Scopus ID: 7003906961
Prof Biffi is the Chief of the Division of Pediatric Hematology, Oncology and Stem Cell Transplant at Padua University and at the Padua University Hospital since October 2018, and coordinates the Research Area on Onco-Hematology, Stem Cell Transplant and Gene Therapy at the Pediatric Research Institute in Padua. Previously, she was the Director of the Gene therapy Program and clinical attending in Stem Cell Transplant at the at Dana-Farber/Boston Children’s Cancer in Boston (2015-2018), and Head of unit at the San Raffaele Telethon Institute for Gene Therapy in Milano, where she also practiced as attending physician and head of a clinical unit in Pediatric Stem Cell Transplant and Immunohematology (up to 2015). She trained over 50 fellows and post-doctoral fellows and numerous residents and medical students in her laboratory and clinics, the majority of whom are still in academic medicine. She received a Consolidator ERC grant award and several other awards, including a Chair in Hematology at Boston Children’s Hospital. She has extensive clinical experience in pediatric stem cell transplant and in early phase cell therapy clinical trials. She is actively involved in advanced allogeneic transplant protocols for metabolic conditions, primary immunodeficiencies and hemoglobinopathies, as well as in gene therapy trials for neurological genetic diseases, hemoglobinopathies, immunodeficiencies and cancer. Her preclinical and clinical research and clinical activity are dedicated at developing innovative treatment modalities for monogenic disorders based on hematopoietic stem cell (HSC) transplantation and gene therapy. Her research is devoted at enhancing the efficacy of HSC-based therapeutic approaches for disorders with severe nervous system involvement by fostering brain microglia replacement by donor cells after HSC transplantation upon detailed understanding of this phenomenon, and enhancing the potential of protein delivery to the affected nervous system by means of the gene corrected progeny of the transplanted and engineered HSCs. Her seminal work on Metachromatic Leukodystrophy provided first obvious evidence of therapeutic efficacy of hematopoietic stem cell gene therapy for this disease and other similar disorders. This innovative treatment modality is now being examined for registration as new drug at EMA. Similar promising data were generated in her laboratory in other neurometabolic and adult-onset neurodegenerative diseases, and new IP associated to these findings was generated and licensed. Over the past 15 years of independent translational research she received over 10M€ of funding from competitive academic grants, including prestigious grants from the ERC (Consolidator grant) and NIH (R01 grant). Moreover, she deposited multiple patents, most of which were licensed to the industry. She established proficient collaborations and sponsored research agreements with biotech companies in the gene therapy space. She also founded a biotech company, Altheia Science, spin off of the University of Padua, devoted to the development of gene therapy for autoimmune disorders.
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