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Benchling for Gene Therapy and Gene Editing R&D

Track genetic materials, design vectors, model genetic engineering workflows, and quickly iterate on workflows to optimize process development.
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Several hurdles persist for companies seeking to develop novel gene therapies

Gene editing is an area of significant research interest, especially with the recent approval of breakthrough therapies like Luxturna. This paper outlines the critical needs and complexities of gene therapy R&D, and how Benchling has helped address these challenges for leading gene therapy and gene editing companies.
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Gene Therapy and Gene Editing Research

  • Manage a wide range of genetic material and vectors

    • Perform CRISPR guide design with built-in on-target / off-target scoring
    • Create a library of genetic material and link with physical inventory
    • Design, create, and map relationships between components of gene therapy

  • Support a variety of genetic engineering and vector design workflows

    • Build custom workflows to manage a variety of gene editing workflows
    • Tailor viral and non-viral vectors to fit the needs of your programs
    • Track editing efficiency, transfection efficiency, yield, and other metrics

  • Prevent genotoxicity from integrating vectors or off-target genome editing

    • Track genotoxic sequences including specific enhancers and promoters
    • Study different vectors and their distinct integration profiles
    • Design vectors with lower risk of insertional mutagenesis

Gene Therapy and Gene Editing Development

  • Support product characterization concurrent to process optimization

    • Establish relationship between critical quality, material, and process attributes
    • Integrate process monitoring technologies for better in-process control
    • Centralize in-process, product release, and investigational assay results

  • Integrate upstream and downstream processes for vector product

    • Seamlessly integrate upstream and downstream vector production operations with built-in flexibility
    • Track process output such as transfection efficiency, viability, purity, potency, and yield
    • Establish a continuous real-time feedback loop throughout the entire process

  • Improve production efficiency and number of manual steps

    • Design and map entire production process for the gene therapy product
    • Integrate with semi-automated and automated bioreactors for vector production
    • Create a custom dashboard for production metrics and analytics to improve process

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