CELL AND GENE THERAPY CATAPULT (HTTPS://CT.CATAPULT.ORG.UK)

Advanced-therapy medicinal products (ATMPs) hold much promise for patients living with cancers and rare diseases. In the future, increased availability of cell and gene therapies also could ease the burden of disease on national healthcare systems such as the UK National Health Service (NHS). Such potential has attracted significant business interest and investment within the ATMP sector. The Cell and Gene Therapy Catapult (CGT Catapult) seeks to facilitate and accelerate industry growth by supporting efforts to overcome technological and process barriers throughout advanced-therapy development and manufacturing. Our organization also trains life-science professionals to help them acquire new skills and equips companies with the tools needed to develop impactful medicines.

Enabling ATMP Development

Innovative treatments could reap significant savings for healthcare systems by reducing or eliminating the need for costly long-term care for a breadth of conditions that currently have high unmet medical needs. Although the global number of ATMP clinical trials dropped by 10% between 2018 and 2023, the number in the United Kingdom has remained steady, with 175 ongoing trials in cell and gene therapy (1). The country finds itself in a strong position to capitalize on high demand for such therapies worldwide and become a global hub for ATMP research.

In 2012, Innovate UK established the CGT Catapult as an independent innovation and technology organization to accelerate advancement in the ATMP industry. The CGT Catapult’s business model is to collaborate with drug companies, academic research centers, and healthcare providers to drive innovation in advanced therapies and related manufacturing technologies.

Innovation organizations such as the CGT Catapult have a strong value proposition for small- and medium-sized enterprises (SMEs), start-ups, and even established companies within the sector. For instance, companies can collaborate with innovation organizations to reduce financial and delivery risks to acceptable levels. Analytical testing, process development, and scale-up activities require a depth of knowledge and experience that small companies often do not have the time or resources to build. Thus, many such companies choose to partner with other organizations.

In cases with well-understood and predictable processes, working with contract research (CROs) and development and manufacturing organizations (CDMOs) can be a cost-effective solution. But when a promising program comes with high risks and unpredictable processes, then it might be difficult to achieve a desired outcome by contracting directly with a CRO or CDMO. Collaborations that share resources and expertise can be a faster and more cost-effective approach. Innovation organizations often specialize in such partnerships, knowing how to share risk and combine expertise, facilities, and experience to achieve groundbreaking advances. Support from innovation organizations helps to convert early stage ideas into valuable products and services by bridging the gap between research and industry — and breaking down barriers to innovation will bring more therapies to the market more quickly than would otherwise be possible.

Key Industry Challenges

The UK advanced-therapies industry has reached an important stage in its growth. The potential of such therapies has been proven, and products are coming steadily into the market. However, the industry needs to accelerate growth to deliver on the benefits it has promised.

According to the European Blood and Marrow Transplantation (EBMT) Registry, 2500 patients received chimeric antigen receptor (CAR) T cell therapy in 2021. However, that represents just 1% of European patients for whom at least one CAR-T product is indicated (2). Thus, ~250,000 patients could be eligible for CAR-T therapy.

The CGT Catapult has identified conditions that can support the advanced-therapies sector in bringing products to patients. First, the sector needs a strong in-flow of new therapies, technologies, and ideas. Many innovative experimental therapies have demonstrated proof of concept, and the sector must continue to invest in their development. Bringing them to patients requires a robust technology cycle in which the costs of development and production come down over time. That will open commercial opportunities.

Companies must be able to manufacture advanced therapies quickly and at large scales while ensuring product quality and safety. Close collaboration with regulators can help to establish a high-quality supply chain for ATMP production.

The industry also needs significant uptake of such therapies in the healthcare sector. Insufficient adoption represents another prominent limiting factor today. Integration of public systems that capture therapy demand from hospitals and other healthcare centers will optimize therapy purchase and use, in itself reducing cost of goods (CoG). In turn, increased demand will create competition within the industry, leading to innovation and, over time, reduced prices. However, that outcome is possible only if infrastructure is in place to sustain ATMP delivery and if manufacturers can produce therapies in a commercially viable way.

One role of the CGT Catapult is to help key players and up-and-coming companies in the UK advanced-therapy landscape to fulfill those conditions.

Data, Digitalization, and Automation

Advanced data analytics and automation systems are influencing operations in almost every industry, and the ATMP sector is no exception. Such technologies hold great promise for helping drug companies to meet critical needs, notably in reducing costs and manufacturing high-quality products. Innovation organizations have a vital role to play in realizing the benefits of digital technologies.

