Shire to collaborate with Fondazione Telethon

Published: 25-Oct-2012

Will provide US$22m over five years for research into rare diseases


Shire has entered a long-term, multi-indication research partnership in rare diseases with Fondazione Telethon, an Italian biomedical charitable foundation.

The Telethon Institute of Genetics and Medicine (TIGEM) will carry out research on 13 undisclosed rare disease indications, which has the potential to add multiple, novel therapeutic candidates into Shire’s early stage pipeline.

The collaboration brings together Shire’s expertise in developing therapies for patients with rare diseases and TIGEM’s research capabilities in gene therapy and other novel therapeutics.

Under the terms of the agreement, Shire will provide US$22m (€17m) funding over five years for several research projects that collectively address 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.

‘As a leader in rare diseases, Shire’s partnership with Fondazione Telethon is another way for us to ensure that we expand into new disease areas and enhance our collaborative relationships with academic institutions,’ said Philip Vickers, senior vice president of research and development at Shire HGT. ‘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.’

Francesca Pasinelli, general manager of Fondazione Telethon, said: ‘Alliances like this one represent, for a not for profit organisation like ours, an actual opportunity to fulfil 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.’

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