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