Cell and Gene Therapy Development

Cell therapy techniques aim to treat diseases by harnessing cells to deliver treatment. Alternatively, cell therapy can be used to repair or replace damaged tissue. In cell therapy, cells are cultured and if required modified outside a patient’s body before being infused into the patient. The cells can originate from the patient themselves (autologous cells) or from a healthy donor (allogeneic cells). Stem cells are one of the most widely used cell types harnessed for cell therapy today. These undifferentiated cells bring great value to regenerative medicine for their ability to differentiate into almost any type of cell in the body.

Gene therapy aims towards restoring abnormal or dysfunctional expression patterns of a disease-related gene by introducing genetic material to replace, enhance or silence its function. Gene therapy intends to achieve long-lasting treatment or prevention of diseases including certain types of cancer, inherited disorders, and viral diseases. It has been estimated that approximately 10,000 human diseases are caused by a mutation in a single gene, and gene therapy could represent a therapeutic option for many of these. Ex vivo gene therapy is the removal of specific target cells from a patient, which then undergo genetic modification, before being transplanted back into the patient. This is an important crossover with cell therapy, as applications like CAR-T cell therapy rely upon ex vivo gene editing. In vivo gene therapy involves gene transfer directly into a patient and is ideally suited for monogenetic disorders. It involves the transfer of genetic material into a patient’s cells with the aim of restoring normal function.

Some cell and gene therapies take a hybrid approach to combatting diseases and can be considered both cell and gene therapies. These work by editing genes within specific types of cells and inserting them into the body. Chimeric antigen receptor (CAR) T cell therapy is a novel technique used in the treatment of blood cancers including lymphomas, some types of leukemia, and multiple myeloma. T cells are isolated from a patient’s blood, and then genetically engineered with the addition of a gene for a special receptor (CAR), which is highly personalized for a patient’s specific cancer phenotype. Once administered back to the patient, the newly engineered CAR-T cells are able to recognize and attack cancer cells. Similar engineered immune cell approaches include tumor-infiltrating lymphocytes (TILs) and tumor-specific T cell receptor (TCR)–modified T cells.