Associate Dir, Upstream Process Development – Gene Therapy (Biotech)

Job Description Summary:

Job Description:

The Associate Director, Upstream Process Development (PD) – Gene Therapy leads, and may actively contribute to, the engineering of upstream process development activities for gene therapy vector production. This includes, but may not be limited to, cell and virus culture as well as small-scale bioreactor process DOE design and execution.

This individual leads the design and execution of 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 internally executed processes, as well as external, Contract Development Manufacturing Organizations (CDMOs)-executed processes, to ensure their proper execution.   S/he interacts extensively with downstream Process Development, Analytical Development and Manufacturing personnel to deliver high-quality and robust upstream processes in a regulated environment.  

S/he manages direct reports.

The incumbent works cross-functionally with internal departments and external resources as appropriate on upstream process development-related issues.

The Associate Director, Upstream Process Development – Gene Therapy supports adherence to relevant regulatory requirements and company Standard Operating Procedures (SOPs) as appropriate.

Primary duties/responsibilities:

• Provides leadership for, and may actively contribute to, upstream process development activities for both internal and external projects.

• Functions as the PIP at CDMOs to monitor, and assist with, on-going upstream process development and execution activities.

• Leads, and may also design and execute, small-scale shake flask, 2-D multilayer flask and stirred tank bioreactor studies to support the development of cell, virus and plasmid DNA-based gene therapy upstream manufacturing processes.

• Applies experience with fed-bath and perfusion cell culture in transfection- and infection-based viral vector gene therapy process development and assists his/her direct reports (as appropriate) to develop/further develop their experience in these skills.

• Applies expertise with single-use bioprocessing equipment and its application to gene therapy upstream process development and GMP manufacturing and assists his/her direct reports (as appropriate) to develop/further develop their experience in these skills.

• Works closely with Clinical Manufacturing to transfer upstream processes from PD to manufacturing.   

• Leads scale-up of upstream processes, supports process characterization and validation activities, writes reports and trains manufacturing personnel as required.

• Collaborates with Downstream to build process understanding and design space via design and execution of in-process testing and control assays

• 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.

• Leads, assures, and may actively contribute to, 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.

• Leads, and actively contributes to writing and/or performing the QC review of relevant sections of global regulatory filings. These include, but may not be limited to, INDs, BLAs and ex-US filings

• Manages, mentors, coaches 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 6 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 9 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 upstream 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 upstream 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 upstream 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 downstream 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 upstream process development

• Ability to present to and interact with Senior Leadership

EEO Statement:

PTC Therapeutics is an equal opportunity employer. We welcome applications from all individuals, regardless of race, color, national origin, gender, age, physical characteristics, social origin, disability, religion, family status, pregnancy, sexual orientation, gender identity, gender expression, disability, veteran status or any unlawful criterion under applicable law. We are committed to treating all applicants fairly and avoiding discrimination.

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