Miguel Forte, President, ISCT, board member of ARM and CEO of Kiji Therapeutics
Many believe that C> is inherently expensive and complex from a science and production perspective. That may be true today, but it is already beginning to change. That will require confidence, investment and forward-looking vision, as in most new technologies, in order to democratize the technology. The value is immense, the effort commensurate, and the reward significant if we are all prepared to keep moving the field forward.
John Maher, Chief Scientific Officer, Leucid Bio
Regulators often argue, with some justification, that their statutory remit is quality, safety and efficacy – not price, reimbursement, or affordability. EMA, for example, states that it has no role in pricing and reimbursement decisions, and FDA states that it has no legal authority to control drug prices. I understand the legal distinction, but I disagree with the implication that regulatory decisions are therefore neutral with respect to cost and access. In cell and gene therapy, regulatory expectations around manufacturing, comparability, release testing, evidence generation and post-authorization commitments can profoundly affect cost of goods, operational complexity, and the number of centers able to deliver a therapy. So, while regulators may not set the price, they absolutely help shape whether a therapy can be developed and delivered in a way that is scalable and equitable.
My view is not that standards should be lowered. Patients must be protected, and advanced therapies need rigorous oversight. But regulation should be proportionate, science-led, and explicitly conscious of its downstream consequences for access. Regulators are public bodies, and their role should include enabling responsible innovation – not only preventing unsafe or poor-quality products from reaching patients, but also helping ensure that transformative therapies can realistically reach the patients who need them.
Cell and gene therapies should not become medicines only for the few. If regulatory frameworks unintentionally make development so complex and expensive that only the best-funded organizations can participate, or only a narrow group of patients can benefit, then we need to ask whether the system is serving the public interest as well as it should.
Edwin Stone, CEO of Cellular Origins
One assumption I disagree with is the idea that low-volume manufacturing processes can simply be duplicated to achieve higher output volumes. In reality, scaling-up fundamentally changes how processes perform.
For example, efficient manufacturing depends on balancing throughput capacity across all steps of the process. At small scale, these constraints are often invisible; at commercial scale, they become the defining constraint.
I also think our industry can underestimate the importance of reliability. Automation is essential to the future of cell and gene therapies, but no system will function perfectly all the time. The real question is whether the manufacturing system can continue operating when something goes wrong. This requires detection, redundancy and recovery strategies built into system architecture from the beginning.
At small scale, people can step in to manage issues manually as they arise. At higher production volumes, reliability will define success or failure. That is why we need to design manufacturing systems with the same robustness expected in other advanced industries.
Daria Donati, Chief Scientific Officer of Genomic Medicine at Cytiva
One common misconception is that autologous therapies are inherently unscalable and will inevitably be replaced by allogeneic approaches.
I think this is an oversimplification. Autologous therapies are complex, however, they are also biologically powerful and clinically validated. The assumption that scalability must mean uniformity overlooks the possibility of digitally-enabled, distributed manufacturing models, where complexity is managed rather than eliminated.
The future is unlikely to be binary. Instead, we will see a coexistence of modalities, each optimized for specific indications. The real question is not autologous versus allogeneic. It is how we engineer systems that make both viable where they deliver the most value.
Daniel Vitt, Chief Executive Officer of Immunic Therapeutics
One widely held belief I disagree with is the idea that diseases can be addressed through a single, highly targeted intervention. There's often an assumption that identifying and blocking a single pathway is enough. Biology is usually far more complex.
Most diseases are multidimensional. Effective therapies need to account for that complexity, balancing efficacy, safety and long-term outcomes. We have seen this evolution over time. Even in well-established areas such as hypertension, treatments have improved significantly not by targeting one mechanism alone, but by better understanding the broader system and minimizing side effects.
This more holistic view of treatment is critical if we want to deliver meaningful and durable benefits for patients.
Ann Cleeves, VP of Application Science, BioPharmics Division, at Optibrium
There is a belief among many that the chemical matter required to address serious unmet therapeutic needs either already exists or is a minor structural modification from something that does. This assumption takes two forms. First, relying on drug repurposing as a strategy for drug discovery is perhaps too optimistic. This assumes that the cure for something like Alzheimer's disease, multiple sclerosis, or some form of cancer has already been made and has gone through regulatory approvals for some other disease. It is certainly true that nearly all drugs have multiple off-targets and that those are often quite different from the intended therapeutic targets. However, it seems extremely unlikely that an Alzheimer's cure is in a jar on a pharmacy shelf, and we just need to find the right jar.
Second, complex models that contain millions (or even billions) of parameters that have been trained on vast quantities of data, such as that in the PDB and ChEMBL, are very good at absorbing, indexing, and interpolating within the training corpus. Using such models can be valuable, but, as with drug repurposing, the challenges posed by serious unmet patient needs are likely to require structurally novel molecules that will require significant extrapolation beyond what has already been made and experimentally tested.
This is not to say that strategies that involve drug repurposing or mega-parameter AI models are not valuable. But drug discovery researchers should understand the assumptions that allow such approaches to be successful in some cases, and that those assumptions begin to break down in challenging and therapeutically important discovery scenarios.
Ali Pashazadeh, CEO of Treehill Partners
That drugs fail. They don’t, at least not most of the time. Management fails. We review hundreds of situations every quarter where clinical programs are going or have gone wrong, and in the overwhelming majority of cases the molecule was not the problem. The study design was wrong, the comparator was wrong, the endpoints were not commercially relevant, or nobody had assessed whether the competitive landscape would still support the product by the time the data arrived. Our analysis of 1,200 Phase II and III studies found that 80 percent contained at least one material design error. Only 5 percent of the companies had a commercial target product profile relevant at time of launch. These are not scientific failures. They are operational and strategic failures made by the ecosystems around the asset, and they are largely preventable.
