Date: 2012-10-24

Type of information: R&D agreement


Company: Shire (UK) Fondazione Telethon (Italy)

Therapeutic area: Rare diseases - Neurodegenerative diseases - Metabolic diseases

Type agreement:


Action mechanism:

Disease: 13 undisclosed rare disease indications related to lysosomal storage disorders and neurodegenerative diseases


Shire has announced a long-term, broad based, multi-indication 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. The collaboration brings together Shire’s established capabilities in developing and distributing effective, life-altering therapies for patients with rare diseases and TIGEM’s world renowned research expertise in gene therapy and other novel therapeutics.  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.

Financial terms:

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.

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Is general: Yes