Medical Director, Experimental Medicine

Role Summary

Fully remote role, US-based candidates only. BioMarin’s Experimental Medicine team is seeking a physician-scientist to serve as a key matrixed clinical leader focused on the preclinical planning, execution, and design and execution of highly informative first-in-human clinical experiments. Leveraging deep experience in biomarkers and translational clinical research, the Medical Director will partner with Preclinical Pharmacology, Biology, Clinical Pharmacology, Preclinical Safety, Clinical Safety, Regulatory and Clinical Operations to ensure that BioMarin assets are optimally positioned at the time of IND filing to inform a well-designed clinical strategy. Clinical Development plans will be designed to gain maximal insights during initial clinical experiences with new therapeutic agents. The Medical Director will have widespread impacts on the BioMarin early clinical pipeline. Key contributions will include informing preclinical data requirements prior to IND, delivery of initial clinical development plans, preIND and IND documents, and well-designed clinical trial protocols and supportive documents. In addition, this role will contribute to clinical assessments of new project opportunities (both internal projects and supporting business development activities).

Responsibilities

• Inform preclinical experimental plans for in vitro and in vivo pharmacology and toxicology packages.
• Develop biomarker strategy for preclinical assets and inform execution of experiments to inform their downstream clinical usage in advance of asset entry to the clinic.
• Formulate clinical development plans that serve as the strategic basis for full clinical development of an asset, with emphasis on demonstrating proof of mechanism in first in human and first in patient trials as well as subsequent demonstration of proof of concept in the clinic.
• Serve as clinical development team lead for assets in the research phase through Phase 1 clinical trial study completion.
• Lead clinical contributions to pre-IND and IND submissions (and other stage-appropriate Regulatory submissions).
• Lead clinical contributions to Phase 1 study protocols and trial-related documents.
• Design and execute natural history studies that enable (1) deepened understanding of proposed targeted patient populations and (2) development of endpoints that enable efficient demonstration of POC and/or approval.
• Serve as clinical expert to aid new project identification (both on internal new programs and business development opportunities).
• Represent Experimental Medicine in scientific advisory and academic meetings.
• Serve as Medical Monitor on BioMarin clinical trials.

Qualifications

• MD required. MD/PhD preferred.
• Ability to work collaboratively in a matrixed, fast-paced environment.
• Interest and curiosity in development challenges.
• Effective and impactful in cross-functional team structure.
• Excellent communication skills, with a desire to develop strong internal and external collaborations.