For Genethon, an altruistically-managed laboratory created by an association of patients and relatives of patients, the interests of patients and their families come first. Our goal is to develop treatments for rare diseases for which therapies don’t already exist and to make them accessible to all those who need them.
After having created the first maps of the human genome and making them available to the global scientific community to help accelerate the identification of disease-causing genes, Genethon has now begun developing gene therapies for rare and ultra-rare diseases so that one day everyone will be able to be treated.
More and more clinical trials are currently underway, and the first gene therapy drugs born of our research or the technology we have developed are arriving on the market. So many patients and their families are seeing their hopes being realized, but we are faced with the major challenge of making our innovative treatments accessible to as many people as possible.
Genethon is a leader in overcoming the scientific and technological barriers presented by gene therapy. We also need to improve bioproduction methods in order to reduce costs and facilitate the large-scale production of drugs for all eligible patients.
Genethon is in favor of “fair and controlled” pricing: the market price of gene therapy drugs should align with the capacities of our healthcare systems and should never prevent patients from getting treatment. We are particularly forceful on this issue when we form industrial partnerships to develop our products.
Such challenges are part and parcel of the mission entrusted to us by people suffering from chronic illness and their families: to find a cure.