Vice President, Global Medical Expert – Gene Therapies at Ultragenyx in

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:

ultraimpact – Make a difference for those who need it most

Ultragenyx is seeking a dynamic and experienced physician leader to create a Gene Therapy Treatment Team to bring the application of gene therapy to patients in the commercial setting at launch. The leader 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 2 years in the US requiring insightful management of clinical centers implementing gene therapy, including dosing, administration, and immunomodulation. A successful candidate will assure that patients with these diseases receive their one-time treatment in the most effective manner and are managed to achieve their optimal therapeutic outcome.

The position reports into the VP Global/US Medical Affairs and Strategy within the Medical Affairs organization. This is an innovative high-profile position that requires a “hands on” adaptable and clinically capable leader, comfortable working in a fast-paced intellectual environment, who understands patient care, immunomodulation and will 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:

Establish and lead a small effective Gene Therapy Treatment Team to provide medical expertise directly 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
Provide strong scientific and medical leadership to ensure the successful implementation of gene therapies in the commercial setting
Proactively identify and take a “hands on” approach to resolve clinical challenges and prevent decisions which impact the optimal outcomes for patient, and compliantly deliver timely scientific information and education to peers to inform clinical decision-making
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 communication, 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
Oversee newborn screening development and implementation
Advise on and/or lead 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 (MD PhD preferred) with Board Certification (or equivalent) in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology or Neurology (required)
Proven expertise in the management of patients receiving systemic AAV gene therapy, including immunomodulation, in the clinical trial and/or post FDA approval setting
"Hands-on leader" 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 communication, onsite support to clinical teams requesting medical affairs input, triage and as needed directly provide robust and compliant information 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 (+10 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: 50-70% 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$315,400—$389,700 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 talentacquisition@ultragenyx.com.

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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.