Curriculum Vitae

Alessandra Biffi is in charge of the Pediatric Hemathology, Oncology and Stem Cell Transplant Division (clinics, diagnostic and research laboratories) at University Hospital of Padua since October 2018 and she coordinates the research area on Oncohematology, 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 has trained over 40 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 has published over 80 peer-reviewed manuscripts and textbook chapters. She has extensive clinical experience in paediatric stem cell transplant and in early phase cell and gene therapy clinical trials. Her preclinical and clinical research is dedicated at enhancing the efficacy of HSC-based therapeutic approaches for inherited disorders with severe nervous system involvement by i) fostering brain microglia replacement by donor cells after HSC transplantation upon detailed understanding of this phenomenon (Capotondo et al., PNAS 2012), and ii) enhancing the potential of enzyme delivery to the affected nervous system by means of the gene corrected progeny of the transplanted, lentiviral vector (LV)-transduced HSCs (Biffi et al., Science 2013; Sessa et al., Lancet 2016).

Additional research activities comprise novel exploratory projects on the therapeutic role of engineered microglia in neurodegenerative diseases, HSC gene therapy application to autoimmune disorders and novel exploratory projects on targeted cancer therapy in collaboration with local PIs. She is actively collaborating with biotech companies in the gene therapy space, she is one of the founders and scientific advisors of Altheia Science s.r.l., a spinoff of the University of Padua that established sponsored research agreements with the Women and Child Health Department, and of Genething s.r.l., a gene therapy consulting company, also a spinoff of Padua University.

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