Assistant Professor in Pediatrics, Harvard Medical School Faculty, Division of Hematology/Oncology, Boston Children's Hospital Faculty, Department of Pediatric Oncology, Dana-Farber Cancer Institute Associate Faculty, Harvard Stem Cell Institute
Dr. Pietro Genovese, Ph.D., is Principal Investigator at the Gene Therapy Program of Dana-Farber/Boston Children’s Cancer and Blood Disorder Center, and Assistant Professor of Pediatrics at Harvard Medical School. In the last 15 years, he dedicates his efforts to develop genome editing tools that improve the safety and efficacy of adoptive immunotherapy or promote safer applications of human stem cell gene therapy.
Working with the group of Luigi Naldini at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), he contributed to pioneer this field since when ZFNs were first shown to enhance gene targeting and be useful for genetic engineering of somatic cells for therapeutic purposes. In 2007, he contributed to a break-through work where they demonstrated for the first time the possibility to exploit ZFN to direct the integration of exogenous DNA sequences into a predetermined genomic locus of several human cell types (Lombardo, Genovese et al., Nat Biotech 2008). During his Ph.D. studies, he extended his knowledge and skills on this technology by developing the T cell receptor gene editing strategy to improve safety and efficacy of cancer adoptive immunotherapies (Provasi* Genovese* et al., Nat Med. 2012). This innovative approach is now widely used in the immunotherapy field for generating allo-compatible T cells or to express CAR genes under the control of endogenous TCR promoter. As post-doctoral associate, he engaged an ambitious study aimed to correct inherited mutations and developed the first protocol that allow targeted transgene integration in human hematopoietic stem cells (HSC) capable of long-term multilineage repopulation (Genovese at al., Nature 2014). As Project Leader, he coordinated a work team of scientists towards the goal to perform pre-clinical development and proof of feasibility of these novel medical treatments for some candidate diseases, chosen as paradigmatic for testing their therapeutic potential. His first project conducted as principal investigator focused on the HDR-mediated correction of HIGM1 is now in advanced phases of manufacturing development, and first-of-this-kind trial is expected to be opened at SR-Tiget in two years from now. Recently, he established his own independent laboratory at the Dana-Farber/Boston Children’s Cancer and Blood Disorder Center, and been appointed assistant professor at Harvard Medical School. As faculty member, his current efforts are aimed to move these advanced genetic engineering strategies towards an effective therapeutic treatment for inherited and malignant diseases. Dr. Genovese is author of several high-profile publications, co-inventor of more than 8 patents on gene editing technology, recipient of several awards, from which the Young Investigator Award from the ESGCT society (2016).