300 million people worldwide are affected by rare genetic diseases. Since 2013, Novartis Gene Therapies (formerly AveXis) has one focus: bringing change to those devastated by a rare genetic disease which frequently affect the nervous system with chronic, progressive, and degenerative pathology that disproportionately (~ 83%) affects children.
What you'll be doing; * Provide clinical leadership and medical strategic input for all clinical deliverables (e.g. clinical sections of individual protocols, Integrated Development Plans, clinical data review, regulatory documents/registration dossiers, and publications). * Lead development of clinical sections of trial and program level regulatory documents (e.g., Investigator's Brochures, briefing books, safety updates, submission dossiers, and responses to Health Authorities) * May act as study medical monitor * Support (Sr.) GPCH in ensuring overall safety of the molecule for the assigned section, may be a core member of the Safety Management Team (SMT), and support overall program safety reporting in collaboration with Patient Safety * As a medical expert, support the (Sr.) GPCH or TAH in interactions with external stakeholders (e.g., regulatory authorities, key opinion leaders, data monitoring boards, advisory boards, patient advocacy groups), internal stakeholders (e.g., CTT, Research, Translational Medicine, Global Medical Affairs, Marketing, HE∨), and internal decision boards * May work with NIBR (Novartis Institute of Biomedical Research)/ Translational Medical Sciences) to drive transition of pre-PoC (Proof of Concept) projects to DDP (Development Decision Point) and with BD&L (Business Development & Licensing) including target identification and due diligences together with other medical matters, as needed * Ensure career development of Program reports and other clinical colleagues through active participation in the performance management and talent planning processes. Provides on-boarding, training, & mentoring support * Gene Therapy