Director, Regulatory CMC
Company: AveXis Inc.
Posted on: June 18, 2019
OverviewReady 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.Oversees regulatory CMC activities and
maintains the regulatory CMC applications. Responsible for
executing the regulatory CMC strategy of submissions to the US FDA,
EMA, and other regulatory authorities as well as managing
interactions with regulatory authorities on CMC issues. Provide
leadership and direct oversight of a team of CMC regulatory
- Implement regulatory CMC strategy for early and late stage
clinical product candidates to attain rapid regulatory
- Lead and drive CMC content development for submission
activities (planning, authoring, reviewing, coordination,
submission) for original INDs, IND amendments, investigational
medicinal product dossiers (IMPD) for inclusion in Clinical Trial
Applications, BLAs and MAAs.
- Serve as RA CMC liaison with various internal functions (e.g.,
Manufacturing, MS&T, R&D).
- Participate in multidisciplinary efforts to prepare CMC updates
for regulatory documents including IND CMC updates, Regulatory
Authority meetings briefing documents, etc.
- Aid in development of regulatory dossier in support of AveXis
products in development for worldwide submissions.
- Respond to CMC queries to advance AveXis clinical
- Aid in establishing, managing, and maintaining a knowledge base
of current and emerging CMC regulatory requirements, guidelines,
and best practices.
- Assure compliance with regulatory standards and guidance
- Ensure that CMC-related changes are reported to authorities in
accordance with regulatory requirements.
- Conduct risk assessments of global CMC regulatory issues.
- Assist in building a world class Regulatory CMC function to
meet AveXis' growing pipeline.
- Maintains and continues to build AveXis' excellent reputation
with the regulatory authorities.
- Manages and mentors, providing leadership to two or more
employees who are direct reports.
- Develops departmental policies and oversees and/or authorizes
their implementation. Qualifications
- BS/MS degree in related discipline and a minimum of 12 years of
related experience with at least 5 years at a manger or above
- Experience and a proven track record of successes in biological
drug submissions (e.g., IND, BLA, MAA), is preferred.
- Experience with biologics required with experience in cell/gene
therapy drug development highly desirable.
- Knowledge of the drug development process and global
regulations and requirements.
- Strong analytical skills, problem solving ability and
- Flexibility and ability to prioritize and manage multiple tasks
- Effective oral and written communication skills.
- Effectively applies strong analytical and business
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. #LI-GK1
Keywords: AveXis Inc., Greenville , Director, Regulatory CMC, Executive , Durham, North Carolina
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