Shire, Fondazione Telethon ink research collaboration

Companies to collaborate on 13 undisclosed rare disease indications

Kelsey Kaustinen
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NYON, Switzerland—Shire plc and Fondazione Telethon, anItalian biomedical charitable foundation, have established a long-term,multi-indication research collaboration in rare diseases for research performedat the Telethon Institute of Genetics and Medicine. The alliance will combinethe companies' talents on research for 13 undisclosed rare disease indications,and stands to add multiple novel therapeutic candidates to Shire's early-stagedevelopment pipeline.
"The collaboration with Shire proves yet again the value ofTelethon-funded research which is able to attract interest in thepharmaceutical market, even in a critical phase like the current one,"Francesca Pasinelli, general manager of Fondazione Telethon, said in a pressrelease regarding the deal. "We are particularly pleased because alliances likethis one represent, for a not for profit organization like ours, an actualopportunity to fulfill the promise made to patients and donors who have beensupporting us; translating the excellent results of scientific research intotherapies that are accessible to people suffering from genetic diseases stilllacking a cure. Those are the people for whom Telethon was born and stillexists and keeps working."
Per the terms of the agreement, Shire will provide €17million ($22 million) in funding over the course of five years for a number ofresearch projects that address a range of different lysosomal storage disordersand neurodegenerative diseases. The bulk of the research will be conducted atthe Telethon Institute of Genetics and Medicine's facility in Naples, Italy,and successful projects resulting from the collaboration will be incorporatedinto Shire's existing development pipeline.
"As a leader in rare diseases, Shire's partnership withFondazione Telethon is another way for us to ensure that we expand into newdisease areas and enhance our collaborative relationships with academicinstitutions," Philip J. Vickers, senior vice resident of Research andDevelopment for Shire Human Genetic Therapies, said in a statement. "Shire'sdevelopmental expertise combined with TIGEM's early stage research capabilitieswill enable us to accelerate our discovery and development efforts. Thisresearch collaboration is evidence of Shire's commitment to patients with rarediseases and our intent to work with the best institutions to achieve this."
Fondazione Telethon was founded in 1990 with the goal ofcuring rare genetic diseases, with a particular focus on muscular dystrophy.The Telethon Institute of Genetics and Medicine is the first intramuralresearch institution founded by the company, and is a world leader for itsresearch in genetic diseases.
"Our institute was founded in 1994 thanks to the vision ofTelethon deciding to invest the funds donated by the Italian people in anintramural research institute dedicated to basic studies on genetic diseases,"Andrea Ballabio, director of The Telethon Institute of Genetics and Medicine,said of the deal with Shire. "After almost twenty years, we believe their trusthas been paid off; today more than half of our funding is awarded to us byinternational granting agencies. An industrial partnership such as that withShire goes along with this trend, plus it provides us with a unique opportunitytranslation wise."
SOURCE: Shire press release

Kelsey Kaustinen

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