Global Medical Expert - Gene Therapies

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: ultrafocused – Work together to fearlessly uncover new possibilities Ultragenyx is seeking a dynamic and experienced physician to join the Gene Therapy Treatment Team to bring the application of gene therapy to patients in the commercial setting at launch. The physician will play a key role within the gene therapy portfolio, initially focused on Inborn Errors of Metabolism. The company is in position to launch two gene therapies over the next year, requiring insightful education of clinical centers implementing gene therapy, including dosing, administration, and immunomodulation. A successful candidate will educate treating sites as part of the Gene Therapy Treatment Team to ensure that patients with these diseases receive their one-time therapy in the most effective manner and achieve their optimal therapeutic outcome. The position reports into the VP Global Medical Expert – Gene Therapies within the Medical Affairs organization. The Gene Therapy Treatment Team is an innovative high-profile team that utilizes a “hands on” adaptable approach. 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: Provide medical education to the gene therapy treatment centers to ensure those centers are well prepared to manage patients throughout their gene therapy treatment journey, including the administration process and the post-treatment monitoring including the use of immunomodulation Proactively identify barriers to treatment and gaps in knowledge at treating sites and provide scientific information and education tailored to each site’s needs Respond to inquiries and questions from treating sites with appropriate level of urgency Represent Ultragenyx at key scientific and external stakeholder meetings to advance awareness and deep understanding of our science and therapies Provide expertise and contribute as needed to key regulatory agency communications, including filings and information request responses 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 support external medical engagement As an expert, support 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  Serve as a key therapeutic area advisor to medical and other functions including providing advice to the Business Development and Translational Research on feasibility, unmet medical need and Target Product Profiles for potential new products Requirements: Medical Doctor required with Board Certification preferred in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology or Neurology (required) Experience in the management of patients receiving systemic AAV gene therapy in the clinical trial and/or post FDA approval setting  Proactive team player who is ready to get involved and take action in all aspects of post-approval gene therapy team collaboration, including but not limited to cross functional field team meetings and site communications, support to clinical teams requesting medical affairs input, triage and compliant site support to enable timely and informed clinical decision making Availability for some out-of-hours work is an expectation Demonstrated success working in a cross functional or multidisciplinary setting is preferred Academic/Faculty experience (+5 years) with a track record of high-quality publications An established broad network of key experts and leaders within the rare disease community  Pharmaceutical experience in drug development and clinical trials or Medical Affairs desirable but not required  Travel: 25-50% but may reduce with time as launches progress and team grows #LI-AM1 #LI-Remote 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. See our CCPA Employee and Applicant Privacy Notice. See our Privacy Policy. 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.