Principal Scientist, Muscular and Skeletal Disease

San Rafael, California
Oct 07, 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 Muscular and Skeletal Therapeutic Area Research Group at BioMarin is seeking a Senior or Principal Scientist with extensive experience researching genetic skeletal diseases using a broad range of in vitro and in vivo models, particularly in the context of evaluating novel therapeutic approaches.  The ideal candidate will be known and respected scientist in the field of skeletal biology and disease. 

The Scientist will assume a crucial role in developing novel therapeutic concepts and will contribute at all stages of a project, from idea evaluation, project inception, early project validation, optimization, candidate selection and through to early clinical development of novel medicines for patients with skeletal disorders.  They will work collaboratively across the Research and Early Development Department, the broader company, and the external scientific collaborators and consultants to develop project strategies, identify key drug discovery issues, propose and implement creative solutions, execute and guide teams to success and decision points.

The Scientist will be a key scientific leader in the Muscular and Skeletal Therapeutic Area Research Group. They will influence the development of therapeutic strategies and contribute to the leadership and development of programs targeting skeletal diseases. The Scientist will manage direct reports.

The successful candidate will have a strong background in skeletal biology, with demonstrated experience in the application of novel therapeutic paradigms for genetic diseases. Experience in cellular and/or in vivo models of disease is critical.  Experience with therapeutic modalities and managing contract research organizations is desirable.


  • Conduct early research for genetic skeletal diseases
  • Develop novel therapeutic programs based on innovative, ground-breaking discoveries that could lead to new, high-impact opportunities for patients
  • Lead cross-functional preclinical program team in the development of a novel therapeutic
  • 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 Muscular and Skeletal Therapeutic Area Research Group, 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
  • Absent a Medical or Religious reason that prohibits vaccinations, all our incoming employees must be vaccinated


  • Required:  Ph.D. with strong experience in skeletal biology and disease or a related subject area with 5+ years (Senior Scientist) or 10+ years (Principal Scientist) of relevant experience.
  • 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 musculoskeletal diseases
  • Experience supporting the development of gene therapies, small molecules, biologics and/or other therapies, including determining preclinical program strategy and design. 
  • Strong critical, scientific, and strategic thinking skills; strong problem solving and decision-making abilities

Absent a Medical or Religious reason that prohibits vaccinations, all our incoming employees must be vaccinated.

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.