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.
Sarepta Therapeutics Inc.