In recent years, the development of cell and gene therapies has provided a new avenue for the treatment of rare disorders. This article will discuss the current and future scenarios of cell and gene therapies in rare disorders with cancer indications as an exception, in Japan. Cell therapy is a treatment in which live cells are transplanted into a patient to replace or repair damaged tissue. Gene therapy, conversely, is inserting genetic material into a patient's cells to fix or replace a malfunctioning gene. Cell therapy aims to replace damaged or diseased cells with healthy ones, while gene therapy targets the underlying genetic cause of a disease for a more targete...
