Medical Director, Early Clinical Development

BioMarin is a leading rare disease biotechnology company focused on genetically defined conditions. Guided by our purpose to develop medicines that make a profound impact on people’s lives, our global teams have delivered a portfolio of therapies since our founding in 1997. Our revolutionary treatments for conditions like achondroplasia, PKU, CLN2, and various forms of MPS offer new possibilities for patients and families who previously had few, if any, available options. More recently, with the close of the Amicus acquisition, our portfolio has expanded to include therapies for Fabry disease and Pompe disease.

Our success comes from our unwavering commitment to excellence, our deep understanding of patient needs, our scientific expertise, and our world-class manufacturing capabilities. At the heart of BioMarin is a dedicated team of the brightest minds in the industry working together to deliver innovative therapies to patients and families around the world.

BioMarin’s Early Clinical Development team is seeking an experienced Medical Director to serve as a key matrixed clinical leader focused on the design and execution of highly informative Phase 1 and Phase 2 trials. This role is central to translating early scientific insights into clinical strategies that maximise learning in first-in-human and early patient studies, enabling efficient progression to proof of concept.

The Medical Director will develop early clinical strategies and development plans that establish the foundation for full program advancement, while leading protocol design, clinical oversight of execution, and medical monitoring to deliver high-quality, insight-rich trials. The role requires close collaboration across Research, Translational Sciences, and Development, along with external engagement in scientific, regulatory, and advisory settings.

A strong focus is placed on embedding translational science, biomarkers, and experimental medicine approaches into clinical programs to deepen mechanistic understanding and inform development decisions. In parallel, the Medical Director mentors colleagues and contributes to organisational capability building.

Additionally, the role supports evaluation of internal and external opportunities by providing clinical and scientific insight for business development decision-making. Through a combination of individual contribution and cross-functional leadership, this position has broad impact across BioMarin’s early clinical portfolio.

Key Responsibilities:

• Develop and lead clinical development plans that establish the strategic foundation for full clinical development of assets.
• Define strategies to demonstrate proof of mechanism in early clinical trials and enable proof of concept in subsequent studies.
• Translate overarching program strategy into actionable clinical plans and study designs.
• Lead clinical strategy contributions to pre-IND, IND, and other regulatory submissions.
• Provide clinical leadership and decision-making within matrixed program teams.
• Lead clinical efforts for 1 to 2 assets (in general).
• Lead the design and execution of Phase 1 and Phase 2 clinical study protocols and associated trial documentation.
• Serve as Medical Monitor for selected studies, ensuring appropriate oversight of safety, study conduct, and data interpretation.
• Design and execute natural history studies to enhance understanding of target patient populations.
• Partner closely with Early Clinical Development, Biomarkers, Clinical Pharmacology, Preclinical Safety, Clinical Safety, Regulatory Affairs, and Clinical Operations to align on development strategy and execution.
• Represent Early Clinical Development in Health Authority interactions, scientific advisory boards, and academic and external meetings.
• Integrate translational science and experimental medicine approaches into early clinical development programs.
• Mentor and develop junior colleagues across Early Clinical Development and Biomarkers functions.
• Serve as a clinical expert to support internal program prioritisation and external business development opportunities.

Qualifications:

• MD or MD/PhD required.
• 5 years of industry experience in Phase 1/2 clinical development, with demonstrated ownership of at least two assets from IND through proof of concept.
• Significant experience contributing to IND submissions.
• Demonstrated expertise in Phase 1 clinical trials, Phase 2 trials, and clinical proof-of-concept generation.
• Strong ability to operate effectively in a matrixed, fast-paced environment.
• Demonstrated collaboration and communication skills, including engagement with internal and external stakeholders.
• Intellectual curiosity and interest in addressing complex development challenges.

This description is not intended to be all-inclusive, or a limitation of the duties of the position. It is intended to describe the general nature of the job that may include other duties as assumed or assigned.