Gene therapy targets the underlying cause of a genetic disease. In gene therapy, a functioning gene is delivered to a targeted tissue in the body, enabling the cell or tissue to produce a protein that is either missing or is not functioning. This type of treatment has the potential to be used instead of drugs or surgery.
The safety and efficacy of gene therapy is being studied in clinical trials.
We believe that gene therapy is a potentially transformative treatment approach that, if successful, may help reduce the burden of disease and of ongoing treatments for the people who are affected by rare diseases.