Associate Director, Process Science (Analytical Science) -Gene Therapy

Overview

Ready to join a team committed to developing and commercializing gene therapies for patients and families devastated by rare neurological genetic diseases? AveXis is advancing cutting-edge science, starting with our proprietary gene therapy for the treatment of spinal muscular atrophy (SMA). We are in the midst of an incredible journey and are looking for passionate individuals to join us on this important mission.

AveXis, a Novartis company, is dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Our initial product is a proprietary gene therapy approved by the US Food and Drug Administration for the treatment of pediatric patients with SMA. In addition to developing a treatment for SMA, AveXis also plans to develop other novel treatments for rare neurological diseases, including Rett syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene.

AveXis is seeking an experienced and exceptional Associate Director to join the dynamic and growing analytical team to establish bioassays suitable for lot disposition and characterization of gene therapy drug products and substances.

The Associate Director will oversee testing and support the design and development of tests, studies, procedures, and specifications for the AveXis pre-clinical material programs. This position will lead a group of staff who will have expertise in execution of analytical testing. Responsibilities will also include execution of complex assays and supporting lab operations. The successful candidate will have extensive experiences in AAV analysis and will be adept and perceptive with passion for new technology and science.

Responsibilities

• Oversees/manages pre-clinical testing labs working closely with the Analytical Development, Quality Control, MS&T, and Manufacturing organizations.
• Perform complex experiments in support of process development using a variety of techniques covering cell biology, biochemistry and molecular biology such as immunocytochemistry, western blot, PCR, ddPCR, flow cytometry, and amino acid analysis.
• Optimize and troubleshoot assays using a variety of platforms including but not limited to ELISA, MSD, high-content imaging and luminescent/fluorescent plate reader in support of developing release and characterization assays for gene therapy products.
• Thoroughly document optimization and troubleshooting processes and develop methods and procedures for the team to use.
• Actively engage in planning and designing of assays/experiments and ownership of assays/experiments one is performing by understanding the purpose, rigidly following through every step of execution, thoroughly analyzing data and ensuring the data quality.
• Act as the subject matter expert to support investigations and resolution of out-of-specification (OOS) and out-of-trend (OOT) testing results.
• Collaborate with Analytical Development for knowledge transfer during assay development and work closely with Quality Control for testing of pre-clinical material.
• Participate in establishment of drug substance and drug product specifications for IND, IMPD, and BLA submissions.
• Author protocols, reports, and analytical sections of regulatory submissions (IND, IMPD, BLA).
• Recruit develop and mentor staff providing support to ensure successful accomplishment of business goals.

Qualifications

• B.S, M.S, or PhD in biology discipline is required.
• Minimum 12 years of relevant experience in pharmaceutical testing laboratory is required for B.S; minimum 10 years of biopharma industry experience is required for M.S.; minimum 8 years of biopharma industry experience is required for PhD.
• Solid understanding of regulatory agency guidance such as FDA, ICH and EMA and hand-on experience.
• Demonstrated track record of successfully optimizing and executing AAV quantification using various assay platforms including but not limited to HPLC, Mass Spectrometry, ELISA, MSD, ddPCR and high-content imaging.
• Broad experiences and demonstrated success in cellular, biochemical and molecular biological methods including but not limited to immunostaining, flow cytometry, protein gel, Western blot, PCR and qPCR.
• Prior experience with biologics, Analytical method development, QC, stability, IND submissions, BLA or NDA submissions is important for success in the position.
• Understanding of applicable USP, ICH, and Ph Eur regulations as they relate to QC testing is highly desirable.
• Enjoy learning and challenges. Must be able to learn new instruments and master their software quickly.
• Ability to work in a fast-paced small but growing team environment and to prioritize work from multiple projects. Must be highly reliable and goal/deadline oriented.
• Demonstrates a respect for others and is open to new/different ideas.
• Must have exceptional organizational skills.
• Excellent written and verbal communication skills.

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.

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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.