Shire and Telethon partner in rare diseases research

Shire plc has announced a research collaboration in rare diseases with Fondazione Telethon, a major Italian biomedical charitable foundation, for research carried out at the Telethon Institute of Genetics and Medicine (TIGEM). This alliance will facilitate research on 13 undisclosed rare disease indications and has the potential to add multiple, novel therapeutic candidates into Shire’s early stage pipeline.

Under the terms of the agreement, Shire will provide $22 million (€17 million) funding over five years for several research projects that collectively address a number of different lysosomal storage disorders and neurodegenerative diseases. The majority of the research will be conducted in TIGEM’s facility in Naples, Italy under the direction of Andrea Ballabio. Successful projects that arise from this research will be incorporated into Shire’s development pipeline and benefit from additional Shire investment and resources.

“Philip J. Vickers, Senior Vice President, Research and Development, Shire HGT said: “Shire’s developmental expertise combined with TIGEM’s early stage research capabilities will enable us to accelerate our discovery and development efforts. This research collaboration is evidence of Shire’s commitment to patients with rare diseases and our intent to work with the best institutions to achieve this.”

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 into 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,” said Francesca Pasinelli, General Manager of Fondazione Telethon.