Global Medical Expert - Gene Therapies

Position Summary:

ultrafocused – Work together to fearlessly uncover new possibilities

Ultragenyx is seeking a dynamic and experienced physician to join the Gene Therapy Treatment Team to bring the application of gene therapy to patients in the commercial setting at launch.  The physician 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 year, requiring insightful education of clinical centers implementing gene therapy, including dosing, administration, and immunomodulation. A successful candidate will educate treating sites as part of the Gene Therapy Treatment Team to ensure that patients with these diseases receive their one-time therapy in the most effective manner and achieve their optimal therapeutic outcome.  

The position reports into the VP Global Medical Expert – Gene Therapies within the Medical Affairs organization. The Gene Therapy Treatment Team is an innovative high-profile team that utilizes a “hands on” adaptable approach. The successful candidate will be comfortable working in a fast-paced intellectual environment, understand patient care including immunomodulation, and is able to 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:

• Provide medical education 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 

• Proactively identify barriers to treatment and gaps in knowledge at treating sites and provide scientific information and education tailored to each site’s needs 

• Respond to inquiries and questions from treating sites with appropriate level of urgency 

• 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 communications, 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 

• Advise on 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 required with Board Certification preferred in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology or Neurology (required) 

• Experience in the management of patients receiving systemic AAV gene therapy in the clinical trial and/or post FDA approval setting  

• Proactive team player 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 communications, support to clinical teams requesting medical affairs input, triage and compliant site support 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 (+5 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: 25-50% but may reduce with time as launches progress and team grows  

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