Although the cell and gene therapies (CGTs) industry continues to advance the approvability, accessibility, and affordability of this new generation of treatments, it remains a constant challenge to create not only safe and effective products but also commercially viable ones. Escalating development costs and complex supply chains have manufacturers increasingly exploring new production models to streamline logistics, increase throughput, and enhance patient access.
Centralized production of CGTs—where therapies are manufactured at a few large-scale facilities serving multiple geographically dispersed treatment centers—works well for the small molecule and biologics manufacturing processes they were built for. However, for living and often personalized medicines such as CGTs, this approach is falling far short.
Unlike chemically synthesized small molecules, CGTs are large, complex molecules derived from a patient’s cells, which are genetically modified and (usually) personalized for an individual patient. This level of personalization and biological complexity requires unique handling, storage, and administration protocols that differ vastly from the manufacturing and distribution models suited for more traditional therapies. Applying a centralized manufacturing approach to CGT production, therefore, has resulted in high costs of goods (COGs), limited throughput, and variable quality (eg, high out-of-spec rates).
The resulting logistical and economic challenges have created significant access challenges for patients. Less than 20% of patients in the United States who could benefit from genome-targeted or cell-based therapies are actually able to access them.1,2 For the lucky few who do get access, vein-to-vein times often exceed 4 to 5 weeks, which can prove fatal for patients with aggressive, advanced cancers. Doctors at large treatment centers, such as The University of Texas MD Anderson Cancer Center, Mayo Clinic, and Dana-Farber Cancer Institute, see as many as 20% of their patients die while waiting for therapy.3
Vein-to-vein times are not just extended by manufacturing logistics; geographical distance between the manufacturer and treatment facility can also increase the time it takes for a treatment to reach a patient. A patient is often left with no other option than to stay close to a treatment center because cell therapies are not available at community cancer centers, increasing logistical complexities and costs (eg, gasoline, airline tickets, temporary housing, lost wages, etc). Even then, the complexities of multistep supply chains, international logistics, and manufacturing itself can leave patients in limbo.
An increasing number of manufacturers recognize that adopting different manufacturing approaches to meet the unique requirements of CGTs is one of the best ways to potentially lower COGs, improve scalability, and ultimately get these therapies to more patients faster. Given the limitations of the centralized model to deliver affordable, accessible treatments, alternative regional or point-of-care (together called “decentralized”) manufacturing models are evolving. For example, chimeric antigen receptor T-cell developer Galapagos, among the pioneers in decentralized manufacturing, announced a partnership with the Blood Centers of America in 2024 to create a multisite, decentralized clinical manufacturing network.4
Decentralized manufacturing is an emerging alternative manufacturing model with the potential to bring significant benefits to both manufacturers and patients. In this model, manufacturing sites can be located closer to local community treatment centers, increasing accessibility to a wider range of patients and dramatically reducing vein-to-vein times. Additionally, decentralized manufacturing reduces the need for complex and costly transportation logistics, both for the cell therapies and the patients. By manufacturing therapies near the point of care (ie, by colocating manufacturing facilities near major academic medical centers or hospitals), providers can minimize costs and delays associated with shipping cells by car or plane over long distances, enabling faster access to treatment and likely better outcomes. More time closer to home, reduced travel costs, and fewer disruptions to daily life for patients and caregivers will also enhance the overall patient experience by reducing costs and logistics, as well as emotional and physical stress.
These advancements could pave the way for decentralized manufacturing to meet the demands of CGTs while maintaining regulatory compliance, operational efficiency, and commercial viability... After all, what is the point of having cures for cancer and potentially many other conditions if patients cannot get access to them?
Despite the promise of decentralized manufacturing models, where production occurs closer to the point of care, widespread adoption has been slow to gain traction. Although a few organizations, such as Galapagos and Caring Cross,5 are pioneering decentralized approaches for CGTs, most manufacturers remain reliant on historical manufacturing models utilizing centralized facilities. Many hesitate to adopt a decentralized approach because process variability between sites may hinder their ability to maintain Good Manufacturing Practices (GMP) standards across sites and could compromise the quality and consistency of their product.6 Adhering to stringent regulatory requirements also becomes more complex with multiple sites, as each location must demonstrate compliance with health authority standards, often adding time and cost to ensure consistent oversight and quality control.7
