Scientist 1 or 2, Molecular Medicine, BioAnalytical Sciences

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 within the Translational Sciences Department at BioMarin Pharmaceutical is seeking a Scientist to help drive understanding of the underlying mechanisms for successful gene therapy with recombinant Adeno Associated Viral (rAAV) vectors. The ideal candidate has a flexible mindset, natural curiosity and motivation to understand biology at the molecular and biochemical levels. The Molecular Medicine group aims to creatively apply the complete molecular and cellular toolkit to understand the contributing mechanisms of action or effect, in collaboration with colleagues across the company. We are currently particularly interested in interrogating mechanisms that control the magnitude, durability and variability of expression of our gene therapy candidate(s). We are investigating the nature, formation, and impact of various rAAV structures formed in the host, as well as other mechanisms driving cross-species and -individual variance in expression, to evaluate translatability to the clinic. The Molecular Medicine group strives to improve technologies to enable analysis on small samples with definitive results, including at the single cell/nuclei level. The group collaborates with the entire set of Translational Sciences functions to understand the molecular and cellular actions of our therapeutics in preclinical experiments through clinical delivery to our patients. As our expanding clinical stage pipeline evolves, this Scientist will have the opportunity to contribute to molecular investigations on a variety of therapeutic modalities and the rare diseases that BioMarin strives to treat.

This position will be primarily laboratory focused, with some mentoring responsibilities.  The Scientist will be responsible for the design and execution of technically complex experiments to support multiple projects, with strategic input and guidance from Molecular Medicine and Translational Sciences leadership. S/he will provide quantitative data analysis, interpretation of results, and contribute to technical reports and scientific papers. S/he 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 Scientist also evaluates new lab equipment, interacts with vendors, and provides assistance to the Assay Strategy and Development group, including troubleshooting and validation. Scientists in the group function independently and contribute their own expertise and initiative, while also interacting with the Molecular Medicine group, the Bioanalytical Sciences colleagues, and Translational Sciences teammates to share ideas, and seek and receive feedback from their teammates.

RESPONSIBILITIES

• Design and execute experiments to understand molecular and cellular mechanisms underlying rAAV gene therapy efficacy, variability, and durability – in a collaborative team environment.
• Develop and perform a variety of analytical methods to detect biomarkers, drug, and antibodies to protein therapeutics in biological matrices to support pre-clinical and clinical programs. 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.
• Other duties as assigned.

EXPERIENCE

• 6+ years experience in biomedical sciences.  Advanced degree or bachelor’s degree with demonstrated experience as an independent scientist in a biotech setting.
• 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.

EDUCATION

• Bachelor (BS / BA) degree in a biological or biochemical field, with 6+ years experience, Masters (MS) degree in a biological or biochemical field, with 4+ years work experience, or Ph.D. with 1+ years post-doctoral experience with clinical/non-clinical biomarkers, molecular biology, cell biology, immunoassays, and/or other analytical methods.

 Scientific Level Differentiation:

• Scientist 1: PhD or equivalent relevant experience, plus 0-2+ years industry experience
• Scientist 2: PhD or equivalent relevant experience plus 2-5+ years industry experience

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.