Position Summary:

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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 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 and EMEA Regional Medical Affairs Leads to develop and implement the EMEA Gene Therapies Treatment Team Framework  

• Lead education initiatives for the broader EMEA regional medical affairs and cross functional teams preparing or potential gene therapy launches 

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

• Stay abreast of internal and external developments, trends and dynamics and provide direction for strategic implications to the development and commercialization of gene therapy  

Requirements:

• Medical Doctor (MD, MBBS or equivalent with PhD preferred) with subspecialty training and certification in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology or Neurology (required) 

• Academic/Faculty experience (5+ years) with a track record of high-quality publications   

• Pharmaceutical experience in drug development and clinical trials or Medical Affairs (3+ years) 

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

• Demonstrated ability to prioritize and lead complex post‑approval gene therapy activities, applying sound scientific judgment while enabling cross‑functional decision‑making 

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

• Demonstrated success working in a cross functional or multidisciplinary setting is essential 

• An established broad network of key experts and leaders within the rare disease community  

• Travel: Estimated at 25%-35% but may reduce with time as launches progress and team grows  

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