Innovation lies at the heart of our mission to enable our customers to meet challenging deadlines and address their patients’ needs. Research & Development (R&D) plays a central role in how we approach innovation. It provides us with a long-term vision and ensures we make appropriate investments into extending our technology and capabilities to develop the tools we need for our future success.
Our scientific mindset, 360º view of industry trends and experience in transforming discovery into manufacturing applications, have been applied on multiple fronts throughout 2020. Our 14 R&D sites worldwide are an integral part of our global network and support innovation across the business.
Innovation does not happen in isolation. It is underpinned by strong synergies across all our R&D focus areas. Our R&D group is active in viral vectors, cell line construction, cell culture media, cell and gene therapies, capsule design and manufacturing, other oral dosage forms, chemical active pharmaceutical ingredients (APIs) and parenteral formulation and delivery. It is also involved in bio manufacturing and digital technology. Our key areas of interest are summarized here.
Scalable Personalized Medicine
Personalized medicine represents the next leap towards individualized therapeutic solutions tailored to each patient’s needs. In many cases, these treatments are lifesaving and potentially curative. The availability of personalized medicine is growing in response to a deeper understanding of complex disease biology and new technological innovations. These are driving the emergence of new modalities that show significant clinical benefit. Many of these therapies address areas of significant unmet medical need, including oncology, where few targeted treatment options are available for certain patients.
As this market continues to grow and evolve, our in-house R&D team has developed and launched a patient-scale cell therapy manufacturing platform. The Cocoon® Platform, a closed and scalable cell therapy manufacturing platform, provides a flexible solution with improved end-to-end automation. These attributes are crucial to enable efficient manufacturing scale-out to meet commercial demand for innovative therapies such as autologous cell therapies. In adopting the Cocoon® Platform, it becomes possible to reduce manufacturing costs and deviations while enhancing process control.
The long-term goal of personalized medicine is to provide individualized therapies at a commercial scale to large patient populations. In September 2020, we reached a significant milestone in our partnership with Sheba Medical Center in Israel. Our collaboration delivered the first autologous CAR-T cell therapy manufactured using the Cocoon® Platform with a cancer patient. This was a major achievement and additional patients are now being treated at Sheba as part of a clinical trial. The Cocoon® Platform is being assessed by other cancer centers and research institutes and is also being used in Lonza’s own R&D facilities.
We remain committed to continued R&D efforts focused on expanding the features and functionality of the Cocoon® Platform, including integrated magnetic separation, non-viral transfection using our proprietary Nucleofector® technology and enhanced analytics. In addition, we are developing a multiplex solution called the ‘Cocoon® Tree™’ which will enable the scale-out of manufacturing.
We are committed to supporting growth across our operations with new digital technologies. The continuing COVID-19 pandemic has accelerated our strong advance towards our digitalization vision. Digitalization across the business enables data collection, analysis and modelling, providing tools that allow us to make better business decisions, follow trends, and provide valuable real-time information. The added security, reliability, time, and resource savings are set to bring benefits to our current and future processes.
In the area of training, virtual reality (VR) will be increasingly used in the future, enabling employees to be prepared for their tasks in production at their own learning speed, regardless of time and place. Experts and trainers can be called in for this training without having to travel to a specific production site. Instead, they can meet the employee in a virtual production room. There are already five VR training modules with specific “standard work units” successfully deployed; a further 34 modules are currently in development.
Another field of focus is the analytics of manufacturing data. The use of machine learning algorithms allows the optimization of processes and yields, while ensuring that our products are of the highest quality. Besides analytical aspects, detailed production process data allow increased transparency for internal monitoring and improvement programs. Moreover, they can allow us to better integrate Lonza into our customers’ supply chain ecosystem.
Inventive Capsule and Small Molecule Projects
Our R&D team is advancing with new products that have the capacity to improve patient experiences. As an example, our lipid multiparticulate (LMP) technology can customize drug delivery for patients of all ages as easy-to-swallow, palate-neutral microspheres, which fit both flexible and fixed dosage forms. We strive to develop products that will leave positive first impressions and empower patients to self-administer their own medications. Multiple pediatric products in the pipeline are being prepared for commercial filings, providing novel, taste-masked, and life-saving medicines to the pediatric market.
Many innovative small molecule-based therapies have poor solubility and therefore have poor bioavailability. Spray drying of amorphous dispersions is a mature technology that helps overcome bioavailability challenges and has led to more than 15 commercial products. Our R&D team has been working to improve this and has developed a temperature-shift process resulting in more than a ten-fold increase in solubility. This process has been successfully applied across pre-clinical, clinical and commercial scales, enabling challenging ‘brick dust’ compounds to reach patients. Recently, this new proprietary technology has been commercialized to enable a breakthrough oncology medicine.
The last decade has brought major innovations in the area of capsule manufacturing. These no longer serve solely as a carrier for the active pharmaceutical ingredient (API) but bring other functionalities. For example, our team has developed an innovative capsule system that can be used to control cargo release timing and location, improving delivery and bioavailability. Applications of this technology include enteric protection, multiple ingredient release and services for dosage form development.
Towards Next-Generation Modalities
Drug manufacturing represents one of the greatest future challenges, especially in the context of novel modalities. With complex diseases and a need for advanced diagnostic tools, the demand for innovative approaches is on the rise. We are witnessing fast progress in material science, nanotechnology and regenerative medicine. Advances in these areas are fueling progress towards next-generation modalities, which bring a transformative potential in medicine and diagnostics.
Induced Pluripotent Stem Cells (iPSCs), exosomes, live bio-therapeutic products, or mRNA-based vaccines require inherently complex starting materials, comprehensive purification and characterization.
From a manufacturing viewpoint, the unique needs and challenges reside in production, characterization and stable formulation development. Our R&D team addresses these challenges as they arise to allow these innovative therapies to advance and scale. With our extensive experience in current good manufacturing practice (cGMP) manufacture and using innovative technologies, our R&D team works to improve product stability, process efficiency and speed to market.