What was your biggest professional highlight of the past 12 months?
My leadership role in the field of cell and gene therapy as President of ISCT has been challenging, stimulating and rewarding. I have had the opportunity to help shape the field while contributing to the growth of ISCT by strengthening the Society's global presence and local impact. One achievement I am particularly proud of is establishing the society's clinical pillar through the appointment of its inaugural Chief Medical Officer, alongside the existing leadership in regulatory, academia, and industry.
What is the biggest bottleneck slowing progress in the cell and gene field right now – and how do we fix it?
The greatest challenge in cell and gene therapy (C>) at present is patient access. Limited patient access – driven by constraints in manufacturing capacity, clinical delivery, and above all reimbursement – is restricting both treatment and confidence in the sector's business models. This, in turn, is restricting investment, further limiting the growth and maturation of the field. New business models and greater confidence in the long-term value of these therapies are needed if we are to expand access for patients with unmet medical needs, many of whom could achieve long-lasting, potentially curative outcomes.
Where is the cell and gene industry making real progress that isn’t getting enough attention?
Allogeneic cell therapies, particularly those derived from induced pluripotent stem cells (iPSCs), continue to make important progress through improvements in manufacturing and genetic engineering. Although they hold enormous promise, they are not receiving the same level of attention as other areas of cell and gene therapy. I believe the future of cell and gene therapy will rest on three complementary approaches: autologous ex vivo therapies, where we have already seen significant success; in vivo therapies, which are attracting considerable attention; and allogeneic therapies, where much of the untapped potential lies.
What’s one widely held belief in your field that you disagree with – and why?
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 democratise the technology. The value is immense, the effort commensurate, and the reward significant if we are all prepared to keep moving the field forward.
What’s the smartest way to navigate today’s funding and commercial landscape in cell and gene therapy?
In the current risk-averse environment, traditional investment is becoming more challenging. It is important to strengthen the message and the value proposition, and often clinical data, derisking and validating the project is required. I am concerned that we may not be investing enough in the initial steps of the translational path, where there is high risk and little supporting evidence, and in doing so not feeding the portfolio of companies. Some will disappear but we need a natural evolution and selection also in company creation and growth. At the same time, existing companies are pursuing alternative paths to investment mainly via business partnerships, licensing, and mergers and acquisitions as well as other financial approaches, to enable survival and progression towards further validation and de-risking of the value proposition.
If you could introduce one policy change to accelerate progress in your field, what would it be – and why?
Operational changes may be even more important than policy changes. One would be faster clinical trial approval and start-up. This is critical to accelerate evidence generation and enable project progression and funding. Another would be greater pragmatism and a broader view when making Health Technology Assessment decisions so that multiple perspectives on evidence could be considered, such as small sample sizes and single arm trials, particularly in orphan indications.
Payment models should better reflect the long-term value of these therapies, including future cost savings, while supporting more flexible health technology assessment and reimbursement decisions.
What will look completely different about drug development in 10 years?
The merging of improved gene editing capabilities and AI will drastically transform the field over the next decade. Development will be proactive and by design rather than exploratory. AI models will be able to modulate and reduce experimental need.
Do you think your younger self would be surprised to see the career you’ve had?
I'm sure I would be surprised. Whether pleasantly or not is another question! I could never have imagined progressing from working with some of the first patients treated during the emergence of HIV to using related viruses to genetically engineer cells for therapeutic purposes. I guess I would be pleased and proud to have progressed from single patient clinical management to population and field impact!
Newsletters
Receive the latest pharmaceutical news, personalities, education, and career development – weekly to your inbox.
