Ready to join a team committed to moving gene therapies into the clinical and commercial settings for patients and families devastated by rare neurological genetic diseases? AveXis is advancing cutting-edge science to treat rare and life-threatening genetic diseases starting with our clinical-stage, proprietary gene therapy candidate, AVXS-101 (ZOLGENSMA®). We are in the midst of an incredible journey and are looking for passionate individuals to join us on this important mission.
AveXis, Inc., a Novartis Company, is a clinical-stage gene therapy company, dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Our initial product candidate, AVXS-101, is our proprietary gene therapy product candidate currently in development for the treatment of spinal muscular atrophy, or SMA, Type 1, the leading genetic cause of infant mortality, and for the treatment of SMA Type 2. It has been granted Orphan Drug and Fast Track Designations by the FDA, and is currently under review by the FDA, EMA and PMDA. We have product candidates for other diseases that are approaching first in human studies. AveXis is a subsidiary of Novartis that is being run as an independent entity, leveraging resources from Novartis where it makes sense.
AveXis is seeking a Medical Director who will, as part of the Translational Medicine team in R&D, be responsible for the successful translation of gene therapy products from the pre-clinical proof of concept stage in R&D to first-in human trials. This includes ensuring trial readiness with regards to the optimization of natural history dataset, biomarkers and clinical outcomes. This also includes stakeholder engagement with clinicians, researchers and patient groups, as well as the design of potentially label-enabling first-in-human trials. Building on AveXis gene replacement therapy technology platform, the Translational Medicine team drives the company’s IND engine to accelerate the development of novel treatments for patients suffering from one of the many rare and life-threatening neurological genetic diseases.
The current focus of the role is on the company’s programs that are nearing IND (AVXS-201 for Rett syndrome and AVXS-301 for genetic amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene). Starting in 2019, it is anticipated that there will be 2 – 5 gene therapy programs in the clinic each year.
Travel is estimated to be 30%, most being domestic and some international.
The level of this position will be based on the final candidate’s qualifications.
Please note this job description is not designed to cover or contain a comprehensive listing of activities, duties or responsibilities that are required of the employee for this job. Duties, responsibilities and activities may change at any time with or without notice.
AveXis is committed to creating a diverse environment and is proud to be an equal opportunity employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, national origin, genetics, disability, age, sexual orientation or veteran status.