This month, we are featuring Natalia Gomez-Ospina, who was awarded a $375,000 Rosenau Family Research Foundation grant in 2025 for her project titled “Autologous Transplantation of Genome-Edited Hematopoietic Stem and Progenitors Cells for Krabbe Disease”, which is working to develop a safer and more effective therapy using a patient’s own stem cells.
Dr. Natalia Gomez-Ospina is an Assistant Professor of Pediatrics (Medical Genetics) at Stanford University and a board-certified medical geneticist. As a physician-scientist, she bridges clinical care and laboratory research to improve the diagnosis and treatment of lysosomal storage disorders, including Krabbe disease. At Lucile Packard Children’s Hospital, she leads the enzyme replacement therapy service and directs the Stanford Medicine Children’s Health – NORD Center of Excellence for Rare Disorders, advancing gene- and cell-based therapies for children with rare genetic conditions.
Dr. Gomez-Ospina’s research focuses on harnessing the hematopoietic system to deliver lysosomal enzymes to the brain, addressing one of the most critical challenges in treating neurodegenerative lysosomal diseases like Krabbe. Her laboratory has developed an adaptable genome-editing platform for autologous transplantation of corrected hematopoietic stem cells, enabling systemic and central nervous system enzyme delivery. This approach is being applied to Mucopolysaccharidosis type I (Hurler syndrome), Gaucher disease, and now Krabbe disease.
Although early in her career, Dr. Gomez-Ospina has made significant scientific contributions, with publications in The New England Journal of Medicine, Cell , Nature Communications , and the American Journal of Medical Genetics . Her work has been recognized through numerous awards, including the Genetics and Genomic Medicine Innovation Award from the ACMG, the Outstanding Young Investigator Award from ASGCT, the Young Scientist Award from ASCI, the Young Investigator Award from WSPR, and the William K. Bowes Jr. Award in Medical Genetics from Partners HealthCare Personalized Medicine.