Gene Therapy is an advanced therapeutic approach that involves modifying or manipulating the expression of a person’s genes to treat or prevent disease. Instead of using drugs to treat symptoms, gene therapy targets the underlying genetic cause of a disorder. It works by introducing new, functional genetic material into a patient’s cells to replace a faulty gene, inactivate a disease-causing gene, or introduce a new gene to fight disease.
The most common delivery method involves using a modified viral vector, often an adeno-associated virus (AAV), to carry the therapeutic genetic payload into the target cells. Gene therapy is highly promising for treating inherited disorders (like certain forms of blindness or muscular dystrophy), cancers, and infectious diseases. It is a core component of the rapidly growing field of Cell and Gene Therapy (CGT), which has seen a surge in regulatory approvals and commercial investment. The successful development and regulatory pathway for these potentially curative treatments require significant expertise in Biomanufacturing and complex clinical development strategies.
