Somagenetix is addressing the treatment of autosomal inherited diseases using the last generation of self-inactivating lentiviruses. The company is also developing diagnostic tools and other innovative therapeutic approaches based on CAS-CRISPR technology.

The lead project is a novel gene therapy vector to cure p47phox and x-linked Chronic Granulomatous Disease (CGD). CGD is an inherited disorder of phagocytic cells in which those cells are unable to kill bacteria and fungi because of a gene defect which does not allow the necessary respiratory burst (NAPDH complex). The patient is subject to recurrent chronic infections and die the first five years of life.

The second objective is to create a gene therapy platform for other diseases such as fronto-temporal dementia (FTD) and certain type of cancers.