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Sr. Scientist I or II - Toxicologist with experience in Gene Therapy & Oncology

BioMarin Pharmaceutical Inc.
San Rafael, California
Start date
Sep 9, 2022

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Science/R&D, Biology, Biotechnology, Molecular Biology
Required Education
Masters Degree/MBA
Position Type
Full time
Biotech Bay
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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 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 Pharmacology and Toxicology Department at BioMarin is seeking an experienced Senior Scientist I/II to provide nonclinical pharmacology and/or toxicology expertise for the development of BioMarin’s novel drug candidates.  This individual will provide scientific leadership and play a pivotal role providing nonclinical expertise in support of all phases of the drug development process from research through market application and commercialization.

The successful candidate will contribute to the strategic development of BioMarin’s therapeutics by creating a best-evidence synthesis of existing knowledge and by designing, implementing, interpreting, investigating and reporting data that elucidates the pharmacological / toxicological profile of drug candidates.  This individual will work collaboratively with internal and external cross-functional sub-teams, collaborators and consultants to contribute to integrated development pathways throughout the drug development cycle. 

The successful candidate will have a strong pharmacology background with experience in molecular biology/genetics (especially related to oncology) as well as a strong understanding of pharmacological mechanisms of disease and will provide broad-based support to align nonclinical efforts with the company’s opportunistic philosophy of drug development.  The candidate will interact with collaborators and scientists across the company in order to provide a balanced assessment as to the risk/benefit profile associated with a drug candidate’s advancement. The individual will be responsible for the planning, implementation and organization of nonclinical studies along with the use of drug exposure to bridge across species, presentation of data (written and verbal) to cross-functional core teams and sub-teams, external collaborators, and the scientific community.  This individual will actively participate in representing Pharmacological Sciences on cross-functional project teams and/or lead project sub-teams; the Pharmacology & Toxicology team at BioMarin is willing to train this individual to achieve to that competency if required. 


  • Contribute to and implement an overall drug development strategy to support nonclinical and clinical pharmacology development during all stages of drug development 
  • Work closely with collaborators on animal models of disease including an in-depth understanding of disease etiology
  • Be able to serve as a nonclinical consult in tumor development based on the molecular biology of genetic alteration(s) following gene therapy (or other modalities) treatments
  • Help Design, implement, report and interpret nonclinical study data of gene therapy, small molecule, oligonucleotides, biologic or other novel drug candidate constructs across all stages of development
  • Prepare study synopses, protocols, study reports and regulatory documents to support nonclinical development of BioMarin’s drug candidates
  • Interact/collaborate with various BioMarin functions, CROs, university or analytical laboratories, business partners, as necessary to ensure appropriate/timely progression of BioMarin projects
  • Effectively communicate nonclinical findings and their relevance to senior management, project teams, collaborators, and regulatory agencies
  • Participate in multi-disciplinary drug development teams including lead sub-teams and serving as a program representative at cross functional program meetings
  • Excellent written and verbal communication skills
  • Ability to build/foster productive cross-functional collaborations both within and external to BioMarin.


  • Nonclinical pharmacology with molecular biology/genetics background and with 5+ years of experience in the pharmaceutical, CRO, and/or biotechnology field at a Scientist level.
  • Expertise in in vivo pharmacology or experience with animal models of disease
  • Strong understanding of drug development and regulatory process, as well as a good understanding of Good Laboratory Practices (GLPs)
  • Experience in the design, conduct, and interpretation of GLP or non-GLP nonclinical pharmacology and toxicology studies
  • Experience supporting the development of gene therapy, small molecules, oligonucleotides, biologics, gene therapies and/or other therapies, including determining non-clinical program strategy and design. Experience in genotoxicity, especially as related to gene therapy is a big plus
  • Experience with the preparation of regulatory submissions (IND/CTDs, NDA/BLA, etc.) and communicating/ interacting with health authorities desirable
  • Strong critical, scientific, and strategic thinking skills; Strong problem solving and decision making abilities
  • Strong communication (verbal and written) skills
  • Experience and ability to work collaboratively in cross-functional teams


  • Required: Masters (M.Sc.) or Doctorate (Ph.D.) or equivalent experience in molecular biology/genetics, pharmacology, toxicology or a related, relevant discipline.  
  • 5+ years of industry experience
  • Intention to obtain Diplomate of the American Board of Toxicology (DABT) certification is a plus

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.
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