Scientist, Technical Development – Upstream

Overview

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. The U.S. Food and Drug Administration, or FDA, has granted AVXS-101 Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation for the treatment of SMA Type 1. In addition to developing AVXS-101 to treat SMA Type 1 and Type 2, we also plan to develop other novel treatments for rare neurological diseases, including Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

The Scientist is responsible for contributing to the development of the upstream manufacturing platform and generating/compiling data and reports to support approval of AveXis gene therapy products. This role will support activity with internal and external organizations to plan, execute, and document experiments that define the process and transfer of the process to the clinical and commercial sites. The Scientist will provide support for process, analytical, and characterization knowledge related to the production of AveXis gene therapy products, and the raw materials needed to make them.

Responsibilities of primary importance are to support the completion of time-sensitive studies and validation at internal and external partners while capturing related data and knowledge, to advance the development of AveXis products from research and development to GMP manufacturing.

Responsibilities

• Support Upstream Process Owner team on process-related issues at internal AveXis sites, as well as with external partners.
• Support trending and monitoring of critical quality attributes/ process parameters to maintain product quality and to control process drift.
• Assist in identifying and implementing potential process improvements in conjunction with manufacturing operations.
• Assist in start-up efforts of new equipment, software or processes in manufacturing.
• Author and support documentation of changes/updates to manufacturing processes.
• Review and provide feedback and technical/scientific support on project deliverables (e.g. remediation initiatives, plan reports, etc.).
• Support investigations in partnership with Manufacturing, Quality, and other business units at the site, to determine root cause for variation, implement solutions, and ensure corrections are effective. Participate in the collection and interpretation of data, and collaboration with other departments on manufacturing related issues to support issue resolution.
• Contribute to the downstream portion of the platform processes, planning to avoid delays, and supporting the activity and team to deliver a robust and effective product and manufacturing process.
• Complete requisite training on all applicable policies and procedures related to the job function.

Qualifications

• Minimum B.S. degree in biochemistry, chemical engineering, bioengineering, or related technical field, Master's degree or PhD preferred.
• Excellent oral and written communication skills; demonstrated technical writing.
• 4-6 years of experience in biopharmaceutical based GMP manufacturing operations and/or process development.
• Experience with mammalian cell culture, cell expansion, and bioreactor technologies.
• Experience with data analysis/interpretation to progress development strategies.
• Working knowledge in DOE and/or statistics is a plus.
• Approximately 10% to 25% travel may be required.

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.

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