Senior Director, 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 curious – Apply your biggest ideas in courageous ways Ultragenyx is seeking a dynamic and experienced physician to join the high-profile and innovative Gene Therapy Treatment Team in EMEA (Europe, Middle East and Africa). The physician will play a key medical affairs leadership role within the gene therapy portfolio, initially focused on Mucopolysaccharidosis Type IIIA and Glycogen Storage Disease Type 1a. This position will be instrumental in ensuring scientifically rigorous, compliant, and effective implementation of one - time gene therapies in EMEA through both pre-approval managed access programs and post-approval commercial treatment. Located in EMEA and reporting to the U.S. based VP Global Medical Expert (GME) – Gene Therapies, the Senior Director will partner closely with the EMEA and U.S Medical teams as well as Clinical Development, Safety, CMC, and Commercial leadership to facilitate medical access and enable treatment of patients with gene therapies. The role requires extensive peer-to-peer interactions with external physicians providing gene therapy treatment to enable successful medical onboarding of treating sites, while also leading education and scientific exchange on safety, efficacy, patient management, including immunomodulation, and outcomes. The successful candidate will be comfortable working in a fast-paced intellectual environment, leverage their direct experience in patient care and gene therapy, and can work effectively at the interface between clinical development and medical affairs both regionally and globally leveraging their expertise to advance our mission to change the future of rare disease medicine. Work Model: Flex: This role will typically require onsite work 2-3 days each week, or more depending on business needs. In many locations, the business will set certain days each week that Flex employees are required to be onsite. Responsibilities: Serve as a senior external medical leader for Ultragenyx gene therapies, leading compliant complex, scientific discussions with treating physicians in EMEA Enable successful implementation of gene therapies in EMEA both within Ultragenyx and with external medical experts Respond to inquiries and questions from treating sites in EMEA with appropriate level of urgency Lead the strategic and scientific development of educational materials and training for the gene therapy treatment centers in EMEA to ensure those centers are well prepared to manage patients throughout their gene therapy treatment journey Provide expertise and contribute as needed to country-level regulatory agency submissions, and data generation initiatives Advise on str…