A new way to address the medical need of orphan diseases
The objective of STOP Orphan is to ensure access to an innovative drug discovery service for orphan patients. We propose, at the request of patient associations, institutions and bio-pharma companies faced with an orphan disease, to develop original orphan disease models based on patient’s cells and test the entire global pharmacopoeia, the main objective being the engagement in a rapid development of a new treatment through drug repurposing.
Why are we testing all drugs from the World Pharmacopoeia?
The repositioning of a drug is the fact of obtaining a new authorization for marketing in a new indication. It is a faster and less risky process because the properties of the active substance, including toxicity and pharmacokinetics, are already known. This allows patients to quickly benefit from 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 unmodified cells and directly taken from the patient, the functional defect that we measure is the cause of symptoms in the patient. The results we get are very relevant to the clinic.
Which diseases are we interested in?
Our technology is quite universal. The main domain we used it is that of Inherited Metabolic Diseases. We propose to make a deep analysis of what is known about the disease to judge if we are able to adapt our technology.