What was your biggest professional highlight of the past 12 months?
When CCRM was established 15 years ago, we were very forward-thinking in recognizing biomanufacturing as a major gatekeeper of success in the emerging field of cell and gene therapy. Over the last 12 months, we have boldly looked ahead again at what will enable advanced therapy industrialization in the future, establishing DeepTech Bio Lab with founding quantum computing leader IonQ. The DTBL will explore how digital tools, AI/ML, quantum computing/sensing, and automation (e.g., deep tech tools) will enable digital twins for the complex biology, manufacturing and logistics/supply chain hampering the widespread adoption of advanced therapies.
What is the biggest bottleneck slowing progress in the cell and gene field right now – and how do we fix it?
The most significant bottleneck slowing progress in the field is access to capital, particularly at early stages, driven by the retreat of investors after an era of overexuberant investing. Attracting investors back to the sector, which has a steady pipeline of amazing clinical outcomes and regulatory approvals, now requires demonstration of durable commercial success. Too many of the sector’s launched products have not achieved expected adoption and sales. Even at the earliest stages of development, we must ask the question: “Will someone pay for the product we are developing?” This requires us to regularly evaluate the “therapeutic headroom” associated with a product, which is tethered to standard of care. Standard of care is not static and will improve over the years-long development of a new product. If the therapeutic headroom of a product is not large enough when a product is finally launched, the market (e.g., patients/health care providers/payors) will assess the balance of benefit, cost, and risk effectively and efficiently, regardless of the “gee whiz” science behind the product.
Where is the cell and gene industry making real progress that isn’t getting enough attention?
The ability to reprogram induced pluripotent stem cells (iPSCs) and then differentiate them into appropriate cell types that can be genetically engineered for safety, manufacturability and enhanced function continues to advance. There is real progress, particularly when viewed in the context of companies such as Bluerock Therapeutics getting these complex products to the clinic, a source of frustration for developers and investors, alike, in recent years. The allogeneic paradigm is still an important pathway to achieving cost-effective, mass adoption of cell and gene therapy products for large markets in the future.
What’s one widely held belief in your field that you disagree with – and why?
I become frustrated with the belief that the development and global adoption of cell and gene therapies is completely different from our historical experience. We continue to debate autologous versus allogeneic and centralized versus decentralized manufacturing; but despite the key difference that advanced therapies are living, we have seen most of the challenges before.
For example, if we think about the treatment of heart disease, the most extreme cases are treated through a heart transplant, which is done on a personalized basis in a highly specialized operating theater by highly trained surgeons at great cost. Alternatively, many patients receive stents to unblock their coronary arteries. These procedures are standardized and still delivered in hospitals by trained physicians, but the stents are not manufactured in the hospitals. Most patients of heart disease are, however, treated with small molecules that are bulk manufactured in centralized facilities at low cost in highly regulated facilities. These drugs are prescribed by physicians through a network of pharmacies.
While it is interesting to envision what the pharmacy of the future might look like, a future enabled by cell and gene therapy will be similar to above: Some indications will be addressed by personalized autologous therapies in decentralized, point-of-care facilities and most, ideally, will be addressed through bulk-manufactured centralized facilities and the “pharmacy of the future.”
What’s the smartest way to navigate today’s funding and commercial landscape in cell and gene therapy?
Even though many emerging products have failed to meet commercial and adoption expectations, the key value inflection in the sector remains demonstrating clinical efficacy as quickly as possible, especially if the clinical indication/target market is chosen in the context of: “Is this an indication for which a cell and gene therapy approach is truly differentiated against standard of care and other modalities?”
If you could introduce one policy change to accelerate progress in your field, what would it be – and why?
Cell and gene therapies are arguably the most complex medicines ever developed, and their complexity will continue to grow. Consequently, it is likely that their mass adoption will require the greatest amount of human collaboration ever seen in therapeutic development. This will require regulatory policy (and other activities) to evolve so that prior collective knowledge and experience can be leveraged in the approval process. It will be unsustainable if personalized medicines or gene therapies for rare disorders are not able to leverage collective, modular development and shared submissions.
What will look completely different about drug development in 10 years?
The development of future medicines will be driven by data, AI and quantum platforms through much greater use of in silico modelling. In addition, the products will be validated by data-enabled, non-animal (human organoid) disease models. While laboratory validation will always remain essential, the transformation will be so stark that “techbio” may be used as much as “biotech” to describe our industry.
Do you think your younger self would be surprised to see the career you’ve had?
I don’t think so. Since I was very young, I have been a daydreamer. I believe that dreaming about your future helps to realise it. What has surprised me is the extent to which our world is driven by networks, including the network of collaborators and teammates required to advance great science to patients.
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