About this role
Primary Responsibilities
- Lead a rapid, tiered cellular assay strategy for small-molecule lead optimization, including target engagement, pharmacodynamics, concentration-response, duration, selectivity, neuronal phenotype, stress, and tolerability
- Lead cellular development of a transferrin-based protein replacement project by establishing quantitative assays for construct uptake, internalization, intracellular trafficking, protein abundance and integrity, mitochondrial response, and disease-relevant phenotypic rescue
- Use orthogonal methods to distinguish surface-bound, internalized, degraded, and functionally delivered protein; compare constructs and define advancement criteria
- Establish fit-for-purpose neuronal culture models, including iPSC-derived neurons or glia where they add decision value, with appropriate banking and quality control
- Define assay controls, acceptance criteria, reproducibility standards, analysis plans, SOPs, sample tracking, and electronic documentation
- Translate data into concise recommendations to advance, modify, or discontinue assets
- Partner across protein engineering, chemistry, bioanalysis, DMPK, pathology, computational biology, and in vivo pharmacology; scientifically manage CROs and assay transfers
- Mentor junior scientists and build workflows that are reproducible across operators. Future scope may include ASO screening in partnership with external experts
Required Skills and Qualifications
- PhD in pharmacology, neuroscience, molecular biology, chemical biology, cell biology, or a related discipline, plus 3 to 6 years of biotechnology or pharmaceutical industry experience
- Demonstrated experience in cellular pharmacology, assay development, and iterative drug discovery or lead optimization
- Hands-on experience with neuronal cell culture models, preferably human iPSC-derived neurons or glia
- Expertise in concentration-response, time-course, target-engagement, pharmacodynamic, quantitative data-analysis, and troubleshooting studies
- Concrete examples of scientific ownership in which an assay, platform, or data package influenced a program decision
- Excellent communication skills and willingness to maintain a substantial hands-on laboratory presence
Preferred Skills and Qualifications
- Enzyme biology, epigenetics, or transcriptional pharmacology; therapeutic proteins or targeted-delivery constructs; cellular uptake, trafficking, or protein quantification; mitochondrial functional assays; high-content imaging or neuronal functional assays; transferrin receptor biology, BBB transport, or biodistribution; laboratory start-up, assay transfer, or CRO management
- Exposure to ASO screening or RNA pharmacology is beneficial
Compensation is dependent on qualifications and experience