Position Summary
The
Senior Scientist, Molecular Biodistribution leads and executes biodistribution and gene expression assessment strategies. This role is responsible for designing fit-for-purpose molecular assays, generating high-quality tissue distribution and transgene expression data, and interpreting results to inform candidate selection, dose strategy, safety assessment, and support regulatory submissions.
The role partners closely with Product Development, Translation Medicine, Tech Ops, and Research and Development among other functions. The successful candidate combines deep hands-on technical expertise with strong scientific judgment in assay development, data analysis and interpretation, and cross-functional communication.
This is an onsite position in RTP, NC and will report to the Director, Product Development Cores.
Job ResponsibilitiesBiodistribution Strategy and Study Support
- Design and support biodistribution and gene expression assessment plans for genetic medicine programs
- Define fit-for-purpose tissue collection, processing, and analytical strategies for biodistribution, persistence, and transgene expression assessments
- Contribute to dose selection, route-of-administration evaluation, and tissue targeting assessments for AAV and other genetic medicine platforms
- Evaluate data in the context of mechanism of action, tissue tropism, vector performance, and nonclinical efficacy
Assay Development and Execution
- Develop, optimize, and troubleshoot molecular assays to quantify vector genomes, transgene expression, and related biomarkers in tissues and biofluids
- Apply and oversee quantitative PCR-based and related molecular methods, including qPCR, ddPCR, RT-qPCR, and other nucleic acid quantification approaches
- Establish assay controls, standards, acceptance criteria, normalization strategies, and data quality practices appropriate for regulated and non-regulated studies
- Collaborate with internal stakeholders external partners to ensure reliable assay execution, method transfer, and sample analysis
- Drive continuous improvement in assay sensitivity, specificity, reproducibility, and operational scalability
Data Analysis and Interpretation
- Analyze and interpret biodistribution and gene expression datasets to support program decisions in research and development
- Assess vector genome copy number, transgene RNA expression, and relationships across dose, tissue, timepoint, and platform
- Identify technical or biological sources of variability and recommend follow-up experiments or analytical refinements
- Summarize findings in clear scientific narratives for study reports, governance discussions, and regulatory documentation
- Present results and recommendations to cross-functional teams and senior stakeholders in genetic medicine
Minimum Requirements - PhD in molecular biology, pharmacology, bioanalytical sciences, genetics, biomedical sciences, or a related discipline with 5+ years' relevant work experience; or Master's degree in molecular biology, pharmacology, bioanalytical sciences, genetics, biomedical sciences, or a related discipline with 10+ years of relevant work experience
- 5+ years of relevant experience in biotech, pharma, CRO, or academic translational research settings supporting genetic medicine or related modalities
- Direct experience designing, executing, and interpreting biodistribution and gene expression studies for gene therapy, especially AAV-based programs
- Strong hands-on experience with nucleic acid extraction from tissues and biofluids and with quantitative molecular assay platforms such as ddPCR, qPCR, and RT-qPCR
- Experience supporting preclinical drug development, including non-GLP and/or GLP studies, assay qualification, and regulated documentation
- Demonstrated understanding of nonclinical development considerations for genetic medicines, including tissue tropism, vector persistence, transgene expression, and safety assessment
Preferred Education, Experience and Skills- Experience with AAV gene therapy development across multiple serotypes, capsids, promoters, or routes of administration
- Experience contributing to regulatory submissions for gene therapy or other advanced therapeutic modalities
- Knowledge of statistical and data visualization approaches used for complex multidimensional nonclinical datasets
