AveXis Inc.

AveXis Inc. Illinois, USA
Mar 18, 2019
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 (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. 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. AveXis is seeking a Biostatistician to support statistical needs and provide statistical expertise to help guide strategy, design and execution of clinical development programs for a dynamic, fast-paced environment. This person will be a key contributor to study design and analysis of clinical data related to company's development programs and support of regulatory and publications functions. In this role, the selected candidate will participate in a variety of duties related to execution of biostatistics deliverables and guide biostatistics consultants/CRO activities. The successful candidate will ensure departmental goals and deliverables are met. This individual will plan, analyze, and summarize the results of individual clinical studies or groups of studies (integrated summaries). Responsibilities Efficiently and effectively coordinate statistical activities for multiple projects simultaneously, including providing expertise and guidance to relevant aspects of protocol development. Develop statistical analysis plans (SAPs), end of study clinical study reports (CSR), and other relevant biostatistics activities. Interact with project team, as well as key internal and external stakeholders. Oversee programming of, and/or review statistical tables, listings, figures, and analysis datasets for clinical trials in accordance with AveXis Standard Operating Procedures (SOPs) or study specific guidelines. Maintain awareness of project tasks and effectively communicate the status of such tasks to the Head of Biostatistics and Head of Clinical Development. Program in SAS and/or R to create output tables and/or listings as needed, including in presentation-ready tables, listings, and figures. Provide guidance for external (CRO) statisticians. Perform other responsibilities as required by the Department and participate in process improvement initiatives. Qualifications Master's or doctoral degree in biostatistics or statistics with 4+ years of experience to successfully perform the key responsibilities of the role. Experience in statistical design, analysis, and reporting methodology for basic and complex studies along with experience in contributing to statistical activities in clinical research. Excellent programming skills in SAS and R, including designing and developing output tables, listings, and figures. Experience with clinical and operational elements of pharmaceutical development. Understanding of the principles of ICH GCP and regulatory requirements is essential. Excellent analytical, verbal, and written presentation skills with a thorough working knowledge of SAS, R, and other relevant statistical software as well as working knowledge of Microsoft Office applications including Word, Excel, PowerPoint, and Visio. Excellent organizational and analytical skills, strong oral and written communication skills, and able to work in a high paced, timeline driven setting. Highly effective interpersonal skills and the ability to continually demonstrate poise, tact, and diplomacy are necessary. 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.
AveXis Inc. Durham, NC, USA
Mar 18, 2019
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 Manager, Incoming Quality Control serves as the owner for all work processes associated with the sampling and testing of incoming raw materials. This position oversees testing activities and ensures compliance with respect to sampling, methods, and procedures. Acts as a subject matter expert for Incoming Quality-related activities during regulatory inspections and as the document owner for procedures in support of the Quality Control Raw Material Programs. Responsibilities include, but are not limited to program management, technical writing, training, metrics, and investigations. A potential candidate will possess a strong understanding of material sampling, analytical techniques, manufacturing operations, GMPs and the principles behind them. The individual must be able to communicate effectively, lead investigations and identify/implement CAPAs. Responsibilities Coordinates and performs timely, accurate and cGMP compliant testing of incoming raw materials. Participates effectively with site functional teams including Manufacturing, Quality Assurance, Supply Chain and MS&T to achieve company objectives. Determines appropriate analytical confirmatory testing of raw materials. Ensures alignment with current relevant monographs. Executes the guidelines to ensure the company has a robust raw materials program. Executes proper investigation into the root cause of material and/or process failures and assists in determining appropriate material disposition and/or process improvements. Ensures that internal and external laboratories comply with cGMP standards. Collaborates with Quality, MS&T and/or outside contract labs to ensure seamless method qualification/validation and transfer. Identifies and qualifies lab equipment. Reviews and approves raw material qualification and testing documentation. Supports interviewing and training of employees. Qualifications Bachelor's degree in Chemistry, Biology or related sciences. Minimum 5 years of relevant experience in GMP testing laboratory with 2 years of supervisory experience. Strong understanding of cGMP regulations. Strong understanding of compendial and non-compendial raw materials analytical testing and associated instrumentation used to perform raw material testing. Strong problem-solving abilities. Good critical thinking, deductive reasoning, and decision-making skills. Deep experience with methods development and validation. Ability to work independently and effectively, prioritizing and delivering on tight timelines. Ability to work effectively within the site and across 3rd party testing laboratories. Approximately 10-15% travel 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. #LI-GK1
AveXis Inc. Illinois, USA
Mar 18, 2019
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 (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. 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. This intern role will allow for a glimpse into the day to day analysis and operational support related to the AveXis accounting and reporting activities. This role will work closely with broader Accounting and Reporting team to ensure accurate month end reporting, controls compliance, coordination of the close process and preparation of materials for review of financials with management. Responsibilities Contribute to month-end financial closing and reporting process. Account Reconciliation and Analysis. Various Journal Entries as required. Preparation of trend and other analysis to support review of Balance Sheet. Assist with resolving accounting related issues as required. Qualifications Enrolled in a 4-year degree program. Ability to deal with time demands. Must possess excellent Word, Excel and interpersonal skills. Must have the ability to build and maintain positive relationships with management and peers. 3.0 GPA. 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.
