Ultragenyx Pharmaceutical
Senior Director, Global Medical Expert - Gene Therapies
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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:
ultracurious – 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.
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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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Full Time employees across the globe enjoy a range of benefits, including, but not limited to:
- Generous vacation time and public holidays observed by the company
- Volunteer days
- Long term incentive and Employee stock purchase plans or equivalent offerings
- Employee wellbeing benefits
- Fitness reimbursement
- Tuition sponsoring
- Professional development plans
Benefits vary by region and country
Ultragenyx Pharmaceutical is an equal opportunity employer and prohibits unlawful discrimination based on race, color, religion, gender, sexual orientation, gender identity/expression, national origin/ancestry, age, disability, marital and veteran status, and any other status or classification protected by applicable federal, state, and/or local laws. Reasonable accommodation will be provided for all protected statuses or classifications protected by applicable law, including individuals with disabilities, disabled veterans, for pregnancy, childbirth, and related medical conditions, and based on sincerely held religious beliefs. Applicants can request an accommodation prior to accepting a job offer. If you require reasonable accommodation in completing this application, or in any part of the recruitment process, you may contact Talent Acquisition by emailing us at talentacquisition@ultragenyx.com.
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It is unlawful in Massachusetts to require or administer a lie detector test as a condition of employment or continued employment. An employer who violates this law shall be subject to criminal penalties and civil liability.
Note to External Recruiters: All candidate activity and open positions are managed strictly through our Human Resources Department. Our Human Resources Department kindly requests that recruiters not contact employees/hiring managers directly in an attempt to solicit business and present candidates. Please note that failure to comply with this request will be a factor in determining a professional relationship with our organization. Submission of unsolicited resumes prior to an agreement set in place between the Human Resources Department and the recruiting agency will not create any implied obligation. Inquiries on developing a recruiting relationship with us, may be directed to: talentacquisition@ultragenyx.com.
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