Executive Director, Global Medical Expert

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:

ultradedicated – Your biggest challenges yield rare possibilities

Ultragenyx is seeking a dynamic and experienced physician to join the Gene Therapy Treatment Team. The physician will play a key medical affairs leadership role within the gene therapy portfolio, initially focused on GSDIa and MPS IIIA, where the company is in position to launch two gene therapies this year. This position will be instrumental in providing clinical and scientific education to gene therapy treating centers to ensure that patients receive their one-time therapy in the most effective manner and achieve their optimal therapeutic outcome.  

Reporting to the VP Global Medical Expert – Gene Therapies, the Executive Director is a senior medical affairs leader responsible for ensuring scientifically rigorous, compliant, and effective implementation of one-time gene therapies. The Executive Director will work closely with Medical Science Liaisons in the high-profile and innovative Gene Therapy Treatment Team through a largely externally facing role. The Executive Director also partners closely with Medical Affairs, Clinical Development, Regulatory, and Commercial leadership to enable successful launch and post launch adoption, while shaping external scientific dialogue around safety, durability, immunomodulation, and patient outcomes.   

The role requires extensive peer-to-peer interactions with external physicians providing gene therapy treatment in the commercial setting. 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:

• Serve as a senior external medical leader for Ultragenyx gene therapies, leading compliant complex, scientific discussions with treating physicians  

• Enable successful implementation of gene therapies globally both within Ultragenyx and with external medical experts 

• Respond to inquiries and questions from treating sites with appropriate level of urgency 

• Lead the strategic and scientific requirements for educational materials and lead the delivery of training for the gene therapy treatment centers to ensure those centers are well prepared to manage patients throughout their gene therapy treatment journey 

• 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 enable external medical engagement and address evidence gaps to inform clinical decision making 

• Work in partnership with VP, GME – Gene Therapies to develop and implement the global Gene The