An Introduction to Gene Therapy
Gene therapy is the introduction of genetic material into cells to replace missing or defective genes. If a gene doesn’t function properly, the introduction of a normal copy of a gene may be able to treat or prevent disease. It is a technique that has the potential to deliver long-term and potentially curative benefits to patients with genetic disorders.
Syncona identified the advent of gene therapy in 2012 as a significant technology discontinuity, which could have dramatic efficacy for patients in areas of high unmet need.
“Very simply, back in 2012 when we saw the initial gene therapy data sets, these were profound… it wasn’t the fact of gene therapy as a technology, it was what gene therapy could do for a patient”
Chief Investment Officer, Syncona