Gene therapy seeks to modify or introduce genes into a patient’s body with the goal of durably treating, preventing or potentially even curing disease, including several types of cancer, viral diseases, and inherited disorders. Gene therapy approaches include replacing a mutated gene that causes disease with a functional copy; or introducing a new, correct copy of a gene into the body in order to fight disease.
Gene therapy may be performed in vivo, in which a gene is transferred to cells inside the patient’s body, or ex vivo, in which a gene is delivered to cells outside of the body, which are then transferred back into the body.
Typically, gene therapy developers introduce new or corrected genes into patient cells using vectors, which are often deactivated viruses. Deactivated viruses are unable to make patients sick, but rather serve as the vehicle to transfer the new genetic material into the cell, where it is incorporated into the chromosomes within the nucleus of the cells. Viruses that have been used for human gene therapy include retroviruses, adenoviruses, herpes simplex, vaccinia, and adeno-associated virus (AAV). Other ways of introducing new genetic material into cells include non-viral vectors, such as nanoparticles and nanospheres.
Gene therapy techniques can also be used to genetically modify patient cells ex vivo, which are then re-introduced into the patient’s body in order to fight disease, an approach known as gene-modified cell therapy. This approach includes a number of cell-based immunotherapy techniques, such as chimeric antigen receptors (CAR) T cell therapies, T cell receptor (TCR) therapies, natural killer (NK) cell therapies, tumor infiltrating lymphocytes (TILs), marrow derived lymphocytes (MILs), gammadelta T cells, and dendritic vaccines.
Examples of ARM members active in developing gene therapies and gene-modified cell therapies include: