Genetic Medicines for Inherited Blood Disorders | Nucleate Translate Session #1 | 2024

Learn about sickle cell disease and genetic therapies that have been approved to treat it with Dr. Daniel Bauer, MD.PhD.
Dr. Bauer is the director of the Gene Therapy Program at Boston Children’s Hospital, an attending physician at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and leading expert in genetic medicine for inherited blood disorders. His postdoctoral work led to the identification of a previously unidentified genetic element crucial for the regulation of fetal hemoglobin expression, a discovery that has directly enabled a number of gene editing therapies for hemoglobinopathies. His research laboratory at Boston Children’s Hospital continues to investigate genetic determinants of blood cell development and disease to enable novel treatment strategies for blood disorders. Dr. Bauer received both his MD and PhD in Cell and Molecular Biology at the University of Pennsylvania.
Nucleate Translate is an open-access lecture series by physicians to help future biotech leaders understand the fundamentals of clinical development and how to make a positive impact on clinical practice.