Senior Director, Rare Disease Translational Center
Core
Lead and scale preclinical programs for genomic-based therapeutics (gene editing, gene replacement, RNA modalities) and small molecules, focusing on in vivo efficacy/safety in genetically engineered mice and iPSC models.
Role type
Senior Director, Preclinical Translational Science
Builds
Pre-IND/IND enabling data packages and preclinical programs for gene therapies and small molecules
Domain
Biopharma / Genomic Therapeutics / Preclinical Drug Development
Deliverable
production ML models | product features | dashboards & analysis | research | client delivery | infrastructure | physical/clinical work
Required skills
mouse genetics, iPSC modeling, DMPK, PK/PD modeling, preclinical toxicology, GLP compliance, team leadership, regulatory strategy
Preferred skills
neurodegenerative disease experience, gene therapy IND packages, vector engineering, AAV manufacturing
Technologies
genetically engineered mice, iPSC-derived organoids, AAV vectors, nanoparticles
Responsibilities
Set scientific strategy and milestones for mixed portfolios; design and interpret efficacy/safety/biodistribution studies; oversee iPSC and organoid applications; manage mouse model programs; ensure operational excellence and GLP readiness; build and mentor multidisciplinary teams; oversee timelines and regulatory reporting
Seniority
Senior Director, strategic leadership with hands-on scientific oversight