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Role Description

Ultragenyx is seeking a dynamic and experienced physician to join the Gene Therapy Treatment Team to bring the application of gene therapy to patients in the commercial setting at launch. The physician will play a key role within the gene therapy portfolio, initially focused on Inborn Errors of Metabolism.

• 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.

Qualifications

• Medical Doctor required with Board Certification preferred in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology or Neurology.
• 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.
• 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.

Requirements

• 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.
• Benefits vary by region and country.