Scientist​/Associate Scientist – Preclinical Design & Development; PDD) Core

Position: Scientist / Associate Scientist – Preclinical Design & Development (PDD) Core
Join our Team!

The Preclinical Design and Development (PDD) Core within the Pediatric Translational Neuroscience Initiative e Children's Research Hospital is seeking a rigorous, execution-focused Scientist to help advance mutation-specific antisense therapies from genetic diagnosis to candidate therapeutic. Our team works at the leading edge of individualized RNA-targeted therapy, developing and evaluating antisense oligonucleotide (ASO) candidates for children with ultra-rare and even one-of-a-kind (n-of-1) genetic conditions.

The successful candidate will carry cases through the pipeline- from patient-derived cellular models, through ASO screening (splice-switching oligonucleotides, gapmers, or related modalities), into functional and molecular validation, working closely with a multidisciplinary team spanning genomics, cell biology, and clinical translation. Cases span diverse genomic and cellular contexts, each requiring adapted assay design and mechanistic reasoning tailored to that context. We are looking for a collaborative scientist who is at ease with the iterative, fast-paced nature of translational work, where rigor and pace are both driven by patient timelines.

This role offers the opportunity to build technical depth in precision-genomics-driven RNA therapeutics as part of a shared, cross-functional, translational team.

Who We’re Looking For The ideal candidate is a skilled molecular biologist with expertise in plate-based cellular screening workflows and working knowledge of splicing/RNA biology. We are looking for a collaborative, methodical scientist who is self-directed and at ease with the iterative, fast-paced nature of translational work.

Preferred Qualifications :

Strong background in molecular biology.

Experience with ASO or RNA-targeted therapeutics, ideally combined with high-throughput screening.

Experience developing and validating quantitative molecular assays, such as Taq Man 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 is highly desirable.

What You Will Do Design and execute cell-based ASO screening and therapeutic evaluation workflows across concurrent patient cases. Derive, characterize, and bank patient-derived cellular models to support screening and functional validation. Interpret assays to distinguish biology-driven effects from technical artifacts and use that understanding to refine assay design and troubleshoot across diverse genomic contexts.

Design and validate quantitative molecular assays (RT-qPCR, ddPCR) and interpret biochemical and imaging-based readouts of therapeutic efficacy. Manage parallel, multi-step experimental workflows across cases. Collaborate closely with genomics, molecular biology, and clinical translation teams to keep cases moving through the pipeline. Maintain rigorous documentation, data integrity, and quality control standards consistent with a translational laboratory environment.

Support day-to-day lab operations, including supply management and equipment upkeep. Contribute positively to a culture of shared responsibility, mutual support, and collective scientific progress.

Prior experience with Janus liquid handlers is a plus.

Minimum Education and/or Training:

Bachelor's degree in relevant scientific area, such as genomics, molecular biology, cell biology, is required.

Prior experience with Janus liquid handlers is a plus.

Scientist

Minimum Experience:

Eight (8) years of relevant work experience is required

Six (6) years of relevant work experience is required with a Master's degree

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