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Medical Director, PKU / Gene Therapy

Location:London, United Kingdom



With accelerated review underway in the United States and Europe for our Hemophilia A drug, a second gene therapy program entering the clinic for PKU patients, and a third program in late research for Hereditary Angioedema, BioMarin continues to pioneer innovative approaches to treating rare diseases. Our commitment to gene therapy is further underscored by having developed the first and largest gene therapy manufacturing facility in the world. The scientific possibilities and the hope we offer to patients with rare diseases is enormous.

BioMarin is a midsize biotechnology company with a market cap of approximately 18 billion. Over the course of our 22 year history we have successfully launched 7 drugs for the treatment of rare diseases. Our success is due to our scientific rigor and our ability to leverage multiple therapeutic modalities. BioMarin is based in the San Francisco Bay Area and offers a comprehensive relocation program.

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Success Profile

Check out the traits we’re looking for and see if you have the right mix.

  • Decision Making
  • Influence
  • Collaboration
  • Agility
  • Leadership
  • Intellectual Curiosity

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  • Patient Focused
  • Results Oriented
  • Pioneering Science
  • Cross Functional Collaboration
  • Willing to try new ways to look at problems


  • Healthcare

  • Medical

  • Dental

  • Vision

  • Health & Wellness Programs

  • 401(k)/Retirement plans

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" BioMarin has this, in their own DNA, an approach: they only work on projects for which they know the underlying biology."
Lon C., Chief Scientific Officer
"We’ve got to understand: What are the problems other people are trying to solve? And do we have a better way of fundamentally doing that?"
Brinda B, GVP, Corporate and Business Development


BioMarin is the world leader in delivering therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We target diseases that lack effective therapies and affect relatively small numbers of patients, many of whom are children. These conditions are often inherited, difficult to diagnose, progressively debilitating and have few, if any, treatment options. BioMarin will continue to focus on advancing therapies that are the first or best of their kind.

BioMarin’s Research & Development group is responsible for everything from research and discovery to post-market clinical development. Research & Development 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. Come join our team and make a meaningful impact on patients’ lives. 

The Medical Director is a critical strategic and executional role in the Clinical Science function, leading one or more studies as a Medical Monitor. The Medical Monitor is responsible for the medical and scientific integrity during study planning and execution and is accountable for the day-to-day Clinical Science medical monitoring deliverables. Key responsibilities may include providing medical and scientific input into the creation and review of all Clinical Science study deliverables; monitoring subject eligibility, safety and data and initiating appropriate action; and representing Clinical Science as a member of the cross functional study execution team.

Responsibilities may include:

  • Design and maintain clinical development plan
  • Design and write clinical protocols, clinical sections of regulatory submissions, clinical study reports
  • Provide clinical leadership and medical monitoring for human clinical trials
  • Develop and maintain relationships with academic thought leaders. Design and initiate strategically meaningful investigator initiated studies.
  • Provide input on due diligence assignments evaluating in-licensing or acquisition targets.
  • Contribute to the peer review process, both internally and externally, to help assure BioMarin clinical science is conducted at the highest level of quality, integrating business and development objectives.
  • Interact with key opinion leaders to assure implementation of latest clinical thinking and guidelines into the clinical development plan
  • Serve as medical and technical resource for clinical issues raised by internal and external collaborators, investigators, consultants and contract resources
  • Represent the company at scientific, commercial and financial community meetings and presentations, as well as other public relations opportunities
  • Attend and provide scientific support for investigator and consultant meetings and other study implementation workshops
  • Participate in management of studies and serve as expert on clinical and medical issues to other colleagues and study site staff
  • Participate in medical monitoring and the review of study data
  • Assists in the development of publications, abstracts, and presentations
  • Provide clinical input into design and endpoints for relevant nonclinical, translational proof of principle animal studies
  • Provide internal clinical leadership by distilling and communicating translational research concepts and clinical management strategies to multi-disciplinary scientific and non-scientific project teams
  • Provide clinical input and partnering with research on IND candidate selection, supporting pre-clinical pharmacologic and toxicology studies to support human clinical studies
  • Develop relevant clinical and molecular biomarkers for nonclinical and clinical studies
  • A minimum of 3 years experience in the pharmaceutical/Biotech industry is highly desired, or equivalent academic and clinical experience.
  • Track record of having directly contributed to the success of an enterprise through scientific accomplishment.
  • Clinical trial design and implementation experience
  • Skills in written and verbal communication, with an emphasis on dynamic oral presentations
  • Passion for continued learning and helping others learn
  • Collaborative, team-oriented, consistently looks for win-win solutions to problems
  • Able to adjust to new challenges as dictated by the requirements of the job
  • Experience with writing filings/briefing books and CSRs would be a plus
  • MD, MD/PhD, DO with appropriate country specific licensure
  • 3 or more years of relevant experience in clinical science/clinical development of genetic diseases, metabolic diseases, specialty care, and/or rare diseases desired

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

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Meet Our Team

From internationally renowned scientists to patient advocates, BioMarin Hemophilia has brought together the right people.

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BioMarin has entered into a preclinical collaboration and license agreement with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies.

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