The cell and gene therapy (CGT) sector has seen unprecedented growth, emerging as a transformative force in disease treatment. With around 2,960 drug candidates in the pipeline and more than 40 products already in the market (see List of marketed CGT drugs, Page 23), the industry is at a point of inflection following extensive innovation and clinical progress in recent years.
This paper offers institutional investors key considerations and spotlights opportunities and risks in the space.
Huge growth potential backed by unmet medical needs and technological advancements
The global CGT market size was valued at $18 billion in 2023 and is projected to reach ~$97 billion by 2033, translating to a compound annual growth rate (CAGR) of 18%1.
That’s significantly higher than the CAGR of 8.2% the biopharma market is expected to log between 2023 and 20322. CGT drug approvals in 2024 are expected to rise to double digits from six in 2023.
Unmet medical needs: Rare diseases currently afflict 300 million people worldwide and 30 million people in the US alone, but 95% of these diseases lack Food and Drug Administration (FDA)-approved treatment. With eight of 10 rare diseases having a genetic origin, it becomes evident that effective treatments are vital for the well-being of patients. Notably, 21% of CGT sales between 2021 and 2028 were from the treatment of rare genetic disorders3
Technological advancements
Gene editing advancements, such as CRISPR-Cas9, hold immense potential for developing better CGT. Biopharma companies are actively leveraging these technologies and applications that offer promising avenues for safer and more effective treatments in the future
Emerging advancements in the field include novel nucleic acid formats, innovative techniques for viral biosynthesis, non-viral gene therapy delivery methods, specialised equipment, such as cell therapy manufacturing units (good manufacturing practice boxes or GMP boxes), microfluidics-based or encapsulation-enhanced cell culture systems, in vivo cell therapy approaches, non-chromatographic separation technologies, advanced GMP-compliant cell sorting methods and state -of-the-art optical tools for nanoscale analytics
