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 Therapies Treatment Team Framework  

• Mentor and develop Global Medical Expert physicians and MSLs, helping build a scalable, high‑performing 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  

Requirements:

• 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 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 

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

• 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: 25-50% but may reduce with time as launches progress and team grows  

#LI-CS1 #LI-Remote

The typical annual salary range for this full-time position is listed below. This range reflects the characteristics of the job, such as required skills and qualifications and is based on the office location noted in this job posting. The range may also be adjusted based on applicant's geographic location.

This position is eligible for annual bonus and equity incentives. Actual individual pay is determined by demonstrated experience and internal equity alignment.

Pay Range$328,400—$405,700 USD  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 [email protected].

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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: [email protected].

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