Expert Insights On CGT Formulation

  1. Strained Manufacturing, Complexity Stymie In Vivo Progress 12/4/2025

     Explicitly mapping delivery challenges to the associated GMP process steps converts biological risk into operational risk.

  2. To Fight Cancer, Gene Editing Needs To Solve Its Delivery Problem 12/4/2025

    The choice of delivery dictates the complexity of manufacturing, which in turn determines the eventual cost and accessibility of the therapy.

  3. Formulation And Delivery Considerations For Cell And Gene Therapies 11/25/2025

    How can we ensure cell and gene therapies are truly transformative for patients? Success depends on the right combination of formulation, preparation, and delivery.

  4. Rethinking The "3:1 Rule" In LNP Production 11/21/2025

    A new two-step, ideal-mixing strategy from MIT researchers shows how operating briefly in unstable regions of LNP assembly can unlock precise control of particle size and shape—advancing next-gen RNA manufacturing.

  5. Emerging RNA Delivery Strategies For Solid Tumor Oncology 11/20/2025

    RNA shows promise in solid tumors, but delivery remains the core challenge. New targeting and delivery strategies aim to overcome tumor barriers and unlock RNA’s full therapeutic potential.

  6. When One Gene Isn't Enough: The Case For Gene-Agnostic Vision Rescue 11/7/2025

    The genetic diversity of retinitis pigmentosa challenges the one-gene-one-therapy model and positions RP as a proving ground for gene-agnostic treatments designed to preserve or restore visual function across genotypes.

  7. Unlocking Method Insights For Novel Engineered AAV Capsids 9/18/2025

    Microfluidic platforms, such as labchip, are widely used for assessing wild type capsids. Now, engineered capsids are challenging these established workflows.

  8. Tune Therapeutics' 2025 Epigenome Editing Outlook 1/10/2025

    Chief Scientific Officer Derek Jantz talks about recent key innovations in gene therapy and RNA technology while considering what milestones we might see in 2025.

  9. FDA Issues New Guidance: Nonclinical Safety Assessment Of Oligonucleotide-Based Therapeutics 12/11/2024

    FDA's CDER recently issued a draft guidance: Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics. This article provides a summary and analysis. The public comment period ends January 14, 2025.

  10. Points To Consider For Assessing Lipid Quality For Lipid Nanoparticle Manufacturing 5/29/2024

    Lipid nanoparticles have generated a ton of buzz, but they pose risks that, perhaps, industry and regulators have not taken seriously enough so far.