FORMULATION ARTICLES
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Analytical Platforms: The Foundation Of The Next Generation Of RNA Therapeutics
RNA therapeutics are entering a new era where analytical platforms drive scalability, regulatory confidence, manufacturing flexibility, and the development of increasingly complex products.
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A New Purification Strategy For The Removal Of dsRNA Impurities From IVT mRNA Products
NIST research introduces a gentle SEC method that separates dsRNA impurities from IVT mRNA by tweaking buffer pH, achieving 81.8% target recovery without transcript damage.
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The Second Holy Grail Of Gene Editing: Large Knock-Ins
Exploring the race to achieve large gene knock-ins, emerging editing platforms, and how overcoming this challenge could unlock therapies for many inherited diseases.
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From Baby KJ To Global Access: The 3 Pillars Of Next-Gen mRNA Expansion
Obviously, the transition from RNA vaccines to therapeutics is an “expansion” all on its own; but to do this successfully, we need to expand our science, our products’ deliverability, and our patient access. In the following article, I highlight a few of the best practices and/or innovations that came up in conversation during this event that will play a significant role in helping us expand mRNA’s reach, both scientifically and therapeutically.
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The Preclinical Gauntlet: Securing Capital And Designing Regulatory Pathways For Ultra-Rare ASO Drugs (Pt. 2)
SynaptixBio CEO Dan Williams explains why funding, regulatory strategy, toxicology, and disciplined execution — not just science — determine success in advancing ultra-rare ASO therapies.
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Reprogramming The Tumor Microenvironment: Why RNA Immunotherapy Is Targeting Liver Metastases (Pt. 1)
RNA/DNA immunotherapy may overcome liver metastases by reprogramming innate immunity, restoring interferon signaling, and improving responses to checkpoint inhibitors.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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Targeted LNPs that bypass the liver and home to the spleen could reshape in vivo CAR T cell manufacturing. See the data behind the approach.
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Prioritize operational efficiency over early-stage flexibility to scale advanced therapies. Standardized workflows and qualified shipping lanes reduce variability for predictable late-phase success.
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In vivo CAR-T approaches aim to reprogram T cells directly in the body using targeted lipid nanoparticles. Improved delivery to immune organs highlights new potential for scalable cell therapies.
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Cell therapies, using reprogrammed immune cells, offer innovative treatments for diseases. Discover how lipid nanoparticles (LNPs) enhance these therapies by delivering RNA for gene modulation and next-gen treatments.
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Learn about one path forward to navigate the pitfalls with mRNA transfection and a medium to assist in lentivirus-mediated transduction of human mesenchymal stromal/stem cells (hMSCs).
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Learn how a robust understanding of the RNP’s critical process parameters and CQAs is crucial for ensuring product consistency, enhancing patient safety, and maintaining regulatory compliance.
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The existing regulatory system is ambiguous for RNA therapeutics. Leverage this Investigational New Drug (IND) guide to help accelerate and strengthen the process IND filing of novel nanomedicines.