The CGT field is undergoing a critical transformation from boom to thoughtful reset. While early enthusiasm brought a rush of investment and regulatory attention, the sector is now refining its ambitions, weeding out unviable approaches, and laying the foundations for long-term impact. Companies, regulators, and healthcare systems are rethinking what it will take to make these life-changing therapies scalable, sustainable, and accessible.
CGT’s Staying Power
Far from being a fleeting trend, CGTs have proven they are here to stay. What began as a niche area has emerged as a core pillar of biomedical research.
After an initial boom that drew large amounts of cash, the space is still driving R&D efforts with over 4,000 therapies currently in development, encompassing everything from CAR-T cell immunotherapies to gene editing and tissue engineering.
Regulatory agencies have tried to keep pace with innovation and have registered record numbers of CGT approvals. The FDA greenlit seven new therapies and three expanded indications in 2024 after giving the nod to seven others the previous year.
But the global regulatory environment remains fragmented. Many agencies still lack the frameworks and evaluative tools needed to assess the unique manufacturing and clinical profiles of CGTs. The result: uneven access and delayed timelines depending on geography.
Investment Uptick and Big Pharma Stake
The initial CGT explosion sparked huge investor interest and capital flows peaking in the space in 2020 and 2021 with USD 19.9 billion and USD 22.7 billion respectively.
After a dramatic slump in 2022 and 2023, the sector appears to have recalibrated, incorporating lessons learned from its first wave. As a result, investments are on the rebound. According to the Alliance for Regenerative Medicine (ARM), they reached USD 10.9 billion in the first half of 2024 alone, with funds particularly pouring into later stage assets.
“This pattern is characteristic of emerging technologies with transformative potential,” says Daniel Palmacci, head and president of Lonza’s Specialized Modalities division, which includes cell and gene therapies. “Initial enthusiasm attracts widespread investment, but not all of it is deployed optimally or addresses genuine patient needs.”
For Palmacci, the adjustments the field have seen are synonymous with maturation, a process that has managed to weed out less workable development programmes and players, leaving those with real promise. “It filters out less viable approaches whilst strengthening those with genuine therapeutic value. The companies and technologies surviving this scrutiny demonstrate more robust scientific foundations and clearer paths to commercial viability.”
Meanwhile, Big Pharma, facing the looming patent cliffs on its blockbuster drugs, is all in. No less than 13 of the world’s 15 largest pharma companies have established a firm footing the CGT arena, having snapped up a variety of promising assets and platforms.
One of the hottest dealmaking areas at the moment is for “off-the-shelf” CGT therapies. Roche, for example, purchased Poseida Therapeutics with its allogeneic CAR-T platform late last year, while AstraZeneca snapped up EsoBiotec and its lentiviral vector platform in May.
A New Rule Book
This reset process has also meant recognising that CGTs are fundamentally different from traditional pharmaceutical products. With cells needing to be extracted, modified externally, and returned as a personalised treatment, new rules must be applied.
Recognising these particularities, Roche for one has built a dedicated, integrated technical operations unit for CGTs to unite regulatory, manufacturing, quality, and supply chain functions under one roof. “Its purpose is to ensure the right level of focus, structure, and expertise to manage this complexity, while also fostering the kind of cross-functional understanding needed to transform scientific progress into real, accessible medicines,” says the firm’s global head of cell and gene therapy, Ralf Altenburger.
Scaling the Manufacturing Mountain
The biggest piece of the CGT rethink is manufacturing. Manufacturing CGTs is complex. Producing living cells and viral vectors involves delicate, time-consuming processes that demand precision and introduce variability that traditional pharma products do not have to face.
“One of the most important realisations was that cell and gene therapies cannot be treated like traditional pharmaceuticals; they demand new frameworks both technically and procedurally,” says Altenburger.
Maintaining consistent quality remains an important objective for securing patient safety and scalability. “Working with living cells brings a level of biological complexity that simply does not exist with small molecules or even antibodies, and this makes full standardisation an ongoing challenge,” he notes.
Consequently, production costs are high, contributing to the prohibitive price tags of these therapies. Biotech players are looking to improve the inefficiency and lower the costs of CGT production. Two Swiss companies are at the forefront of these efforts.
NewBiologix for example has developed a platform for the advanced engineering of cell lines used in the production of CGTs. “By addressing the inefficiencies inherent in current manufacturing techniques, we aim to lower production costs and enhance consistency,” says CEO Igor Fisch. “In doing so, we hope not only to improve access to existing therapies but also to enable the expansion of gene therapies into broader indications.”
Limula, on the other hand, is building a system where cell therapies are made inside a single, closed device with no pipettes, no clean rooms, no human hands. “This novel technology “combines the functionality of a bioreactor, where cells are incubated and grown, with a centrifuge, which concentrates and separates cells,” explains CEO Luc Henry. “We are the only platform that integrates both into a single system where the cells remain in the same container throughout.”
Access, Affordability, and Systemic Integration
Perhaps the final frontier as these therapies find their footing is access. Due to their complex manufacturing process, CGTs often come with a seven-figure price tag.
Most CGTs today cost between USD 1 and 2 million per dose, and in some cases even more. CSL Behring’s Hemgenix, for example, is priced at USD 3.5 million for a single treatment.
For many health systems, that is simply impossible to afford. Global spending on CGTs hit USD 5.9 billion in 2023, but reimbursement remains fragmented and unpredictable. In Europe, Bluebird Bio withdrew its β-thalassaemia gene therapy, for example, after failing to reach a pricing agreement with European authorities.
In many countries, payers have yet to determine their policies relating to CGTs, requiring healthcare systems to work closely with their finance and payer management teams to create single case agreements.
In the US, insurers are setting tight limits on coverage. A report from Cardinal Health highlights the core barriers: restrictive payer policies, high upfront costs, and limited treatment infrastructure. Meanwhile, in low- and middle-income countries, access is practically non-existent.
Healthcare systems may still need time to adapt to these cutting-edge therapies. “They’ve just begun to enter the healthcare system, which was designed decades ago. Changing the status quo takes time, and this is just the first chapter in a long story,” says Stephen Majors of the Alliance for Regenerative Medicine.
Beyond cost, CGTs require specialised treatment centres, adding an additional burden to healthcare systems.
Beyond Rare Diseases
Another adaptation that has taken place in the cell and gene sphere is looking beyond the initial focus on rare diseases to address diseases with higher incidence. “The early development of gene therapies naturally centred on rare conditions. These cases offered a well-mapped therapeutic pathway, but the limited patient populations made it difficult to build a commercially viable model around rare diseases alone,” says Altenburger.
“What we are now seeing, across the industry and within Roche, is a deliberate shift towards broader indications. While rare diseases continue to be an important focus, they cannot be the sole basis for sustainable innovation in this space,” he explains. “This evolution reflects a growing understanding that the promise of cell and gene therapies must be matched by models capable of reaching more patients, more consistently, and at scale.”
