World leaders in
gene therapy

A milestone achievement by SR-TIGET – San Raffaele Telethon Institute for Gene Therapy in Milan was the approval in 2016 of the first ex-vivo stem cell gene therapy to treat patients with ADA-SCID, a severe congenital immunodeficiency, once known as “the bubble boys disease”

In 2016, GSK, Fondazione Telethon and Ospedale San Raffaele announced the approval of Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with ADA-SCID.
It is the first corrective gene therapy for children to be awarded regulatory approval anywhere in the world and one of the world’s first instances of efficacious gene therapy.
The gene therapy for the treatment of ADA-SCID was originally developed at SR-Tiget and was taken forward by GSK through a strategic collaboration formed in 2010 between GSK, OSR and Fondazione Telethon.

In 2013 the Institute’s contribution to establishing gene therapy as a promising innovative therapeutic approach for genetic diseases was definitively acknowledged by the international scientific community with the success of the clinical trials on another immunodeficiency, Wiskott-Aldrich syndrome (WAS), and a metabolic disease leading to severe neurodegeneration in children, metachromatic leukodystrophy (MLD).

The results from the trials, published in two independent back-to-back articles in Science, are universally renowned as a breakthrough achievement with significant potential impact beyond the treatment of rare pediatric diseases previously without a cure, like WAS and MLD.