Scientist, CNS Biology Research
Core
Identify and validate novel siRNA drug targets for neurodegenerative diseases using human genetics, literature, and genomic approaches; design and execute proof-of-concept experiments in animal and iPSC models.
Role type
Senior CNS Biology Research Scientist (Neurodegenerative Therapeutics)
Builds
Novel siRNA drug candidates for neurodegenerative and neuromuscular programs
Domain
Biotechnology / CNS Biology / Neurodegenerative Diseases
Deliverable
production ML models | product features | dashboards & analysis | research | client delivery | infrastructure | physical/clinical work
Required skills
siRNA drug target identification, human genetics analysis, genomic approaches, in vitro assay design (RNA/protein quantification, high-content imaging, functional assays), single-cell RNA sequencing, in vivo mammalian disease model expertise, intracerebroventricular (ICV) injections, necropsy, behavioral analysis, ex vivo tissue processing, molecular and histological assessments
Preferred skills
ALS expertise, postdoc experience, lead discovery oversight, CTA/IND filing support, CRO/academic collaborator management, mentoring junior scientists
Technologies
iPSC models, high-content imaging systems, single-cell RNA sequencing platforms, animal models
Responsibilities
Identify new siRNA drug-targets based on human genetics and genomic approaches; Design and execute proof of concept experiments in animal and in vitro iPSC disease models; Design studies, coordinate activities, and lead from the bench; Serve as Biology Lead on select neurodegenerative/neuromuscular programs; Work with multifunctional teams to shepherd a drug candidate through lead selection and development; Support programs through CTA/IND filing processes; Manage relationships with contract research organizations (CROs) and/or academic collaborators; Mentor junior scientists
Seniority
Senior, hands-on IC with mentorship responsibilities