Senior Scientist, Technical Development Laboratory – Downstream

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 Senior Scientist is responsible for contributing to the development and improvement of the downstream manufacturing platform processes used to manufacture, as well as the data and reports to support approval of, AveXis gene therapy products. This individual will lead and support activity with internal and external organizations to plan, execute, and document experiments and early manufacturing that define the process, and method of delivery to the clinical and commercial sites. The Scientist will provide leadership and 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 ensure the execution of time-sensitive experiments, studies, and validation at external partners while capturing related data and knowledge, to advance the development of AveXis products from Research to Development, and on to GMP manufacturing. Complete requisite training on all applicable policies and procedures related to the job function. The successful candidate will take responsibility to ensure all documentation and reports are accurate, complete, and suitable for using in support of production, characterization, and regulatory approval of AveXis products.

Responsibilities

• Provide scientific and technical input for process-related issues at internal Avexis sites, as well as with external partners.
• Performing trending and monitoring of critical quality attributes/critical process parameters to maintain product quality and to control process drift.
• Identifying and implementing potential process improvements in conjunction with manufacturing operations.
• Participating in start-up efforts of new equipment, software or processes in manufacturing.
• Assisting in documenting changes/updates to manufacturing processes and working with manufacturing, engineering and validation to implement those changes.
• Reviewing/providing feedback and technical/scientific support on project deliverables, i.e. remediation initiatives, plan reports, etc.
• Establishing small-scale production processes and using scaled-down lab processes to enable process troubleshooting.
• Participate in leading investigations in partnerships 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 all manufacturing related issues to drive issue resolution and support AveXis goals.

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.
• 6-10 years of experience in biopharmaceutical based GMP manufacturing operations including direct experience in cell culture.
• Previous experience with AAV manufacturing or Process Development is required
• Specific laboratory experience desired includes, but is not limited to:
• In depth knowledge of AKTA chromatography systems
• In depth knowledge of Chromatography, Depth Filtration, Tangential Flow Filtration, and Ultracentrifugation
• Familiarity with TFF scale up including flux, sheer, and TMP in hollowfiber and flat sheet membranes
• Familiarity with chromatography scale up
• Ability to effectively and efficiently analyze and interpret data to further progress development strategies.
• Working knowledge in DOE is a significant 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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