Scientist - Yen Lab/TGEn Group (Hematopoietic Stem Cell, Genome Editing)
Core
Develop genome editing strategies for human hematopoietic stem cells (HSC) to treat sickle cell disease and other hematological diseases, advancing from pre-clinical to IND-enabling studies.
Role type
Senior Scientist (Genome Editing & Hematopoietic Stem Cell Biology)
Builds
Pre-clinical genome-edited HSC cellular therapy products for sickle cell disease
Domain
Biotechnology / Gene Therapy / Hematology
Deliverable
production ML models | product features | dashboards & analysis | research | client delivery | infrastructure | physical/clinical work
Required skills
Genome editing technologies, Human hematopoietic stem cell (HSC) biology, Multicolor flow cytometry and FACS, Next-generation sequencing (NGS) library preparation and analysis, RT-PCR, ddPCR, Single-cell sequencing, Process development and scale-up, In vitro and xenotransplantation assay design
Preferred skills
Experience leading research experiments, Lab and scientific project coordination, Training laboratory personnel
Technologies
CRISPR/Cas systems, Flow cytometers, NGS platforms, qPCR systems, Xenotransplantation models
Responsibilities
Design and execute IND-enabling experiments to characterize pharmacology of genome-edited HSC products, Characterize efficacy, safety, and potency of HSC cellular drug products in in vitro and xenotransplantation assays, Develop experimental methods including RT-PCR, ddPCR, NGS, and single-cell sequencing, Evaluate scale-up and close-system processes to ensure reproducibility, Conduct exploratory research on novel in-vitro and in-vivo delivery strategies, Draft documentation to standardize and transfer processes to GMP facility
Seniority
Senior, hands-on IC with project leadership
