Global Medical Expert - Gene Therapies

Ultragenyx Pharmaceutical

1d•Remote

About The Position

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.

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

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