
501 - 1000 funcionários
Fundada em 2010
🧬 Biotecnologia
💊 Farmacêutico
Biotechnology • Pharmaceuticals
A Ultragenyx é uma empresa biofarmacêutica dedicada a transformar a vida de pessoas com doenças genéticas raras e ultrarraras. A companhia foca no desenvolvimento e na disponibilização de tratamentos para condições em que não existem terapias atuais, utilizando uma variedade de modalidades terapêuticas adaptadas a cada doença. A Ultragenyx conta com um pipeline clínico robusto e diversificado, com terapias em desenvolvimento para doenças genéticas como a Doença de Wilson, a osteogênese imperfeita e a Síndrome de Angelman. Com uma equipe apaixonada e um compromisso com o advocacy de pacientes, a Ultragenyx busca liderar a vanguarda em terapias pioneiras que gerem impacto significativo nas comunidades de pacientes com doenças raras.
🕒 Abril 29
🇺🇸 Estados Unidos – Remoto (EUA)
💵 $328.400 - $405.700 / ano
⏰ Tempo Integral
🔴 Especialista
👔 Diretor
🦅 Patrocina Visto H1B
🗣️🇺🇸🇬🇧 Inglês obrigatório
Melhore suas chances de conseguir uma entrevista verificando sua pontuação de currículo antes de se candidatar.

501 - 1000 funcionários
Fundada em 2010
🧬 Biotecnologia
💊 Farmacêutico
Biotechnology • Pharmaceuticals
A Ultragenyx é uma empresa biofarmacêutica dedicada a transformar a vida de pessoas com doenças genéticas raras e ultrarraras. A companhia foca no desenvolvimento e na disponibilização de tratamentos para condições em que não existem terapias atuais, utilizando uma variedade de modalidades terapêuticas adaptadas a cada doença. A Ultragenyx conta com um pipeline clínico robusto e diversificado, com terapias em desenvolvimento para doenças genéticas como a Doença de Wilson, a osteogênese imperfeita e a Síndrome de Angelman. Com uma equipe apaixonada e um compromisso com o advocacy de pacientes, a Ultragenyx busca liderar a vanguarda em terapias pioneiras que gerem impacto significativo nas comunidades de pacientes com doenças raras.
• Serve as a senior external medical leader for Ultragenyx gene therapies, leading compliant complex, scientific discussions with treating physicians • Enable successful implementation of gene therapies globally both within Ultragenyx and with external medical experts • Respond to inquiries and questions from treating sites with appropriate level of urgency • Lead the strategic and scientific requirements for educational materials and lead the delivery of training for the gene therapy treatment centers to ensure those centers are well prepared to manage patients throughout their gene therapy treatment journey • 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 enable external medical engagement and address evidence gaps to inform clinical decision making • Work in partnership with VP, GME – Gene Therapies to develop and implement the global Gene Therapies Treatment Team Framework • Mentor and develop Global Medical Expert physicians and MSLs, helping build a scalable, high-performing medical organization to support current and future gene therapy launches • Provide expertise and contribute as needed to key regulatory agency communications, including filings and information request responses • Represent Ultragenyx at key scientific and external stakeholder meetings to advance awareness and deep understanding of our science and therapies • As an expert, lead 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
• Medical Doctor (MD required, MD plus PhD preferred) with Board Certification (or equivalent) in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology or Neurology (required) • Academic/Faculty experience (+7 years) with a track record of high-quality publications • Pharmaceutical experience in drug development and clinical trials or Medical Affairs (+7 years) • Experience in the management of patients receiving systemic AAV gene therapy in the clinical trial and/or post FDA approval setting • Demonstrated ability to prioritize and lead complex post-approval gene therapy activities, applying sound scientific judgment while enabling cross-functional decision-making • Demonstrated ability to lead through ambiguity and make strategic medical decisions in first or best-in-class therapeutic settings • Availability for some out-of-hours work is an expectation • Demonstrated success working in a cross functional or multidisciplinary setting is essential • An established broad network of key experts and leaders within the rare disease community • Travel: 25-50% but may reduce with time as launches progress and team grows
• 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
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