A frequent point of confusion is the role of companies like Lonza versus public bone marrow registries. While both are part of the broader ecosystem of cellular medicine, they operate in separate spheres and do not interact with the same people. Public registries focus on recruiting and matching volunteer donors with patients who need a transplant. In contrast, Lonza is a manufacturing partner for pharmaceutical firms developing new cell-based therapies and does not work with public donors.
The Role of Public Bone Marrow Registries
Public bone marrow registries connect volunteer donors with patients who have life-threatening illnesses requiring a transplant. Organizations like the National Marrow Donor Program (NMDP), which operates Be The Match, manage databases of willing donors. To join, eligible volunteers complete a registration kit that includes a consent form and cheek swabs for DNA collection.
The cheek swab samples are sent to a lab for human leukocyte antigen (HLA) typing. HLA are proteins on cell surfaces that the immune system uses to recognize the body’s own cells, making a close match between donor and patient necessary for a successful transplant. Once typed, the donor’s information is entered into a searchable database for transplant centers worldwide.
If a person on the registry is identified as a potential match, they are contacted for more detailed blood testing to confirm they are the best donor. If confirmed, the donor proceeds to one of two donation methods. The most common is peripheral blood stem cell (PBSC) donation, a non-surgical procedure. The donor receives injections for several days to increase the number of stem cells in their bloodstream, which are then collected from the blood using a machine.
The second method is a surgical procedure to donate marrow directly. Performed under anesthesia, doctors use needles to withdraw liquid marrow from the back of the pelvic bone. This procedure is often chosen for pediatric patients. In both cases, the collected cells are immediately transported by a medical courier to the patient’s location, and all donation-related costs are covered by the registry.
What Lonza Does in Cell Therapy
Lonza is a contract development and manufacturing organization (CDMO), a specialized manufacturing partner for pharmaceutical and biotech companies. These clients design and develop new treatments, including advanced cell and gene therapies. Lonza then provides the expertise, facilities, and processes to produce these therapies at a clinical or commercial scale.
A helpful analogy is to view a pharmaceutical company as an architect designing a new building, and Lonza as the specialized construction firm that builds it. Lonza does not recruit public donors or manage registries. Instead, it receives starting cellular materials from its clients and uses its manufacturing platforms to create a final therapeutic product.
Lonza’s services span from development to commercialization, including process development, regulatory support, and large-scale cGMP (Current Good Manufacturing Practice) manufacturing. The company works with various cell types, such as hematopoietic stem cells (HSCs), T-cells, and induced pluripotent stem cells (iPSCs). This work is performed under strict regulatory oversight to ensure the safety and quality of the final medical product, such as producing newly approved CRISPR-based cell therapies for client companies.
Connecting the Dots: From Donor to Therapy
The path for a traditional bone marrow transplant is straightforward: a volunteer from a public registry donates cells directly to a matched patient. A different path exists for novel cell therapies. For these treatments, the starting cells may come from the patient (autologous) or a healthy third-party donor (allogeneic). The biotech company developing the therapy is responsible for sourcing these initial cells, a process separate from the public registry system.
After acquiring the starting cells, the biotech firm may hire a CDMO like Lonza for manufacturing. The firm provides Lonza with the cells and the therapy’s “recipe.” Lonza then uses its specialized facilities to engineer, expand, and formulate the cells into a final product. This finished product is then cryopreserved and shipped to a treatment center.
The engineered cell therapy is then administered to the patient. This completes a highly coordinated supply chain involving the biotech firm, Lonza, and the treatment center. These two distinct systems, public donation for transplants and commercial manufacturing for novel therapies, represent different but vital contributions to cell-based medicine.