Sr. Scientist - Molecular Biologist, Vector Core/Gene Therapy

Sarepta Therapeutics Columbus , OH 43216

Posted 4 months ago

The Position:

As a member of the Vector Core group this molecular biologist position will help drive the development of Sarepta's Gene Therapy programs and be responsible for the design and characterization of plasmid constructs for early-stage production processes for AAV vector research pipeline. The team will collaborate with Analytical Development, Process Development and additional internal and external stakeholders to ensure the development and transfer of robust processes to facilitate fit for purpose clinical and commercial AAV vector production.

Primary Responsibilities include:

  • Provide leadership role in building and optimizing the design of AAV gene therapy constructs

  • Responsible for hands-on experimental design, execution, data analysis and reports for AAV vector core pipeline research and development programs

  • Develop molecular assays to characterize AAV genome integrity, identity, vector variants, residual DNA quantification, as well as in-vitro potency to support product development and manufacturing of gene therapy products

  • Identification and implementation of operational improvements and novel technologies to improve AAV vector expression and key quality attributes

  • Participate in project related teams and other internal/external collaborations

  • Prepare and review technical reports and SOPs to support CMC regulatory activities

  • In collaboration with Gene Therapy Technical Operations Leadership, develop strategic and operational vision for the organization

  • Select, develop, and evaluate personnel to ensure the efficient operation of the function and represent the department internally/externally

  • Work seamlessly with cross functional stakeholders including Process Development, Analytical Development and Translational Research to help advance the AAV vector production portfolio

  • Ensure the development of robust, well-characterized, high quality and high yielding vector expression system

  • Ensure that scientific and technical challenges are resolved in a timely manner

  • Contribute to budget and goal planning

  • Contribute to a safe, efficient and effective environment with personal accountability

Education and Skills Requirements:

  • Ph.D. with 6+ years related experience or MS with 10+ years related experience with degree in Molecular/Cellular Biology, Virology or a related discipline

  • Deep understanding of plasmid expression cassettes, vector biology and molecular techniques

  • Extensive hands-on experience with molecular and cellular assays critical for biologics drug discovery or gene therapy development research including plasmid cloning, E. coli screening, restriction digestion mapping, DNA sequencing, qPCR, RT-qPCR, gel or capillary electrophoresis, and in vitro plasmid expression

  • Experience in ELISA and western blot is required

  • High-throughput qPCR experience is preferred

  • Experience in ddPCR and NGS is desirable

  • Experience in process design utilizing QbD and DOE principles via high-throughput small-scale approaches for optimization of viral vectors or biologics production is required

  • Able to gather internal/external benchmarks for best practices in the scientific field

  • Familiar with upstream techniques used in the growth of biologics including suspension and adherent culture of mammalian or insect cells

  • Solid communication and interpersonal skills to help build strong relationships within and across various drug discovery and development functions

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.

icon no score

See how you match
to the job

Find your dream job anywhere
with the LiveCareer app.
Mobile App Icon
Download the
LiveCareer app and find
your dream job anywhere
App Store Icon Google Play Icon

Boost your job search productivity with our
free Chrome Extension!

lc_apply_tool GET EXTENSION

Similar Jobs

Want to see jobs matched to your resume? Upload One Now! Remove
Scientist I Gene Therapy Research

Sarepta Therapeutics

Posted 2 months ago

VIEW JOBS 8/22/2019 12:00:00 AM 2019-11-20T00:00 The Position: This is an exciting opportunity for a highly motivated and creative individual to work in a key drug discovery group in our research organization and play a central role in applying Sarepta's DMD expertise to gene therapy drug development for LGMD. The successful candidate will participate in target discovery and validation efforts, characterization of mechanism of action of gene therapy through preclinical proof of concept, and translation of LGMD gene therapies through pre-clinical development into clinical trials. Primary Responsibilities Include: * Serve as subject matter expert on gene therapy pre-clinical development for existing and new programs. * Conducts laboratory activities in Sarepta in-house laboratory, interacts with research, translational biology and bioanalytical groups. * Create and maintain clear and concise lab records and documentation * Author development reports, study protocols, and pre-validation reports * Complete data gap studies for sarcoglycan programs * Complete LGMD2L (ANO5) pre-clinical dose finding studies * Restoration of DAPC, transgene and functionality of the DAPC * Development of in vitro potency assays * Development of immunological transgene ELISA assays for each LGMD program * Coordinate with TD on clinical biomarkers to assess same biomarkers in Limb Girdle animal model * Validate the biodistribution and shedding assay for the respective drug product * Deliver study reports in a timely fashion to support regulatory filings * Contribute to pre-clinical development of new LGMD GT programs * Perform other related duties incidental to the work described Education and Skills Requirements: * Ph,D. with 0-3 years experience or M.S. degree in Biology 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). * Experience with pre-clinical animal models is preferred. * Experience in gene therapy or related field is useful. * Must have effective written and verbal communication skills * Operate as part of a team; assisting fellow lab associates in providing a functional, safe working environment while maximizing laboratory efficiency 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 Columbus OH

Sr. Scientist - Molecular Biologist, Vector Core/Gene Therapy

Sarepta Therapeutics