Alliance between Fondazione Telethon and biopharmaceutical company Shire for research and development of new therapies for rare genetic diseases

Telethon research proves once again its ability to attract international funding; the Italian foundation focused on rare genetic diseases has signed an important agreement to collaborate with the international pharma company Shire plc.

The company has committed to invest more than 17 million euros over a 5 year period to support research performed at the Telethon Institute of Genetic and Medicine (Tigem) in Naples, under the direction of Andrea Ballabio.

Thanks to the synergy between Tigem’s scientific standing and Shire’s proven track record in the field of drug development and distribution, the agreement represents an important step forward toward the common goal of making effective therapies available to patients suffering from rare genetic diseases. Through this virtual changeover, with the economic support by Shire, scientists at the Institute in Naples will continue to perform their independent research on several projects aimed at developing gene and drug therapy strategies on lysosomal and neurodegenerative disorders.

The company will eventually be able to pick up the most interesting results and guarantee their development into effective therapies to be delivered to patients around the world.

According to Telethon’s general manager Francesca Pasinelli «the collaboration with Shire proves yet again the value of Telethon-funded research which is able to attract interest in the pharmaceutical market, even in a critical phase like the current one. We are particularly pleased because alliances like this one represent, for a not for profit organization like ours, an actual opportunity to fulfill the promise made to patients and donors who have been supporting us; translating the excellent results of scientific research in therapies that are accessible to people suffering from genetic diseases still lacking a cure. Those are the people for whom Telethon was born and still exists and keeps working».

Andrea Ballabio, Tigem’s director, also expressed great satisfaction: «our institute was founded in 1994 thanks to the vision of Telethon deciding to invest the funds donated by the Italian people in an intra-mural research institute dedicated to basic studies on genetic diseases. After almost twenty years, we believe their trust has been paid off; today more than half of our funding is awarded to us by international granting agencies. An industrial partnership such as that with Shire goes along with this trend, plus it provides us with a unique opportunity translation wise».«For a leader company in the fight against rare diseases like ours, the partnership with Fondazione Telethon is a great opportunity to strengthen our position in the field and to productively collaborate with academic research» said Philip J. Vickers, senior vice-president, and director of Business Development at Shire Human Genetic Therapies.

«Thanks to the synergy between complementary expertise along the different phases in drug development– from basic studies to the actual therapeutic development– we can significantly speed up our efforts. The agreement with one of the most important research institutes in the rare disease field shows ones again to the patients’ community that Shire has a real interest for these severe conditions».

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