Insights on Discovery

  1. Variant-Aware Therapeutic Guide Selection 2/21/2025

    Discover a technology that identifies CRISPR guide RNAs with minimal off-target effects for therapeutic gene editing by screening thousands of genomes and incorporating genetic diversity across populations.

  2. SAFER Detection For Efficient Interrogation Of DNA Rearrangements 2/21/2025

    Learn about SAFER Detection™, a Next-Generation Sequencing (NGS) based method for the efficient identification of DNA rearrangements in gene-edited human cells.

  3. A Standardized T Cell Backbone Panel For Immune Monitoring Of Adoptively Transferred Cellular Therapies 2/14/2025

    This validated 22-marker flow cytometry panel enables deep immune profiling to identify key biomarkers influencing ACT efficacy — optimizing cell therapy outcomes and improving patient responses.

  4. Biodistribution Of AAV6.2-mCherry In C57BL/6J Mouse Dosed Via Nose-Only Inhalation Route 2/13/2025

    Explore how a rodent nose-only AAV inhalation model can optimize the safe and effective delivery of aerosolized gene therapies for monogenic pulmonary diseases.

  5. Limiting Off-Target Effects Of CRISPR-Based Products 2/11/2025

    Find out how to enhance CRISPR gene editing specificity by using Next Generation Sequencing to improve sgRNA quality control, addressing off-target effects and ensuring higher sequence fidelity.

  6. Applying Next Generation Sequencing To Accelerate CGT Product Development 2/11/2025

    Next Generation Sequencing (NGS) revolutionizes gene therapy by enhancing the development and safety of viral vectors like AAV and lentivirus. Discover more about its impact.

  7. Adapting A Hybrid Analysis For AAV Characterization 2/11/2025

    Gain insights into utilizing hybrid long-read and short-read sequencing to analyze adeno-associated virus capsids to enhance biosafety testing and genome assembly while achieving high identity mapping.

  8. Integrating Analytical Method Development And Quality Control 2/11/2025

    As cell and gene therapies continue to advance, a well-integrated analytical framework will be critical for accelerating development timelines and optimizing regulatory submissions.

  9. Comparing Synthesis Processes To Establish rAAV Batch Quality 2/5/2025

    In this study, we present a comprehensive analysis of various published and newly developed dsDNA conversion processes that were tested using the same sequencing chemistry.

  10. Targeted CRISPR/Cas9 Screen Identifies Superior HEK293 Cell Lines For AAV9 Production 2/5/2025

    In this study, we developed a proprietary clonal suspension-adapted HEK293 cell line (AC001.230) through clonal isolation and high-throughput screening, resulting in improved productivity for 7 out of 10 AAV serotypes compared to 293F cells.