Heme, Research Fellow
Core
Lead drug discovery and translational research for in vivo gene editing therapies targeting hemoglobinopathies, bridging clinical expertise with preclinical science to advance programs from proof-of-concept to Phase 1/2 trials.
Role type
Senior physician-scientist research project lead
Builds
In vivo gene editing therapeutics for hematopoietic diseases
Domain
Biotechnology / Gene Therapy / Hematology
Required skills
hemoglobinopathies expertise, translational science, drug development strategy, IND-enabling studies, cell and gene therapy, genome editing, stem cell biology, pharmacology/toxicology integration, cross-functional leadership
Preferred skills
sickle cell disease/thalassemia experience, first-in-human strategy, delivery systems optimization, HSC phenotyping, conditioning strategies, regulatory pathways (FDA/EMA), matrixed environment leadership
Technologies
CRISPR, AAV, lentivirus, NGS, flow cytometry, animal models (mouse/non-human primate), organoid models
Responsibilities
Define scientific strategy for editing technologies and delivery approaches; guide in vitro, ex vivo, and in vivo studies; lead cross-functional teams on timelines and budgets; interpret complex datasets for program recommendations; represent the organization in external collaborations and publications.
Seniority
Senior, hands-on IC with strategic oversight