Scientist, Rare Disease Biology
Amgen
Summary
In this vital role you will contribute to the discovery and advancement of innovative therapies for rare diseases by serving as a core member of multidisciplinary disease-focused research teams. You will apply experimental and data-driven approaches to investigate disease mechanisms, evaluate novel therapeutic targets, and generate insights that inform portfolio decisions. This role offers a unique opportunity to establish and leverage advanced human cellular disease models, including iPSC-based systems, to support discovery efforts across rare diseases.
Job description
What you will do
- Design, execute, analyze, and interpret in vitro and ex vivo studies to investigate disease mechanisms and evaluate therapeutic hypotheses.
- Generate high-quality experimental data to support target identification, validation, and progression of rare disease research programs.
- Investigate molecular and cellular mechanisms associated with transcriptional dysregulation, protein aggregation, cellular stress responses, and impairment of neurological and neuromuscular processes.
- Establish scalable experimental platforms that can support multiple rare disease programs.
- Integrate experimental findings with published literature, human genetics, clinical observations, and other data sources to develop mechanistic insights and inform scientific decision-making.
- Serve as an active member of disease-focused research pods, collaborating closely with disease biologists, computational scientists, translational researchers, and partner functions.
- Participate in scientific discussions, target reviews, and portfolio evaluations by contributing data-driven insights and recommendations.
- Build and maintain productive relationships with internal and external collaborators, including academic partners, foundations, and scientific experts when appropriate.
- Present scientific findings and recommendations to project teams, governance forums, and departmental meetings.
- Maintain accurate documentation and contribute to organizational knowledge-sharing efforts.
- Embrace and contribute to a culture of scientific curiosity, collaboration, continuous learning, and operational excellence.
Basic Qualifications
- PhD in Immunology, Neurology, Cell Biology, Molecular Biology, Pharmacology, Biomedical Sciences, or related discipline
- OR Master's degree and 3 years of scientific research experience
- OR Bachelor's degree and 5 years of scientific research experience
Preferred Qualifications
- Experience in neurobiology, genetic disease biology, or related therapeutic areas.
- Experience studying mechanisms associated with protein aggregation, RNA toxicity, gene silencing, cellular stress responses, or neurological and neuromuscular disease processes.
- Demonstrated expertise developing, maintaining, and utilizing iPSC-derived cellular models.
- Experience with neuronal differentiation, glial cell systems, organoid models, or other advanced human cellular systems.
- Experience working with patient-derived cellular models and genetically engineered cellular systems.
- Familiarity with CRISPR-based approaches, gene editing, or functional genomics technologies is desirable.
- Scientific curiosity and enthusiasm for exploring emerging disease areas and technologies.
- Demonstrated ability to combine experimental and computational approaches to scientific problem-solving.
- Experience working effectively within highly collaborative and matrixed research environments.
- Ability to balance scientific rigor with urgency and adaptability in a dynamic research environment.
- Demonstrated commitment to teamwork, knowledge sharing, and continuous improvement.
- Familiarity with AI-enabled scientific tools, image analysis workflows, computational biology approaches, or advanced data analytics.
Requirements
- PhD in Immunology, Neurology, Cell Biology, Molecular Biology, Pharmacology, Biomedical Sciences, or related discipline
- OR Master's degree and 3 years of scientific research experience
- OR Bachelor's degree and 5 years of scientific research experience
- Experience in neurobiology, genetic disease biology, or related therapeutic areas
- Experience studying mechanisms associated with protein aggregation, RNA toxicity, gene silencing, cellular stress responses, or neurological and neuromuscular disease processes
- Demonstrated expertise developing, maintaining, and utilizing iPSC-derived cellular models
- Experience with neuronal differentiation, glial cell systems, organoid models, or other advanced human cellular systems
- Experience working with patient-derived cellular models and genetically engineered cellular systems
- Familiarity with CRISPR-based approaches, gene editing, or functional genomics technologies
- Demonstrated ability to combine experimental and computational approaches to scientific problem-solving
- Experience working effectively within highly collaborative and matrixed research environments
- Ability to balance scientific rigor with urgency and adaptability in a dynamic research environment
- Demonstrated commitment to teamwork, knowledge sharing, and continuous improvement
- Familiarity with AI-enabled scientific tools, image analysis workflows, computational biology approaches, or advanced data analytics
Benefits
- Health insurance
- Dental coverage
- Vision coverage
- Life insurance
- Disability insurance
- Retirement and savings plan
- Flexible spending accounts
- Annual bonus program
- Stock-based long-term incentives
- Paid time off
Company overview
Amgen Inc. is a leading American multinational biopharmaceutical company headquartered in Thousand Oaks, California, founded in 1980. As one of the world's largest independent biotechnology companies, Amgen specializes in discovering, developing, manufacturing, and delivering innovative human therapeutics. The company focuses on six key therapeutic areas: inflammation, oncology/hematology, bone health, cardiovascular disease, nephrology, and neuroscience. Amgen's portfolio includes blockbuster drugs such as Enbrel for autoimmune diseases, Prolia and XGEVA for osteoporosis, Repatha for cholesterol management, Otezla for psoriasis, and Neulasta for chemotherapy-induced neutropenia. With approximately 28,000 employees worldwide, Amgen operates as a component of both the Dow Jones Industrial Average and Nasdaq-100 indices. The company maintains a robust research and development pipeline with numerous clinical programs across various stages, leveraging cutting-edge biotechnology and human genetic data to address some of the world's most challenging diseases. Amgen's manufacturing capabilities span multiple global locations, and it distributes products through pharmaceutical wholesale distributors and direct-to-consumer channels.