RARE & ORPHAN diseases
Fight against monogenic diseases
Because we are aware that current technologies can not meet the medical needs of all, we believe that the repositioning of medication is the approach that, in the long term, will treat the greatest number.
Based on a systematic approach, we discover unsuspected drug activities by measuring their effect on the cellular defects that cause symptoms in patients with rare monogenic diseases.
Our technology allows us to evaluate the effect of several thousand molecules per individual. Thus, if a drug has a corrective effect of the causal defect of the disease, it can be identified and quickly evaluated clinically in the patient.
diseases and fields of application
It is estimated that three hundred million people worldwide suffer from a disease that is inscribed in their genome, transmitted by their parents or spontaneous. This deleterious genetic polymorphism is said to be responsible for more than seven thousand rare diseases, most of them untreated.
Through the dozens of cell screens that we have already carried out, we have gained solid experience in the field of inherited metabolic diseases that we are now using in dermatological, muscular and neurological programs.
Les Petits MecP2 is an association of parents whose children are affected by a rare and serious genetic abnormality: the MeCP2 gene duplication syndrome. In France, there are about a hundred people who would be affected by this genetic anomaly still too little known by the medical community. APTEEUS ran its whole process in less than 24 months to identify several promising drug candidates.