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Role Description

This role offers a unique chance to broaden your experience, expand your knowledge, and contribute meaningfully to AskBio’s mission: to advance gene therapy and change the lives of patients around the world.

• Lead and execute the clinical development strategy for gene therapy programs across early- and late-stage development (Phase 1–3)
• Provide medical and scientific input into clinical trial design, protocol development, dose escalation strategies, and endpoint selection, particularly for CNS and movement disorders
• Oversee all clinical trials from initiation through completion, ensuring data integrity, patient safety, and regulatory compliance
• Collaborate with internal cross-functional teams and external partners, including CROs, investigators, vendors, and regulatory agencies
• Conduct clinical risk assessments and implement appropriate mitigation strategies, including safety monitoring and benefit–risk evaluations
• Contribute to regulatory interactions, including briefing documents, investigator brochures, and responses to health authority questions
• Present clinical data and development updates to senior management, governance committees, and external stakeholders
• Supervise, mentor, and support clinical team members and contribute to a high-performing development organization
• Establish and maintain oversight of clinical monitoring activities across studies in a highly regulated environment, ensuring compliance with ICH-GCP, regulatory requirements, and internal quality standards
• Author and review key clinical and regulatory documents, including clinical protocols, investigator brochures, clinical study reports, and regulatory submissions, ensuring scientific rigor, clarity, and compliance with applicable regulatory requirements and internal standards
• Drive innovation in clinical trial execution, particularly in rare disease, CNS, and gene therapy settings

Qualifications

• MD from an accredited U.S. medical school or equivalent international medical degree with 6+ years’ relevant work experience
• Completion of clinical training with demonstrated clinical experience; board certification or eligibility in Neurology or a closely related specialty
• Minimum of 3 years of industry experience or related experience in clinical research or clinical development within the pharmaceutical, biotechnology, medical device, CRO/CDMO, or closely related academic or translational research setting
• Direct experience contributing to the design and execution of clinical trials, including protocol development and clinical data review
• Demonstrated working knowledge of Good Clinical Practice (GCP), including clinical monitoring, safety oversight, and data quality standards
• Experience supporting inspection readiness activities, including preparation for or participation in FDA or other health authority inspections, audits, or internal quality reviews
• Ability to independently perform the core duties of a Medical Director in a regulated drug development environment
• Ability to provide medical oversight for FDA inspection readiness, including review of key trial documentation, monitoring outputs, and safety narratives
• Experience reviewing and interpreting monitoring reports, audit findings, protocol deviations, and CAPAs
• Strategic oversight of risk-based monitoring and trial quality management approaches
• Understanding of gene therapy–specific safety risks, including delayed adverse events and durability considerations requiring extended follow-up
• Strong analytical and problem-solving skills applied to clinical, safety, and compliance issues
• Excellent written and verbal communication skills, including interaction with senior leadership and regulatory agencies

Preferred Education, Experience and Skills

• Prior experience in CNS, neurology, or movement disorders clinical development
• Experience with gene therapy, cell therapy, or other advanced therapeutic modalities
• Experience supporting or leading early Phase development programs including Phase 1 and 2 and/or Phase 3 trials, including registrational strategies
• Experience with long-term follow-up (LTFU) studies, including post-treatment safety monitoring required for gene therapy products
• Direct experience partnering with CROs and CRAs on monitoring strategy, risk-based monitoring, and vendor oversight
• Experience working in rare disease or small patient population studies