Although early in its development, personalized medicine, like cell and gene therapy, has already shown remarkable advancements in recent years as the customized nature of these treatments has made them highly successful in treating historically complex and grim diagnoses. In fact, the global cell and gene therapy market is expected to reach $15.48 billion by 2025, up from $4.39 billion in 2020. Unfortunately, the ability to scale therapies to more patients is impeding research from the mainstream, as controlling and regulating the quality of mass-produced treatments has become increasingly challenging.
The Food and Drug Administration (FDA) is enforcing stricter standards on developers to maintain consistency in the development of cell and gene therapies, which demands labor-intensive and costly manufacturing. And for good reason — cell and gene therapies require meticulous and exact processes to be proven successful. As such, developers are leveraging technology to help them scale and ease bottlenecks in their operations. Multiply Labs recently announced a new robotic manufacturing platform that automates many processes in cell and gene therapy development that have traditionally required highly skilled scientists.
Developers are also leveraging connected infrastructure like ultra-low temperature (ULT) and cryogenic freezers to achieve their objectives, while remaining compliant with regulations, as these storage units can provide them with real-time data from changes in temperature to the condition of the product.
As the industry moves forward, automation can provide a valuable tool to helping manufacturers meet regulatory requirements and efficiently scale-up.
Obstacles ahead
The development of cell and gene medicine generally requires human involvement at every step, from extraction to distribution, but this method is highly prone to inefficiencies and errors that make these therapies difficult to scale. Creating and distributing personalized treatments requires careful extraction of a biological specimen from the patient that must then be transferred to a lab for development and finally transported back to the patient in a byproduct form of the original specimen. For this process to be effective, it’s critical that biomaterials are stored at ULT or cryogenic temperatures while being consistently monitored and controlled throughout the entire journey. Any disruption in the process could severely impact the patient from both a cost and health standpoint. Additionally, this becomes increasingly complex when every treatment is personalized and has its own individual requirements.
The implementation of proper infrastructure for sample preservation in supply chains, research labs and long-term storage facilities will be a top bottleneck preventing these treatments from being administered to a wider range of patients. The use of adequate infrastructure has already increased rapidly with the introduction of mRNA COVID-19 vaccines, as ultracold chain storage became crucial to the vaccine supply chain. However, because cell and gene therapies are customized for and distributed to individual patients, developers must constantly track and monitor samples to preserve their integrity. Governing bodies are currently in the process of creating standards to ensure consistency in the development of these treatments, and guarantee that they are being stored and handled exactly the way they were designed to be. This will assure that a product is properly stored throughout the entire journey, delivered to the right patient and safe to administer. Standardizing as many components as possible can streamline the process, mitigate errors and save developers’ significant time. To make this a reality, developers must leverage flexible and reliable infrastructure, while maintaining a system that enables them to trace and capture every data point in the process.
The benefits of connected and automated technologies
With rising regulatory requirements, monitoring technology in biopharma has gradually moved from an optional resource to a crucial asset. Regulators are holding developers accountable for properly preserving samples as they were created to be for the entire duration of clinical trials and after FDA approval. Due to the high price value and delicate nature of these samples, it’s crucial that developers are following these regulations. Fortunately, connected technology like integrated data management can provide research teams with a single, comprehensive view of all of their assets. This has become essential to monitor, track and preserve the integrity of cell and gene therapies throughout downstream manufacturing, clinical trials, cold chain distribution and administration. Consequently, the demand for digital and cloud-based data management systems is increasing in every aspect of making innovative therapies a reality and administering them to patients. This facilitates the development of these treatments and ensures their temperature and conditions are being continuously tracked and monitored as they travel down the supply chain to the patient.
Automated cell and gene therapy development not only streamlines repetitive processes that would otherwise require regular human interaction and maintenance, but it also significantly reduces the chances for human error, while providing improved safety and efficacy through digital tracking and monitoring. Integrating robotics with data management platforms will provide developers with automated data capture and traceability of their products, while ensuring data visibility for regulators to make critical decisions about approving treatments.
The automation of cell and gene therapy development offers countless benefits, but unfortunately machines are still subject to data integrity errors. Machines are simply incapable of the same level of expertise a scientist has when it comes to creating and producing an effective treatment. Additionally, the personalized nature of every individual treatment requires multiple machines and highly specific programming that are far too costly to implement and maintain.
However, humans can leverage these machines and connected technologies to produce greater precision medicine at scale. For instance, sample management systems linked with ULT freezers can provide researchers with a roadmap of their products’ whereabouts to identify where samples are located prior to opening the freezer door, as well as where to put them back. This system of guided access and retrieval can prevent sample quality from diminishing, mitigate the opportunity for human error and simplify the development and delivery of the final product. Meanwhile, all of this data is being captured and stored for examination in real-time.
Precision medicine still has a long way to go in terms of providing treatments to the masses, reducing labor and costs, and minimizing any room for errors. Nonetheless, the industry has shown immense progress in effectively treating rare, life-threatening diseases with the use of cell and gene therapy treatments. By integrating modern technologies with infrastructure like ULT and cryogenic storage, developers can confidently, effectively and permanently treat diseases that were once considered incurable.
