Over the past 10 years, the cell and gene therapy (C>) space has grown rapidly, with the number of innovative medicines entering the development pipeline steadily rising year on year.

The potential of C&GTs continues to expand as our understanding of molecular biology and genetic engineering techniques grows and manufacturers adopt innovative technologies. As well as improving the safety, efficacy and manufacturability of these therapies while helping to broaden patient access, advancements in the C> space are driving trends and introducing new challenges. From meeting changing regulatory requirements to adopting strategies to minimise manufacturing costs, C> developers must adapt to navigate these novel difficulties and continue to provide life-changing medicines to current and future patients.

In this article, Andelyn Biosciences examines the drivers behind current trends in the C> space and the obstacles developers and manufacturers face on their journey to market. Leveraging its unique insight, Andelyn Bioscicences emphasizes the importance of implementing specific strategies to quickly adapt to changing C> needs as they emerge.

Three Decades of Rapid Expansion

Following the success of the first approved gene therapy procedure in 1990 to treat a patient suffering from severe combined immunodeficiency (SCID), the biopharma industry has seen a steady increase in resources dedicated to unlocking the potential of C&GTs.¹

Over the last decade, the biopharma industry has intensified its focus on next-generation technologies such as C&GTs and precision medicines. There are now 26 gene therapies and 63 non-genetically modified cell therapies approved for clinical use globally.²

These revolutionary therapies target a wide variety of indications, from rare diseases to cancers and neurological disorders.2 Prominent factors driving the demand for C&GTs include the rising prevalence of cancer worldwide and the expanding population of chronic disease patients.³

This growing demand is reflected in the projected expansion of the global C> market from US$13 billion in 2022 to US$62.5 billion by 2032 at a compound annual growth rate of 22.8%.⁴

Many of the therapeutics behind the recent growth in the C> market require an extremely skilled workforce, state-of-the-art technologies, and advanced consumables, including plasmids and viral vectors. As a result, meeting the expanding demand for C&GTs has increasingly relied on outsourced development, manufacturing and testing organisations offering the necessary capacity, technical capabilities, and expertise to support production.

Navigating the Challenges of a Novel Therapeutic Space

Although the C> market has grown immensely in the last 30 years, it is still relatively new compared with other therapeutic areas. Our understanding of the treatments’ mechanisms of action and delivery has rapidly transformed in this time. As a result of this transformation, novel challenges have also surfaced in the development and manufacturing of these new medicines.

A Lack of Necessary Expertise and Experience

Staffing has been a persistent challenge in the biopharma industry, and it has been felt strongly in the burgeoning C> space. Even now, few companies possess the technologies needed to scale commercially, and even fewer have experience working with multiple vectors and different serotypes in those systems. As a result, finding staff with relevant C> experience is a significant challenge for developers.