Scientist, Rare Disease Biology
Core
Investigate disease mechanisms and evaluate therapeutic hypotheses for rare diseases using experimental and data-driven approaches, including advanced human cellular models.
Role type
Scientist, Rare Disease Biology
Builds
Innovative therapies for rare diseases
Domain
Biotechnology / Rare Disease Biology
Deliverable
production ML models | product features | dashboards & analysis | research | client delivery | infrastructure | physical/clinical work
Required skills
[PhD in Immunology/Neurology/Cell Biology/Molecular Biology/Pharmacology/Biomedical Sciences OR Master's + 3 years research OR Bachelor's + 5 years research], experimental design, in vitro/ex vivo study execution, data analysis, mechanistic insight generation, iPSC-based system utilization, collaboration in multidisciplinary teams
Preferred skills
neurobiology, genetic disease biology, protein aggregation mechanisms, RNA toxicity, gene silencing, cellular stress responses, neurological/neuromuscular disease processes, iPSC-derived cellular models, neuronal differentiation, glial cell systems, organoid models, patient-derived cellular models, genetically engineered cellular systems, CRISPR-based approaches, functional genomics, AI-enabled scientific tools, computational biology, advanced data analytics
Technologies
iPSC-based systems, CRISPR, organoid models, AI-enabled scientific tools
Responsibilities
Design and execute in vitro and ex vivo studies to investigate disease mechanisms; Generate high-quality experimental data to support target identification and validation; Investigate molecular and cellular mechanisms of transcriptional dysregulation and protein aggregation; Establish scalable experimental platforms for multiple rare disease programs; Integrate experimental findings with literature and clinical data to inform decision-making; Present scientific findings to project teams and governance forums
Seniority
Mid-Senior, hands-on IC
