Cell and gene therapy

The potential of cell and gene therapies to either prevent disease progression or provide a cure offers a great clinical benefit over existing therapies which are often geared toward the treatment of symptoms.

Gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uptake of the gene into the appropriate cells of the body. This can include the insertion of a new copy of a gene, gene editing using e.g., CRISPR technology, and gene silencing. Gene therapy is usually performed by viral-mediated gene transfer with Adeno-Associated Virus (AAV) or lentivirus or alternatively by using plasmids. Cell therapy involves the transfer of cells with the relevant function into the patient. The cells are modified outside the body before being injected into the patient, and can come from the patient (autologous cells) or a donor (allogeneic cells). Examples of cell therapy include CAR T-cells.