Gene therapy is a promising field in medicine that is experiencing unprecedented dynamic development. Unlike the majority of drugs on the market, which merely treat symptoms, this approach attempts to correct the underlying genetic cause of a disease and thereby cure patients. Somagenetix seeks to harness the power of gene therapy to develop treatments for life-threatening diseases.
The first aim is to address an inborn immunodeficiency of phagocytes, which can lead to life‐threatening bacterial and fungal infections as well as to hyperinflammation. Life expectancy in affected children and young adults is compromised, and the disease burden is high. To date, the only curative option is to transplant blood-building stem cells from donors. In the absence of a compatible donor, the patient’s own stem cells can be gene corrected. Somagenetix is developing a treatment that comprises isolation of patient stem cells from bone marrow, the addition of a therapeutic gene using lentiviral vectors to compensate for the inborn genetic defect, and transplantation of gene-corrected cells back into the patient.
In later phases, the aim would be to apply this platform technology to cure other monogenetic defects found in various diseases of the immune system, as well as in other fields of medicine.