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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Polyethylene glycol and similar variants have driven innovative research for therapies. However, the continued use of PEG poses significant drawbacks.
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Explore the details supporting LNP-mediated mRNA delivery, as well as the challenges facing mRNA based therapeutics in the future.
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Building regulatory readiness early in cell and gene therapy development protects product integrity, prevents costly delays, and secures long-term program viability.
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Learn about the key stages of drug discovery and screening as well as how to overcome bottlenecks in screening mRNA-LNPs to rapidly develop genomic medicines.
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Precision In Nanoparticle Processing And Monodispersed Lipid Nanoparticles For Advanced Therapeutics
Precision in nanoparticle processing is key for drug delivery and vaccines. Learn how monodispersed formulations enhance reproducibility and stability for next-generation therapeutics, like RNA-LNPs.
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One main mRNA technology challenge is the formulation of delivery systems like lipid nanoparticles (LNPs). Learn about evading impurities in lipids, the future of mRNA-based genomic medicines, and more.
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Discover how mRNA technologies open the doors to a whole range of new gene therapies and the role of lipid nanoparticles (LNPs) in current and future developments.