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Sr Research Associate - OR - Research Associate II, Molecular Medicine, BioAnalytical Sciences (Gene Therapy)

Location :San Rafael, California

Workstyle :Onsite Only



With accelerated review underway in the United States and Europe for our Hemophilia A drug, a second gene therapy program entering the clinic for PKU patients, and a third program in late research for Hereditary Angioedema, BioMarin continues to pioneer innovative approaches to treating rare diseases. Our commitment to gene therapy is further underscored by having developed the first and largest gene therapy manufacturing facility in the world. The scientific possibilities and the hope we offer to patients with rare diseases is enormous.

BioMarin is a midsize biotechnology company with a market cap of approximately 18 billion. Over the course of our 22 year history we have successfully launched 7 drugs for the treatment of rare diseases. Our success is due to our scientific rigor and our ability to leverage multiple therapeutic modalities. BioMarin is based in the San Francisco Bay Area and offers a comprehensive relocation program.

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Success Profile

Check out the traits we’re looking for and see if you have the right mix.

  • Decision Making
  • Influence
  • Collaboration
  • Agility
  • Leadership
  • Intellectual Curiosity

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  • Patient Focused
  • Results Oriented
  • Pioneering Science
  • Cross Functional Collaboration
  • Willing to try new ways to look at problems


  • Healthcare

  • Medical

  • Dental

  • Vision

  • Health & Wellness Programs

  • 401(k)/Retirement plans

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" BioMarin has this, in their own DNA, an approach: they only work on projects for which they know the underlying biology."
Lon C., Chief Scientific Officer
"We’ve got to understand: What are the problems other people are trying to solve? And do we have a better way of fundamentally doing that?"
Brinda B, GVP, Corporate and Business Development


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 Molecular Medicine Group within the Translational Sciences Department at BioMarin Pharmaceutical is seeking a Research Associate with industry experience in molecular biology to help drive understanding of the underlying mechanisms for successful therapeutic candidates in gene therapy and oligonucleotide modalities. The ideal candidate has a flexible mindset, natural curiosity and motivation to understand biology at the molecular and biochemical levels. We are investigating the uptake, distribution, structure, variability and durability of therapeutic candidates by expanding expertise in nucleic acid biology, to creatively develop and apply assays for detection of nucleic acids. 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 Research Associate 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 laboratory focused, and technically enriching.  The Research Associate will be responsible for the design and execution of technically complex experiments to support multiple projects, and will provide quantitative data analysis, and interpretation of results. S/he will collaborate with colleagues in the Assay Strategy and Development and BioAnalysis groups, including to troubleshoot and validate assays.  The Research Associate evaluates new lab equipment, evaluates complex methods and technologies, identifies new areas requiring process improvement and adapts existing processes or develops new ones with direct input and supervision from a manager. They may contribute to scientific publications and regulatory submissions as required.  Molecular Medicine team values learning agility, open communication skills, willingness to support others’ experiments, and ability to troubleshoot experiments on multiple platforms.

We are searching for a self-motivated Research Associate with 4+ years of hands-on laboratory experience. The position will involve working directly with Scientists in the Molecular Medicine to develop and implement novel assays to contribute to scientific investigation into unexpected findings in our clinical and preclinical studies.


  • 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 nucleic acids, biomarkers and drug in biological matrices to support pre-clinical and clinical programs. Techniques may include (but are not limited to) nucleic acid extraction and quantitation, single cell sequencing, qPCR, multi-color droplet digital polymerase chain reaction (ddPCR)
  • Optimize, troubleshoot, and perform nuclei isolation from animal tissues followed by sequencing.
  • Perform quantitative analysis within and across multiple experiments, including basic statistical and graphical analysis.  Use mid-level bioinformatics tools to analyze DNA sequencing data.
  • Develop new techniques, or bring in new technology from the outside, to enhance the analytical capabilities of the group.
  • Organize efforts across multiple collaborating Research Associates to obtain consistent high-quality data on large analysis efforts
  • 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 maintenance 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.


  • Expertise in Molecular Biology techniques including DNA/RNA extractions and quantitation, reverse transcription, qPCR, ddPCR, and next generation sequencing.
  • Experience in method development for single cell RNA sequencing from animal tissues.
  • The position requires organizational skills, attention to detail, self-motivation, teamwork, and dedication to quality in a fast-paced setting.
  • Committed to overcome challenges and ability to troubleshoot.
  • Ability to solve complex scientific and technical issues independently.
  • Demonstrated experience initiating new methods or technologies in an experimental laboratory setting.
  • Effective verbal and written communication skills to support collaboration among scientists and documentation of experiments.
  • A strong team-player who wants to contribute to innovative science.
  • Ability to use computer effectively for data analysis and presentation.


  • Familiar with single cell platforms (ie. droplet-based or nano well technologies)
  • Basic knowledge on next generation sequencing analysis


  • Bachelor’s degree in molecular biology, biochemistry or a related scientific field and at least 4+ years of relevant experience OR Master's degree with at least 2+ years of relevant experience.


Research Associate 2:

  • Bachelor’s degree and at least 4 years of experience; Master’s degree with at least 2 years of relevant experience

Senior Research Associate 1:

  • Bachelor’s degree and at least 6 years of experience; Master’s degree with at least 4 years of relevant 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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Meet Our Team

From internationally renowned scientists to patient advocates, BioMarin Hemophilia has brought together the right people.

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In the News

BioMarin has entered into a preclinical collaboration and license agreement with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies.

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