Rare Partners announces a three parties alliance for thalassemia
RP announcement
February 2011 - Rare Partners, the Italian non profit company dedicated to rare diseases, announced its first product development project, related to the use of rapamycin (sirolimus) in thalassemic patients and based on the research results obtained and patented by Professor Roberto Gambari and coworkers at the University of Ferrara (Italy).
Rare Partners finalized an agreement with the patent's inventors and assignees (University of Ferrara and Associazione Veneta per la Lotta alla Talassemia), aimed at completing the preclinical studies and proceeding with Orphan Drug Designation and clinical development in thalassemic patients. Rapamycin, already used as an immunosuppressant in transplanted patients, should act by inducing erythroid differentiation and expression of embryo-fetal hemoglobin (HbF). It is known that an increase of HbF in thalassemic patients may result in a relevant clinical improvement, such as reduced transfusion frequency.
"The acquisition of this intellectual property represents a major milestone in our development. We are particularly pleased to have reached this agreement and to have the possibility to manage the development of a compound which such a great potential of cilnical benefit. The preclinical development for the new therapeutic indication has already started, with beginning of clinical trials planned for second half of 2012” said Marco Prosdocimi, Managing Director at Rare Partners.
Associazione Veneta per la Lotta alla Talassemia
University of Ferrara