Scientist I, Gene Therapy R&D

Sarepta Therapeutics is seeking a motivated and creative individual to work in a key drug discovery group in our research organization and play a central role in gene therapy research and development for rare diseases. The successful candidate will participate in target discovery, characterization of pre-clinical animal models of neuromuscular and cardiac diseases, and demonstration of the mechanism of action of gene therapies through in vitro and in vivo pre-clinical studies. This person will be expected to work within the framework of a GLP-compliant laboratory, maintain good documentation practices, and adhere to standard chemical and biohazard safety guidelines while working in the laboratory. Key activities will include designing and conducting critical pre-clinical efficacy and safety studies to support development of neuromuscular and additional gene therapies through early stage research and into clinical development.Primary Responsibilities Include:Serve as subject matter expert on gene therapy pre-clinical research for existing and new programs.Serve as subject matter expert on equipment for molecular assays.Participate in new or ongoing gene therapy programs to drive pre-clinical research through stage gates and key milestones to support regulatory and clinical development.Assist in the execution of pre-clinical study sample analysis using a wide variety of assay formats such as ddPCR, RT-ddPCR, Jess, Western Blot, and ELISA.Assist in the method development of novel assays.Work with the extended gene therapy research teams to execute data gap studies for programs under development.Investigate disease pathogenesis, new biomarkers and other outcome measures of safety and efficacy for use in gene therapy studies.Work with manager to design, plan, and execute pre-clinical gene therapy efficacy and safety studies in collaboration with vivarium staff, project management, and other key stakeholders.Author PowerPoint presentations, study protocols, and pre-clinical study reports to communicate ongoing activities internally and externally.Document work in accordance with good documentation practices.Desired Education and Skills:Ph.D. in biological sciences or related field with 0-3 years of experience or M.S. in biological sciences or related field with 5+ years relevant lab experience.Expertise in molecular biology across a wide range of technologies (qPCR or ddPCR, Western blot or Jess, SDS-PAGE, ELISA, immunofluorescence) is required.Experience working with animal models, muscle biology and/or in gene therapy or related field is required.Experience with plasmid design is preferred.Experience conducting in vitro and ex vivo functional experiments on skeletal and/or cardiac muscle cell models is preferred.Experience or expertise with neuromuscular disease and mechanism of action discovery studies preferred.Experience with cell culture techniques and protocols is preferred.Ability to plan and carry out experiments independently with strict adherence to laboratory methods and SOPs.Demonstrates excellent written and verbal communication skills and a documented ability to troubleshoot technical issues.Experience with statistical and computer productivity software (i.e., Word, Excel, Outlook, GraphPad).Desire to work in a dynamic, fast-paced team environment and to make a difference in the lives of people through our innovative medicines.Recognized by former peers, colleagues, managers, and direct reports for attributes congruent with Sarepta Values.This position requires work on site at one of Sarepta's facilities in the United States.The targeted salary range for this position is $91,200 - $114,000 per year. Sarepta is making a good faith effort to be transparent and accurate around our hiring ranges. The salary offer is commensurate with Sarepta's compensation philosophy and considers factors including, but not limited to, education, training, experience, external market conditions, criticality of role, and internal equity.Candidates must be authorized to work in the U.S.Sarepta Therapeutics offers a competitive compensation and benefit package.Sarepta Therapeutics is an Equal Opportunity/Affirmative Action employer and participates in e-Verify.

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