Senior Director, Global Medical Expert - Gene Therapies

Pharmaceutical experience in drug development and clinical trials or Medical Affairs (3+ years)

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

approval gene therapy activities, applying sound scientific judgment while enabling cross

Demonstrated ability to lead through ambiguity and make strategic medical decisions in first

class therapeutic settings

An established broad network of key experts and leaders within the rare disease community

Ultragenyx is seeking a dynamic and experienced physician to join the high-profile and innovative Gene Therapy Treatment Team in EMEA (Europe, Middle East and Africa). The physician will play a key medical affairs leadership role within the gene therapy portfolio, initially focused on Mucopolysaccharidosis Type IIIA and Glycogen Storage Disease Type 1a.  This position will be instrumental in ensuring scientifically rigorous, compliant, and effective implementation of one

time gene therapies in EMEA through both pre-approval managed access programs and post-approval commercial treatment.

Located in EMEA and reporting to the U.S.

based VP Global Medical Expert (GME) – Gene Therapies, the Senior Director will partner closely with the EMEA and U.S Medical teams as well as Clinical Development, Safety, CMC, and Commercial leadership to facilitate medical access and enable treatment of patients with gene therapies.

The role requires extensive peer-to-peer interactions with external physicians providing gene therapy treatment to enable successful medical onboarding of treating sites, while also leading education and scientific exchange on safety, efficacy, patient management, including immunomodulation, and outcomes.  The successful candidate will be comfortable working in a fast-paced intellectual environment, leverage their direct experience in patient care and gene therapy, and can work effectively at the interface between clinical development and medical affairs both regionally and globally leveraging their expertise to advance our mission to change the future of rare disease medicine.

Serve as a senior external medical leader for Ultragenyx gene therapies, leading compliant complex, scientific discussions with treating physicians in EMEA