Regulatory Pathways for Cell and Gene Therapies: Balancing Innovation with Compliance

Abstract

Gene and cell therapies are relatively advanced types of medicine characterized by the ability to cure a variety of diseases, where the disease has genetic basis or complex nature. These therapies involve altering a patient’s DNA, or using modified cells to replace missing or damaged function or cells, boost immune responses or stimulate tissue repair. The rules that surround these therapies are complex and disparate, which is why agencies such as the FDA, EMA, PMDA and NMPA have introduced frameworks to maintain safety, efficacy and to protect patients. The existing and developed pathways like the breakthrough therapy and the priority review designs are in effort to fast track delivery of such promising treatments. these advanced forms of medicine are used in genetic disorder, cancer, viral and bacterial infections, auto immune diseases, neurological diseases, cardio diseases, and regenerative medicine. However, there are obstacles that many of these therapies face, including the manufacturing of the therapy, the cost and, of course, long term effects monitoring. However, as more research is carried out and new legislation implemented cell and gene therapies are expected to transform the map of medicine by providing a cure for diseases that were formerly untreatable.