Principal Scientist, Quality Control - Gene Therapy at PTC Therapeutics, Inc. in Pennington, New Jersey

Job Description:

Job Description Summary:

Job Description:

In this newly created role, the Principal Scientist, Quality Control (QC) – Gene Therapy leads and provides Subject Matter Expertise (SME) for the testing of Gene Therapy clinical and commercial products. Actively contributes to and provides oversight for the assays required to perform QC activities. This involves raw data review and approval to support the manufacturing of gene therapy products.

She/he will manage direct reports.

The incumbent works cross-functionally with internal departments and external resources as appropriate on issues involving assigned QC assays.

The Principal Scientist, Quality Control – Gene Therapy supports adherence to relevant regulatory requirements and company Standard Operating Procedures (SOPs) as appropriate.

Primary duties/responsibilities:

• Provides leadership for, and actively contributes to, raw data review and approval for validated assays performed at Contract Testing/Manufacturing Organizations and in-house validated methods.

• Supports gene therapy programs by routinely performing data trending and analysis of Stability data generated for the assigned gene therapy program(s).

• Provides oversight for assigned assay-related activities conducted in-house or by Contract Research Organizations (CROs)/Contract Manufacturing Organizations (CMOs).

• Provides assigned assay-related technical expertise in cross-functional team(s) to review analytical data and provide guidance on quality events impacting data for the validated assays.

• Contributes to authoring and/or reviewing method development reports, SOPs, validation protocols/reports, comparability protocols/reports, justification of specifications (JoS), and other relevant sections of regulatory filings. These include, but may not be limited to, Investigation New Drugs (INDs), Investigational Medicinal Product Dossier (IMPDs) and Biological License Application (BLAs).

• Assists in advancing PTC’s scientific and regulatory capabilities via active participation and leadership across industry trade groups and conferences.

• Builds the QC capabilities and strategies in gene therapy product GMP testing that enable fast to clinic/fast to market product development and commercialization.

• Actively contributes to, and assures strict adherence to, Good Manufacturing Practices (GMPs) and regulations to support clinical development and commercial manufacture.

• Contributes to leading Health Authority analytical related observation responses.

• Provides leadership for Out of Specification (OOS)/Out of Trend (OOT) laboratory investigations.

• Participates in the implementation of a Laboratory Information Management System (LIMS).

• Manages, coaches and mentors direct reports. This includes providing scientific and technical support to help troubleshoot and solve scientific and Quality challenges.

• Performs other tasks and assignments as needed and specified by management.

* Minimum level of education and years of relevant work experience.

• PhD degree in a Molecular Biology, Virology or Biochemistry related discipline and a minimum of 8 years of Quality Control experience in a pharmaceutical, biotechnology or other related industry OR a Master’s in a Molecular Biology, Microbiology or Biochemistry related discipline and a minimum of 10 years of Quality Control experience in a pharmaceutical, biotechnology or other related industry.

* Special knowledge or skills needed and/or licenses or certificates required.

• Prior Current Good Manufacturing Practices (cGMP) experience in a QC laboratory, which supports the testing of biological and/or gene therapy products

• Demonstrated understanding of current Good Manufacturing Practices (cGMPs).

• Applied knowledge of mammalian cell culture.

• Expert knowledge of cell-based assays, qPCR/ddPCR, HPLC, SEC-UPLC, ELISA, CE-SDS , SDS-PAGE, mass spectrometry, DLS, AUC and other relevant molecular biology and bioanalytical techniques.

• Prior experience the late stage clinical and commercial release testing conducted by CROs/CMOs.

• Demonstrated experience in developing strategic plans based on business needs and balancing Quality Control testing needs for multiple programs.

• Strong understanding of Quality Control in pharmaceutical/biotechnology industries.

• Proficient with statistical tools like JMP, MiniTab, etc.

• Strong knowledge of QC Analytical testing methodologies, method validation and method transfers.

• Strong knowledge of quality systems and ability to interpret Quality standards for implementation and review.

• Demonstrated leadership ability and experience managing, coaching and mentoring direct reports.

• Demonstrated ability to remain flexible and to adapt to company growth and change.

• Proficiency with Microsoft Office.

• Excellent verbal, written and presentation communication skills.

• Ability to work independently and collaboratively, as required, in a fast-paced, matrixed, team environment consisting of internal and external team members.

• Detail-oriented and 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 multitask, support and prioritize multiple projects at various stages of development.

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

• Experience in interacting with CROs/CMOs.

• Experience successfully managing/directing CMO relationships.

• Small company and/or start-up experience.

• Prior experience with reviewing/ authoring Standard Operating Procedures, work instructions, protocols, methods, method validations.

**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.

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