Dr. Biffi is the current director of the Gene therapy Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. Her previous position was in Milan, at the San Raffaele Telethon Institute for Gene Therapy where she trained and developed a Research and Clinical Unit dedicated to the treatment of lysosomal storage disorders (LSDs) by means of hematopoietic stem cell (HSC)-based approaches. She has trained over 30 fellows and post-doctoral fellows and numerous residents. She has published over 45 peer-reviewed manuscripts and textbook chapters. She is actively involved in gene therapy trials for neurological genetic diseases, hemoglobinopathies and immunodeficiencies. Her specific research is dedicated to enhancing the efficacy of HSC-based therapeutic approaches for LSDs with severe nervous system involvement by fostering brain microglia replacement by donor cells after HSC transplantation and 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. Additional research activities comprise the identification and exploitation of novel biomarkers and therapeutic targets for lysosomal storage, and novel exploratory activities on the therapeutic role of engineered microglia in neurodegenerative diseases. She is also involved in other gene therapy clinical trials active in the Gene Therapy Program and in advanced allogeneic transplant protocols for LSDs.
Alessandra Biffi, MD