Clinical Scientist

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.

About Skyhawk

Skyhawk is committed to discovering, developing and commercializing small molecule therapeutics that modify RNA expression. We use our novel SkySTAR® platform (Skyhawk Small molecule Therapeutics for Alternative splicing of RNA) to develop drug candidates directed toward targets for some of the world’s most intractable diseases including neurological conditions, cancer, and traditionally “undruggable” targets.