Scientist / Associate Scientist – Preclinical Design & Development (PDD) Core
About The Position
The Preclinical Design and Development (PDD) Core within the Pediatric Translational Neuroscience Initiative at St. Jude Children's Research Hospital seeks a rigorous, execution-focused Scientist to advance mutation-specific antisense therapies from genetic diagnosis to candidate therapeutic. The team develops and evaluates antisense oligonucleotide (ASO) candidates for children with ultra-rare and one-of-a-kind genetic conditions. The successful candidate will carry cases through the development pipeline, from patient-derived cellular models and ASO screening—including splice-switching oligonucleotides, gapmers, or related modalities—to functional and molecular validation. Work is tailored to diverse genomic and cellular contexts and involves close collaboration with teams in genomics, cell biology, and clinical translation. The role is part of a shared, cross-functional translational team and requires adapting assay design and mechanistic reasoning to each case while working rigorously and at a pace responsive to patient timelines.
Requirements
- Bachelor's degree in a relevant scientific area, such as genomics, molecular biology, or cell biology.
- Scientist: 8 years of relevant work experience; 6 years with a master's degree; or 3 years with a PhD.
- Associate Scientist: 7 or more years of research experience; 5 or more years with a master's degree; or 2 or more years with a PhD.
- Skilled molecular biologist with expertise in plate-based cellular screening workflows and working knowledge of splicing and RNA biology.
- Collaborative, methodical, and self-directed approach, with comfort working iteratively in a fast-paced translational setting.
Nice To Haves
- Experience with ASO or other RNA-targeted therapeutics, ideally combined with high-throughput screening.
- Experience developing and validating quantitative molecular assays, such as TaqMan qPCR, ddPCR, and allele-specific probe assays.
- Strong understanding of RNA splicing biology and splice-site regulation.
- Experience with patient-derived cellular models, such as primary cells, fibroblasts, or iPSCs.
- Experience in rare disease, neurogenetics, and/or translational therapeutic research.
- Prior experience with Janus liquid handlers.
Responsibilities
- Design and execute cell-based ASO screening and therapeutic evaluation workflows across concurrent patient cases.
- Derive, characterize, and bank patient-derived cellular models for screening and functional validation.
- Interpret assays to distinguish biology-driven effects from technical artifacts; refine assay design and troubleshoot across diverse genomic contexts.
- Design and validate quantitative molecular assays, including RT-qPCR and ddPCR, and interpret biochemical and imaging-based readouts of therapeutic efficacy.
- Manage parallel, multi-step experimental workflows across cases.
- Collaborate with genomics, molecular biology, and clinical translation teams to advance cases through the pipeline.
- Maintain rigorous documentation, data integrity, and quality control standards in a translational laboratory environment.
- Support day-to-day laboratory operations, including supply management and equipment upkeep.
- Contribute to a culture of shared responsibility, mutual support, and collective scientific progress.
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What This Job Offers
Job Type
Full-time
Career Level
Mid Level