AveXis Inc. Libertyville, IL, USA
Mar 17, 2019
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 (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. AveXis is seeking a highly motivated individual to join us as a Quality Control Microbiology Technician. This position will work with the Quality Control team in supporting our efforts in this exciting new area of gene therapy, responsible for environmental monitoring and product testing. Responsibilities Perform environmental and utilities monitoring in clean rooms. Perform bioburden, endotoxin and cell culture assays. Perform general laboratory duties such as stocking and ordering supplies. Perform other duties as assigned. Qualifications Bachelor's degree in Microbiology or related discipline or Associate's degree with a minimum of 2 years relevant experience working in a GMP QC Microbiology laboratory. Ability to gown for entry into Aseptic core and supporting areas and lift approximately 25 pounds. Proficient in Microsoft Word, Excel, Power Point and other applications. Ability to communicate and work in a team environment. Comfortable in a fast-paced small company environment with proven ability to adjust workload based upon changing priorities. Self-motivated, detail-oriented, and willing to accept temporary responsibilities outside of core duties. Ability to think critically and demonstrate troubleshooting and problem-solving skills. Excellent interpersonal, verbal and written communication skills. Ability to work in a cleanroom environment. 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.
AveXis Inc. San Diego, CA, USA
Mar 16, 2019
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 (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. AveXis is seeking a Manager of Manufacturing Process Science that will be responsible for organizing, managing, and development of manufacturing operations and processes at the AveXis manufacturing Process Science site. This individual will be directly responsible for a team of Process Science Engineers whose direct responsibilities include development and production of pre-clinical and clinical material. The manufacturing manager will be the first line of contact to handle process and equipment deviations on the floor. Areas of responsibility could include both upstream (cell culture), downstream (Purification), and fill/finish activities. Responsibilities Independently design and conduct experiments to support the development and implementation of a cell culture scale-up, recovery, and downstream purification processes for gene therapy. Work cross-functionally to obtain feedback and alignment regarding projects and potential modifications to current processes. Capable of evaluating and summarizing data using analytical methodologies, interpreting results, drawing conclusions and recommending options for future experiments to achieve project goals Produce pre-clinical and clinical material on an annual basis that meets the site's strategic objects and is compliant with cGMPs and safety regulations. Schedule daily operations (finite scheduling) for manufacturing and cleaning processes. Ensure manpower resources are adequate to complete operations. Lead investigations as related to the manufacturing process. Partner with Quality to address these issues effectively and compliantly. Ensure documentation (batch records and SOPs) are accurate and updated as required. Participate in tours or information requests for all agency and internal audits of the manufacturing facilities/processes and respond to any observations received per procedure. Provide monthly manufacturing metrics as determined by management. Lead areas of tech transfer of new products and processes into the manufacturing area. Provide manufacturing feedback on engineering related projects. Identify and implement continuous improvement opportunities. Lead and mentor staff. Write performance reviews and annual goals, hold one-on-ones, and handle HR related matters. Adhere to all EH&S policies, procedures and guidelines. Qualifications Minimum B.S degree in biochemistry, chemical engineering, bioengineering, or related technical field, or equivalent industry experience. A minimum of 8-10 years of experience in biopharmaceutical based GMP manufacturing operations including experience in cell culture, recovery, purification (TFF, chromatography), aseptic fill/finish with: Solid knowledge of FDA regulations and GMP systems. Excellent oral and written communication skills. Strong technical writing ability required. Excellent computer skills, proficient with Microsoft Word, Excel, PowerPoint and Visio, and working knowledge of JMP or equivalent statistical software. Ability to motivate and mentor peers, staff, foster a culture of continuous improvement and operational excellence. Demonstrated leadership skills. Project management skill set with experience in strategic/tactical planning, team building, and meeting budgets. Experience with viral manufacturing and transfection a plus. 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-HJH1
AveXis Inc. Libertyville, IL, USA
Mar 16, 2019
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 Manufacturing Quality Supervisor (MQ) will provide critical quality expertise to transition a novel gene therapy into a commercially approved product and is responsible for implementing robust Quality Systems for a new viral gene therapy manufacturing site and being quality on the manufacturing floor. Responsibilities Deliver a robust overall quality systems, strategy, and ultimate plan resulting in a fully compliant facility with respect to procedures and processes. Quality decision making on the manufacturing floor SOPs/Document Management: Approve all instruction sets, specifications, batch records etc. and other Quality procedures directly related to operations and other cGMP activities On the floor Batch record review: Ability to review controlled documentation during processing for GMP and GDP Deviation/CAPA Management approval: Ensure all deviations from established procedures are appropriately documented and investigated to determine and fix root cause. Requires direct input on the creation and effectiveness of Corrective and Preventive Action plans. Assist with implementation and supporting on-going continuous improvements. In process sampling/ AQL sampling: Ensure all required assessment and testing is carried out to support Quality approval or rejection, as appropriate, for starting materials, packaging materials, intermediates, bulk and finished product Training: Ensure all personnel, including contractors, consultants and temporary employees have the proper qualifications, skill sets, education, and training to perform their job in accordance with applicable procedures and regulations Supervise Manufacturing Quality Associates by providing work direction, performance management and professional development. Qualifications Minimum B.S. degree in preferably microbiology, chemistry or biochemistry A minimum of 3-5 years of experience in biopharmaceutical based GMP manufacturing operations including direct experience in cell culture, purification and aseptic fill/finish Experience with viral gene therapies and/or orphan disease indications is a plus Comprehensive knowledge of FDA and EMA regulations and experience in US and international regulatory agency inspections Direct experience reviewing and/or authoring standard operating procedures and partnering with operations on product related investigations and deviations Excellent oral and written communication skills with strong technical writing experience required. Experience leading both internal audits, identify findings, drive resolution and provide closure report Ability to synthesize data and summarize outcomes to provide recommendations on compliant path forward Knowledge of AQLs and visual inspection Strong knowledge and application of the CFR's and cGMP's. Approximately 10% travel 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.
AveXis Inc. Durham, NC, USA
Mar 14, 2019
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 (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. AveXis is seeking a Human Resources Generalist for our Durham site to carry out HR responsibilities in the areas of benefits enrollment, training, facilitating time tracking, performance management, onboarding, new hire orientation and legal compliance. This individual will work closely with the HR team both locally and remotely to define and accomplish HR goals that support business success. Responsibilities Review and make recommendations to site policies and procedures. Organize meetings and partner with external vendors to execute. Respond to employee inquiries regarding policies, procedures, benefits and general questions. Coordinate training on HR topics (i.e. new manager training, harassment training). Ensure onboarding and new employee orientation properly introduces employees to the culture. Identify opportunities to improve and scale orientation to accommodate the rapid growth of the company. Provide guidance and support to managers in the areas of performance management and employee relations, with oversight from Site HR Head. Investigate accidents and work closely with EH&S and Occupational Health on workers compensation claims. Champion the Avexis Core Values and identify ways to improve employee satisfaction. Partner with HRIS to ensure that employee data is up to date and accurate. Conduct Exit Interviews to determine reason for separation. Resolve benefits-related issues. Assist with new hire processes and questions. Work with other corporate support functions to handle the proper processing of employee-level processes such as payroll and employment verification. Maintain responsibility for company compliance with federal, state and local law pertaining to all employee topics. Qualifications 4-year college degree required. Master's degree in labor Relations, Industrial Relations, or similar discipline a plus. Minimum 3 years of related Human Resources experience required, preferably in pharma, biopharma, or biotechnology. Experience working in a manufacturing, operations, or supply chain environment a plus. Exceptional communication skills and demonstrated ability to work with all levels of people within our organization required. Strong analytical skills and attention to detail and judgmental capability. Demonstrated proficiency working in a matrix team environment and multitasking. Demonstrated ability to develop work processes and establish standards. Must demonstrate a high-level of accommodation and flexibility with a "hands on approach" in our fast-paced environment of constant change; able to perform well under pressure and deadlines. The ideal candidate will be well organized and confident to work independently but also a strong team player. Outstanding customer service skills. Demonstrated ability to effectively communicate with all levels of the organization. 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-RL1
AveXis Inc. Illinois, USA
Mar 14, 2019
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(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. This position will be on the Biostatistics team within the Clinical Development department at AveXis. Clinical Development is the department within AveXis that is responsible for the planning, execution, and oversight of studies conducted in humans (clinical studies). The purpose of these studies is to determine the safety and efficacy of new treatments for disease. The ultimate goal of evaluating new treatments for diseases is for potential approval by the Food and Drug Administration and other regulatory authorities OR to determine at an early time point that the new treatment is not effective or has unacceptable risks. The Summer Intern will collaborate with one or more biostatisticians who work with clinical scientists in the planning, execution, and analysis of clinical studies. Potential activities may include the derivation of data sets from clinical studies, completed and ongoing, and then the subsequent analyses of those data sets with R or SAS. The intern will then assist with the evaluation of the analysis results and provide additional statistical support to the biostatisticians and clinical scientists. Responsibilities Assist senior biostatisticians with study design and analysis of clinical data related to company's development programs and support of regulatory and publications functions. Participate in database evaluation, programming in R and SAS, and help prepare biostatistics deliverables. Review statistical tables, listings, figures, and analysis datasets for clinical trials in accordance with AveXis Standard Operating Procedures (SOPs) or study guidelines. Review statistical analysis plans (SAPs), end of study clinical study reports (CSRs), and other relevant biostatistics documents. Program in R and/or SAS to create output tables and/or listings as needed. Perform other responsibilities as required by the Department and participate in process improvement initiatives. Qualifications Enrolled in a college degree program for an academic major appropriate for biostatistics or statistics. This could include mathematics, bioinformatics, or data science. Programming skills in R and/or SAS. Preferred Skills: Candidates must have excellent verbal and written skills. Working knowledge of Microsoft Office applications including Word, Excel and PowerPoint. Effective organizational, analytical & interpersonal skills and the ability to work in a high paced timeline driven setting. 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.
AveXis Inc. Illinois, USA
Mar 13, 2019
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 (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. AveXis is seeking a Human Resources (HR) Specialist to support the HR team on a variety of data, reporting and project activities. The Specialist will be a resource for employee inquiries and facilitate several HR processes. Responsibilities Provide support to the HR Business Partners on a variety of deliverables including reporting, HR system support, data changes and job description development support. Provide high touch candidate support from offer to onboarding including welcome communication, monitoring background check statuses, and coordinating New Employee Orientation at multiple sites. Maintain personnel files in compliance with applicable legal requirements. Keep employee records up-to-date by processing employee status changes in Workday in a timely fashion. Maintain employee documentation electronically in Workday as well as in paper files as necessary. Create and maintain positions and organization structure in Workday. Conduct data audits within Workday, partnering with the HR team and correcting errors as needed. Participate in HR projects to improve processes such as Workday integrations to other systems. Facilitate human resources processes such as biweekly time tracking. Monitor the HR mailbox, assisting employees with questions and requests; provide information regarding AveXis HR policies or resources as appropriate; refer questions and issues as necessary to appropriate HR staff member. Verify information for employment verifications and other HR related requests. Qualifications Bachelor's degree or equivalent experience. 3 years of experience in an HR support role or equivalent experience. Ability to effectively communicate policies and information, knowing when to escalate questions or issues. Excellent attention to details and accuracy of data, ability to prioritize and follow through on requests. Must be able to handle HR/Payroll related information with strict confidentiality and professionalism. Well organized, able to multi-task, meet deadlines and recommend process improvements. Solid verbal and written communication skills with proven customer service focus. 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.
AveXis Inc. Illinois, USA
Mar 12, 2019
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. We are looking for a positive, high-energy and agile marketer who will be responsible for the development and execution of initiatives aimed at Healthcare Professionals, with an emphasis on digital channel management and execution. This position will play a key role in the development and implementation of AveXis first commercial product launch, including advertising campaigns, digital strategy, collateral development, congresses, and vendor management. Areas of focus may include one or more of the following: professional HCP marketing, brand website development, CRM development and implementation, paid media, social media, and congresses. The position requires a highly motivated individual who can work independently, think strategically, and collaborate transversally to accomplish complex deliverables. Responsibilities Establish deep expertise in market and customer unmet needs across key markets. Ensure voice of customer is continuously monitored and included in defining market and product requirements. Support the development and deployment of HCP branded and patient unbranded and branded initiatives including objective setting, content development, internal Promotional Review Committee (PRC) review, QC, production, deployment/distribution to the field and performance measurement; focus on HCP or consumer or both. Work closely with field teams (advocacy, sales and reimbursement) to understand barriers to adoption, develop strategies to grow penetration, and improve commercial execution. Initiate and analyze qualitative and quantitative market data to continually gain a deeper understanding of the HCP and patient customers, disease area, and market. Propose informed strategies and tactics to address key unmet needs. Participate in cross functional sub-teams to ensure alignment and appropriate execution on brand plan; become a trusted partner in this setting. Manage relationships with strategic partners and vendors (include agencies of record) to ensure that projects are completed in a timely and efficient manner while remaining on budget; condition partners for executional excellence and hold them accountable for success metrics. Work with advanced analytics to set up measurement plans to assess program performance optimize spend across marketing mix. Collaborate on Non-Personal Promotional efforts targeting HCPs to supplement and complement Sales Force efforts. Manage vendor contract processing (purchasing requisitions, invoices, and approvals). Collaborate with multiple functional groups across the organization, including but not limited to Sales, Regulatory, Market Access, Market Research, Commercial Operations, Legal, Patient Advocacy, and Medical Affairs. Drive the brand's digital marketing strategies, priorities and goals and ensure that digital tactics are aligned with brand business objectives, including integration of digital strategy into overall brand marketing strategy. Develop and execute build of brand website and related digital initiatives, including CRM. Review reporting and KPIs on brand's digital initiatives to develop recommendations and implement future digital marketing programs. Plan and oversee execution of activities related to congresses. Manage materials, projects and resources through the internal review committee process (PRC). Qualifications BA/BS degree in biological sciences, pharmacy, marketing or business administration or a minimum of 7 years of related experience in a prior role; Minimum 4-5 years of US pharmaceutical brand marketing experience. US Launch experience in orphan and/or CNS required. Ability to quickly understand and communicate all clinical data / publications to support business objectives. Strong HCP marketing background with emphasis on digital channel management and execution (websites, SEO/SEM, paid media, CRM, analytics, social media). Proven ability to work under pressure, prioritize and manage multiple projects, and effectively influence, collaborate, and communicate with internal and external partners. Experience working with external brand agencies and vendors on a day-to-day basis. Demonstrated financial and business acumen. A high interest in working in a "roll up the sleeves" environment of a small bio-pharma company, without the resources of larger companies. Up to 25% of US domestic travel. 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.
AveXis Inc. Illinois, USA
Mar 12, 2019
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. We are looking for a positive, high-energy and agile marketer who will be responsible for the development and execution of initiatives aimed at Healthcare Professionals. This position will play a key role in the development and implementation of AveXis first commercial product launch. Areas of focus may include one or more of the following: professional HCP marketing, brand website development, CRM development and implementation, paid media, social media, sales training liaison and congresses. The Product Manager position requires a highly motivated individual who can work independently, think strategically, and collaborate transversally to accomplish complex deliverables. Responsibilities Contribute to the development of annual business plans, product positioning and strategies, and marketing program creation. Work to ensure company objectives through on-strategy execution of the annual Marketing plan in every area of responsibility (Advertising and Promotion, Conferences, Medical Education, and/or Segment Marketing, etc.). Develop promotional campaigns, messages, programs and communication tools. Engage with key customers, speakers, and Key Opinion Leaders (KOL's) as needed to ensure creation and seamless roll out of high impact, high value marketing programs. Plan and oversee execution of activities related to conferences. Manage and coordinate the work of ad agency, medical education partners and various external vendors including acquiring necessary budget estimates, contracts, purchase orders, invoices, etc. Collaborate with Sales Training to ensure effective sales force training and pull through of promotional messaging, tools and programs. Effectively interact with sales force to understand needs and handle requests. Communicate brand performance and strategies effectively to senior management. Provide product evaluation and performance tracking for products of responsibility. Budget responsibility including creation, spend, reconciliation and developing metrics to monitor effectiveness and/or program success. Interpret and translate scientific clinical data into commercial applications. Monitor competitive activity and identify customer needs. Assist in establishing and maintaining consistent corporate image throughout product lines, promotional materials, and events. Travel to conferences / rep-rides (as needed). Other duties and responsibilities as assigned. Qualifications BA/BS degree in biological sciences, pharmacy, marketing or business administration or equivalent experience. Minimum 3 years of US pharmaceutical product management preferred with additional experience desired, including pharmaceutical field sales, advertising agency and/or pharmaceutical market research. Ability to coordinate interdisciplinary teams (internal and external) to drive issues and projects to conclusion. Success in introducing new pharmaceutical products to market preferred. Demonstrated financial and business acumen. Strong organizational, analytical and problem-solving skills essential. Excellent interpersonal and negotiation skills required. Team and project leadership skills. A high interest in working in a "roll up the sleeves" environment of a small bio-pharma company, without the resources of larger companies. Up to 35% of US domestic travel. 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.
AveXis Inc. San Diego, CA, USA
Mar 11, 2019
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 (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. AveXis is seeking an experienced and exceptional Scientist II 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 individual will play a crucial role for the development and optimization of cell-based potency and other functional bioassays in a team environment. In addition, he/she will participate (or lead) method qualification/validation in a cGMP setting and may be involved in the method transfer to internal and/or external QC laboratories. Responsibilities will also include execution of routine assays and supporting lab operation. Successful candidate will have extensive experiences in tissue culture work and will be a fast-learner with passion for new technology and science Responsibilities Optimize and troubleshoot cell-based assays as well as other plate-based assays using 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. Actively engage in planning and designing of assays/experiments and take 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. Maintain clean and fully functional cell culture area by implementing TC room policy and take ownership of on-going cultured cells by thoroughly documenting cell passage, morphology and behavior and routinely performing tests for contamination. In addition, create cell banks and oversee their inventory. Play an important role in qualifying and validating cell-based assays. Help documenting assay development, qualification and validation processes as well as SOPs. Perform experiments in support of assay development using variety of techniques covering cell biology, biochemistry and molecular biology such as immunocytochemistry, western blot, qPCR, flow cytometry, and immunoprecipitation. Qualifications B.S or M.S in biology discipline is required. Minimum 8 years of biopharma industry experience is required for B.S and minimum 5 years of biopharma industry experience is required for M.S. Basic understanding of regulatory agency guidance such as FDA, ICH and EMA and hand-on experience is a significant plus. Must have good cell culture techniques with good understanding of cell behavior and biology. Demonstrated track record of successfully optimizing and executing cell-based assays using various assay platforms including but not limited to ELISA, MSD, ELISpot and high-content imaging in 96 well plate setting. Broad experiences in cellular, biochemical and molecular biological methods including but not limited to immunostaining, flow cytometry, protein gel, Western blot, PCR and qPCR. Enjoy learning and challenges. Especially, must be able to learn new instrument 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 goals/deadlines oriented. Intelligent individual who respects 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. 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.
AveXis Inc. San Diego, CA, USA
Mar 11, 2019
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. He/she will collaborate closely with the internal R&D team to co-develop prototype bioassays and identify suitable assay formats for further optimization with demonstrated independence and with ability to make solid scientific and technical judgements. He/she will lead from the lab with hands-on experiments and data interpretation and may represent Bioassay group in cross-functional teams. Main responsibilities also include method qualification and validation under phase-appropriate GMP as well as method transfer to internal or external QC laboratories under the guidance by his/her supervisor. The individual will have the ability to supervise 1 or more junior members in Bioassay group to guide appropriate and effective experimental designs, execution, trouble-shooting as well as data analysis and interpretation. Responsibilities will also include supporting the director of Bioassay group on authoring tech reports, SOPs and qualification/validation protocols and reports. Responsibilities Key driver to develop, optimize and qualify/validate MOA-reflective, bioassays suitable for lot disposition and stability study based on thorough understanding of the scientific background of the products, applicability of the emerging or existing bioassay platforms and phase-appropriate GMP requirements. Independently design and execute effective experiments with clear goals of answering key questions to drive assay development and troubleshooting in a very face paced environment. Interpret obtained data for follow-up studies and decision making with guidance of his/her supervisor. One of the key liaison to work closely with R&D team for knowledge and methodology learning and transfer to ensure win-win collaboration in co-developing bioassays and other functional assays suitable for characterization, stability, and release testing purpose. Represent the Bioassay group to attend cross-functional CMC and project meetings if needed and actively participate in strategic plan for the projects, execution, data presentation and follow-up. Authors technical reports and SOPs and assists director of Bioassay to prepare relevant sections of regulatory filings if needed. Mentor and guide junior members of the group on daily laboratory work, experimental design, and data interpretation. Qualifications Ph.D. or M.S. in biology discipline is required. 5-8 years (Ph.D) or 12-15 years (M.S) of biopharma industry experience with demonstrated leadership and track record of successfully developing different spectrum of bioassays by utilizing appropriate methodologies (i.e cell imaging systems, flow cytometry, ELISA, MSD and Luminex etc) Minimum 1 year of GxP experience is preferred but not required Experience in working on antibodies, vaccines, viruses or gene therapy drugs are preferred but not required Good understanding of high-content imaging system and image analysis is highly preferred. Technical writing skills and experience in authoring development reports and SOPs with detail-oriented trait are required. Ability to work in a fast-paced team environment with highly goal-oriented approaches and to prioritize work from multiple projects with can-do attitude is required. Ability to bring in and assess value-added platforms based on special needs associated with characterization of the gene therapy drug products. Proven track record of solid scientific achievement to allow in-depth understanding of mechanism of action of the investigational drugs for the development of the MOA-reflective bioassays. Ability to work in a fast-paced small but growing team environment and to prioritize work from multiple projects. Must be highly reliable and goals/deadlines oriented. Excellent written and verbal communication skills. Intelligent individual who respects others and is open to new/different ideas. Prior supervisory experience is preferred. This position is located out of our La Jolla area facility and may require up to 10% travel between La Jolla, third party vendors (domestic) and our HQ and Manufacturing sites located in the Chicago area. 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.
AveXis Inc. San Diego, CA, USA
Mar 11, 2019
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 (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. AveXis is seeking an experienced and exceptional Senior Scientist to join the dynamic and growing analytical team to establish analytical methods suitable for lot disposition and characterization of gene therapy drug products and substances. He/she will be mainly responsible for the analytical method development, qualification and validation under phase-appropriate GMP and will also be part of method transfer and training team for successful transfer of the in-house qualified or validated assay to internal or external QC laboratories under the guidance by his/her supervisor. The individual will have the ability to mentor/train junior group members to ensure appropriate and effective experimental design, execution, data interpretation and trouble-shooting. Responsibilities will also include authoring tech reports, SOPs and qualification/validation protocols and reports, with minimum guidance. The role will involve laboratory work and provide representation of the chemistry team on cross-functional teams. Responsibilities Independently develop, optimize and qualify/validate HPLC, CE, FFF based analytical methods suitable for gene therapy characterization, lot release and stability study based on a thorough understanding of the scientific background of the products. Proactively identify assay issues, new assay needs and provide scientific suggestions and solutions. Serve as SME to work with CROs for method development/qualification/validation, actively participate in the scientific discussion and provide input/feedback. Work closely with functional groups (for example, R&D, QC/QA, process development and manufacturing groups) to ensure win-win collaboration in developing/transferring analytical methods suitable for characterization, stability, and release testing purpose. Effectively communicate results both internally and externally by presentations or written reports. With minimum guidance, author/review SOPs and technical protocols/reports; contribute to data generation/interpretation/summarization in support of regulatory filings including IND and BLA. Mentor/guide junior group members on daily laboratory work, experimental design, data interpretation and troubleshooting. Train new group members on basic analytical lab skills. Actively participate and give suggestions on lab designing, instrumentation purchasing and strategic planning. Qualifications Ph.D. or M.S. in analytical chemistry or related scientific discipline is required. 5-8 years (Ph.D) or 8-10 years (M.S) of biopharma industry experience is required. Extensive knowledge and experience with a broad spectrum of analytical techniques (including but not limited to SEC, FFF, IEC, RP-HPLC, CE-SDS, iCIEF) for lot disposition and characterization of biologics drug product candidates. Good understanding of biophysical characterization methodology using MALS, DLS, AUC, DSC, TEM type of techniques is highly desired. In-depth understanding and troubleshooting capability of analytical instrumentation and software (including but not limited to HPLC, CE, FFF, MALS, DLS, Chromeleon, Empower, Chemstation, Astra, 32 Karat). Previous working knowledge on mass spectrometry (MS) characterization of protein therapeutics is a plus. Working experience on antibodies, vaccines, gene therapy drugs or nanoparticle drug deliveries are required Strong technical writing skills and experience in authoring development reports and SOPs with detail-oriented trait. 1-3 years of GxP experience in qualifying and/or validating analytical assays is preferred. Highly motivated, hard-working and detail oriented. Strong ability to work in a fast-paced team environment with highly goal-oriented approaches and to prioritize work from multiple projects with can-do attitude is required. Ability to proactively bring in and assess value-added platforms based on special needs associated with characterization of the gene therapy drug products is essential. Excellent written and verbal communication skills. This position is located out of our La Jolla area facility and may require up to 10% travel between La Jolla, third party vendors (domestic) and our HQ and Manufacturing sites located in the Chicago area. 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-EC1
AveXis Inc. Durham, NC, USA
Mar 11, 2019
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. AveXis is seeking a Maintenance Technician (Electrician) who is responsible for performing electrical system operation and maintenance throughout the site. Facilities Maintenance & Engineering are directly responsible for reliable, efficient and sustained operation of the facility and all equipment. Additionally, through daily tasks, Facilities supports current project operations and site goal objectives and ensures compliance with regulatory, corporate, and site requirements. Responsibilities Operate and maintain electrical power generation and distribution systems. Operate and maintain backup power/emergency power generation systems and Uninterruptible Power Systems (UPS). Perform troubleshooting and repair of electrical equipment and systems. Support operation and maintenance of Building Fire Alarm systems including operating the system, disabling points, and performing troubleshooting activities. Interpret and update loop sheets, electrical schematics, P&ID's, I/O wiring, and panel drawings. Ensure activities are accomplished in a safe manner. Support 24x7 site-based operations after startup. Write/revise accurate operational procedures, training documents, and maintenance procedures for various electrical systems. Supply information and technical data for securing spare parts. Provide responsive customer support with emphasis on customer satisfaction. Perform startup and commissioning activities as required. Work in a collaborative team environment and interact with other support groups such as Engineering, Manufacturing, Metrology, Quality Assurance and Validation. Complete and provide accurate documentation, as required in cGMP operations. Qualifications Associate Degree and 4 years of Electrical Specific Maintenance experience or High school diploma and 6 years of Electrical Specific Maintenance experience 8+ years of combined education, training, and experience in industrial electric operations and maintenance and instrumentation/measurement and controls State of NC Unlimited Electrical Contracting License Ability to effectively communicate ideas through verbal and written form Theoretical understanding of control, analog and/or digital electronics, microprocessors and/or computers Working in a team environment, with excellent communication and organizational skills. Proficient computer skill utilizing MS Office suite applications, Building Management Systems, and Computerized Maintenance Management Systems (CMMS). Ability to climb ladders and lift up to 50 lbs. 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.
AveXis Inc. Durham, NC, USA
Mar 11, 2019
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 (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 BioProcess Engineer I is responsible for assisting with organizing, running, and sustaining the manufacturing operations process at the plant/site during off hours. This individual will work on the manufacturing floor to produce product, media/buffer preparation, learn to troubleshoot equipment, participate in interviews on deviations, stocking of items in supply/ production / warehouse area, standardizing equipment and cleaning production area. Responsibilities Assist in manufacturing led investigations through partnerships with Quality and other business units at the site. Assist BPEs to determine root cause, implement a solution and a verification check to ensure the fix was effective. Assist with creation and on-going maintenance of all pertinent equipment, policies, and procedures. Be flexible in rotations between upstream and downstream to learn aseptic techniques, cell culture, recovery, purification, aseptic fill/finish. Assist in producing clinical and commercial material on an annual basis that meets the site's strategic objectives and is compliant with cGMPs. Support the product requirements to ensure that all products are produced according to plan. Learn cGMP and cGDP and ensure cGMP documentation is being filled out correctly, training is current and all Quality requirements are being followed. Responsible for maintaining quality standards to meet cGMP requirements, CFR's, and internal company policies directly related to the manufacturing process. Partner with Quality to ensure a quality and compliant manufacturing environment. Assist the technical operations team to resolve any issues related to production. Qualifications 0-2 years' experience in GMP environment with Bachelors of Science Degree in Biology, Chemistry, Biotechnology or applicable field or equivalent experience. Excellent oral and written communication skills. Entry level into the biopharmaceutical based GMP manufacturing operations, no experience necessary. Approximately 10% travel required. Must be able to routinely lift over 35 lbs. Must be able to work alternate 12 hour shifts and weekends, as needed. 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.
AveXis Inc. Libertyville, IL, USA
Mar 11, 2019
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 (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. AveXis is seeking a highly motivated and solution-driven individual to join the dynamic and growing Quality Control (QC) team. The individual will collaborate closely with Analytical Development team to qualify/validate analytical methods and establish the site QC lab operations to support development and commercialization of new gene therapy medicines. The QC Analyst is responsible for routine sample testing under GMP or non-GMP modes, laboratory maintenance, and participating in method qualification/validations. A potential candidate should have enriched experience in culturing transformed and primary cells and banking cells. In addition, experience in testing samples using qualified cell-based assays (e.g. virus infection assays and proliferation assays) is desired. Additional responsibilities include adherence to all GMP requirements, a basic understanding of FDA/EMEA regulations, effective interactions/communication with Quality management and support of investigations. The QC Analyst may also support and identification and implementation of corrective and preventive actions. He/she should be able to work effectively within the group, within Quality, and across site. Responsibilities Perform cell-based potency and impurity assays for in-process, release, or stability samples. Perform other established analytical methods such as qPCR and CE to support QC operations. Participate in assay troubleshooting, qualification and validation if needed. Manage the procurement, use, and maintenance of scientific equipment, instrumentation, and computer systems. Write and revise documents such as SOPs and technical reports. Support investigations to determine root cause of deviations and non-conforming results and implement appropriate corrective and preventative actions in a timely manner. Liaise with other departments including Analytical Development, Quality Assurance, and Manufacturing to address compliance issues and to implement corrective actions and to improve programs Qualifications Minimum B.A. or B.S. in biochemistry or related scientific field. Minimum of 1 year GMP lab experience and at least 2 years of industry experience working on tissue culture and cell-based assays associated with biologics products. Excellent oral and written communication skills with strong technical writing experience 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.
AveXis Inc. San Diego, CA, USA
Mar 10, 2019
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 (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 Process Science Engineer II is responsible for designing, planning and executing experiments to develop and characterize adherent and suspension cell culture manufacturing processes, recovery, and downstream purification operations. In addition, the individual will support new technology evaluation and development, technology transfer and process validation. These activities will support the cGMP manufacturing operations and production of pre-clinical and clinical material. Responsibilities Conduct experiments supporting the development and implementation of a closed system, scale-up, recovery, and purification processes for gene therapy. Capable of evaluating and summarizing data using analytical methodologies, interpreting results, drawing conclusions and recommending options for future experiments to achieve project goals. Produce pre-clinical and clinical material on an annual basis that meets the site's strategic objectives and is compliant with cGMPs. Support the product requirements to ensure that all products are produced according to plan. Appropriately document experimental procedures and results according to established guidelines. For cGMP manufacturing, ensure documentation is being filled out correctly, training is current. and all Quality requirements are being followed. Participate in information requests for FDA and internal audits of the manufacturing facilities. Responsible for maintaining quality standards to meet cGMP requirements, CFR's, and internal company policies directly related to the manufacturing process. Support tech transfer of new products and processes to ensure smooth transition from process development into GMP manufacturing. Look for opportunities to implement operational excellence and continuous improvement. Partner with Quality to ensure a quality and compliant manufacturing environment. Assist the technical operations team to resolve any issues related to production. Adhere to all EH&S policies, procedures and guidelines. Qualifications 2-4 years' experience in Biological Manufacturing environment. Minimum B.S degree in biochemistry, chemical engineering, bioengineering, or related technical field, or equivalent industry experience. Excellent computer skills, proficient with Microsoft Word, Excel, PowerPoint and Visio, and working knowledge of JMP or equivalent statistical software. Excellent oral and written communication skills, written and oral, and aptitude for problem solving. Preferred: 2-4 years of experience in biopharmaceutical based GMP manufacturing operations including direct experience in cell culture, recovery, purification, aseptic fill/finish with working knowledge of FDA regulations and GMP systems, strong technical writing ability and proven ability to effectively lead and participate on teams. 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.
AveXis Inc. Libertyville, IL, USA
Mar 10, 2019
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 (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 Quality Control Microbiology Specialist provides Environmental Monitoring/Microbiological support at the AveXis GMP Manufacturing facility. The QC Microbiology Specialist responsibilities include, but are not limited to, training, data review, routine testing, method qualification/validations, investigations, and technical writing. A potential candidate will possess a strong technical understanding of QC Microbiological/Cell Culture/Environmental Monitoring testing techniques. The QC Specialist should be able to work effectively within the group, within Quality, and cross functionally. Responsibilities Perform Cell Culture Assays, Environmental Monitoring, and Microbiological testing. Review and trend data and publish quarterly and annual trend reports. Conduct thorough investigations to determine root cause of OOS investigations and other non-conforming results and implement appropriate corrective and preventative actions in a timely manner. Liaise with other departments including Quality Assurance, Manufacturing, and Facilities to address compliance issues, implement corrective actions and improve programs. Participate in assay troubleshooting, qualification, and validation. Manage the use and maintenance of scientific equipment, instrumentation and computer systems. Write and revise documents such as SOPs and technical reports. Qualifications Minimum B.A. or B.S. Microbiology or in scientific related field. Minimum of 5 years in a GMP Quality Control Microbiology/ Environmental Monitoring laboratories, preferably in a biologics aseptic processing facility. Strong knowledge of Environmental Monitoring and Microbiological Assays (e.g. Bioburden, Endotoxin, Gram Stain, Microbial ID, and Sterility). Ability to gown for entry into Aseptic core and supporting areas, and lift approximately 25 lbs. Experience with method and instrument qualification. Experience performing laboratory investigations and environmental monitoring excursion investigations. Experience with classroom and on the job training (i.e. Microbiological Assays, Aseptic Techniques, and Aseptic Gowning). Good understanding and demonstration of aseptic techniques. Experience leading small teams and projects. Working knowledge of quality systems and regulatory requirements (21 CRF Part 11/, ISO 14644-1, USP, EP, JP). Proficient in MS Word, Excel, Power Point and other applications. Ability to communicate and work in a team environment. Comfortable in a fast-paced small company environment and able to adjust workload based upon changing priorities. Self-motivated, detail-oriented, and willing to accept temporary responsibilities outside of initial job description. Ability to think critically, and demonstrate troubleshooting, and problem-solving skills. Excellent interpersonal, verbal and written 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.
AveXis Inc. San Diego, CA, USA
Mar 10, 2019
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 (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 Process Science Engineer III is responsible for organizing, running, sustaining, and continuously improving the manufacturing operations process at the plant/site. This individual will work on the manufacturing floor to produce product, troubleshoot equipment, and provide ownership for specific pieces of processing equipment. Responsibilities Design and conduct experiments supporting the development and implementation of a closed system, scale-up, recovery, and purification processes for gene therapy. Work cross-functionally to obtain feedback and alignment regarding projects and potential modifications to current processes. Capable of evaluating and summarizing data using analytical methodologies, interpreting results, drawing conclusions and recommending options for future experiments to achieve project goals. Produce pre-clinical and clinical material on an annual basis that meets the site's strategic objectives and is compliant with cGMPs. Appropriately document experimental procedures and results according to established guidelines. For cGMP manufacturing, ensure documentation is being filled out correctly, training is current, and all Quality requirements are being followed. Participate in tours or information requests for all FDA and internal audits of the manufacturing facilities/processes and respond to any observations received per procedure. Responsible for maintaining quality standards to meet cGMP requirements, CFR's, and internal company policies related to the manufacturing process. Support tech transfer of new products and processes to ensure smooth transition from process development into GMP manufacturing. Provide hands on technical leadership to the manufacturing staff members. Look for opportunities to implement operational excellence and continuous improvement. Partner with Quality to ensure a quality and compliant manufacturing environment. Support Director of Operations to meet information requirements as needed for quality, compliance, and management reporting. Assist the technical operations team to resolve any issues related to production. Adhere to all EH&S policies, procedures and guidelines. Qualifications 4+ years' experience in Biological Manufacturing environment. A minimum of 4-7 years of experience in biopharmaceutical based GMP manufacturing operations including experience in cell culture, recovery, purification, aseptic fill/finish with working knowledge of FDA regulations and GMP systems. Excellent oral and written communication skills. Strong technical writing ability required. Ability to motivate peers and staff, foster a culture of continuous improvement and operation excellence. Experience with 3rd parties (equipment vendors, and contract manufacturing insourcing/outsourcing). Project management skill set with experience in strategic/tactical planning, team building, and meeting budgets. Approximately 10% travel required. Must be able to routinely lift over 35 lbs. 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.