Pennington, New Jersey

Job Description Summary:

Job Description:

The Scientist II, Downstream Process Development (PD) - Gene Therapy actively contributes to the engineering of downstream process development activities for gene therapy vector production. This includes establishing robust purification processes for Gene Therapies using sound DOE design and develop and execution of robust fill and finish process.

This individual contributes to the design, and actively executes, scale-up studies from lab/pilot scale through full clinical and commercial scale. S/he may also function as PTC's Person in the Plant (PIP). In this capacity, s/he may provide Subject Matter Expertise (SME) for external, Contract Development Manufacturing Organizations (CDMOs)-executed processes to ensure their proper execution. S/he interacts with Upstream Process Development, Analytical Development and Manufacturing personnel to deliver high-quality and robust downstream processes in a regulated environment.

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

The Scientist II, Downstream Process Development - Gene Therapy supports adherence to relevant regulatory requirements and company Standard Operating Procedures (SOPs) as appropriate.

Primary duties/responsibilities:
  • Actively contributes to downstream process development activities for both internal and external projects, including tangential flow filtration, chromatography, formulation, drug substance/product fill/finish automation and freezing.
  • Contributes to the design and execution of small-scale DOE studies to support the development and optimization of robust purification processes with optimal product yields and high quality.
  • Applies experience with separation technologies, focusing on the most current industry recognized resin chromatography and clarification processes used in Gene Therapy manufacturing.
  • Applies experience with single-use bioprocessing equipment and its application to gene therapy downstream process development and GMP manufacturing.
  • Actively contributes to the scale-up of downstream processes. May write reports and train manufacturing personnel as required.
  • May function as the PIP at CDMOs to monitor, and assist with, on-going downstream process development and execution activities.
  • Works closely with Clinical Manufacturing to transfer downstream processes from PD to manufacturing.
  • Collaborates with Upstream and 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. 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 2 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 7 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.

  • Demonstrated ability to lead downstream process development activities internally and externally conducted by Contract Research Organizations (CROs)/Contract Manufacturing Organizations (CMOs).

  • Expert level knowledge of chromatography and membrane filtration, and familiar with GLP and GMP requirements as they pertain to downstream process development and clinical manufacturing for gene therapy products

  • Experience in liquid or lyophilized drug product process development and clinical manufacturing for vaccines, biologics, or gene therapy products are preferred.

  • Understanding of cell-based assays, qPCR, HPLC, ELISA, SDS-PAGE, mass spectrometry and nucleic acid and protein sequencing, UV spectrophotometry, fluorescence, light scattering, DSC, SEC, capillary electrophoresis, microflow imaging, etc.

  • 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.

  • 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.

  • Excellent verbal and written communication skills.

  • Proficiency with Microsoft Office.

*Special knowledge or skills and/or licenses or certificates preferred.

  • Experience with formulation process development and drug substance/product fill/finish.

  • Experience with downstream purification of biological molecules such as preparative chromatography and tangential flow filtration.

  • Demonstrated experience developing, executing, and documenting process validation protocols and validation activities in support of regulatory filings.

  • 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

**At PTC Therapeutics we are seeking candidates in the United States who are fully vaccinated against COVID-19 or who will be fully vaccinated by the time they begin the position.

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.

Click here to return to the careers page

Pennington, New Jersey

PTC is a science-driven, patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for patients with rare disease.

Similar jobs