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.

Education/Skills/Experience 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.
• Strong understanding of clinical trial design and execution, including endpoint selection, study population definition, natural history considerations, feasibility, recruitment challenges, safety monitoring, and interpretation of early clinical data.
• Sound understanding of biomarker strategy and implementation, including translational rationale, assay readiness, biospecimen collection, laboratory/vendor interface, data quality, and integration of biomarker results into clinical decision making.
• Experience contributing to regulatory submissions and interactions with health authorities; working knowledge of GCP, ICH guidelines, pharmacovigilance expectations, and ethical conduct of clinical research.
• Ability to lead cross-functional teams and communicate clearly with scientific, clinical, operational, regulatory, executive, and external audiences.
• Excellent judgment, attention to detail, organizational skills, and ability to balance strategic leadership with hands-on execution in a fast-paced biotechnology environment.
• Commitment to advancing therapies for patients with serious neurological and orphan diseases.

Preferred Experience:

• Experience with genetically targeted, RNA-based, small-molecule, or precision-medicine approaches.
• Familiarity with global rare disease development pathways, expedited regulatory designations, patient-reported outcomes, caregiver-reported outcomes, functional endpoints, and natural history studies.
• Prior involvement in advisory boards, investigator meetings, data review committees, or regulatory meetings for neuroscience or orphan disease programs.