The CGT Catapult is working with companies to develop, optimize, and apply such tools. In 2023, we opened a new laboratory dedicated to developing techniques that monitor and control ATMP manufacturing processes in real time (3). Our process analytical technology (PAT) laboratory uses state-of-the-art automation and digitalization to evaluate manufacturing difficulties involved in scaling up ATMP production.

In June 2023, Autolomous Ltd. — a market leader in developing critical ATMP manufacturing-management systems — and the CGT Catapult were awarded a £1.2 million (US$1.5 million) grant to elucidate ways to increase production, support rapid product release, and reduce CoG at industrial scales (4). Through this project, Autolomous and the CGT Catapult aim to demonstrate the benefits of leveraging digitalization to automate data collection in ATMP manufacturing.

Since then, the CGT Catapult has collaborated with Rentschler Biopharma and Refeyn Ltd. to apply PATs in ways that improve manufacturing of adenoassociated virus (AAV) (5), a frequently used vector for delivering gene therapies with therapeutic applications in ophthalmology, neurology, hematology, and metabolic and muscular disorders (6).

The CGT Catapult hopes that facilities such as the PAT laboratory and partnerships in data and automation such as that with Autolomous can relieve bottlenecks and enable safe and effective production of therapies.

Once-in-a-Lifetime Opportunity

Advanced therapies are potentially life-changing for patients and could be extremely valuable to global healthcare systems. Additionally, breakthroughs in ATMP manufacturing not only will strengthen the UK economy by attracting investment, but also will generate high-skilled jobs that will position the country at the forefront of transformative healthcare discoveries.

The United Kingdom is in an excellent position to establish itself as a world leader in advanced therapies. The CGT Catapult can support the country’s ATMP industry on that journey by providing the skills, facilities, and expertise needed to take promising treatments from concepts to products. This is a once-in-a-lifetime opportunity for the sector to revolutionize healthcare, and at the CGT Catapult, we are eager to work alongside industry, healthcare systems, and regulators to make that change a reality.

References

1 UK 2023 ATMP Clinical Trials Database: Highlights. CGT Catapult: London, UK, 2024; https://cgt.ams3.cdn.digitaloceanspaces.com/Cell-and-Gene-Therapy-Catapult-ATMP-Clinical-Trials-Database-2023-Highlights.pdf.

2 Giorgioni L, et al. CAR-T State of the Art and Future Challenges: A Regulatory Perspective. Int. J. Mol. Sci. 24(14) 2023: 11803; https://doi.org/10.3390/ijms241411803.

3 Press Release: Cell and Gene Therapy Catapult Opens New Process Analytical Technologies Laboratory. CGT Catapult: London, UK, 14 March 2023; https://ct.catapult.org.uk/news/cell-and-gene-therapy-catapult-opens-new-process-analytical-technologies-laboratory.

4 Press Release: Autolomous and the Cell and Gene Therapy Catapult Awarded £1.2m Grant by UK Research and Innovation. CGT Catapult: London, UK, 12 June 2023; https://ct.catapult.org.uk/news/autolomous-and-the-cell-and-gene-therapy-catapult-awarded-1-2m-grant-by-uk-research-and-innovation.

5 Press Release: Cell and Gene Therapy Catapult, Rentschler Biopharma, and Refeyn Collaborate To Improve Process Analytical Technologies and AAV Manufacturing for Gene Therapies. CGT Catapult: London, UK, 23 August 2023; https://ct.catapult.org.uk/news/cell-and-gene-therapy-catapult-rentschler-biopharma-and-refeyn-collaborate-to-improve-process-analytical-technologies-and-aav-manufacturing-for-gene-therapies.

6 EMA/CAT/60852/2018. Report on the Committee for Advanced Therapies (CAT) Expert Meeting on Scientific and Regulatory Considerations for Adeno-Associated Viral Vector (AVV)-Based Gene Therapy. European Medicines Agency: Amsterdam, the Netherlands, 19 July 2018; https://www.ema.europa.eu/en/events/committee-advanced-therapies-cat-expert-meeting-scientific-regulatory-considerations-adeno-associated-viral-vector-avv-based-gene-therapy.

Matthew Durdy is chief executive of the Cell and Gene Therapy Catapult, 12th Floor Tower Wing, Guy’s Hospital, Great Maze Pond, London SE1 9RT, UK; https://ct.catapult.org.uk/contact.