Transformative cell and gene therapies represent a new frontier of medicine. They offer hope for many patients, particularly those affected by rare diseases. Gene therapies are a success story of the 21st century, with exciting treatment options in the pipeline for many diseases, such as rare bleeding disorders or different forms of neuromuscular disorders. Likewise, cell-based products are offering new hope for several conditions (e.g. CAR-Ts for certain blood cancers).
Cell and gene therapies have fundamentally different properties than medicines and surgery in that they treat the core underlying genetic causes of a disease rather than treating symptoms.
Gene therapy is the use of genetic material to treat genetic diseases. This may involve adding or replacing a wild type copy of the gene (gene addition or gene replacement) or altering a gene with mutation to the wild type gene (gene editing). The treatment may take place outside of the body (ex vivo) or inside the body (in vivo).
Cell therapy is the transfer of intact, live cells into a patient to help lessen or cure a disease. The cells may originate from the patient (autologous cells) or a donor (allogeneic cells). The cells used in cell therapy can be classified by their potential to transform into different cell types. Pluripotent cells can transform into any cell type in the body and multipotent cells can transform into other cell types, but their repertoire is more limited than that of pluripotent cells.
POLITICAL PRIORITIES FOR CELL AND GENE THERAPIES IN THE CONTEXT OF THE PHARMACEUTICAL STRATEGY: European Alliance for Transformative Therapies