Cell and gene therapies (CGTs) have established themselves as revolutionary treatment methods in the world of oncology, where they have transformed prospects for patients with haematological malignancies. Remarkable results achieved in conditions such as acute lymphoblastic leukaemia and non-Hodgkin’s lymphoma have set a high benchmark for medical breakthroughs, but recent years have witnessed a surge of interest and success in a wider array of previously untreatable conditions.
According to GlobalData, the CGT market was valued at approximately $8.7 billion in 2024 and is forecasted to reach $76 billion by 2030. Oncology will continue to dominate the sector, accounting for more than half of the market share. However, substantial expansion is anticipated in metabolic disorders, projected to reach $8.1 billion, along with notable growth in haematological ($5.4 billion) and central nervous system disorders ($5.2 billion).
CGT breakthroughs in genetic disorders
Spinal muscular atrophy (SMA) is a severe neurodegenerative disorder that once carried a bleak prognosis. However, a groundbreaking AAV-based gene therapy called Zolgensma is fundamentally altering outcomes for SMA patients. With a single infusion, Zolgensma replaces a defective gene, dramatically improving survival rates, motor function, and overall quality of life for infants diagnosed with SMA.
Another striking example is metachromatic leukodystrophy (MLD), a genetic disorder that causes rapid neurological deterioration in young children. Until recently, this disorder had limited and mostly palliative treatments. Libmeldy, an innovative gene therapy, introduces a functioning copy of the defective gene into a patient’s own stem cells, which effectively halts disease progression when administered early.
Gene therapies also have the potential to transform treatment for haemophilia, a life-threatening disorder characterised by the inability to form blood clots effectively. Traditional management involves lifelong infusions, significantly impacting patients’ daily lives. However, with AAV-based therapies such as Roctavian for haemophilia A and Hemgenix for haemophilia B, patients now have the potential for lasting protection through a single infusion.
One of the most exciting advancements in CGTs is the advent of CRISPR gene editing, with the approval of Casgevy for sickle cell disease and beta-thalassaemia marking a significant milestone in expanding its potential. Unlike traditional gene therapies, CRISPR-based approaches can make precise edits to DNA, correcting genetic defects at their root. This precision opens doors to treating many more genetic diseases and sets the stage for a new era of personalised medicine.
Overcoming the hurdles in CGT growth
Despite optimistic projections, the investment landscape for CGTs has faced turbulence. Venture capital funding saw a notable 28% decrease in 2024, reflecting investor caution amidst regulatory complexities and market uncertainties. Nonetheless, confidence in the sector’s long-term prospects remains high, evidenced by strong IPO activity and targeted investment in groundbreaking technologies such as CAR T-cell therapies for autoimmune diseases.
Looking towards the future, greater use of automation, more standardisation, and scalable logistics will be key for making advanced therapies more accessible and affordable. As the market for CGTs grows, logistical challenges have become prominent, particularly when it comes to specialised cryogenic storage and transportation. As many of these treatments require early intervention and precision handling, improving logistical capabilities is critical to ensure timely delivery. CGTs are highly valuable and, since they are tailored to specific patients in certain cases, often irreplaceable. The strategic integration of technology within logistics processes can mitigate some operational complexities. Ultimately, however, choosing the right third-party logistics partner with which to trust these high-value medicines is one of the most important decisions that a manufacturer approaching the commercialisation stage can face.
A promising horizon
The rapid evolution of CGTs brings hope to millions suffering from genetic and chronic conditions previously deemed untreatable, but the healthcare sector must adapt swiftly to support these pioneering therapies. The convergence of scientific innovation, robust logistical solutions, and sustained investment will be crucial to fully realise the transformative potential of cell and gene therapies, ultimately changing countless lives beyond oncology.
Part of the Cencora group, ICS is a leading provider of integrated logistics solutions tailored for pharmaceutical manufacturers and cell and gene therapy innovators. The company offers a comprehensive suite of services to support the full commercialisation journey of advanced therapies across North America, including a complete cryogenic supply chain, fully automated technology and temperature-controlled transport.
“ICS has shown the market that we can build the facilities required to support cell and gene therapy logistics quickly and efficiently on demand,” says Krystal Haynes, Senior Director International 3PL Business Development. “We just need pharmaceutical manufacturers’ commitment, and we will develop a ‘fit for solution’ network within our 3PL infrastructure, helping to get scientific breakthroughs to market faster. Our oversight and client-designed outsourced warehouse model reduces cost and complexity for our customers, with dedicated continuous improvement for scalability.”
“The Cencora network is beyond reproach as we scale globally, providing anything that a cell and gene therapy manufacturer needs within the US or Europe,” she adds. “Our services can unlock back-to-back launches for our customers and enable fast cash flow for the next innovation.”
To find out more about the role of 3PL logistics in the biopharma landscape, download the whitepaper below.
