Scientist 1, Cell Biology For Neuroscience Research

Who We Are
For more than two decades, going our own way has led to countless breakthroughs, bettering the lives of those suffering from rare genetic disease. In 1997 we were founded to make a big difference in small patient populations. Now we seek to make an even greater impact by applying the same science-driven, patient-forward approach that propelled our last 25 years of drug development to larger genetic disorders, as well as genetic subsets of more common conditions. Through our unparalleled expertise in genetics and molecular biology, we will continue to develop targeted therapies that address the root cause of the conditions we seek to treat. Applying our knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most. The end goal has always been better lives and now we can reach more.
And the more people we reach, the more our impact can grow. We transform lives through genetic discovery.
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.
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 or best-in-class therapeutics that provide meaningful advances to patients. Come join our team and make a meaningful impact on patients’ lives.
The Neuroscience Research group at BioMarin is seeking a Senior Research Associate with experience in utilizing iPSC and other relevant culture systems to investigate neurological disease mechanisms and advance novel treatments.
The Senior Research Associate in Neuroscience Cell Biology will join and assist a team of neuroscience-focused cell biologists in advancing early-stage therapeutic programs targeting the underlying genetic causes of neurological diseases. Employing cutting-edge in vitro approaches, he/she will assist in the development of novel, advanced cellular models of human disease in order to screen therapeutic compounds, and to identify, optimize and progress therapeutic leads. In doing so, the Senior Research Associate will establish themselves as a thought leader on method development and work diligently across functions to contribute to diverse areas such as biomarker identification, functional assay development and patient-specific disease modeling.
The successful candidate will ideally have a strong background in neuroscience, cell and molecular biology, iPSC culture, and pharmacology/drug discovery. He/she will have excellent interpersonal skills and a strong track record of utilizing collaboration to advance therapeutic programs. The candidate will interact with the Head of Neuroscience, Director of Cell Biology in Neuroscience, and Research and Early Development Team to provide critical updates on the Neuroscience portfolio and specific programs.
Responsibilities

• Establish in vitro screening platforms and disease models for neurological indications
• Evaluate complex new methods and culture systems, identify new areas requiring method improvement and adapt existing methods or develop new ones with direct input and supervision from a manager
• Employ cellular models to identify and validate new targets
• Assist in the development of cellular screening platforms for lead identification and optimization
• Contribute to the establishment of cutting-edge iPSC disease models for in vitro proof-of-concept studies

• Contribute to scientific publications and regulatory submissions as required
• PhD neuroscience, molecular biology, biochemistry or related field
• Assist lead optimization and development candidate selection using advanced (co-culture/organoid) and patient-specific disease models
• Cross-team collaboration and engagement, data analysis and organizational skills
• Identify biomarkers and develop clinical strategy
• Contribute to productive cross-functional collaborations both within and external to BioMarin
• Thoroughly record and document experimental methodology and results
• Assess the potential safety of development candidates and other leads using cell types representing key tissues of interest.
• Use patient-derived disease models to predict pharmacodynamic requirements for efficacy
• Perform quantitative analysis within and across multiple experiments, interpret results and provide feedback on implications for program progression
• Excellent written and verbal communication skills
• Execute experiments to understand molecular and cellular mechanisms underlying therapeutic efficacy, variability, and durability
• Committed to overcome challenges and ability to troubleshoot
• Collaborate with various BioMarin functions, CROs, university or analytical laboratories, business partners, as necessary to ensure appropriate/timely progression of projects
• Promote the identification of lead compounds utilizing cellular screening platforms
• Identify and optimize lead compounds for early-stage neuroscience programs
• Identify and validate biomarkers for target and mechanism engagement using in vitro disease models
• Ability to solve complex scientific and technical issues independently
• At least 0-2 years of relevant postdoctoral or industry research experience with a track record of productivity