Global Medical Expert - Gene Therapies

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: ultra focused – Work together to fearlessly uncover new possibilities 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. The company is in position to launch two gene therapies over the next year, requiring insightful education of clinical centers implementing gene therapy, including dosing, administration, and immunomodulation. A successful candidate will educate treating sites as part of the Gene Therapy Treatment Team to ensure that patients with these diseases receive their one-time therapy in the most effective manner and achieve their optimal therapeutic outcome. The position reports into the VP Global Medical Expert – Gene Therapies within the Medical Affairs organization. The Gene Therapy Treatment Team is an innovative high-profile team that utilizes a “hands on” adaptable approach. The successful candidate will be comfortable working in a fast-paced intellectual environment, understand patient care including immunomodulation, and is able to work at the interface between clinical development and medical affairs leveraging their expertise to advance our mission to change the future of rare disease medicine. 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 medi ... (truncated, view full listing at source)