Executive Director, 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 join the Global Medical Affairs team to lead the implementation of gene therapy programs at launch. The leader will play a key role within the gene therapy portfolio, initially focused on Inborn Errors of Metabolism. The company is launching over the next 2 years two gene therapies in the US requiring insightful management of clinical centers implementing gene therapy.

Reporting to the VP Global/US Medical Affairs and Strategy, this leader will partner closely with the US Field Medical, Global/US Medical Strategy Leads and Clinical Development through a largely externally facing role which requires peer-to-peer interactions with KOLs who are conducting gene therapy treatments. It will also involve clinical and scientific education, evidence creation and dissemination and addressing information needs of physicians treating patients as well as patient organizations or others who may be stakeholders for commercialized Ultragenyx gene therapies. This in an innovative high-profile position that requires a “hands on” adaptable and clinically capable leader, comfortable working in a fast-paced intellectual environment, understanding patient care, immunomodulation and collaborating 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:

Be a core member of the Medical Affairs Gene Therapy Treatment Team, collaborating closely with Clinical Development to proactively identify and resolve challenges, and compliantly deliver timely scientific information and education regarding the use of gene therapies to inform clinical decision-making. We need every site doing these critical therapies to be well prepared to conduct treatment and manage treated patients
Collaborate closely with Global/US Medical Leads and US Field Medical team across the Inborn Errors of Metabolism (IEM) portfolio to ensure execution of medical plans by leading/supporting initiatives to expand the knowledge of KOLs and centers of excellence
Provide the leadership both internally and externally to drive successful implementation of gene therapy and not just be a passive contributor
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
Through partnership with Patient Advocacy, Government Affairs and Market Access teams, meet the engagement and education needs of stakeholders relevant to patient education, advocacy and access
Represent Ultragenyx at key scientific meetings to achieve conference strategy objectives including: leading engagement meetings with KOLs, gathering and sharing of strategic insights, executing education initiatives, presenting data
Stay abreast of internal and external developments, trends and dynamics and provide direction for strategic implications to the development and commercialization of gene therapy
Play a leadership role in Ultragenyx training and education on relevant information through partnership with global medical and commercial training teams
Advise on and/or lead strategic development of medical materials to support external medical engagement and publications
Advise on life-cycle management for label/indication expansion
Oversee newborn screening development and implementation
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)
Academic/Faculty experience (+10 years) with a track record of high-quality publications
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
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
To ensure the continuum of peer-to-peer clinical and experiential conversations, the continuance of up to one day a month in a clinical practice setting
Travel: 50-70% #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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