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Senior Scientist, Molecular Medicine, BioAnalytical Sciences

Location San Rafael, California
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 Molecular Medicine Group at BioMarin Pharmaceutical is looking for a Sr. Scientist to help drive understanding of the underlying mechanisms for successful gene therapy with rAAV vectors.  The ideal candidate has a flexible mindset, natural curiosity and motivation to understand biology at the molecular and biochemical level. The Molecular Medicine group aims to creatively apply the complete molecular and cellular toolkit to understand the contributing mechanisms of action or effect of our therapeutics, in collaboration with colleagues across the company.  At this time, we are particularly interested in interrogating mechanisms that control the magnitude, durability and variability of expression of our gene therapy products.  Some of the initial experiments taken on by this individual will be aimed at understanding the molecular details of rAAV mRNA expression on the single-cell, and single-molecule level. We are also investigating the nature and mechanisms of formation of the various rAAV structures found in the cell, including in the context of species-specific changes in mRNA expression, to evaluate translatability to the clinic. This position may include both laboratory and supervisory roles.  The Sr. Scientist will be responsible for the design and execution of technically complex experiments to support these projects, with strategic input and guidance from Molecular Medicine and Translational Science leadership. Individuals will provide quantitative data analysis, interpretation of results, and contribute to technical reports and scientific papers, as well as mentor junior members of the team. They will evaluate existing complex methods and technologies, develop new solutions, and collaborate across functions to solve technical problems.  The Molecular Medicine team values learning agility, open communication skills, willingness to support others’ experiments, and ability to trouble-shoot experiments on multiple platforms. The Sr. Scientist also evaluates new lab equipment, recommends vendors, and provides assistance to assay development, troubleshooting, and validation.   Scientists in the group function independently and contribute their own expertise and initiative, while also interacting with the Molecular Medicine group to share ideas, and seek and receive feedback from their teammates, including those in the broader BioAnalytical Sciences function, Translational Sciences, and Research & Early Development.

RESPONSIBILITIES:

  • Design and execute experiments to understand molecular and cellular mechanisms underlying rAAV gene therapy efficacy, variability, and durability – in a collaborative team environment, making use of skills and efforts of other team members.
  • Develop a variety of analytical methods to detect biomarkers, drug, and antibodies to protein therapeutics in biological matrices. Techniques may include (but are not limited to) nucleic acid extraction, single cell sequencing, multi-color droplet digital polymerase chain reaction (ddPCR), sequence-tagged molecular tracing, ultrasensitive protein assays, cell-based and ex vivo culture assays.
  • Perform quantitative analysis within and across multiple experiments, including basic statistical and graphical analysis.
  • Develop new techniques, or bring in new technology from the outside, to enhance the analytical capabilities of the group.
  • Present results, conclusions, and implications to Molecular Medicine, BioAnalytical Sciences, and Translational Sciences groups and cross-functionally.
  • Meticulously adhere to electronic data recording standards.
  • Share responsibility for lab management duties.
  • Participate and present data as a member of BioAnalytical teams, and share responsibility for project milestones.
  • Present data and participate in discussion in Translational Sciences/Research and Early Development team meetings and as necessary.
  • Participate in external scientific meetings and contribute to original peer-reviewed publications.
  • Opportunity to manage one direct report
  • Other duties as assigned.

EXPERIENCE

  • D.-level training and experience, including 2+ years demonstrated experience leading an effort as an independent scientist.
  • Broad technical proficiency.
  • Ability to solve complex scientific and technical issues independently.
  • Excellent verbal and written communication skills: ability to cogently prepare and present data in group settings, prepare formal reports, communicate across functions.
  • Strong teamwork skills – ability to solicit, receive, and give useful feedback.
  • Ability to use computer effectively for data analysis and presentation.
  • Strong working knowledge of molecular and cell biology and biochemistry, with deeper expertise in at least one area.
  • Demonstrated experience initiating new methods or technologies in an experimental laboratory setting.
  • Management skills are a plus

Level Differentiation:

Sr. Scientist 1:

  • PhD or equivalent relevant experience plus 5+ years industry experience

Sr. Scientist 2:

  • PhD or equivalent relevant experience plus 8+ years of industry experience
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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We treat rare diseases, and that gives us rare opportunities. Chris Brodeur, Director, Corporate Compliance
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