Bone marrow failure (BMF) disorders are catastrophic diseases of childhood that result in failure to produce hematopoietic cells leading to severe morbidity and eventually mortality. Treatment options are limited to supportive care and allogeneic bone marrow transplantation. We are interested in developing curative therapies for patients with these rare but severe disorders. Using a combination of disease model systems such as induced pluripotent stem cell-derived hematopoietic stem cells and engineered hematopoietic stem cells from healthy donors to model patient mutations, we have advanced development of gene therapy for Diamond-Blackfan anemia which is anticipated to move to clinic in the coming months. Now we are developing individualized genome editing-based treatments using base editors and prime editors for BMF disorders. As part of an academic/industry team, we recently received funding from ARPA-H to advance development of in vivo genome editing for BMF disorders (https://www.stjude.org/research/departments/hematology/drive.html). This translational project, called DRIVE which stands for Delivering Rare-disease In Vivo Editors, will involve development of preclinical models and pipelines to demonstrate efficacy and safety and accelerated development of personalized bespoke genomic medicines for patients with BMF disorders such as Diamond-Blackfan anemia and GATA2 deficiency. You will work with a multidisciplinary team of scientists with expertise in genome editing, high throughput screens, hematology, cell biology, LNP formulation, technology development and commercialization, cGMP practices, technical writing, regulatory affairs, clinical trial design and project management.