Who We Are
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 (the most common form of dwarfism), PKU (phenylketonuria), CLN2, a form of Batten disease, and a number of forms of MPS (mucopolysaccharidosis) 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, expanding our ability to reach more people living with rare genetic conditions.

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.

About Worldwide Research and Development
From research and discovery to post-market clinical development, our R&D engine involves all bench and clinical research and the associated groups that support those endeavors. Our teams work on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with rare diseases.

Medical Director, Early Clinical Development
Location: London (hybrid role 2 days per week onsite)
Closing date: 30 June 2026

Overview
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 maximize learning in first-in-human and early patient studies, enabling efficient progression to proof of concept.

Leveraging experiences in translational clinical research and early‑phase trial design, 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. The role partners closely with colleagues in Early Clinical Development, Biomarkers, Clinical Pharmacology, Preclinical Safety, Clinical Safety, Regulatory, and Clinical Operations to ensure BioMarin’s assets are optimally positioned to generate high‑value insights that inform downstream development and regulatory strategy.

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 organizational 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 and enable development of clinically meaningful endpoints.
• Ensure studies are designed to maximize clinical, pharmacologic, and mechanistic insights.
• 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.
• Partner with Biomarker teams to define and implement strategies for patient selection, pharmacodynamic assessments, and response biomarkers.
• Mentor and develop junior colleagues across Early Clinical Development and Biomarkers functions.
• Serve as a clinical expert to support internal program prioritization 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 (industry, CRO, or Phase 1 units) and Phase 2 trials.
• Experience leading cross-functional clinical development teams and developing clinical development strategies.
• 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.
• Experience supporting business development evaluations is a strong plus.

Equal Opportunity Employer/Veterans/Disabled. All qualified applicants will receive consideration for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability.