Global Medical Expert - Gene Therapies

Position Summary:

ultra focused – Work together to fearlessly uncover new possibilities

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:

• Provide medical education to the gene therapy treatment centers to ensure those centers are well prepared to manage patients throughout their gene therapy treatment journey, including the administration process and the post-treatment monitoring including the use of immunomodulation
• Proactively identify barriers to treatment and gaps in knowledge at treating sites and provide scientific information and education tailored to each site’s needs
• Respond to inquiries and questions from treating sites with appropriate level of urgency
• Represent Ultragenyx at key scientific and external stakeholder meetings to advance awareness and deep understanding of our science and therapies
• Provide expertise and contribute as needed to key regulatory agency communications, including filings and information request responses
• Provide expertise and contribute as needed to protocol development, steering committee meetings, data generation initiatives of Disease Monitoring Programs, and life‑cycle management for label/indication expansion
• Advise on strategic development of publications and medical materials that support external medical engagement
• As an expert, support the education and training of Ultragenyx internal teams
• Stay abreast of internal and external developments, trends and dynamics and provide direction for strategic implications to the development and commercialization of gene therapy
• Serve as a key therapeutic area advisor to medical and other functions including providing advice to the Business Development and Translational Research on feasibility, unmet medical need and Target Product Profiles for potential new products

Requirements:

• Medical Doctor required with Board Certification preferred in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology, or Neurology (required)
• Experience in the management of patients receiving systemic AAV gene therapy in the clinical trial and/or post FDA approval setting
• Proactive team player who is ready to get involved and take action in all aspects of post‑approval gene therapy team collaboration, including but not limited to cross‑functional field team meetings and site communications, support to clinical teams requesting medical affairs input, triage and compliant site support to enable timely and informed clinical decision making
• Availability for some out‑of‑hours work is an expectation
• Demonstrated success working in a cross‑functional or multidisciplinary setting is preferred
• Academic/Faculty experience (+5 years) with a track record of high‑quality publications
• An established broad network of key experts and leaders within the rare disease community
• Pharmaceutical experience in drug development and clinical trials or Medical Affairs desirable but not required
• Travel: 25–50% but may reduce with time as launches progress and team grows

Benefits:

• 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

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

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.