financial boom and seem focused on seeing ROI from that. It’s hard to blame them, when you consider the expense of moving to CRISPR, which many believe is unproven and costly.
BPR: More than 80 percent of your survey’s responses reported having a cell therapy product in their pipeline. There was a lot of excitement in the report about some of the breakthroughs that are elevating cell therapy from boutique research to “industry mainstay.” What do you think is behind this?
In the early days of cell therapy research and development, it was mostly small- and medium-sized companies that were pioneering cell therapy. But now large companies have moved in. Our data suggests that commercial readiness and decentralized manufacturing are probably behind the speed-up toward efficient CGMP manufacturing.
But there’s another factor getting attention – the role of new gene editing technologies. Our data reveals a kind of inversion, suggesting the focus on viral vector manufacturing will soon expand to include a future of RNA-based gene editing. The regulatory impacts of these technologies will help speed these products to market, and our report explores those impacts in-depth.