Senior Medical Director, Early Clinical Development in London

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, 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.

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.

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 lead (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.

Clinical Development Execution: 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.

Translational and Biomarker Strategy: Design and execute natural history studies to deepen understanding of targeted patient populations and enable development of endpoints that support efficient demonstration of POC and/or regulatory approval.

Portfolio, Talent, and Organizational Development: 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.

Business Development Support: Serve as a clinical expert supporting new project identification, including internal pipeline opportunities and business development assessments.