Alessandra Rufini

Dr. Alessandra Rufini received her PhD in Immunology at the University of Rome “La Sapienza” in 2001. During her PhD she focused her studies on the cross-talk between caspases and tyrosine-kinases in the control of the apoptotic pathway. She trained at the Brown University (Providence, RI) in Cristina Alberini’s laboratory and, as a recipient of an EMBO fellowship, she performed part of her PhD studies at the EMBL (Heidelberg) in Giulio Superti-Furga’s laboratory. She then received a post-doctoral fellowship from the Italian Association for Cancer Research (AIRC) and she continued her studies at the University of Rome “Tor Vergata”. She is currently Senior Investigator in the Laboratory of Signal Transduction (www.labst.org), Rare Diseases Unit, at the University of Rome “Tor Vergata”, Italy. In the last fifteen years her studies have been focused on the identification of therapeutic strategies for Friedreich ataxia, a rare disease that has no cure, following both drug discovery and drug repurposing strategies The drug discovery programs build on the original hypothesis and discovery of the involvement of the ubiquitin-proteasome system in the degradation of frataxin, the protein defective in Friedreich ataxia, and the identification of the E3 ligase responsible for frataxin degradation, and lead to the design of new potentially therapeutic molecules. The drug repurposing programs, based on the idea to identify a new therapeutic indication for an already approved drug, lead to the identification of the anti-retroviral drug etravirine as a promising drug candidate for Friedreich ataxia. Both programs lead to the creations of patents and publications, of which she is author. In 2015 Dr. Alessandra Rufini participated in the creation of Fratagene Therapeutics srl (www.fratagene.com), a spin-off company located in the premises of the University of Rome “Tor Vergata” with the aim to attract investments to develop a therapy for Friedreich ataxia. She is currently the Scientific Director of Fratagene Therapeutics.