REGENXBIO is searching for a research scientist to join the Translational Research laboratory to design and conduct appropriate preclinical studies to support the development of gene therapy clinical candidates.
Support the lead scientist in the Translational Research lab supporting the REGENXBIO portfolio.
Support pharmacology studies to support the development of a clinical candidate to initial clinical studies.
Assist in lab management tasks, such as material inventory, ordering and equipment maintenance.
Perform in vitro experiments to address key mechanistic and pharmacological scientific questions relating to AAV biology that is relevant to the disease target.
Design, complete experimental studies, carry out data analyses and interpretation, record results, and report findings to the team.
Interact with a team of scientists and engage in both target exploration and driving established projects in our portfolio.
Present, discuss and review data within R&ED and with other stakeholders.
Work cross-functionally across the organization, including Regulatory, CMC, Clinical, Quality Assurance, Bioanalytical Sciences and other groups.
Present/attend scientific conferences.
SKILLS, KNOWLEDGE AND ABILITIES REQUIRED
Ph.D. in Biomedical Sciences or BS/MS in Biomedical Sciences with experience
Proficiency with standard assays (e.g. qPCR, flow cytometry, imaging, luminescence-/fluorescence-based cellular readouts).
Experience with animal models and cell culture
Ability to work independently and take responsibility for the progression of small projects.
Experience with gene therapy, in particular working with AAVs.
Demonstrated ability to analyze and resolve problems.
Must possess excellent oral and written communication skills.
Knowledge of molecular biology techniques such as DNA, RNA, and protein isolation; qRT-PCR, Western blot, ELISA, Immunohistochemistry.
Desire to constantly review, consider and develop emerging cell biology technologies.
Experience working with animal models of disease
Experience of working on several projects simultaneously and the ability to work independently as well part of a team.
Suggest and implement changes to experimental procedures and data analyses to increase quality of output and minimize time.
WORKING CONDITIONS AND/OR PHYSICAL REQUIREMENTS
Normal working conditions
Internal Number: RE-18-00010
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, our gene therapy product candidates are designed to provide long-lasting effects, potentially significantly altering the course of disease and delivering improved patient outcomes. We are currently developing gene therapy product candidates for the treatment of retinal, metabolic, and neurodegenerative diseases. Our product candidates all utilize AAV viral vectors from our proprietary gene delivery platform, which we call our ...NAV Technology Platform. In addition to our internal product candidate programs, we also selectively license our NAV Vectors to other leading biotechnology companies.