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Senior Research Scientist - In Vivo Pharmacology

Vertex Pharma
United States, Massachusetts, Boston
October 05, 2022

Job Description

General Summary:

In June 2019, Vertex announced the establishment of a new research site in the Boston area where research, development, and clinical manufacturing for cell and genetic therapies will be primarily based. Cell and genetic therapies represent two rapidly emerging therapeutic modalities with the potential to treat-and even cure-several of the diseases Vertex is focused on, including, Duchenne muscular dystrophy, type 1 diabetes, and sickle cell disease. Further investment in a research site specifically designed to advance these programs will be key to Vertex's continued and sustained leadership in these exciting approaches. At Vertex Cell and Genetic Therapies (VCGT) our research teams will bring together the best biology, technologies, and enhanced manufacturing capabilities to ensure we bring these cutting-edge transformative therapies to patients as quickly as possible.

Our Preclinical In Vivo Pharmacology Team is looking to hire a Senior Research Scientist. The candidate should be adept at designing and executing focused preclinical projects to support the development of CRISPR-Cas9 based gene-editing therapies. The scientist will have the opportunity to lead various preclinical in vivo studies and work together with other Scientists and Research Associates to evaluate, develop and advance lead assets for our therapeutic programs. The ideal candidate is expected to display a high level of passion, drive, and determination, complete work in a resourceful and proactive manner and will be able to design studies and troubleshoot results to achieve the projected goals in a timely manner.

Key Duties and Responsibilities:



  • Apply scientific and technical expertise to gene-editing gene therapy discovery programs to impact novel drug candidates' development.
  • Conceptualize and design experiments; organize and conduct in vivo/ex vivo/in vitro studies independently to evaluate novel gene-editing tools and candidate therapeutic assets.
  • Analyze, interpret, and present scientific data in matrixed team settings. Translate experimental results to advance the understanding of how genetics and complex physiological systems are altered in diseases and how they respond to therapeutic interventions to guide Project decisions and new research activities.
  • Maintain awareness of scientific and technical advancements in the field. Contribute to literature-based assessment of novel therapeutic concepts.
  • Interface with an interdisciplinary team of scientists at VCGT and engage in both disease understanding and driving established programs in the portfolio.
  • Analyze data, draw conclusions, and author summary reports that can be used to support IND filings
  • Maintenance of laboratory notebook records and adherence to safety policies is strictly required.
  • Exercise solid judgment to prioritize studies, manage multiple projects, and complex workflow. Consider feasibility and Project impact.
  • Performs other duties as assigned


Knowledge and Skills:



  • Proficient at working with rodent models of disease, at IP, IV, SC injections, cardiac puncture, blood collection (tail nick, SM), necropsy, and tissue harvesting and as well as other laboratory techniques (for example, NGS, cell culture, Western Blots etc.)
  • Demonstrates a broad working knowledge of fundamental molecular biology and pharmacology principles to design and execute more complex, challenging experiments that measure the action of gene editing assets on in vitro and in vivo disease biology assays
  • Displays greater ability to productively disseminate ideas across discipline- and project-boundaries
  • Communicates effectively in verbal presentations and in writing technical reports
  • The candidate will have daily interactions with a team of creative and innovative scientists in a supportive environment.
  • Excellent communication and interpersonal skills with the proven ability to build open and collaborative relationships and work effectively as a member of a multidisciplinary team are essential.


Education and Experience:



  • A Ph.D. in Genetics, Molecular Biology, or related disciplines with at least two years of relevant post-doc or pharmaceutical industry experience.
  • Experience in muscle biology, muscular dystrophies, and AAV gene therapy is preferred.
  • Experience in CRISPR/Cas9 gene editing and next-generation sequencing is preferred.
  • Expertise in preclinical muscular dystrophic disease models is a plus.
  • A successful track record of scientific achievements through top-tier scientific publications, presentations, or equivalent industry accomplishments.


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.

Please note that Vertex requires COVID-19 vaccination for all employees working in the U.S. as a condition of employment at Vertex, subject to medical, religious or other accommodations or exemptions required by law. We believe this requirement is vital to keeping everyone at Vertex and in our communities safe. This requirement applies to both existing and new employees who may at any time work on-site at a Vertex facility or visit another location on behalf of Vertex (e.g., conferences, customer visits, etc.), including all remote employees. 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 (see application details).

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