Senior Medical Director, Early Clinical Development

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.

Senior Medical Director, Early Clinical Development

Remote - United Kingdom

Overview 

BioMarin’s Early Clinical Development (ECD) team is seeking an accomplished Senior Medical Director to serve as a senior clinical leader responsible for shaping and executing early clinical development strategies across one or more assets within the early pipeline. This role encompasses end‑to‑end clinical leadership from first‑in‑human studies through demonstration of clinical proof of concept, with accountability for scientific rigor, strategic clarity, and development decision‑making during the highest‑risk phases of development.

Leveraging deep expertise in translational clinical research and early‑phase trial design, the Senior Medical Director will lead integrated clinical development planning, design and oversee highly informative Phase 1 and Phase 2 studies, and guide key investment and governance decisions based on emerging safety, pharmacology, biomarker, and efficacy data. 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.

In addition to asset‑level leadership, the Senior Medical Director will contribute broader portfolio‑level clinical perspective across BioMarin’s early pipeline, support evaluation of new internal programs and external business development opportunities, and provide mentorship and leadership within the ECD organization. This position is expected to interact regularly with senior governance bodies, health authorities, and external scientific experts, and to represent BioMarin as a thought leader in early clinical development.

Key Responsibilities:

Clinical Strategy and Leadership 

• Develop clinical development plans that serve as the strategic foundation for full development of assigned assets, with emphasis on demonstrating proof of mechanism first‑in‑human and first‑in‑patient studies, and subsequent demonstration of clinical proof of concept.
• Serve as clinical development team leader (CDTL) for assets from the research phase through completion of human POC studies. 
• Provide scientific and clinical leadership across multiple assets, balancing strategic priorities and resource allocation. 
• Make recommendations at key development and governance decision points, incorporating safety, efficacy, and benefit‑risk considerations. 

• Lead clinical contributions to pre-IND, IND, and other stage‑appropriate regulatory submissions. 
• Collaborate with Research and Translational Science colleagues to integrate innovative experimental designs that inform clinical development strategy and regulatory filings.Serve as Medical Monitor on selected studies, as appropriate.

• Design and execute natural history studies to (1) deepen understanding of targeted patient populations and (2) enable development of endpoints that support efficient demonstration of POC and/or regulatory approval. 

• Partner with Biomarker and Clinical Pharmacology colleagues to ensure robust plans for patient selection, pharmacodynamic assessments, response biomarkers, and potential approval endpoints. 

• Provide matrixed leadership and mentorship to Early Clinical Development colleagues. 

• May directly manage and develop more junior Medical Directors. 

• Define capability needs for the ECD function and contribute to recruitment, training, and retention strategies. 

• Establish and evolve standard operating procedures, decision frameworks, and best practices across the ECD organization. 

• Serve as a clinical expert supporting new project identification, including internal pipeline opportunities and business development assessments.
• Translational and Biomarker Strategy
• Design and execute natural history studies to (1) deepen understanding of targeted patient populations and (2) enable development of endpoints that support efficient demonstration of POC and/or regulatory approval.
• Partner with Biomarker and Clinical Pharmacology colleagues to ensure robust plans for patient selection, pharmacodynamic assessments, response biomarkers, and potential approval endpoints.

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.