APTEEUS is a clinical stage biotech company addressing monogenetic disorders. Focusing on the molecular causes of diseases, APTEEUS dedicates its strong experience to the discovery and the development of Disease Modifying Therapies.

APTEEUS most advanced drug-candidate is targetting the common underlying cause of a 6th of patients with a monogenic disease. It is the first therapy that target the mechanism responsible for the decay of mRNA transcripts containing a premature termination codon. APTEEUS harnesses its unique mode of action to address several monogenetic recessive conditions, the first ones being genodermatoses. Phase 1/2 clinical trials are planned for 2019.

APTEEUS implemented a unique technology using patient cells to rapidly expand the use of its drug candidate. APTEEUS is also collaborating with patient associations and Pharmas into Drug Discovery projects for molecule screening on patient primary cells and drug repurposing.