Daniel E. Bauer, MD, PhD
At-Large Director (2023-2026)
Daniel E. Bauer, MD, PhD, is the director of the Gene Therapy Program, staff physician, and principal investigator in Pediatric Hematology/Oncology at the Boston Children’s Hospital and Dana-Farber Cancer Institute; the Donald S. Fredrickson, MD, associate professor of Pediatrics at Harvard Medical School; principal faculty at the Harvard Stem Cell Institute; and associate member at the Broad Institute of Harvard and MIT.
Dr. Bauer is a physician-scientist whose laboratory research focuses on genetic determinants of blood cell development and disease and opportunities for innovative therapies for hematopoietic disorders. He has discovered the BCL11A erythroid enhancer as a favorable target for therapeutic genome editing for the β-hemoglobinopathies sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT), now validated by several clinical trials (NCT03745287, NCT03655678, NCT04211480, NCT03653247).
Dr. Bauer has developed methods for highly efficient, penetrant, and specific nuclease and base editing in human hematopoietic stem cells and advanced functional genomic methods to correlate genotypes with molecular, cellular, and organismal phenotypes with high precision and resolution. He has identified a therapeutic gene editing strategy for universal amelioration of ELANE-mutant severe congenital neutropenia, described a core NuRD subcomplex and uncovered ZNF410 as a transcription factor critical for fetal hemoglobin (HbF) repression, and demonstrated how human genetic diversity alters off-target outcomes of therapeutic gene editing. He is developing investigator-initiated clinical trials, as sponsor, for hemoglobinopathies and inherited bone marrow failure disorders.
Dr. Bauer's clinical work focuses on the care of patients with nonmalignant blood disorders. He teaches undergraduate, graduate, medical students, residents, and fellows. He directs a hospital-based gene therapy program and chair an IRB scientific review committee for cell and gene therapies. Dr. Bauer peer reviews grants and manuscripts and serve on the NHLBI SCD Advisory Committee. For the Society, he is a member of the editorial board of Molecular Therapy, chair of the Stem Cell Committee, and has served as abstract reviewer, poster reviewer, committee member, and workshop organizer.