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Scientist I or II - Functional Genomics, Human Genetics Team

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.

We are seeking a highly motivated, innovative and team-oriented individual to join the Human Genetics research group. Within the Human Genetics group, the Functional Genomics group is expanding in order to carry out its mission of applying genetic engineering and other functional genomic technologies to discover and validate novel therapeutic targets.  The successful candidate will utilize genomic perturbation and next generation sequencing technologies to advance our therapeutic research and discovery efforts.  Expertise should include the use of NGS and gene editing technology to assess genomic functions in vitro. The position requires adaptability, creativity, and a capacity for constant innovation in pursuit of company goals. 


  • Utilize NGS technologies to interrogate genetic variants in therapeutically relevant models, including organoids and iPSC-derived cultures.
  • Utilize gene editing technologies to develop in vitro functional models and perform high-throughput scRNA-seq on pooled genetic perturbation screens (e.g., Perturb-seq).
  • Develop complex phenotypic endpoints using transcriptomics and other high-throughput imaging or luminescence-based screening assays to study genetic variants in the context of disease biology.
  • Design, develop, execute, and analyze experimental research projects in collaboration with cross-functional teams.


  • Utilizing genomic perturbation (ASOs, RNAi, CRISPR) with mammalian cell models for identification and validation of therapeutic targets.
  • Next-generation sequencing based endpoints (Perturb-seq, CRISP-seq, CROP-seq). Implement transcriptomic (scRNAseq, RNAseq, Iso-seq) and epigenome profiling assays such as ATAC-seq, ChIP-seq/CUT&RUN to support the expanding portfolio.
  • Working knowledge of liquid handling robotics (Hamilton, BioMEK, Tecan) for mammalian cell culture or NGS library preparation is preferred.
  • Design and execution of genetic screens (ie MPRA or STARR-Seq) for target identification and MOA characterization.
  • Strong microscopy skills, including immunohistochemistry, 3D imaging, in situ hybridization, live cell imaging, and/or high content imaging.


  • Ph.D. in Molecular/Cell Biology, Genomics, Bioengineering, or related discipline with research experience in either an academic or industry lab.
  • Strong analytical, written, and verbal communication skills.
  • Track record of productivity and effective projects.
  • Interested in learning new techniques and approaches. Attention to detail, time management and organization skills are critical.
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.

About our location

San Rafael


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We treat rare diseases, and that gives us rare opportunities. Chris Brodeur, Director, Corporate Compliance
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