Gene Therapy

Gene therapy involves delivering genetic material to replace, edit, or delete faulty, disease-causing genes. These efficient treatments — sometimes involving just one session — have the potential to provide lasting cures.

For gene therapy to work, the gene causing a disease must first be identified and a corrective gene made in the lab. Genes are made of DNA. The corrective DNA is placed into a delivery vehicle, known as a “vector”. The “vector” is injected into a patient and carries the new DNA into a cell for delivery. Once inside the cell, the new DNA is inserted into the patient’s chromosomes. The cells than replicate as usual, but now with the corrective gene instead of the faulty one.

152 drugs in the pipeline

60 companies

Scientists have been working on gene therapy for 30 years and only now are overcoming the obstacles faced in delivery and packaging the curative DNA. The gene therapy approach will take us away from daily pills, or weekly injections, to what could potentially be just a single shot. Millions of patients' lives could be transformed in the near future with this new technology, as today many gene-based diseases leave patients without any treatment option.