Study Director, Rare Disease Translational Center
Core
Planning, execution, and oversight of scientific studies involving mouse models and therapeutic evaluation for rare diseases.
Role type
Senior Study Director (Rare Disease Translational Research)
Builds
Clinically relevant mouse models, gene therapy/RNA-based therapeutic candidates, and molecular biology/biomarker analysis technologies.
Domain
Biomedical research / Rare diseases / Genetics / Molecular biology
Required skills
Mouse physiology expertise, Transgenic and genome editing technologies, Therapeutic evaluation (gene therapy, ASO, small molecules), Team leadership, Project management, Budget management, Scientific publication record
Preferred skills
Post-doctorate experience, Experience with emerging therapeutic approaches
Technologies
Mouse models, Gene therapy, RNA-based treatments, ASO, Small molecules, Molecular biology platforms
Responsibilities
Characterize and validate new and existing mouse models; Evaluate potential therapeutics for clinical use; Develop molecular biology and biomarker analysis technologies; Manage a multi-project portfolio and publish key results; Provide leadership to staff including Research Assistants and Molecular Biology Specialists
Seniority
Senior, hands-on IC with management responsibilities