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Principal Scientist, Cellular Biology Lead, Neuroscience Research

Location San Rafael, California Workstyle Onsite Only

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 Neuroscience Research group at BioMarin is seeking a Cellular Biology Lead (Director-level role) with extensive experience in utilizing iPSC and other relevant culture systems to investigate neurological disease mechanisms and advance novel treatments.

The Cellular Biology Lead in Neuroscience will direct a team of neuroscience-focused cell biologists in advancing early-stage therapeutic programs targeting the underlying causes of genetic neurological diseases. Using cutting-edge in vitro approaches, he/she will help identify new disease targets and drive therapeutic programs to critical milestones and research inflection points. The Cellular Biology Lead will oversee the establishment and utilization of cutting-edge, patient-specific iPSC models of neurological disease that enable critical insights into human biology. He/she will also direct in vitro studies to identify and validate targets, select lead compounds, perform lead optimization and development candidate selection, predict exposure levels needed for clinical efficacy, and identify biomarkers. The Cellular Biology Lead will play a key role in partnering with cross-functional teams within BioMarin, including Discovery Medicine, Biomarker Discovery, Center of Technology, Product and Portfolio Development, Business Development, and IP.

The successful candidate will have a strong background in neuroscience, cellular and molecular biology, iPSC culture, pharmacology and drug discovery and development, with demonstrated experience in the field of genetics and/or precision medicine for neurological diseases. He/she will have experience or a strong interest in interneuron biology. The candidate will interact with the Head of Neuroscience, Directors of In Vivo Biology and External Collaboration, and Research and Early Development Leadership Team to provide critical updates on the Neuroscience portfolio and specific programs.


  1. Establish in vitro screening platforms and disease models for neurological indications
    • Direct the development of cellular screening platforms for lead identification and optimization
    • Lead the establishment of cutting-edge iPSC disease models for in vitro proof-of-concept studies
    • Employ cellular models to identify and validate new targets

  1. Identify and optimize lead compounds for early-stage neuroscience programs
    • Drive the identification of lead compounds utilizing cellular screening platforms
    • Guide lead optimization and development candidate selection using advanced and patient-specific disease models

  1. Identify biomarkers and develop clinical strategy
    • Identify and validate biomarkers for target and mechanism engagement using in vitro disease models
    • Use patient-derived disease models to predict pharmacodynamic requirements for efficacy
    • Assess the potential safety of development candidates and other leads using cell types representing key tissues of interest.

  1. Team building and cross-functional partnering
    • Build and lead a cohesive in vitro research team in Neuroscience
    • Build and enable strong, productive partnerships across RED and WWRD, including with Discovery Medicine, Project and Portfolio Development, Biomarkers, and Manufacturing
  • Drive in vitro model-based thought leadership to support BioMarin’s R&D programs in neuroscience
  • Provide scientific leadership and help guide the strategic direction of the company’s efforts in the Neuroscience therapeutic area, including developing and evaluating new internal and external therapeutic opportunities
  • Manage, mentor and guide a focused team of in vitro neurobiologists
  • Collaborate with various BioMarin functions, CROs, university or analytical laboratories, business partners, as necessary to ensure appropriate/timely progression of projects
  • Excellent written and verbal communication skills
  • Ability to build/foster productive cross-functional collaborations both within and external to BioMarin


  • Required: Ph.D. with strong experience in neuroscience research, molecular and cellular biology, pharmacology or a related subject area.
    • D. or M.D. / Ph.D. physician-scientist, with a major focus in basic neuroscience research preferred
    • Experience with interneuron biology a plus

Education & Experience Level:

  • PhD or equivalent relevant experience plus 7+ 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.

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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