Research Associate Ii, Molecular Medicine, Translational Sciences

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.

Our Culture

Our desire to make a positive impact on our patients extends to our employees and BioMarin is committed to fostering an inclusive environment where every person feels seen, valued, and heard - so employees can thrive in all areas of their lives, in and outside of work. We seek to provide an open, flexible, and friendly work environment to empower people and to provide them with the ability to develop their long-term careers. Ultimately, we want to be an organization where people enjoy coming to work and take pride in our efforts to help patients.

Position Summary

TheMolecular Medicine Group within the Translational Sciences Department at BioMarin Pharmaceutical is seeking a Research Associate II to help drive understanding of the underlying mechanisms for successful treatment with recombinant Adeno Associated Viral (rAAV) gene therapy and antisense oligo (ASO) modalities. 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 positioned to help solve technically challenging questions that present as unexpected findings and/or have cross-program impact. We are currently particularly interested identifying early indicators and mechanisms that modulate the magnitude, durability and variability of expression of our gene therapy candidate(s). We work with cross-functional teams to develop and evaluate nucleotide integration capabilities for safety assessments.

The Molecular Medicine group strives to improve technologies to enable analysis on small samples with definitive results, including at the single cell and 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 RAII will have the opportunity to contribute to molecular investigations on a variety of therapeutic modalities and the genetic diseases that BioMarin strives to treat.

This position will be primarily laboratory focused. The Research Associate II 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 RAII also evaluates new lab equipment, interacts with vendors, and provides assistance to the Assay Strategy and Development group, including troubleshooting and validation. Members of the group function independently and contribute their own expertise and initiative, while also interacting with Molecular Medicine, Bioanalytical Sciences and Translational Sciences colleagues 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.
• Provide scientific and intellectual input to guide project-related experiments within the context of interdisciplinary project teams
• Able to troubleshoot experiments based on thorough understanding of assay principles, and assist junior teammates in trouble shooting.
• 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

• 2+ years of experience in biomedical sciences. Bachelor's or advanced degree with demonstrated experience as an independent Research Associate in a biotech setting.
• Broad technical proficiency.
• Ability to solve complex scientific and technical issues.
• 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's degree in Biology, Chemistry, or like field with 2+ yr, Master's degree with 0-2 yr of relevant work experience in molecular biology, cell biology, clinical/non-clinical biomarkers, immunoassays, and/or other analytical methods.