The history of medicine shows that innovations in pharmacotherapy follow close behind developments in cutting edge science and technology platforms. The full sequencing of the human genome, from rough draft in 2000 to full sequence in 2003, and advances in insights on the immune system lead to the better understanding of the aetiology of diseases, brought significant advances in treatment, including the immuno-oncological "checkpoint inhibitors" which have brought huge advances in the treatment of certain cancers. This knowledge is now also being used to develop highly sophisticated gene and cell editing techniques which marks a new era of drug development: the era of Advanced Therapies (ATs).
Over the last decade, Advanced Therapies have emerged as novel treatments for conditions including cancers and rare diseases. Unlike traditional pharmacotherapies, based on small molecules or biopharmaceuticals, ATs are intact, living, human cells or segments of DNA or RNA, which are injected into the patient for therapeutic purpose. Within the human body, such therapies replace, repair or regenerate cells, tissues or organs, restoring or establishing normal function1. According to the European Medicine Agency (EMA), tissue engineered products †, cell therapy, gene therapy are considered as ATs. In this white paper, we will not focus on tissue engineering, and will use the term Advanced Therapies to describe cell and gene therapies only.
Gene therapy involves the use of specific nucleic acid (DNA or RNA) to modify the expression of a patient’s defective gene or to interfere with viral replication. Vectors, either viral or non-viral, are required for the delivery of nucleic acids. Cell therapies use intact living cells, often modified outside the body, and injected into the patient to, for example, specifically target and kill cancerous cells. The source of cells used in cell therapies can be autologous (same individual) or allogeneic (another individual).