Sr. Clinical Science Specialist, Gene Therapy for Hemophilia A

Location:  San Rafael, California Category: Clinical


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.

As a vital member of the valoctocogene roxaparvovec (previously known as BMN 270) Clinical Science (CLS) team, the Clinical Science Specialist partners with a Medical Director to uphold medical and scientific integrity during study planning and execution and participates in the day-to-day CLS medical monitoring deliverables. Key responsibilities, as delegated by the Clinical Development Team Lead, include:

  • Monitor patient eligibility, safety, and data and initiate appropriate action, as needed
  • Ensure detail-oriented contributions are of high-quality and in compliance with regulatory requirements
  • Provide CLS input in all stages of the clinical trial, from start-up to close-out
  • Represent CLS as a member of the Study Execution Team (SET)


  • Bachelors Degree required
  • Advanced Clinical/Science Degree preferred (e.g., PharmD, PhD, MSN, MPH, etc.)
  • At least 4 years of clinical trial experience (minimum of 2 years of clinical trial experience in industry)
  • Extensive experience with data listing review
  • Experience in hemophilia and/or gene therapy desirable but not required
  • Strong computer skills, including Microsoft Office Suite (Word, PowerPoint and Excel), EDC RAVE, Spotfire


  • Impeccable ethics
  • Outstanding attention to detail
  • Demonstrated, through past experience, ability to competently manage majority of CLS deliverables associated with assigned clinical studies
  • Strong interpersonal, verbal communication and influencing skills: can influence without authority and has proven experience building and cultivating relationships with key partners and stakeholders, both internally and externally
  • Comfortable working in a fast-moving, evolving environment
  • Outstanding written communication skills
  • Strong business presentation skills: highly effective at summarizing and presenting key considerations and decision points
  • Confident and competent when interacting with varying levels of internal/external management, KOLs, etc.: stays focused and on-point, and is able to raise problems or challenges in a productive and mature manner
  • Proven teamwork skills: has a proven track record of working highly effectively, efficiently and collaboratively with others. Has proven experience and skills working with multidisciplinary teams
We are an equal opportunity employer and all qualified applicants will receive consideration for employment without regard to race, color, religion, sex, national origin, disability status, protected veteran status, or any other characteristic protected by law.