Postdoctoral Scientist – Gene Therapy Biodistribution & Molecular Biology (CNS)

• Independently design and execute CNS gene therapy–focused biodistribution studies, applying sound experimental design and Design of Experiments (DoE/DOR) principles.
  • Diagnose and troubleshoot complex technical challenges related to vector detection, tissue processing, assay performance, or data variability.

• Apply strong foundational knowledge of CNS biology, molecular biology, and biochemistry to interpret biodistribution and expression data in a therapeutic context.
  • Integrate biodistribution results with an understanding of pharmacokinetics and pharmacodynamics (PK/PD) to inform dose selection, delivery route optimization, and therapeutic index assessment for viral and non-viral gene therapies.

• Independently interface with external CROs to design, execute, and oversee in vivo gene therapy biodistribution studies, including work in higher vertebrate CNS models.
  • Provide scientific oversight for tissue collection, sample processing, and downstream molecular analyses conducted internally or externally.

• Apply best practices for managing and analyzing large, multi-dimensional biodistribution datasets generated from CNS gene therapy studies.
  • Perform statistical analysis and generate clear, insightful data visualizations to support project teams and decision-making forums.

Requirements

• PhD in Molecular Biology, Biochemistry, Pharmacology, Neuroscience, Bioengineering, or a closely related life sciences discipline.
  • Demonstrated in-depth experience in AAV biodistribution analysis and/or molecular bioanalytical assay development.
• Strong hands-on expertise in PCR/qPCR/ddPCR, ELISA, IHC, NGS, and/or related molecular biology techniques.
  • Solid understanding of CNS gene therapy delivery, molecular mechanisms of transgene expression, and/or tissue-specific biology.
• Proven ability to independently design experiments, troubleshoot technically challenging assays, and maintain rigorous scientific documentation.
  • Highly motivated, proactive, and able to thrive in a collaborative, team-based research environment.

Preferences

• Knowledge of pharmacokinetics and pharmacodynamics as applied to gene therapy biodistribution and expression.

• Spatial transcriptomics / RNAscope as a preferred qualification for CNS transgene expression mapping.

• Direct experience working with CNS tissues and CNS-relevant animal models for gene therapy research.

• Experience integrating biodistribution data with imaging, functional, or efficacy readouts.

• Experience collaborating with CROs and supporting in vivo gene therapy studies.

• Familiarity with FDA (2020) long-term follow-up guidance and EMA biodistribution reflection papers as preferred qualifications.

• Knowledge of current FDA guidance on intrathecal/systemic AAV safety signals.