Cell and gene industry considers Trump agenda amidst positivity over growth

Krishna Kakkaiyadi of Pinsent Masons said a ‘patient first’ approach will help industry build on the growth opportunities it sees for CGTs and meet major challenges that still exist to taking CGTs through the development lifecycle and into commercialisation.

Kakkaiyadi was commenting in the aftermath of the recent cell and gene state of the industry briefing hosted by the Alliance for Regenerative Medicine (ARM). Discussion at the event ranged from: the growing pipeline of ‘blockbuster’ CGTs, to the level of investor interest in CGTs, how the policy agenda of US president Donald Trump will impact on market growth, and the significant opportunities for commercialising CGTs in countries outside of the US.

The briefing from the US is an annual event attended by nearly 2,000 of the CGT sector’s top stakeholders and runs parallel to the larger JP Morgan annual healthcare conference in San Francisco. In addition to the widely anticipated industry update from Tim Hunt, the chief executive of ARM, this year’s event included a session on updates on the US Food and Drug Administration’s efforts to accelerate advances in the CGT sector, which were provided by Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), as well as a panel session on navigating opportunities and challenges in the sector.  

“There is an overall positive outlook for the CGT sector, despite various challenges and ‘headwinds’,” said Kakkaiyadi. “Analysts are likening the current climate in the CGT sector to that of the monoclonal antibodies development in the 1990s, where there were periods when investor interest in the sector waned due to setbacks. Today, however, the monoclonal antibodies space is a mature, thriving sector drawing significant investments and revenues. There was a general consensus amongst those at the briefing of a similar outlook for the CGT space in the short- to medium- term, with some going as far as saying that we are at an ‘inflection point’.”

Kakkaiyadi said real scientific progress, especially in gene-editing/platform technologies like CRISPR, which will propel the development also of in vivo cell therapies and drug delivery modes, coupled with data on trends shared at the ARM event, supports this positive outlook within industry.

According to that data, while just two CGTs with estimated global sales value of greater than $1 billion have been brought to market in the past six years, that number is expected to rise to more than 10 by 2030. Other data shared with delegates highlighted how 13 of the 15 largest biopharmaceutical companies in the world have invested in the development or commercialisation of CGTs. Kakkaiyadi said this is reflective of large pharma’s buy in to the CGT sector and the potential of new disease breakthroughs – much of the first wave of CGTs brought to market or in the latter stages of development are for treating cancers, but CGTs to address a wider range of diseases, from blood disorders to muscular dystrophy, are now emerging in greater number.

The US market is by far the largest market for CGTs, globally – 62% of the $5.9 billion spent on CGTs by payers globally in 2023 was spent in the US, and further data shows that the proportion of new products being approved by the US Food and Drug Administration (FDA) constituting CGTs has been steadily increasing over recent years.

Peter Marks’ session highlighted some initiatives that the agency is engaging in to support CGTs development, referencing the Rare Diseases Hub that promotes collaboration between two national bodies – the Center for Drug Evaluation and Research and CBER – as well as methods for accelerating regulatory approvals.  

On the US market, there was also a focus at the ARM event on how the new Trump administration’s agenda will impact the sector.

Some of the measures that president Trump appears set to pursue that were discussed include his broad aim of addressing the root cause of disease and reducing the need for treatment of chronic conditions; his support for regulatory flexibility, accelerated approvals and enabling gene-editing platforms; and strengthening of manufacturing capabilities in the US, which could align well and present real opportunities for the development and maturation of the CGT sector

Part of the positive outlook of industry is also reflected by the scope for growth of CGTs outside of the US, such as in Europe and the Asia Pacific region, Kakkaiyadi said. Just 35% of CGTs revenue globally in 2024 were generated in non-US markets, but data presented at the briefing suggested strong year-on-year growth across all regions.

Speaking at the ARM event, Marks highlighted an international pilot that the FDA is participating in – the Collaboration on Gene Therapies (CoGenT) Global Pilot – in which it is working with other authorities around the world to explore how a review of CGTs by multiple regulators could run concurrently. The FDA has been working with the World Health Organization (WHO), among others, on the initiative. However, Trump issued an executive order shortly after coming to office for the second time to signal that the US would withdraw from WHO membership, casting the future of the pilot project into doubt.

Kakkaiyadi said: “It is clear that ARM and the CGT industry in general see how some developments expected under the Trump administration could support the sector, but it remains to be seen how his ‘America first’ agenda will see the US lead or participate in wider, global initiatives to expand and develop the CGT sector in other markets.”

“There is real opportunity for all countries to collaborate and leverage developments and breakthroughs happening across the world. For instance, a recently approved CAR T-cell therapy (QARTEMI, varnimcabtagene autoleucel) in India, for adult B-cell non-Hodgkin lymphoma, came out of collaboration between Immuneel, a CGT start-up in India, and Hospital Clinic Barcelona. It can reportedly be manufactured using an automated platform that has been approved by the US FDA and the European Medicines Agency. It is all the more interesting that the drug’s price tag is reported to be a fraction of other CAR T-cell therapies,” he said.

“While there is optimism and positivity about what the future holds for CGTs, the industry is not blind to the real challenges and obstacles it faces, from attracting investment to dealing with regulatory burdens, pricing pressures, and scaling up manufacturing,” Kakkaiyadi said. “Concerted policy and regulatory action in the US and in other big markets, like the EU and UK, and collaboration will support the industry in maximising the opportunities for growth.”

“Ultimately, industry is best served putting patients at the heart of what it does – at the ARM event, there was a message of optimism and a ‘call to arms’ for investors and stakeholders, especially when there is a chance to reflect on CGT’s transformative capabilities for patients, to help move CGTs towards becoming a ‘first-line’ treatment for large populations of patients across appropriate diseases,” he added.