Associate Scientist or Scientist - Yen Lab/TGEn Group (Lipid Nanoparticle Formulation)
Core
Develop and optimize lipid nanoparticle-based delivery technologies and genome editing strategies for human hematopoietic stem cells to treat sickle cell disease and other hematological disorders.
Role type
Associate Scientist / Scientist (Lipid Nanoparticle Formulation & Genome Editing)
Builds
Pre-clinical genome editing strategies, IND-enabling studies, and cellular therapy products for hematological diseases.
Domain
Biotechnology / Gene Therapy / Hematology
Deliverable
production ML models | product features | dashboards & analysis | research | client delivery | infrastructure | physical/clinical work
Required skills
Lipid nanoparticle formulation, genome editing strategies, IND-enabling experiments, in vitro and xenotransplantation assays, multicolor flow cytometry, FACS, RT-PCR, ddPCR, next-generation sequencing, single-cell sequencing, NGS library preparation, process scale-up, GMP documentation
Preferred skills
Leading research experiments, lab and scientific project coordination, training laboratory personnel
Technologies
Lipid nanoparticles, flow cytometry, FACS, RT-PCR, ddPCR, NGS, single-cell sequencing
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 assays; Develop experimental methods including NGS and single-cell sequencing; Evaluate scale-up processes to ensure reproducibility; Draft documentation to standardize and transfer processes to GMP facility.
Seniority
Mid-to-Senior level research scientist