Global Medical Expert - Gene Therapies

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

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

Long term incentive and Employee stock purchase plans or equivalent offerings

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.

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 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