Heme, Research Fellow

Role: Lead drug discovery and translational research for in vivo gene editing therapies targeting hemoglobinopathies, with responsibilities spanning target selection, preclinical, IND-enabling studies, and Phase 1/2 trials. Responsibilities include shaping scientific strategy, guiding in vitro/ex vivo/in vivo studies, supporting target and candidate optimization, and leading cross-functional teams toward development candidate nomination. Requirements: MD and/or PhD with 11+ years in academic, biotech, or pharma research, expertise in drug development, translational science, and disease biology, with experience in cell/gene therapy and genome editing. Preferred: Direct hemoglobinopathy experience (e.g., sickle cell, thalassemia), familiarity with delivery systems, editing efficiency, HSC biology, regulatory pathways (FDA/EMA), and GCP. High-Value: Hemoglobinopathies, gene editing, in vivo pharmacology, IND-enabling programs, clinical translation, and leadership in matrixed environments. Work setup: On-site, five days/week, with ad hoc flexibility.