About gene therapy

What makes gene therapy technology unique is that defective or missing genes can be replaced. With CombiGene’s method, functioning genes are complemented with new functions.

The goal of gene therapy is to treat diseases that are caused by single defective genes. Today, about 2,800 such diseases are known. For many of them, there are no adequate treatment methods. Gene therapy is therefore one of the most exciting fields of development in the pharmaceutical industry, thanks to unique possibilities for targeted therapy and the potential for meeting a very great medical need.

Unlike conventional drug treatment of chronic diseases, which requires continuous medication, gene therapy has the great advantage of being able to achieve a long-term effect after only one or a few treatments. Significant progress in gene therapy has been made over the past 5-10 years and the major pharmaceutical companies are showing considerable interest. To date, in Sweden, four gene-therapy products have been approved for use. Considering the great advantages gene therapy offers and the many research and development projects now under way throughout the world, it is reasonable to assume that the number of approved gene-therapy products will increase dramatically in the years to come.