Why Join Us?
Be a hero for our rare disease patients
At Ultragenyx, we fundamentally believe that taking real impactful action to care for the needs of patients and our people is always the right thing to do. To achieve this goal, our vision is to lead the future of rare disease medicine. For us, this means going where other biopharma companies won’t go – challenging the status quo and creating a new model that advances our field so more patients and caregivers can benefit from life-changing treatments. We do this by following the science, applying a novel rapid development approach, making innovative medicines at fair and reasonable prices, and creating a collaborative ecosystem to reach patients in ways that are most meaningful for them.
Our commitment and care for patients extends to our people, so culture is an essential cornerstone for Ultragenyx. We remain continuously focused on creating a supportive and inclusive environment of profound learning and growth – so employees can thrive in all areas of their lives, in and outside of work. Ultimately, we want to be an organization where we would be proud for our family, friends and children to work.
If you want to have a meaningful impact, do the best work of your career, and grow a lot, both professionally and personally, come join our team.
Position Summary:
Ultragenyx is seeking an experienced physician leader to serve as Executive Director, Global Medical Affairs & Strategy, providing strategic medical leadership across key rare disease programs, including LC‑FAOD, MPS VII, and MPS IIIA.
In this role, you will guide global medical strategy for both approved therapies (Dojolvi and Mepsevii) and an investigational AAV gene therapy for MPS IIIA. You will shape and drive integrated evidence generation, scientific communications, external engagement, global launch preparation, and lifecycle strategy across a diverse and evolving portfolio.
Reporting to the VP, Global & US Medical Affairs and Strategy, the Executive Director will operate as a trusted scientific and strategic leader—collaborating cross‑functionally, influencing decisively, and navigating the complexities of metabolic and neurodegenerative genetic diseases. This is a high‑impact, career‑defining opportunity for a medical affairs leader passionate about advancing the future of rare disease medicine.
ultrainnovative – Tackle rare and dynamic challenges
Work Model:
Remote: Officially documented as working full-time from home, with travel to Ultragenyx's offices or other locations on occasion as needed.
Responsibilities:
- Serve as Global Medical Lead for LC-FAOD (Dojolvi) and the MPS programs, including MPS VII (Mepsevii) and MPS IIIA (UX111)
- Lead the Global Medical Sub Teams (GMSTs), setting the strategy for the programs, ensuring cross regional alignment, and driving timely execution of annual medical plan
- Guide and oversee prelaunch planning, launch execution, and postlaunch optimization across the globe
- Serve as the strategic medical leader on the Program Core Team and related sub-teams, shaping development, commercialization, and lifecycle strategies. Work decisively to overcome challenges and drive program priorities forward
- Own, develop, and update the Integrated Evidence Generation Plans (IEPs) for all assigned programs ensuring cross functional alignment to address global/regional evidence gaps
- Oversee global Real World Evidence initiatives, including ISTs and collaborative research that inform clinical decision making and regulatory objectives.
- Accountable as the medical affairs lead for Disease Monitoring Programs for LC-FAOD and MPSs, providing strategic medical leadership on all aspects of the study and accountable for leading data generation and publication activities aligned to the IEP
- Lead advisory boards, steering committees, and investigator meetings to gather key insights that inform program strategy and activities
- Provide deep disease area and product expertise, ensuring accurate and consistent communication of clinical and scientific data
- Direct global publication strategy and congress planning activities, ensuring timely, high-impact dissemination of data and other education initiatives
- Lead training and provide strategic guidance and tools to regional medical affairs teams to ensure launch readiness and consistent scientific exchange
- Build and maintain relationships with global KOLs, academic centers of excellence, scientific societies and advocacy groups to inform strategy and amplify scientific reach
- Monitor competitors and landscape changes, translating insights into strategic actions.
Requirements:
- Medical Doctor (MD) required. MD, PhD strongly preferred. 7+ years of combined industry and academic experience preferred. Board Certification or equivalent in Clinical/Biochemical Genetics or Neurology preferred
- Proven expertise in the management of patients with inherited metabolic and/or neurodegenerative rare diseases
- Recognized scientific authority with the ability to engage in advanced scientific and clinical discussions with both internal and external experts
- Proven leadership skills including a passion for collaboration and understanding all stakeholder perspectives to drive alignment and decision-making
- High aptitude for attention to detail, commitment to high-quality work, and a sense of passion and urgency to achieve goals and improve the lives of patients
- Proven ability to take initiative and drive successful execution of complex projects
- Highly analytical and strategic thinker with the ability to identify interdependencies, consider medical implications, and understand the broader goals of a program and the organization
- Excellent communication skills, with the ability to effectively articulate scientific and strategic concepts in both written documents and verbal presentations
- Demonstrated success leading cross functional or multidisciplinary teams, ideally in both academia and biotech
- Willingness and ability to travel up to 15%, including domestic and international engagements
#LI-CS1 #LI-Remote
Full Time employees across the globe enjoy a range of benefits, including, but not limited to:
· Generous vacation time and public holidays observed by the company
· Volunteer days
· Long term incentive and Employee stock purchase plans or equivalent offerings
· Employee wellbeing benefits
· Fitness reimbursement
· Tuition sponsoring
· Professional development plans
* Benefits vary by region and country
Ultragenyx Pharmaceutical is an equal opportunity employer and prohibits unlawful discrimination based on race, color, religion, gender, sexual orientation, gender identity/expression, national origin/ancestry, age, disability, marital and veteran status, and any other status or classification protected by applicable federal, state, and/or local laws. Reasonable accommodation will be provided for all protected statuses or classifications protected by applicable law, including individuals with disabilities, disabled veterans, for pregnancy, childbirth, and related medical conditions, and based on sincerely held religious beliefs. Applicants can request an accommodation prior to accepting a job offer. If you require reasonable accommodation in completing this application, or in any part of the recruitment process, you may contact Talent Acquisition by emailing us at [email protected].
It is unlawful in Massachusetts to require or administer a lie detector test as a condition of employment or continued employment. An employer who violates this law shall be subject to criminal penalties and civil liability.
Note to External Recruiters: All candidate activity and open positions are managed strictly through our Human Resources Department. Our Human Resources Department kindly requests that recruiters not contact employees/hiring managers directly in an attempt to solicit business and present candidates. Please note that failure to comply with this request will be a factor in determining a professional relationship with our organization. Submission of unsolicited resumes prior to an agreement set in place between the Human Resources Department and the recruiting agency will not create any implied obligation. Inquiries on developing a recruiting relationship with us, may be directed to
: [email protected].