Insights on Discovery

  1. Design Of Experiment (DoE) Based High Throughput Process Optimization For rAAV Manufacturing Development 2/5/2025

    This study presents the results of a Design of Experiment (DoE) approach to assess how these process parameters impact rAAV yield and quality.

  2. Quality Over Quantity: Where Is The Trade Off? 2/5/2025

    Our study aims to compare two widely used transfection reagents to our standard process, evaluating their impact on final product quantity and quality as part of our ongoing platform innovation efforts.

  3. New Hope For Neuronal Diseases With Polymer Nanoparticle-Based Gene Therapy 1/28/2025

    A new polymer nanoparticle (PNP) system enables targeted delivery of large genetic payloads to neuronal cells, advancing gene therapies for NF1 and other nervous system disorders.

  4. Fit-For-Purpose Biomarker Development 1/17/2025

    Here, we highlight the essential framework of a “fit-for-purpose” biomarker development strategy, emphasizing strategic and transactional engagements with clinical research organizations.

  5. Biomarkers In Oncology Studies 1/17/2025

    Here we provide an overview of oncology biomarker development and strategic considerations.

  6. LC-MS/MS Method Development Of Endogenous Small Molecule Biomarker 1/17/2025

    Biomarkers are essential to improve drug R&D efficiency. Explore some practical solutions for achieving a fit-for-purpose LC-MS/MS biomarker analysis method.

  7. Exploring Progress In Nanomedicine: Unlocking New Possibilities For Treating Rare Genetic Diseases 1/15/2025

    Gene therapy offers hope for rare genetic diseases, with advancements in polymer nanoparticles addressing delivery challenges and paving the way for innovative, effective treatments.

  8. Structural Characterization Of RNA And Detection Of RNA-Ligand Binding 1/13/2025

    Delve into the intricacies of riboswitch behavior and ligand interactions and gain valuable insights into gene regulation and potential therapeutic targets.

  9. Assigning IR Absorption Bands For RNA Building Blocks Using Microfluidic Modulation Spectroscopy 1/13/2025

    Explore the power of MMS in identifying RNA base signatures. This study analyzes vibrational modes of A, G, C, and U, revealing insights into electronic environments and molecular interactions.

  10. Guidelines And Strategies For The Development, Qualification Of Potency Assay 1/7/2025

    Explore key strategies and guidelines to advance the development and qualification of robust potency assays for gene therapy products.