Job Description Summary:
The Senior Scientist, Downstream Process Development (PD) – Gene Therapy leads, and actively contributes to, the engineering of downstream process development activities for gene therapy vector production. T
This individual designs and executes scale-up studies from pilot through full clinical and commercial scale. S/he also functions as PTC's Person in the Plant (PIP). In this capacity, s/he provides Subject Matter Expertise (SME) for external, Contract Development Manufacturing Organizations (CDMOs)-executed processes to ensure their proper execution. S/he interacts extensively with upstream Process Development, Analytical Development and Manufacturing personnel to deliver high-quality and robust downstream processes in a regulated environment.
S/he manages direct reports.
The incumbent works cross-functionally with internal departments and external resources as appropriate on downstream process development-related issues.
The Senior Scientist, Downstream Process Development – Gene Therapy supports adherence to relevant regulatory requirements and company Standard Operating Procedures (SOPs) as appropriate.
- Provides leadership for, and actively contributes to, downstream process development activities for both internal and external projects.
- Functions as the PIP at CDMOs to monitor, and assist with, on-going downstream process development and execution activities.
- Applies expertise with single-use bioprocessing equipment and its application to gene therapy downstream process development and GMP manufacturing.
- Designs and executes small-scale chromatography, ultrafiltration/diafiltration (UF/DF), depth and dead-end filtration and centrifugation studies to support development of cell, virus and plasmid DNA-based gene therapy manufacturing processes.
- Serves as Downstream PD SME for internal and external projects.
- Works closely with Clinical Manufacturing to transfer downstream processes from PD to manufacturing.
- Leads scale-up of downstream processes, writes reports and trains manufacturing personnel as required.
- Collaborates with Analytical Development to build process understanding and design space via design and execution of in-process testing and control assays.
- Works with Analytical Development to build the scientific knowledge, capabilities, and strategies in gene therapy product characterization that enable fast to clinic/fast to market product development and commercialization.
- Actively contributes to, and assures the effective execution of, science-driven, phase appropriate, and risk-based process development strategies to support development projects from candidate nomination to clinical development and commercial manufacture.
- Actively contributes to writing and/or QC review of relevant sections of global regulatory filings. These include, but may not be limited to, INDs, BLAs and ex-US filings
- Supervises, mentors and cross-trains junior scientists to build a highly functional PD Team that is cross-trained in at least two PD areas. This includes providing scientific and technical support to help troubleshoot and solve scientific challenges.
* Minimum level of education and years of relevant work experience.
- A PhD in Chemical Engineering, Biochemical Engineering, Biochemistry or other relevant discipline and a minimum of 5 years of progressively responsible experience in biologics and/or gene therapy process development roles OR a Master's degree in these same disciplines and a minimum of 8 years of relevant experience OR a Bachelor's degree in these same disciplines and a minimum of 10 years of relevant experience.
* Special knowledge or skills needed and/or licenses or certificates required.
- Expert level knowledge of GLP and GMP requirements as they pertain to downstream process development and clinical manufacturing for gene therapy products
- Demonstrated experience developing, executing and documenting process validation protocols and validation activities in support of regulatory filings.
- Demonstrated, applied knowledge of CMC and regulatory requirements, including the development of process and product specifications and writing and reviewing development reports.
- Demonstrated, hands-on experience overseeing and conducting technology transfer-in and transfer-out of downstream gene therapy manufacturing processes in all phases of development.
- Significant experience in laboratory operations, including budgeting, capex procurement and I/O/Q, safety, and status reporting to Senior Management is preferred.
- Demonstrated understanding of, and applied experience with, cell-based assays, qPCR, HPLC, ELISA, SDS-PAGE, mass spectrometry and nucleic acid and protein sequencing.
- Demonstrated ability to lead downstream process development activities conducted by Contract Research Organizations (CROs)/Contract Manufacturing Organizations (CMOs).
- Proficiency with Microsoft Office.
- Excellent verbal and written communication skills.
- 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 excellent problem-solving skills and the ability to adapt to changing priorities and deadlines.
- Excellent planning, organization and time management skills including the ability to support and prioritize multiple projects.
*Special knowledge or skills and/or licenses or certificates preferred.
- Experience in GMP upstream, downstream clinical manufacturing and small-scale product filling is preferred.
- Expert level knowledge of mammalian and insect cell culture, virology, and associated upstream processes is preferred.
- Small company and/or start-up experience.
- Experience in high-level interactions with domestic and international Regulatory agencies
- Ability to make and defend high-level decisions as they relate to downstream process development
- Ability to present to and interact with Senior Leadership
* Travel requirements