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 a cellular therapy from bench-to-bedside.
Role type
Senior Scientist (Genome Editing & Hematopoietic Stem Cell Biology)
Builds
Pre-clinical genome-edited HSC products for Phase 1 clinical trials
Domain
Biotechnology / Gene Therapy / Hematology
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, IND-enabling study design
Preferred skills
Experience leading research experiments, Lab and scientific project coordination, Training laboratory personnel
Technologies
CRISPR/Cas systems, Flow cytometers, NGS platforms, Single-cell sequencers, GMP facilities
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, and single-cell sequencing; Evaluate scale-up and close-system processes to ensure reproducibility; Draft documentation to standardize and transfer processes to GMP facility.
Seniority
Senior, hands-on IC with project leadership