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Scientist I or II: Payload Engineering, Gene Therapy Research

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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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


Scientist I or II: payload engineering

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 looking for a highly motivated team member with extensive experience in non-viral delivery technologies to be a key contributor to the Genetics Research and Delivery Group. The incumbent will be responsible for supporting the ongoing gene therapy efforts within the Genetics Research and Delivery Group.


  • Apply their deep technical knowledge of multiple aspects of gene regulation to design and optimize expression cassettes for therapeutic payloads and/or gene-editing tools, including cell-specific regulation to drive high-level expression in target cells and limit off-target expression.
  • Implement assays for in vitro screening of genetic circuit performance
  • Work collaboratively with delivery and in vivo biology groups to assess performance and implement improvements.
  • Manage direct reports and collaborate with cross-functional teams and external partners
  • Record, document, analyze and present scientific data to senior staff and team members and participate in the writing of SOPs, reports, and manuscripts for external scientific communications


  • PhD in molecular biology, biochemistry, synthetic biology or relevant biological sciences
  • Gene therapy or gene editing experience preferred, including working knowledge of state-of-the-art technologies utilized in the field.
  • Strong technical background with standard laboratory methods, including molecular biology and cloning; (q)PCR; ELISA; intracellular staining; western blotting; mammalian tissue culture; DNA transfection and viral transduction; flow cytometry or microscopy
  • Ability to productively collaborate in cross-functional teams
  • Excellent interpersonal communication and writing skills for presentations, electronic notebook, and report writing
  • Ability to adapt to a new and fast-paced environment and navigate uncertainties
  • Willingness to embrace new challenges, fearlessly innovate, and constantly learn
  • Absent a Medical or Religious reason that prohibits vaccinations, all our incoming employees must be vaccinated

Level Differentiation

Scientist 1:

  • PhD or equivalent relevant experience, plus 0-2+ years industry experience

Scientist 2

  • PhD or equivalent relevant experience plus 2-5+ years industry experience

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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