Job Description

General Summary

The DMD Biology team at Vertex Cell and Genetics Therapy Research (VCGT Research) is seeking a highly experienced and motivated Principal Research Associate to build genetic therapy candidates and to develop and execute assays to support advancing non-viral therapeutics through program stage gates. The successful candidate will bring strong hands-on expertise in molecular biology, cell biology, gene-expression analysis, and protein-based assays. This individual will work closely with cross-functional partners to generate high-quality data that informs discovery-stage decisions and candidate progression. This laboratory-based role will design, execute, optimize, and interpret molecular and cellular assays that characterize functional delivery, intracellular activity, gene expression, and immunological and pharmacological responses.

Key Responsibilities

• Build gene expression constructs using advanced molecular biology tools.
• Design, execute, and optimize in vitro assays supporting non-viral delivery biology and gene replacement strategies for Duchene Muscular Dystrophy.
• Develop and apply cellular and molecular biology workflows to evaluate uptake, dose response, intracellular trafficking, functional delivery, gene expression, and pharmacological activity of therapeutic candidates for Duchene Muscular Dystrophy.
• Perform nucleic acid extraction and downstream gene-expression analyses, including qPCR, RT-qPCR, NGS library construction, and related molecular characterization methods.
• Execute protein and cellular assays, including but not limited to ELISA, MSD, Western blotting/Jess, immunoprecipitation, reporter gene assays, and cell-signaling assays to assess cellular responses and delivery outcomes.
• Support both in vitro and in vivo evaluation of gene-editing and gene-replacement approaches using relevant molecular and cellular readouts.
• Collaborate closely with cross-functional teams and external partners to design studies, communicate findings, and advance program priorities.
• Prepare clear experimental and technical summaries, data reports, and presentations; proactively communicate findings.
• Maintain accurate, timely, and well-organized electronic laboratory notebook records and structured documentation/SOPs.

Knowledge and Skills:

• Significant hands-on laboratory experience in molecular cloning, molecular biology assays, cell biology assays, protein-based assays, and mammalian cell culture.
• Experience with: RNA/DNA extractions, qPCR, plasmid construct design, gene expression analysis, reporter assays, Western Blot/Jess, MSD, immunoprecipitation, ELISA etc.
• Experience with one or more of the following: RNA/DNA therapeutics, Lipid nanoparticle (LNP) dosing, receptor biology and intracellular trafficking, gene expression, cell transfection/transduction experiments, or confocal imaging.
• Strong collaboration skills, with the ability to work in a highly cross functional, fast-paced research environment.
• Strong written and verbal communication skills, including the ability to summarize complex experimental findings clearly.
• Experience with one or more of the following: IND-enabling studies, data visualization, report writing, electronic lab notebook keeping (e.g. Benchling), or structured data documentation.

Education and Experience

• Master’s Degree (or equivalent degree) in biological sciences (or a related field) and 8+ years of relevant employment experience.
• Bachelor’s Degree (or equivalent degree) in biological sciences (or a related field) and 10+ years of relevant employment experience.
  

At Vertex, we embrace continuous learning and encourage employees to stay informed on advances in AI, technology, automation, and industry trends. We seek talent who bring an external perspective, demonstrate curiosity and sound judgment, and thoughtfully adopt new capabilities that improve decision-making, drive innovation, increase efficiency, and help deliver better outcomes for patients.

Pay Range:

Disclosure Statement:

Flex Designation:

Flex Eligibility Status:

In this On-Site designated role, you will work five days per week on-site with ad hoc flexibility.  

Note: The Flex status for this position is subject to Vertex’s Policy on Flex @ Vertex Program and may be changed at any time.

#LI-Onsite

Company Information

Vertex is a global biotechnology company that invests in scientific innovation.

Vertex is committed to equal employment opportunity and non-discrimination for all employees and qualified applicants without regard to a person's race, color, sex, gender identity or expression, age, religion, national origin, ancestry, ethnicity, disability, veteran status, genetic information, sexual orientation, marital status, or any characteristic protected under applicable law. Vertex is an E-Verify Employer in the United States. Vertex will make reasonable accommodations for qualified individuals with known disabilities, in accordance with applicable law.

Any applicant requiring an accommodation in connection with the hiring process and/or to perform the essential functions of the position for which the applicant has applied should make a request to the recruiter or hiring manager, or contact Talent Acquisition at ApplicationAssistance@vrtx.com