Scientist I, Gene Therapy Research

Sarepta Therapeutics Inc. Dublin , OH 43016

Posted 2 months ago

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 drug research and development for neuromuscular diseases. The successful candidate will participate in target discovery, validation efforts, characterization of pre-clinical animal models of neuromuscular diseases, and demonstration of the mechanism of action of gene therapies through in vitro and in vivo pre-clinical proof of concept studies. Key activities will include designing and conducting critical pre-clinical efficacy and safety studies in order to support development of neuromuscular gene therapies through pre-clinical research and into clinical development.

Primary Responsibilities Include:

  • Serve as subject matter expert on gene therapy pre-clinical research for existing and new programs.

  • Conducts laboratory activities in Sarepta in-house laboratory, interacts with research, translational biology and bioanalytical groups.

  • Ownership of a new or ongoing gene therapy program to drive pre-clinical research through stage gates and key milestones in order to support regulatory and clinical development

  • Work with the extended gene therapy research teams to assist in completing data gap studies for programs under development

  • Investigate disease pathogenesis and new biomarker and other outcome measures of safety and efficacy for use in gene therapy studies

  • Assist in transition to becoming a GLP facility

  • Maintains compliant laboratory records, consistently and accurately documents all laboratory activities in laboratory notebooks and internal technical reports

  • 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 development reports and powerpoint slide decks, study protocols, and pre-clinical study reports to communicate ongoing activities internally and externally

  • Perform other related duties incidental to the work described

Desired Education and Skills:

  • Ph,D. in biological sciences or related field with 0-3 years experience or M.S. degree in biological sciences or related field with 5+ years relevant lab experience

  • A strong understanding of molecular biology across a wide range of technologies (qPCR, SDS-PAGE, Western blotting, cloning).

  • Ability to plan and carry out experiments independently with strict adherence to laboratory methods and SOPs.

  • Experience working with animal models and/or in gene therapy or related field is useful.

  • Experience in cell biology, muscle biology, or knowledge of neuromuscular diseases is useful.

  • Demonstrates proficient written and oral communication and presentation skills and a documented ability to troubleshoot technical issues.

  • Experience with statistical and computer productivity software (i.e. Word, Excel, Outlook, etc.).

  • Desire to work in a high-functioning, collaborative team

  • 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: Drive, Excellence, Resilience, Teamwork, Innovation and Compassion

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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