Expert Insights On CGT Formulation

  1. 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.

  2. 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.

  3. 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.

  4. 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    FDA's CDER recently issued a draft guidance: Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics.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.

  5. 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.

  6. Forum Notebook: Quick Takes From USP's mRNA Virtual Summit 5/1/2024

    The United States Pharmacopeia held a forum in February to exchange ideas about analytical methodologies and release testing. Here's a recap of the event.

  7. Tracking 4 Payload Trends In Drug Delivery Development 1/22/2024

    Amid new research and development in nanocarriers, market attention has shifted toward payloads and enhancing compatibility. This discussion explores the latest trends and includes a research database.

  8. FDA Sets Recommendations For Predicting Dangerous Nitrosamine Impurities 10/25/2023

    The FDA recently published a final guidance that sets a recommended framework for predicting the potential of nitrosamine drug substance-related impurities.

  9. Defining The RNA Therapeutics Industry In 2023 1/16/2023

    Four executives outline which industry challenges, strategic considerations, and opportunities should be top of mind as RNA therapeutics companies establish their to-do lists for 2023.

  10. The Expanding Patent Landscape For RNA-Based Therapeutics 11/16/2022

    The COVID-19 pandemic thrust RNA-based therapeutics into the spotlight. Before that, only a handful had received regulatory approval. Why are RNA-based therapeutics seeing more patent activity now and how should biotech companies mitigate patent risks? Attorneys from Neal Gerber Eisenberg share their insights.