Principal Scientist, Biology Research - Hematology (Non-Onc)

San Rafael, California
Jun 15, 2021
Required Education
Position Type
Full time
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 Biology Research group at BioMarin is seeking a Principal / Sr. Scientist with extensive experience researching genetic / inherited non-maliganant hemotology disorders using a broad range of in vitro and in vivo models, particularly in the context of evaluating novel therapeutic approaches.

The Principal / Sr Scientist will assume a crucial role in developing novel therapeutic concepts and will contribute at all stages of a project, from inception, early project validation, optimization, candidate selection and through to early clinical development of novel medicines for patients with rare inherited hematological disorders. He/She will work collaboratively with other teams to develop project strategy, identify key drug discovery issues, propose and implement creative solutions, execute and guide teams to success and decision points. He/She will lead the early development of a pipeline program targeting a genetic hematological disease, including formation of a cross-functional program team and, in partnership with the team, building short- and long-range plans and establishing key go/no-go decision-making criteria.

In addition to leading the early program team, the Principal / Sr Scientist will be a key scientific leader in the group, interfacing with program teams developing other novel therapeutics for inherited hematological diseases and will play a leadership role in helping to set the strategic direction for BioMarin’s interest in genetic hematological diseases, including the assessment of new target/therapeutic concepts and the evaluation of potential business development opportunities. The Sr/Principal Scientist will also manage several direct reports.

The successful candidate will have a strong background in hematology research, with demonstrated research experience in the application of novel therapeutic paradigms for genetic hematological diseases. Experience in the clinical practice will be beneficial.

The successful candidate will have experience providing context of findings to senior management and offering insights to inform continued clinical development of programs for inherited hematological (non-onc) diseases. The candidate will communicate, publish, and present scientific data and conclusions, along with their potential translatability to humans, to project teams, external collaborators, and the scientific community. 


  • Conduct early research for genetic non-malignant hematologic disorders
  • Develop novel programs based on innovative, ground-breaking discoveries that could lead to new, high-impact therapeutic opportunities for patients with unmet medical need
  • Lead a cross-functional preclinical program team in the development of a novel therapeutic for inherited hematological diseases
  • Lead a team of scientists to investigate the mechanisms of action of therapeutics for inherited hematological diseases
  • Direct research and publish findings in high-impact journals and present research at major conferences
  • Effectively partner with the program team, other scientists and the Research and Early Development leadership team to develop and execute on the program’s strategic plan
  • Present program data at a range of internal forums, including Research Seminars and Leadership Team governance forums
  • Provide senior scientific leadership and guide the strategic direction of the company’s efforts in the non-onc hematology therapeutic area, including developing and evaluating new internal and external therapeutic opportunities
  • Manage, mentor and guide a focused team of in vitro / in vivo scientists
  • Review and contribute to study synopses, protocols, study reports and regulatory documents
  • Collaborate with various BioMarin functions, CROs, university or analytical laboratories, business partners, as necessary to ensure appropriate/timely progression of projects
  • Participate in multi-disciplinary drug development teams and may lead sub-teams
  • Excellent written and verbal communication skills
  • Ability to build/foster productive cross-functional collaborations both within and external to BioMarin


Required:  M.D. or PhD in hematology (non-onc) or immunology. Experience in the field of pharmacology, viral gene therapy, non-viral gene therapy or a related subject area.


  • 8+ (Sr Scientist 2) or 10+ (Principal Scientist) years of experience in the research of genetic hematological diseases and novel therapeutic approaches in an academic or industrial setting; clinical practice experience in non-onc hematology is a plus
  • The candidate will have a proven record of accomplishment of driving and supporting multiple research projects, and contributions as an author to high-impact, peer-reviewed publications in the field
  • Experience in performing in-depth functional assays and in vivo efficacy studies for hematological conditions
  • Experience supporting the development of gene therapies, small molecules, biologics and/or other therapies, including determining non-clinical program strategy and design. 
  • Clinical trial design experience to guide seamless transition of early pipeline projects into clinical development
  • 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

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.