Role Summary
Senior Research Scientist to join the Disease Modeling Group. Develop engineered human cell models and integrate them into drug discovery workflows to support translational relevance and advance Vertexβs pipeline. Apply expertise in human cell biology, primary cell systems, iPSC technologies, and gene editing to develop robust in vitro disease models that support target validation, proof-of-concept studies, primary screening, selectivity and efficacy assessments, mechanism of action studies, and medicinal chemistry optimization. This is a full-time, onsite position in San Diego.
Responsibilities
- Design, generate, QC and validate engineered human cell lines; perform functional characterization of genetic edits and prepare documentation for internal review.
- Establish and maintain quality control criteria for different cell types and disease model systems.
- Contribute to the design and development of human in vitro disease models that recapitulate disease mechanisms and phenotypes.
- Perform and optimize primary cell culture, iPSC differentiation, transdifferentiation, and gene-editing workflows.
- Collaborate with cross-functional project teams to integrate disease models into compound screening, target validation, and mechanistic studies.
- Analyze datasets, prepare high-quality experimental summaries, and communicate findings to project teams and functional leadership.
- Maintain awareness of emerging technologies in gene editing, cell engineering, and in vitro disease modeling.
- Contribute to continuous improvement of methodologies, tools, and best practices within the group.
Knowledge and Skills
- Strong ability to analyze complex datasets, review scientific literature, and formulate hypotheses.
- Demonstrated expertise with human cell based systems for disease modeling and therapeutic development.
- Extensive hands-on experience establishing iPSC differentiation workflows across multiple cell lineages.
- Proficiency with CRISPR/Cas9 gene editing and its application in disease modeling.
- Hands-on competence with standard validation methods (RNAseq, qPCR/ddPCR, NGS, Western blot, immunostaining).
- Strong organizational, communication, and interpersonal skills.
- Ability to manage multiple high-impact projects in a fast paced environment, with excellent prioritization and execution.
- Demonstrated ability to work both independently and collaboratively within a matrixed organization.
- Strong publication record and evidence of contributions to drug discovery programs.
Required Qualifications
- Ph.D. in Cell Biology, Molecular Biology, Neuroscience, or related discipline and 2-5 years of relevant post-doctoral employment experience, or
- Masters Degree (or equivalent degree) in molecular biology, cell biology, biochemistry, or related disciplines and 5+ years of relevant employment experience, or
- Bachelors Degree (or equivalent degree) in molecular biology, cell biology, biochemistry, or related disciplines and 8+ years of relevant employment experience