Associate Director, Analytical Development-Molecular (NGS, ddPCR)

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 individual to join the growing, dynamic multidisciplinary Analytical Development (AD) Department. The person will lead the Molecular Biology group with more than two talented scientists responsible for the exploring, development and optimization of phase-appropriate molecular assays for the lot release, stability testing and characterization of drug substance/drug product. The individual will be overseeing and establishing the adequate and value-added assay platforms suitable for the characterization and lot disposition of clinical and commercial gene therapy drug products.

She/he will implement feasible strategies and timelines together with the head of Analytical Development for effectively developing, qualifying the robust molecular assays under phase-appropriate GMP by collaborating with the AveXis R & D and QC teams (and/or CROs) in support of development and commercialization of AveXis/Novartis new gene therapy medicines. The individual will serve as tech lead for the assay transfers both for outsourcing and internal transfer. He/she will also represent the AD team in cross-functional team meetings and be critical part of decision-making processes. He/she will be the key member to support the head of AD and the site leadership team in building the positive culture and continued success of AD, in advancing pipeline projects from IND to commercialization.

This role will involve mentoring the growth of lead scientists to their next level by delegating tasks and providing appropriate advice in experimental design, data analysis and presentation. The individual will serve as SME and key decision maker on behalf of AD for topics related to the advancement of robust molecular assays, in cross-functional teams and external team meetings. The individual will also work closely with on-site project managers and global project management organization to support timely communication and ensure project meet development timelines and critical milestones.

Responsibilities

• Lead, in conjunction with Senior Director of Analytical Development, the Molecular Biology function in establishing near-term and long-term overall strategies, goals, execution plans and timelines, as well as resource allocations and budget planning.
• Key driver to oversee the exploring, development, optimization and qualification/validation of robust molecular assays
  • For example, NGS, qPCR, ddPCR and/or other molecular assay platforms to evaluate DNA characteristics such as genomic integrity and residual packaged impurity DNA, suitable for the characterization and lot disposition of AveXis' AAV-based gene products.
  • Expertise will be based upon:
    • Thorough understanding of relevant molecular assay platforms and applicable bioinformatics tools.
    • Extensive knowledge and strong scientific background related to the products and underlying biology, applicability of the emerging or existing molecular assay platforms and phase-appropriate GMP requirements.
  • NGS experience and molecular biology lab set up experience are highly preferred.
• Establish and maintain an effective analytical laboratory with full capability to carry out the molecular and other analytical methods. The responsibilities include, but not limited to, overseeing the budget of the Molecular Biology group, ordering and setting up the laboratory.
• The key liaison to work closely with AveXis AD- Chemistry, AD-Bioassay, R & D, QC groups and project management organization for win-win collaborations in implementing molecular assays and characterizing the key attributes of new gene therapy pipeline products.
• Represents the analytical team to attend cross-functional CMC and project meetings, and to actively participate in strategy design, execution, data presentation and follow-up.
• Authors technical reports and SOPs and assists the Head of the Analytical Development group to prepare analytical sections of regulatory filings.
• Present data in major conferences and write/submit manuscripts which will result in win-win achievements for both AveXis and the gene therapy community.

Qualifications

• Ph.D. in Molecular Biology or related discipline, such as Immunology, Cellular Biology or Genetics
• Min 10 years of biopharma industry experience with demonstrated track record of successfully developing different molecular biology analytical methods (e.g., next-generation sequencing (NGS) platforms, Sanger sequencing assays, DNA/RNA isolation, primer design, end-point PCR, qPCR, qRT-PCR, droplet digital PCR (ddPCR), , )
• Next-generation sequencing (NGS) experience and proficiency with various molecular biology and sequence analysis software. (e.g., Miseq sequencer, Geneious, Applied Biosystems 7500, QuantaSoft) is required.
• Experiences with bioinformatics programming (e.g., Python, R, etc.), is a plus
• Experiences with analytical and/or statistical software (e.g., JMP, Softmax Pro, XLfit, Gen5 etc.), is highly beneficial.
• Familiarity with virology is a plus.
• GxP experience is preferred, but not required.
• Experience in starting up/constructing a molecular, in particular NGS laboratory, in support of the characterization and lot release of clinical and/or commercial product, is highly beneficial.
• Basic understanding of regulatory agency guidance such as FDA, ICH and EMA and hands-on experience, is a plus.
• Technical writing skills and experience in authoring development reports and SOPs, is required; prior experience in regulatory filings is a plus.
• Ability to work in a fast-paced, team-oriented environment, to prioritize tasks from multiple projects, and high acumen to organize time-lines/be cognizant of project deadlines.
• Proven track record to lead a highly effective team.
• 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.

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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