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Associate Medical Director, Experimental Medicine, Early Clinical Development

BioMarin Pharmaceutical Inc.
Working from Home
Start date
May 29, 2023

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Clinical, Clinical Development, Clinical Research
Required Education
Position Type
Full time

Job Details

Who We Are

From research and discovery to post-market clinical development, our WWRD 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 genetic diseases.

BioMarin’s Experimental Medicine is a group of physician-scientists that support pipeline advancement and growth of the clinical portfolio.  As an Early Clinical Development function, our group of physician-scientists link research initiatives and technical assessments of external innovation to areas of development based on fundamental understanding of genetics, molecular mechanisms, disease phenotypes, and medical need in specific patient populations. We seek a neurology trained physician-scientist to support the early clinical development of a broad portfolio of programs in neurogenetic diseases. 

  • Support neurogenetic programs within Experimental Medicine as a primary early clinical lead
  • Help design and conduct early clinical studies, from natural history through treatment proof-of-mechanism clinical trials
  • Author translational and pre-treatment plans based on research results
  • Interrogate natural history and disease progression of therapeutic indication to plan a development strategy encompassing biomarkers, endpoints, and target patient population
  • Serve as subject matter expertise of genetically driven neurologic disease by keeping current with literature, meetings, and disease pipeline
  • Work collaboratively with research and clinical groups to advance programs
  • Help generate development plans, draft product characterization, first-in-human and proof of concept trial strategy, draft clinical development plans
  • Early clinical representative in investigative new drug (IND) filing team
  • Represent Early Clinical Development in scientific advisory and academic meetings
  • Identify high value therapeutic targets for neurogenetic indications and contribute clinical expertise to groups involved in disease mapping and pipeline strategy
  • Work with Research, Innovation Accelerator and Business Development to facilitate clinical diligence of new development opportunities
  • Participate in scientific advisory boards convened by BioMarin to support the research initiatives in neurogenetics

  • MD or MD/PhD required
  • Strong record of academic, basic and/or translational research productivity
  • Solid foundation and interest in mechanisms of neurogenetic diseases
  • Board certified or eligible in adult or pediatric neurology
  • 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


PLEASE NOTE: Absent a Medical or Religious reason that prohibits vaccinations, all our incoming employees must be vaccinated for COVID-19.

We are an equal opportunity employer and all qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, sexual orientation, national origin, disability status, protected veteran status, or any other characteristic protected by law.


BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. In 1997, the company was founded to make a big difference in small patient populations, focusing on giving much-needed attention to the underserved communities of those with rare diseases. BioMarin sought to help patients whose voices could not always be heard and treat conditions that were not always in mainstream medical conversations. Throughout its history, BioMarin has worked tirelessly to make a difference by pursuing bold science. The company’s first approved therapy was inspired by a father who refused to accept there was no therapy for his son’s rare diagnosis. That relentless pursuit to change the course of his son’s future through the power of science is at the core of who BioMarin is today.

Through the company’s expertise in genetics and molecular biology, BioMarin has developed eight first or best-in-class targeted therapies that address the root cause of the conditions it seeks to treat, helping to better the lives of those struggling with rare genetic disease. The company’s purposeful and scientifically precise team of physicians and scientists has honed each treatment modality to seek transformative therapies.

Now, BioMarin seeks to make an even greater impact by applying the same science-driven, patient-forward approach that propelled the last 25 plus years of drug development to larger genetic disorders, as well as genetic subsets of more common conditions. Each drug candidate pursued is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address. The company believes that applying its knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most. The more innovative solutions developed, the more lives BioMarin can impact.

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