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.

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: Clinical Strategy and Leadership

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)

Clinical Development Execution

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
Enable development of clinically meaningful endpoints supporting POC and regulatory approval
Ensure studies are designed to maximize clinical, pharmacologic, and mechanistic insights.

Cross-Functional and External Engagement

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
Act as a key matrix clinical leader, ensuring alignment across cross-functional stakeholders.

Translational and Biomarker Strategy

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, response biomarkers, and endpoints supporting approval pathways
Ensure programs are positioned to generate deep mechanistic and translational insights during early clinical studies.

Portfolio Talent and Organizational Development

Mentor and develop junior colleagues across Early Clinical Development and Biomarkers functions.
Contribute to building organizational capability in early clinical strategy and execution.
Foster a culture of scientific rigor, collaboration, and continuous learning within the early development organization.

Business Development Support

Serve as a clinical expert to support internal program prioritization and external business development opportunities.
Provide scientific and clinical assessments of new assets, including risks, differentiation, and development feasibility.
Deliver recommendations to senior leadership to inform decision-making on new project 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)
Phase 2 trials and clinical proof-of-concept generation
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.

Workstyle:

This is a virtual position unless you live within 100 miles of our Headquarters in San Rafael, CA (zip: 94901). If so, the role changes to hybrid requiring an onsite presence 2 times per week.

Note: 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.

Equal Opportunity Employer/Veterans/Disabled

An Equal Opportunity Employer. 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.

Early Clinical Development jobs

Medical Director, Early Clinical Development

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