Renee Aguiar-Lucander, CEO, Hansa Biopharma
That “science always wins.” Many factors contribute to making a drug successful. This is often, but not always, driven by the best science. One should not underestimate the impact of informal networks, professional connections, resources and geography.
Michael May, CEO, CCRM
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 centralised versus decentralised manufacturing. 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 personalised basis in a highly specialised operating theatre by highly trained surgeons at great cost. Alternatively, many patients receive stents to unblock their coronary arteries. These procedures are standardised 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 personalised autologous therapies in decentralised, point-of-care facilities and most, ideally, will be addressed through bulk-manufactured centralised facilities and the “pharmacy of the future.”
Rab Prinjha, Chief Research and Development Officer, Curve Therapeutics
That the drug discovery world is divided into just small molecules or antibodies. There is a whole other world between and around them that will be populated by the next generation of innovative cyclic peptides that bring the pharmacological benefits of those two extremes together into an emerging powerful therapeutic space.
Bari Kowal, Senior Vice President, Development Operations and Portfolio Management, at Regeneron
One misconception is that technology alone will transform drug development. In reality, its value is only realised when it is applied with a clear purpose and supported by experienced teams. AI, automation, and analytics can strengthen areas such as protocol design, feasibility, and data review, but they cannot replace judgement. If used poorly, they can create more complexity rather than remove it. The real opportunity is to use these tools to support better decisions, improve efficiency, and free teams to focus on the areas where human expertise matters most.
Paul Peter Tak, President and CEO of Candel Therapeutics
The assumption that building a fully integrated, in-house commercial infrastructure early is the optimal path. In reality, premature fixed investment can reduce strategic flexibility and create unnecessary cost burdens. A more adaptive, partnership-driven model provides immediate access to top-tier expertise without the delays associated with hiring and building large internal teams. It allows companies to access specialist expertise when and where it is needed while maintaining capital efficiency. Importantly, this approach does not preclude building capabilities in-house. Rather, it enables bringing the right resources inside at the right time, typically once approval is secured and the path to commercialisation is clear.
Claudia Zylberberg, PhD, co-founder and board chair of ARScience Bio, founder and chair of Akron Bio, and co-founder and board chair of Kosten Digital
That there will be one dominant model that solves everything.
I don’t believe the future is purely centralised or purely decentralised, or purely ex vivo versus purely in vivo. Advanced therapies are too complex, diverse, and global for a single solution.
The future will likely be a hybrid ecosystem where different manufacturing models, therapeutic modalities, hospitals, healthcare systems, and digital infrastructures coexist and complement one another. The challenge is not choosing one model over another, it is creating the infrastructure, standards, and interoperability that allow all these components to work together effectively.
Jason Bock, CEO of CTMC
One belief I disagree with is that in vivo CAR-T will simply replace autologous ex vivo cell therapy.
I understand why the field is excited. In vivo CAR-T is an elegant idea: deliver the genetic payload directly into the patient and turn their own immune cells into therapeutic cells inside the body. If it works safely, reproducibly, and at scale, it could remove some of the most challenging parts of today’s cell therapy model: leukapheresis, individualised manufacturing, chain of identity, complex logistics, and long vein-to-vein timelines.
But I think it is too simplistic to assume that this will replace autologous cell therapy.
Ex vivo manufacturing gives us something very powerful: control. We can select, activate, engineer, expand, characterise, release, and cryopreserve the product before it goes back to the patient. We can measure identity, potency, phenotype, vector copy number, viability, sterility, and other critical quality attributes. We can intervene in the process. We can learn from the process. And we can link product attributes to clinical outcomes over time.
In vivo approaches will need to solve a different set of hard problems: targeting the right cells in the right tissues, controlling the level and duration of expression, managing biodistribution, avoiding off-target transduction, achieving consistency across very different patients, and understanding how to regulate and reverse the therapy if something goes wrong.
So I do not think the future is “in vivo replaces autologous.” I think the future is more nuanced. Some diseases, targets, and clinical settings may be ideal for in vivo engineering. Others may continue to benefit from the control, characterisation, and flexibility of ex vivo manufacturing. The winning approach will depend on the biology, the patient population, the safety requirements, and the product attributes needed for clinical benefit.
The broader lesson is that we should not confuse convenience with therapeutic superiority. The goal is not simply to make cell therapy easier to administer. The goal is to make therapies that are safe, potent, durable, controllable, scalable, and accessible. In vivo CAR-T may become an important part of that future, but it will not eliminate the need for highly engineered, well-characterised autologous cell therapies.
Ali Tavassoli, Professor of Chemical Biology, University of Southampton; and Former Chief Scientific Officer and Co-founder, Curve Therapeutics
That peptides cannot be oral drugs. For most of my career the conventional wisdom has been that peptides are too large, too polar, too easily chewed up in the gut to ever work as oral therapeutics. That belief shaped which targets the industry was willing to pursue with peptides and which it wasn't, and it kept a huge swathe of biology off the table.
The work of the last fifteen years has steadily dismantled that assumption. Macrocyclisation, N-methylation, intramolecular hydrogen bonding, chameleonic behaviour that lets a molecule shift its properties between aqueous and membrane environments. These are not theoretical curiosities anymore, they have become design principles that practising medicinal chemists use to build orally bioavailable cyclic peptides. Several cyclic peptides coming to market have proved this point and there is a pipeline coming behind that proves the broader scientific one. I think we will look back on "peptides cannot be oral" as a generational assumption that turned out to be a failure of imagination rather than a law of nature.
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