Tippi MacKenzie

Biography
Tippi MacKenzie is Professor of Surgery at the University of California, San Francisco and Director of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research. A pediatric and fetal surgeon, she leads a translational research program developing new approaches to treat genetic diseases, including before birth. Her team has advanced three fetal molecular therapies from the laboratory to FDA-authorized Phase 1 clinical trials: in utero hematopoietic stem cell transplantation for alpha thalassemia, in utero enzyme replacement therapy for lysosomal storage disorders, and in utero gene therapy for GM1 gangliosidosis. She recently co-founded the UCSF Center for Genome Surgery, a multidisciplinary collaboration with the Innovative Genomics Institute that brings together scientists and clinicians to accelerate the development of genomic therapies for patients with single-gene disorders. Her work spans basic maternal-fetal biology, therapeutic development, and clinical translation, with the goal of expanding treatment options for patients with serious genetic diseases.