Thanks to our research, we have developed a number of therapies capable of making big improvements to the lives of genetic disease patients.

One of the most successful of these solutions is gene therapy, deployed against three serious genetic diseases in children. We are rapidly proceeding along this road and may soon discover new remedies for other diseases.

A great result

For all children with ADA-SCID, a rare immunodeficiency, gene therapy is finally available; thanks to an agreement signed in 2010 with GlaxoSmithKline, the outstanding results obtained by researchers from the San Raffaele Telethon Institute for Gene Therapy in Milan have been turned into a treatment accessible to all patients in Europe.

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Strimvelis, new hope

The European Commission has given the green light for the use of gene therapy for ADA-SCID as a drug: see the comments by some of the main protagonists of this huge success.

Read our position paper on Strimvelis

Gene therapy stories


The research carried out at our institutes has pinpointed promising therapeutic strategies for combatting a number of diseases. Such results require years of investigation of the mechanisms underlying genetic diseases, and for development of innovative forms of treatment. Concrete answers are now emerging. For all patients.