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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. 

Remote: Officially documented as working full-time from home, with travel to Ultragenyx's offices or other locations on occasion as needed.

• 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 Therapies Treatment Team Framework  

• Mentor and develop Global Medical Expert physicians and MSLs, helping build a scalable, highperforming medical organization to support current and future gene therapy launches 

• Provide expertise and contribute as needed to key regulatory agency communications, including filings and information request responses 

• Represent Ultragenyx at key scientific and external stakeholder meetings to advance awareness and deep understanding of our science and therapies 

• As an expert, lead 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 

• Medical Doctor (MD required, MD plus PhD preferred) with Board Certification (or equivalent) in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology or Neurology (required) 

• Academic/Faculty experience (+7 years)with a track record of high-quality publications 

• Pharmaceutical experience in drug development and clinical trials or Medical Affairs (+7 years) 

• Experience in the management of patients receiving systemic AAV gene therapy in the clinical trial and/or post FDA approval setting 

• Demonstrated ability to prioritize and lead complex postapproval gene therapy activities, applying sound scientific judgment while enabling crossfunctional decisionmaking 

• Demonstrated ability to lead through ambiguity and make strategic medical decisions in first or bestinclass therapeutic settings 

• Availability for some out-of-hours work is an expectation 

• Demonstrated success working in a cross functional or multidisciplinary settingis essential 

• An established broad network of key experts and leaders within the rare disease community 
• Travel: 25-50% but may reduce with time as launches progress and team grows   

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