Clinical Scientist The Clinical Scientist will provide strategic and hands-on medical leadership for clinical development programs in neuroscience and orphan diseases. This individual will help shape clinical development strategy from translational readiness through early and mid-stage clinical execution, integrating disease biology, patient population definition, study design, endpoint selection, safety monitoring, and evidence generation. The role requires a strong understanding of biomarker strategy, including patient selection, target engagement, pharmacodynamic and proof-of-mechanism readouts, biospecimen planning, and interpretation of translational data.

This position will work closely with cross-functional teams and external investigators to advance high-quality, scientifically rigorous clinical programs aligned with regulatory, patient, and business needs.

Responsibilities

: Drive the design and execution of early and mid-stage clinical studies, including objectives, endpoints, eligibility criteria, dose-escalation or dose-selection logic, safety monitoring, data review plans, and decision criteria. Integrate biomarker strategy into clinical development plans, including patient identification and stratification, target engagement, pharmacodynamic markers, disease progression markers, exploratory efficacy markers, imaging, fluid biomarkers, genetics, and other precision-medicine approaches. Partner with translational science, biometrics, clinical pharmacology, regulatory, clinical operations, data management, pharmacovigilance, CMC, program management, and commercial/strategy colleagues to ensure coherent program plans and timely execution.

Provide medical oversight for clinical studies, including protocol implementation, eligibility and medical queries, safety data review, emerging risk assessment, data monitoring committee interactions, and interpretation of clinical and biomarker findings. Lead or contribute to key clinical and regulatory documents, including clinical development plans, protocols, investigator brochures, informed consent forms, safety management plans, statistical analysis plan input, clinical study reports, IND/CTA submissions, annual reports, and briefing documents. Engage with investigators, key opinion leaders, patient advocacy groups, academic collaborators, CROs, central laboratories, and other external partners to inform study design, enrollment feasibility, scientific communication, and operational success.

Represent the clinical development function at program team meetings, governance discussions, advisory boards, investigator meetings, regulatory interactions, and scientific conferences. Review and synthesize emerging literature, competitive intelligence, natural history data, registries, real-world evidence, and regulatory precedent relevant to neuroscience and rare/orphan disease development. Support publication, congress, and scientific communication strategy in collaboration with medical affairs, translational science, and program leadership.

Mentor clinical scientists and cross-functional colleagues, promoting rigorous clinical thinking, high-quality documentation, strong scientific judgment, and patient-centered decision making.

Requirements

: MD or equivalent medical degree required; board certification or advanced training in neurology, psychiatry, neurogenetics, pediatrics, clinical genetics, or another relevant specialty preferred. MD/PhD is a plus. (title commensurate with experience) Demonstrated expertise in neuroscience drug development; experience in rare/orphan diseases, genetically defined diseases, neurodegenerative disease, neuromuscular disease, movement disorders, or pediatric neurological disorders is highly desirable.