Senior Medical Director, Early Clinical Development

Overview

In this role you will lead the early clinical development strategy for one or more assets at BioMarin, guiding end-to-end translational and clinical work from first-in-human studies through proof of concept. You will partner with cross-functional teams to shape development plans, safety and efficacy assessment, and regulatory strategy, while mentoring future leaders in the ECD organization. Your work will influence portfolio decisions and help translate scientific insights into meaningful patient outcomes. This is a high-impact, cross-disciplinary leadership role at the forefront of rare disease therapeutics.

Responsibilities

• Develop and own clinical development plans for assigned assets, emphasizing first-in-human proof of mechanism and clinical proof of concept.
• Serve as clinical development team leader (CDTL) from research through human POC studies.
• Provide scientific and clinical leadership across multiple assets, balancing priorities and resources.
• Inform development and governance decisions with safety, efficacy, and benefit-risk considerations.
• Lead pre-IND, IND, and other regulatory submissions and collaborate on innovative trial designs.
• Design and execute natural history studies to deepen understanding of patient populations and enable robust endpoints.
• Coordinate with Biomarker and Clinical Pharmacology for patient selection, pharmacodynamics, and response biomarkers.
• Provide matrixed leadership and mentorship within the ECD team and contribute to recruitment and capability building.
• Support business development by acting as clinical expert for internal opportunities and external assessments.

Key requirements

• leadership
• mentorship
• cross-functional collaboration
• translational clinical research
• early-phase trial design (Phase 1/2)
• regulatory submissions (pre-IND, IND)

…