Executive Director, Global Medical Expert, Gene Therapies

Summary

Join Ultragenyx and lead a Gene Therapy Treatment Team, making a difference for rare disease patients. This high-profile position requires a hands-on physician leader to ensure successful gene therapy implementation in the commercial setting. You will establish and lead a team, resolve clinical challenges, represent Ultragenyx at key meetings, and contribute to regulatory communications and protocol development. The role involves overseeing newborn screening, advising on publications, and supporting internal team education. This is a remote position with travel, offering a competitive salary and a comprehensive benefits package.

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

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

Preferred Qualifications

• 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

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