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 about an innovative closed, single-use system and proprietary mixing technology aimed at improving reproducibility and reducing contamination risks in biopharma manufacturing.
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Producing high-quality oligonucleotides demands precise execution across multiple stages. Gain a clearer understanding of the complete mid-scale production workflow and necessary quality control measures.
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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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Gene delivery platforms must support a diversity of genetic engineering strategies for genomic medicine development. Learn how lipid nanoparticles can accelerate development from discovery to scale-up.
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Cell therapy’s future depends on smarter gene delivery. Learn how lipid nanoparticles offer a scalable, cell-friendly alternative to electroporation and viral vectors for manufacturing efficiency.
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Understand how Microfluidic Modulation Spectroscopy (MMS) provides early insights into RNA degradation within LNP formulations, offering a label-free approach to monitoring chemical integrity.
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There are various factors that require careful consideration throughout the process of formulating LNPs and utilizing them for successful mRNA delivery and translation.