The cell and gene therapy (CGT) sector urgently needs common standards. Open dialogue will help achieve the best outcomes for developers, manufacturers and – most importantly – the patients. Aloysius Chong, Project Director at IMAPAC, investigates.

CGT is a modern medical marvel. Treatments that are able to reach a molecular level have the potential to treat a whole host of hereditary diseases, genetic disorders, autoimmune diseases, and cancers.

Needless to say, this represents a revolution in medicine – and the market and scientific community have responded. The broad application of CGT to so many aspects of healthcare creates huge potential for growth. The CGT sector was valued at $848 million in 2021, and is expected to grow by 23.6% before 2028.1

Moreover, even amidst a weak investment landscape, the number of CGT startups has only fallen by 15%, as opposed to an overall decline of 34% in biopharmaceutical startups – indicating that investors remain confident in these technologies and their commercial potential. Sales growth within the CGT sector is projected to significantly outpace that of the wider life sciences industry, and CGT firms represent an increasing proportion of IPOs within this sphere.2

However, a lack of standardisation and clear regulation are preventing the CGT sector from taking advantage of its enormous potential. This speaks to an urgent need for dialogue and discussion to address these questions and take the field forward.

Too bespoke

CGT developers are finding it difficult to scale up their operations to meet growing demand for their treatments, and – indeed – to achieve commercial viability. This can be chiefly ascribed to a lack of standardisation in chemistry, manufacturing, and controls (CMC) of the proteins and tissues involved in CGT.3 There is currently no strong set of international standards governing CMC within the CGT sector. The European Medicines Agency (EMA) and the FDA have issued industry guidelines, but these are mostly non-binding and have been criticised as vague.4

This lack of regulatory clarity has caused a number of problems across the sector. For one, it stymies the manufacturing process. Business-to-business selling is very difficult in absence of clear CMC standards, meaning that developers seldom benefit from any kind of division of labour, technology transfer, sector supply chains, or specialisation. This has meant that most CGT manufacturing has been confined to creating small quantities in-house for individual patients.5 This highly bespoke method raises costs, which in turn makes these therapies harder to market to the general consumer – it is currently almost impossible to find anything approaching an ‘off-the-shelf’ course of CGT. All this serves to limit the commercial viability of CGT in its current state.

Moreover, the lack of clarity over standards creates regulatory problems. The absence of clear and binding standards to judge new treatments against, means that the approvals process is often slow: in 2022, only 27 CGTs won FDA approval.6 The slow pace of standardisation also means that developers have a greater chance of inadvertently falling foul of what binding regulations do exist, saddling them with legal costs and further obstructing growth.

Putting the house in order

To proceed any further, there is an urgent need for common standards within the CGT sector. Governments should consult with stakeholders throughout the sector to produce a binding and precise set of regulations. These would do much to kickstart the CGT sector – accelerating its drive towards commercial viability and general use.

Precise, binding, and widely-known CMC standards would allow for the development of third-party vendors, supply chains, and – ultimately – a large scale-up of manufacturing to meet growing demand. This would allow more entrants into the market, as smaller biotech firms would now be better able to outsource capital-intensive manufacturing to a third party.7

Common standards would also serve to facilitate technology transfer and research cooperation among industry participants, driving innovation. In addition, regulatory clarity would do much to speed up the approvals process, as regulators would now have a clearer criterion by which to assess new patents. The ultimate effect of these changes would see the industry move towards accessible, low-cost CGTs that could be marketed to the general consumer.

A need for dialogue

CGT is a fairly new field. As such, there is a need for discussion and debate among stakeholders to work towards a new system of standards. The current fragmented state of the sector makes it all the more critical that scientists, firms, healthcare professionals, and policymakers come together to engage in a constructive dialogue on CMC standards that could win broad approval, and that could be implemented quickly and effectively. At IMAPAC, we aim to facilitate this dialogue through our industry conferences, annual committee meetings, and speaking events – which provide an essential forum for stakeholders to discuss the future of the CGT sector.

Debate and dialogue within the life sciences have done much to advance the field in the past. Given the enormous potential implications of CGT to healthcare, such dialogues within this sector will prove no exception. Members of the CGT sector should gather to share best practice, and discuss how to balance the need for innovation with questions of safety and medical ethics. Industry participants could also share their insights into what has become a perennial debate within the sector: namely, which elements of CGT manufacturing should be outsourced to a third party, and which others are best carried out in-house.8 One CGT firm that is leading the charge in this regard is UK-based CGT Catapult, which is actively engaging the sector in coming up with a set of agreed-upon CMC standards.9

CGT has matured as a sector, and now stands at a crossroads. There are a host of regulatory, commercial, and ethical issues to be considered as the field advances. Open dialogue across the sector is the best way to engage with the questions that need answering; it will pay dividends not just to CGT developers and manufacturers, but also to the patients who could benefit immensely from CGT that is more widely available and commercially viable. 

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