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: $88,200 - $132,400 Disclosure Statement: The range provided is based on what we believe is a reasonable estimate for the base salary pay range for this job at the time of posting. This role is eligible for an annual bonus and annual equity awards. Some roles may also be eligible for overtime pay, in accordance with federal and state requirements. Actual base salary pay will be based on a number of factors, including skills, competencies, experience, and other job-related factors permitted by law. At Vertex, our Total Rewards offerings also include inclusive market-leading benefits to meet our employees wherever they are in their career, financial, family and wellbeing journey while providing flexibility and resources to support their growth and aspirations. From medical, dental and vision benefits to generous paid time off (including a week-long company shutdown in the Summer and the Winter), educational assistance programs including student loan repayment, a generous commuting subsidy, matching charitable donations, 401(k) and so much more. Flex Designation: On-Site Designated 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 Help Us Achieve Our Mission Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia, acute pain and acromegaly, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, congenital adrenal hyperplasia, ACTH-dependent Cushing's syndrome, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 16 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube, and X We believe our culture is one of Vertex’s greatest strengths and a key enabler of our ability to deliver transformative medicines for patients who need them. Rooted in innovation, purpose and collaboration, our culture empowers people to take smart risks, challenge conventional thinking and do their best work. The diversity of our people is central to that culture. By bringing together individuals with different backgrounds, experiences and perspectives, we foster an environment where new ideas thrive, better decisions are made and breakthrough innovation is possible. We are committed to creating a workplace where everyone feels valued, included and connected to our shared mission, because we know that our unique culture and the collective strength of our people are what set Vertex apart.

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