Despite funding for Cell and Gene Therapies becoming harder to come by in 2022/23, the outlook for pipeline and product approvals still looks strong, and there is an increasing focus on enabling technologies for therapy manufacturing.

Those were the conclusions from a panel discussion hosted by Stuart Lowe, Head of Cell & Gene Therapy at TTP, in January 2023. Alongside the four industry experts on the panel, we were joined by leaders from the UK’s ‘Golden Triangle’, specialising in therapy manufacturing and process technology – with CEOs making up about half the audience.

Our panel started off with a historical perspective, discussing how the excitement of potential treatments for incurable and common diseases, from blindness to cancer to cardiovascular disease, drove investment in cell and gene therapy companies. “It was the promise of new target spaces and powerful therapeutics,” explained Gonzalo Garcia, partner at life science investors Syncona. “The possibilities were endless, everyone piled in.”

The panel highlighted the current industry bottleneck in specialised technologies and supply chains to manufacture cell and gene therapies, hindering widening access to larger patient populations. This has led to a shift in emphasis – while several years ago the investment community pressed start-ups to pursue a therapeutics strategy, some of these firms are now pivoting back to focus on enabling technology, observed Jason C. Foster, CEO of Ori Biotech. “The time for enabling technology companies is now. How do we make these therapies widely available – high throughput, high quality, and low cost? Ultimately, that’s what the industry needs.”

This ecosystem of these enabling technology companies is growing in sophistication, and technologies to address challenges around manufacturing and quality control are in high demand. Molly Stevens, Professor of Biomedical Materials and Regenerative Medicine at Imperial College London, gave the example of Sparta Biodiscovery, one of the companies she has recently co-founded. The company’s recently launched benchtop instrument allows process developers to characterise individual nanoparticles relevant to making cell and cell gene therapies – be they lipid nanoparticles, viral vectors or exosomes. “Understanding what’s happening with your product and being able to perform live quality control for rational design is very enabling”, she said.

Access to cell and gene therapies and the investment landscape

Policymakers are taking a keen interest in the present challenge of therapy cost. Laura Beswick, who is Head of Health Economics and Market Access at the UK’s Cell and Gene Therapy Catapult, outlined the criteria for cost-effectiveness and mechanisms within the UK’s National Health Service for widening access to the cell and gene therapies. Together, the Innovative Medicines Fund and the Cancer Drugs Fund provide £680 million per annum to give patients access to innovative medicines. “I really look forward to seeing cell and gene therapies also for non-rare, more chronic conditions,” she said. They are “a huge burden on the NHS”.

Garcia also noted that the NHS, with its integrated set-up enabling excellent patient tracking and its deep understanding of cell and gene therapies, can make the UK an attractive market to develop or launch cell and gene therapies.

In line with this observation, the investment landscape is becoming more international, with more cross-Atlantic investment flowing into Europe, noted the panellists. Particularly, US-resident specialist funds that cover areas relevant to cell and gene therapy that are not covered by European funds may be worth having conversations with, suggested Foster. A show of hands revealed that, although most of the attendees were from the UK, a significant number had accessed investment from the US.

The current focus on enabling technologies could prove a catalyst for increased investor appetite for therapeutics companies. Building capability and capacity to manufacture these therapies for large patient populations will make the case for cell and gene therapies even more compelling.

Stuart Lowe leads TTP’s Cell & Gene Therapy team, which undertakes custom product development enabling new process analytics, unit operations, and end-to-end processes in cell and gene therapy.

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Stuart Lowe