Lead or Senior Scientist - Genome Editing, Genomics, Biotechnology (Shengdar Q. Tsai Lab)
Core
Develop an individualized therapeutic base editing platform to treat pediatric patients with bone marrow failure disorders by correcting disease-causing mutations in hematopoietic stem cells in vivo.
Role type
Lead or Senior Scientist, in vivo genome editing drug development
Builds
In vivo base editing drug products and IND-enabling studies for rare disease treatments
Domain
Biotechnology / Genomics / Pediatric Therapeutics
Deliverable
production ML models | product features | dashboards & analysis | research | client delivery | infrastructure | physical/clinical work
Required skills
genome editing, next-generation sequencing, protein engineering, high-throughput genomics, laboratory automation, IND-enabling studies, genotoxicity characterization, lipid nanoparticle optimization
Preferred skills
CRISPR genome editors, NGS library preparation, variant-aware analysis, manuscript drafting
Technologies
CHANGE-seq-BE, CHANCE-seq, lipid nanoparticles
Responsibilities
Design and develop in vivo base editing drug products; characterize genotoxicity using genomic assays; optimize potency and specificity of HSC-targeted lipid nanoparticles; coordinate multi-disciplinary teams; manage project technical and drug development risks; analyze experimental outcomes and present updates to DRIVE and ARPA-H teams
Seniority
Senior, hands-on IC with leadership responsibilities
