Gene therapies are used to treat genetic defects or hereditary disorders. Cells are taken from the body and altered by adding, modifying or removing a gene. Then, they are multiplied in the laboratory (ex vivo) and reintroduced into the body. The therapeutic gene is delivered either as an infusion or as an injection directly into the target tissue, e.g. muscle. As a result of this treatment defective genes are repaired or disease-causing genes are switched off.

Gene therapies can be used in patients with hematological disorders, such as sickle cell anemia. In this case, a defective gene for hemoglobin production is replaced, thereby enabling the body to produce healthy blood cells again. Gene therapy can also be administered directly in the body (in vivo) without cell extraction.

In this category of advanced therapies, patients receive living cells – mainly stem cells, immune cells or other specialized cells. Those cells have previously been modified according to specific biological or structural characteristics or physiological functions. In the body, they repair damaged tissue or destroy diseased or infected cells. Cell therapies can be  categorized into autologous and allogeneic.

Autologous therapies use patients own cells, manufactured in one single batch. They are generally well tolerated. As one example, CAR-T cell therapy is used very successfully in cancer patients to specifically target tumor cells. T cells are extracted from the blood and genetically modified in the laboratory to express a chimeric antigen receptor (CAR). The CAR-T cells are then multiplied and injected back into the patient. They recognize cancer cells by the CAR receptor and immediately activate their destruction.

Allogeneic therapies can be manufactured in larger batches. The cells are not derived from patients themselves, but from a different donor. A typical example for allogeneic therapy is stem cell therapy targeting blood cancers. These therapies are often associated with a higher risk of rejection because the immune system does not recognize the cells as the body's own cells.

Allogeneic therapies are a highly interesting area for pharmaceutical research because they are associated with significantly lower costs and less workload than autologous therapies. They are also considered the most suitable for scalable application in large patient groups. The primary goal in developing new allogeneic therapies is to achieve not only maximum tolerability and minimal rejection  but also optimal efficacy, so that as many patients as possible can benefit.

Tissue engineering can be used to repair damaged human tissue or replace missing tissue. This involves placing the body's own cells (e.g. stem cells, tissue-specific cells) or donor stem cells on a scaffold made of artificial material and cultivating them in special bioreactors. The resulting tissues are known as tissue engineered products (TEPs). Areas of application for tissue engineering include skin grafts for burn victims or cartilage implants.