Collaboration agreement between Rare Partners and the Department of Life Sciences and Biotechnology of Ferrara University, Italy

Press release

Milan, December 1st, 2014. Rare Partners and the Department of Life Sciences and Biotechnology of Ferrara University, Italy, have received a Pathfinder Award from the Wellcome Trust to investigate an innovative strategy to improve life quality in beta-thalassemia patients. The Award will fund a study on the characterization of products able to induce higher levels of fetal haemoglobin (HbF) and on development of preclinical models to predict therapeutic response.

Beta-thalassemia treatment can still be considered a major unmet medical need, due to the lack of effective therapies. Median patient survival has increased in most countries, even in patients needing transfusions, in comparison with few years ago, but the quality of life is still poor for many patients and the complication of frequent transfusions is a major problem.

The concept of improving quality of life and possibly increasing survival through HbF increase has found widespread acceptance, but rather limited practical application for the lack of adequate drugs and of methods able to select the most appropriate patients. Our team aims to advance the knowledge and transfer preclinical findings to clinical application in a relatively short time. Among the different drugs potentially useful for the management of thalassemia through HbF increase, the project team will focus on those expected to be tested soon in clinical trials.

Research on thalassemia at the University of Ferrara has been conducted by Roberto Gambari, Professor of Biological Chemistry, and various coworkers for more than 15 years through a collaboration with AVLT (Associazione Veneta per la Lotta alla Talassemia).

Rare Partners is collaborating with the University and AVLT since February 2011. The new project will last 12 months and will be coordinated by Professor Roberto Gambari and by Marco Prosdocimi, Managing Director of Rare Partners.

About University of Ferrara

The University of Ferrara, established in 1391 is one of the oldest universities in Italy, counting more than 18,000 students and with an outstanding track-record of excellence in scientific research, including life sciences. Professor Roberto Gambari is the founder and Director of the Laboratory for the development of genetic and pharmacogenomic therapy of thalassemia, Thal¬Lab at the University of Ferrara.
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About the Wellcome Trust

The Wellcome Trust is a global charitable foundation dedicated to improving health. We provide more than £700 million a year to support bright minds in science, the humanities and the social sciences, as well as education, public engagement and the application of research to medicine.

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