About Gene Therapy
Gene therapy can be used to introduce functioning genes into the cells of a patient, in order to replace defective or missing genes or, as in the case of CombiGene’s platform, to supplement functioning genes and thereby increase the expression of endogenous proteins or peptides with therapeutic effects.
Gene therapy has the potential to cure or alleviate a chronic disease by a one-time treatment, as opposed to drug substances, which must be administered repeatedly.
During the last 5-10 years there has been significant progress in this field, both in academic research and in the commercial development pipeline. Many clinical studies have been initiated in the CNS and other therapeutic areas and, in 2012, the first gene therapy product, Glybera, obtained regulatory approval in Europe.
Read more about gene therapy: