Sr. Scientist/Principal Scientist – CMC Downstream Gene Therapy Development

Sr. Scientist/Principal Scientist – CMC Downstream Gene Therapy Development

Requirements

• BA/BS in Chemical Engineering, Biochemical Engineering, Biochemistry, or other relevant discipline and 5-12+ years of Biotechnology industry experience in downstream AAV purification technologies and theory.
• Proven experience in the scale-up and technology transfer from pilot plant to manufacturing with extensive chromatography experience.
• Downstream experience with writing regulatory documents to support filings (INDs/CTAs, Briefing Materials, and BLAs/MAAs).
• Demonstrated and significant applied experience with technology transfer-in and transfer-out of gene therapy manufacturing process at all levels of development.
• Significant experience in laboratory operations, including budgeting, capital expense procurement and IQ/OQ, safety, and status reporting to Senior Management.
• Demonstrated ability to lead downstream process development activities conducted by Contract Research Organizations (CROs)/Contract Manufacturing Organizations (CMOs).
• Demonstrated and significant experience with single-use bioprocessing equipment and its application to gene therapy downstream process development and GMP manufacturing OR Master’s degree in Chemical Engineering, Biochemical Engineering, Biochemistry, or other relevant discipline and 6-10+ years of similar experience noted above OR PharmD or PhD in Chemical Engineering, Biochemical Engineering, Biochemistry, or other relevant discipline and 4+ years of similar experience noted above.
• Ability to work independently and collaboratively, as required, in a fast-paced, matrixed, team environment consisting of internal and external team members.
• Analytical thinker with problem-solving skills and the ability to adapt to changing priorities and deadlines.
• Demonstrated ability to complete technical assignments by supporting the design, execution, and interpretation of complex experiments.
• Ability to deliver results within defined budget, timing, and resources constraints. Awareness of impact(s) on other functional areas.
• Self-motivated and comfortable working as part of a cross-functional teams, and an ability to meet timelines efficiently and productively.
• Ability to monitor on-going experiments and suggest alternatives or solutions.
• Commitment to continuous process improvement, driving efficiencies in all areas of operation.
• Advanced knowledge with Excel, Word, PowerPoint, and other associated software used in the scientific field.
• Excellent verbal and written communication skills.
• Advanced planning, organization and time management skills including the ability to support and prioritize multiple projects.
• Expert level knowledge of HEK-based AAV based gene therapy downstream process with a good understanding of manufacturing and testing/characterization methods required.

Responsibilities

• Provides leadership for exploratory development of AAV gene therapy purification including all aspects of downstream process and the associated analytical development in a research & development environment.
• Experienced in the development, implementation, and manufacturing of AAV drug substances for pre-clinical and clinical trial use.
• Provides support for exploratory process development for therapeutic antibodies including all aspects of downstream production in a research environment.
• Drives the implementation of downstream suspension-based platform processes for the generation of material as well as the early optimization studies, working with a downstream and analytical team for the characterization and release testing to support research, pre-clinical, and IND-enabling studies.
• Drives the additional process development activities to support development candidates and activities for timely IND, BLA and all relevant ex-US regulatory filings.
• Collaborates with discovery research and product development to build the scientific knowledge, capabilities, and strategies in biotherapeutic, protein and gene therapy product characterization that enable fast to clinic/fast to market product development and commercialization.
• Actively contributes to science-driven, phase appropriate, and risk-based process development strategies to enable further development from candidate nomination to clinical development and commercial manufacture.
• Identifies and resolves technical problems through scientific understanding, options assessment, and application of innovative solutions.
• Works collaboratively with internal staff and collaboration partners to generate project and cost proposals, timelines, and work plans by applying scientific knowledge and business expertise.
• Prepares clear and concise technical reports, participates, or leads project teams.
• Executes on strategy, vision, and direction set by line management.
• Takes a lead in decision-making processes within the project team by actively engaging in scientific and technical discussions.
• Responsible for participating in the day-to-day operations of the project, including conducting and evaluating training for new or current team members and delegating tasks appropriately.
• Ensures adherence to relevant regulatory requirements and company Standard Operating Procedures (SOPs) as appropriate.
• Performs other tasks and assignments as needed and specified by management.

Preferred Qualifications

• Expert level knowledge of the biologics drug development process is preferred.
• Knowledge of GLP and GMP environments as they pertain to gene therapy process development and Quality Control (QC) testing.
• Knowledge of CHO-based antibody expression system optimization is a plus.
• Familiar knowledge of CMC and regulatory requirements, including the development of process and product specifications and writing and reviewing development reports.
• Skilled at AAV purification downstream development, such as UF/DF, affinity chromatography, anion exchange chromatography from small scale to pilot scale.
• Knowledge of cell-based methods associated with bioanalytical analytical method development (e.g., TCID50, Potency assay, viral transduction, and ELISA methods).
• Ability to lead analytical method and downstream process development activities conducted by Contract Research Organizations (CROs)/Contract Manufacturing Organizations (CMOs) is desired.
• Knowledge and experience with novel methods in AAV characterization (e.g., ddPCR, DLS, AUC, NGS, etc.) is preferred.
• Proven expertise with chromatography bioprocessing equipment and its application to gene therapy process development and GMP manufacturing required.