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Scientist 1 , Gene Therapy platform studies

Location:San Rafael, California



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


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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


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 Genetics Research and Delivery group is currently recruiting exceptional individuals at the Scientist 1 level to support gene therapy projects with a strong focus on in vivo biology.  Successful candidates will be adept in interacting with multiple diverse teams, CROs and collaborators to guide the development of novel genetic therapeutics.  Candidate will design and manage in vivo studies to evaluate safety and efficacy of gene therapeutics. Organization, writing skills and ability to analyze and communicate data is essential as the candidate will be expected to generate and manage protocols and compose reports.Applicants should have a PhD degree or a BS with equivalent years of experience in a relevant field, with extensive experience with in vivo studies, cell culture, and molecular biology. Viral-based vector biology, virology, gene therapy, protein expression analysis are desirable knowledge/experience areas but not required. Teamwork, individual-initiative, and meticulous recordkeeping are essential.


This position is to support gene therapy projects with a strong focus on in vivo biology of gene therapies and animal study management.


-Design in vivo studies according to IACUC principles

-Study management, scientific report writing

-Present at internal and external meetings

-Execute and document laboratory procedures and experiments with great attention to detail

-Excellent communication, oral and written.


Required Skills:

Minimum Required: Bachelor’s degree with 8+ years of experience, Masters with 5+ years, or PhD with 2 years animal study experience. 

Experience in designing and writing in vivo study protocols, engaging CROs, data analysis, including statistical analysis, and interpretation

Assay development experience: cell culture and molecular biology techniques. 

Strong organization skills and computer skills.

Excellent communication skills: oral and written

Desired Skills:

-Cell culture work – multiple cell lines, transduction

-Molecular biology – DNA and RNA isolation from tissues, ddPCR

-Protein biology/ chemistry – ELISA, Western

-Strong organization skills and ability to multitask efficiently in a dynamic environment

-Gene therapy experience is a plus but not required.


PhD degree in biological science related field or Bachelor’s or Master’s with additional experience


Laboratory equipment, computerized systems


Must be able to work in a team format and present data at internal meetings.


No directs reports.

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.

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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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