Gene therapy solutions for patients with severe inherited diseases
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, gene therapy corrects the underlying genetic cause of a disease and thereby cures patients.
The initial focus addresses a rare inborn immunodeficiency of phagocytes, which leaves affected children susceptible 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. The only current curative option is a stem cell transplant from a compatible donor, which is not always available.
The Somagenetix team is developing a gene therapy treatment that involves isolating a patient’s stem cells from bone marrow, introducing a therapeutic gene using lentiviral vectors to correct the inborn genetic defect, and transplanting the gene- corrected cells back into the patient to restore normal function. In the future, the team aims to expand the platform technology to cure other monogenetic defects in various diseases of the immune system.
Faculty Mentor
