A collaborative way to address the medical need for orphan diseases
STOP Orphan aims at giving orphan patients access to APTEEUS innovative discovery engine.
In collaboration with patient associations, institutions and bio-pharma companies faced with orphan diseases, we develop in vitro screening assays based on patient’s cells that harbors the molecular cause of the disease.
The main objectives is identify and optimize lead molecule as well as drug candidates and engage them into a rapid clinical development.
Why testing all drugs from the World Pharmacopoeia?
Drug repurposing consists in obtaining a new marketing authorization for a new indication. It is a faster and less risky process because safety and PK properties of the active substance are already known. It accelerate patients’ access to our discoveries.
Why do we commit the patient in search of his own treatment?
The best model of a disease is the patient himself. Working on primary cells directly taken from the patient, allows the measurement of the functional defect that is the cause of disease. The results we get are very relevant to the clinic.
Which disease families the technology is suitable for?
Our discovery engine requires primary and expandable cells in which the defective function is at work. To date, we have worked on many inherited metabolic disorders and genodermatoses. For each new demand, we make a deep analysis of the state-of-the-art on the disease and its compatibility with our technology.