Senior Director, Formulations and Pharmaceutics Research

Employer
AveXis Inc.
Location
San Diego, CA, US
Posted
Feb 07, 2019
Ref
2019-2873
Hotbed
Biotech Beach
Required Education
Doctorate/PHD/MD
Position Type
Full time
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. 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 Director is responsible for leading the formulation studies of pipeline gene therapy products from preclinical through early phase clinical development and lifecycle enhancements. A key accountability of this position is to develop strategic formulation plans consistent with program strategies and provide managerial leadership of external resources to successfully execute formulation studies to support AveXis programs. The successful candidate will work seamlessly with CRO/CMO to ensure robust formulation and stability studies are conducted on time and on budget. This person will also need to design appropriate analytical experiments and assays. Formulation strategy, work-in-process status and results will need to be summarized in writing and presented on occasion to R&D, manufacturing sciences and technology (aka Process Development), Regulatory and Quality groups.

Responsibilities

  • Develop and execute the scientific and technical strategy for formulations development.
  • Collaborate with research/discovery, regulatory, quality, clinical development, manufacturing, and commercial to develop state of art formulations and stability programs.
  • Manage analytical testing of novel gene therapy formulated products from designed screening and stability experiments.
  • Contribute to novel analytical method generation in collaboration with Analytical Development and Research and Development scientists.
  • Formulation and drug product development: develop stable formulations such as liquid or lyophilized; assess product stability during the manufacturing, handling, storage (including container closure) and transportation of the DS and DP.
  • Product development such as combination product (with device and/or other drugs/materials) and room temperature lyophilized product to enhance clinical performance world-wide access.
  • Work with analytical development (and CROs) to support the entire process development efforts with key characterization regarding QC release and stability assays. Under the quality by design (QbD) paradigm, define critical quality attributes (CQA) and critical process parameters (CPP) necessary for process characterization and validation.
  • Provide necessary process development support for the internal and external pilot (non GMP) production.
  • Responsible for reviewing, writing, and editing of technical reports and regulatory documents to support IND and BLA filings.


Qualifications

  • Ph.D. in Biology, Biochemical Engineering or related fields with a minimum of 15 years of experience in the development of cell and gene therapy or other biologics is required.
  • Strong background in biologics process development is required.
  • Hands on experience in one or more biologics process development field such as upstream, downstream, analytical and formulation and CMC regulatory is required.
  • Previous senior leadership experience directing biologics process development is required.
  • Must be familiar with GMP regulations and compliance standards governing the manufacturing of biologics.
  • Ability to manage priorities and make decisions in a fast-paced environment while ensuring high quality deliverables are achieved in a timely fashion.
  • Strong interpersonal skills to influence across multiple functions and interact collaboratively within the team environment.
  • Excellent oral and written communication skills including presentation development and delivery.


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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