Overview As a Scientist in Rare Disease Biology, you contribute to discovering and advancing therapies for rare diseases within cross-functional teams. You will leverage experimental and data-driven methods to uncover disease mechanisms, validate targets, and inform portfolio decisions. The role focuses on pioneering human cellular models, including iPSC-based systems, to support discovery across multiple rare disease programs. You will collaborate, share findings, and help scale experimental platforms while aligning with Amgen's patient-centric mission.
Compensation / Benefits- comprehensive benefits package
- retirement and savings plan with company contributions
- group medical, dental, and vision coverage
- life and disability insurance
- flexible spending accounts
- stock-based long-term incentives
Responsibilities- Design, execute, and interpret in vitro and ex vivo studies to investigate disease mechanisms and test hypotheses
- Generate high-quality data to support target identification, validation, and progression of rare disease programs
- Investigate molecular and cellular mechanisms linked to transcriptional dysregulation, protein aggregation, cellular stress, and neurological/neuromuscular processes
- Establish scalable experimental platforms to support multiple rare disease programs
- Integrate findings with literature, human genetics, clinical observations, and other data sources to derive mechanistic insights
- Collaborate with disease biologists, computational scientists, translational researchers, and partner functions
- Participate in scientific discussions, target reviews, and portfolio evaluations with data-driven recommendations
- Build and maintain relationships with internal and external collaborators, including academic partners and experts
- Present findings to project teams and governance forums; maintain thorough documentation and knowledge sharing
- Contribute to a culture of scientific curiosity, collaboration, learning, and operational excellence
Key requirements- PhD in Immunology, Neurology, Cell Biology, Molecular Biology, Pharmacology, Biomedical Sciences, or related discipline; OR Master's degree with 3 years of research experience; OR Bachelor's with 5 years of research experience
- Experience in neurobiology, genetic disease biology, or related therapeutic areas
- Experience studying mechanisms like protein aggregation, RNA toxicity, gene silencing, cellular stress responses, or neurological/neuromuscular processes
- Expertise with iPSC-derived cellular models and neuronal differentiation, glial cell systems, or organoid models
- Experience with patient-derived cellular models and genetically engineered systems
- Familiarity with CRISPR-based approaches or functional genomics; familiarity with AI-enabled tools and computational biology is a plus
- Strong collaboration skills in matrixed research environments; ability to balance rigor with urgency
- collaboration
- scientific curiosity
- communication
- iPSC-derived cellular models
- neuronal differentiation and organoid models
- CRISPR-based approaches and functional genomics
