FORMULATION ARTICLES
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Why Delivery Is Defining The Next Era Of Neuromuscular Therapeutics
Emerging therapies are being designed to safely deliver oligonucleotides to disease-relevant tissues. Delivery is no longer a technical hurdle to overcome but a strategic lever.
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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.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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Learn more about issued guidelines on Critical Quality Attributes for IVT mRNA vaccines and biotherapeutics to focus on drug substance and LNP-encapsulated drug product assessment using an analyzer system.
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By following some of the best practices in freezing mRNA, you can effectively balance product quality, cost-effectiveness, process safety, and efficiency.
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Fragmented pre‑clinical supply chains create avoidable delays and risks; unifying cryopreservation, logistics, and storage early improves consistency, reduces operational gaps, and supports smoother progression into clinical phases.
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Small, early operational decisions accumulate over time, creating hidden variability that emerges at scale, making standardization essential for stability, reproducibility, and regulatory confidence.
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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.
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We discuss the process of transfection and highlight two highly effective vectors for both in vitro and in vivo gene therapy applications.
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Lipid nanoparticles (LNPs) have played a crucial role in COVID-19 vaccine development as efficient mRNA delivery systems, exhibiting versatility and effectiveness in accelerating the process.