Scientist, Rare Disease Biology

Role: Scientist in Rare Disease Biology focused on discovering and advancing therapies for rare diseases through disease mechanism investigation and target validation. Responsibilities: design and perform in vitro/ex vivo studies, utilize human cellular models—including iPSC-based systems—to generate mechanistic insights, collaborate across multidisciplinary teams, and contribute to portfolio decisions. Qualifications: PhD in immunology, neurology, cell/molecular biology, or related fields; or Master's with 3+ years or Bachelor's with 5+ years of research experience. Preferred skills include expertise in neurobiology, protein aggregation, RNA toxicity, gene editing (e.g., CRISPR), and experience with patient-derived cellular models, organoids, or advanced human cellular systems. High-Value: focus on rare diseases, neurodegenerative and neuromuscular conditions, advanced cellular models, data-driven and mechanistic research, collaborative environment, and development of scalable experimental platforms. Work setup and travel details are not specified.