Scientist, Rare Disease Biology
Core
Investigate disease mechanisms and evaluate novel therapeutic targets for rare diseases using data-driven approaches and experimental studies.
Role type
Senior IC disease biologist (rare disease)
Builds
Innovative therapies for rare diseases
Domain
Biotechnology / Rare Disease Biology
Required skills
[PhD in Immunology/Cell Biology/Molecular Biology/Pharmacology/Biomedical Sciences OR Master's + 2 yrs research OR Bachelor's + 4 yrs research], in vitro/ex vivo study design, data analysis, mechanistic insight generation, scientific communication, collaboration in multidisciplinary teams
Preferred skills
Gout therapeutic area experience, enzyme biology/enzyme replacement therapies, cell-based/molecular/biochemical experiment troubleshooting, disease-relevant cellular systems (primary cells/patient-derived), computational/bioinformatics integration, AI-assisted tools, biotech/pharma research experience
Technologies
[None explicitly listed beyond general 'computational tools' and 'AI-assisted tools']
Responsibilities
Plan, design, execute, analyze, and interpret in vitro and ex vivo studies to elucidate disease mechanisms; Generate reproducible experimental data to support target identification and validation; Integrate experimental findings with human genetics, clinical observations, and literature to develop mechanistic insights; Apply computational tools to evaluate datasets and support target prioritization; Contribute to innovative experimental approaches; Collaborate with disease biologists, computational scientists, and translational researchers; Present scientific findings to project teams and governance forums; Maintain accurate documentation and contribute to knowledge-sharing efforts
Seniority
Senior, hands-on IC