Executive Director, Global Medical Expert, Gene Therapies

Summary

Join Ultragenyx's Global Medical Affairs team as a physician leader to spearhead the launch of gene therapy programs, focusing initially on Inborn Errors of Metabolism. This role involves collaborating with internal and external stakeholders, including KOLs and clinical centers, to ensure successful implementation. Responsibilities include providing scientific information and education, leading initiatives to expand knowledge, contributing to regulatory communications, and advising on strategic development. The position requires a medical doctor with board certification and extensive academic/faculty experience, along with proven expertise in managing patients receiving AAV gene therapy. A remote work model is offered with occasional travel. Competitive compensation and a range of benefits are provided.

Requirements

• Medical Doctor (MD PhD preferred) with Board Certification (or equivalent) in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology or Neurology
• 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
• 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

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
• 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

Preferred Qualifications

• Demonstrated success working in a cross functional or multidisciplinary setting
• 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

Benefits

• 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
• Annual bonus
• Equity incentives