Scientist, Rare Disease Biology

BioSpace; Thousand Oaks (CA); On-site; USD 130,000 - 170,000; Full time

Job summary

Amgen is seeking a Scientist, Rare Disease Biology in Thousand Oaks, CA to advance discovery of therapies for rare diseases. You will design and execute in vitro/ex vivo studies, generate data to support target identification, and collaborate with disease biologists, computational scientists, and translational teams.

Strong emphasis on iPSC-derived models and gene-editing technologies. The role requires a PhD or equivalent with experience in neurobiology, cellular models, and cross-functional

Qualifications

• PhD or equivalent higher degree with a strong research background in relevant biology.
• Experience in neuroscience or related therapeutic areas is required.
• Experience with human cellular models and advanced in vitro methods.

Responsibilities

• Contribute to discovery and advancement of therapies for rare diseases as a core team member.
• Design, execute, and interpret in vitro and ex vivo studies to test therapeutic hypotheses.
• Generate high-quality data to support target identification and progression of programs.
• Investigate molecular/cellular mechanisms including transcriptional dysregulation and protein aggregation.
• Establish scalable experimental platforms supporting multiple programs.
• Integrate experimental findings with literature, genetics, and clinical observations.
• Collaborate with scientists across biology, computation, translational research and partners.

Skills

Neurobiology experience

Genetic disease biology

iPSC-derived models

Neuronal differentiation

Patient-derived cellular models

CRISPR/gene editing

Computational biology

Collaborative research

Education

PhD in Immunology, Neurology, Cell Biology, Molecular Biology, Pharmacology, Biomedical Sciences, or related discipline

Master's degree + 3 years of scientific research experience

Bachelor's degree + 5 years of scientific research experience

Job description

Join Amgens Mission of Serving Patients

At Amgen, if you feel like youre part of something bigger, its because you are. Our shared missionto serve patients living with serious illnessesdrives all that we do.

Since 1980, weve helped pioneer the world of biotech in our fight against the worlds toughest diseases. With our focus on four therapeutic areas Oncology, Inflammation, General Medicine, and Rare Disease we reach millions of patients each year. Amgen is advancing a broad and deep pipeline of medicines to treat cancer, heart disease, inflammatory conditions, rare diseases, and obesity and obesity-related conditions. As a member of the Amgen team, youll help make a lasting impact on the lives of patients as we research, manufacture, and deliver innovative medicines to help people live longer, fuller happier lives.

Our award-winning culture is collaborative, innovative, and science based. If you have a passion for challenges and the opportunities that lay within them, youll thrive as part of the Amgen team. Join us and transform the lives of patients while transforming your career.

Position: Scientist, Rare Disease Biology

What you will do

• Lets do this. Lets change the world. 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.
• 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.

What we expect of you

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.

What you can expect of us

As we work to develop treatments that take care of others, we also work to care for your professional and personal growth and well-being. From our competitive benefits to our collaborative culture, well support your journey every step of the way.

The expected annual salary range for this role in the U.S. (excluding Puerto Rico) is posted. Actual salary will vary based on several factors including but not limited to, relevant skills, experience, and qualifications.

In Addition To The Base Salary, Amgen Offers a Total Rewards Plan, Based On Eligibility, Comprising Of Health And Welfare Plans For Staff And Eligible Dependents, Financial Plans With Opportunities To Save Towards Retirement Or Other Goals, Work/life Balance, And Career Development Opportunities That May Include

• A comprehensive employee benefits package, including a Retirement and Savings Plan with generous company contributions, group medical, dental and vision coverage, life and disability insurance, and flexible spending accounts
• A discretionary annual bonus program, or for field sales representatives, a sales-based incentive plan
• Stock-based long-term incentives
• Award-winning time-off plans
• Flexible work models where possible. Refer to the Work Location Type in the job posting to see if this applies.

careers.amgen.com