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 crosstalk between caspases and tyrosine-kinases in the control of the apoptotic pathway. She trained at the Brown University (Providence, RI) and, as a recipient of an EMBO fellowship, she performed part of her PhD studies at the EMBL (Heidelberg). 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, Rare Diseases Unit, at the University of Rome “Tor Vergata”, Italy. Her research is aimed at the identification of new molecular targets and therapeutic strategies for Friedreich ataxia, a rare disease that has no cure, following both drug discovery and drug repurposing strategies. The drug discovery programs are based on the original finding of the involvement of the ubiquitin-proteasome system in the degradation of frataxin, the protein defective in Friedreich ataxia, and led to the identification of the E3 ligase responsible for frataxin degradation, as a new therapeutic target for Friedreich ataxia. The drug repurposing programs, through the screening of an FDA-approved drugs collection, led to the identification of the anti-retroviral drug etravirine as a promising drug candidate for Friedreich ataxia. A phase II clinical trial to test the efficacy and safety of etravirine in a cohort of FRDA patients has been recently concluded. Both research programs led to the creation of patents and publications, of which she is inventor and author. In 2015 Dr. Rufini participated in the creation of Fratagene Therapeutics srl (www.fratagene.com), a spin-off company located on the premises of the University of Rome “Tor Vergata”, with the aim to attract investments to develop a therapy for Friedreich ataxia. She was the scientific director of the company until 2022. Since 2019, Dr Rufini has been a Contract Professor of Immunology and Immunopathology in Medicine and Surgery Degree and of General Pathology in Dentistry and Dental Prosthetics Degree, at the Saint Camillus International University of Health and Medical Sciences.