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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Treating cryopreservation as a validated, controlled input improves consistency, quality, and patient outcomes by reducing variability across cell therapy manufacturing, storage, and distribution.
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Learn about a continuous manufacturing platform designed to help overcome challenges in domestic pharmaceutical production, including scale-up barriers and production variability.
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As RNA therapeutics become more precise, translational success increasingly depends on humanized preclinical models that accurately replicate human RNA biology.
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The production of lipid nanoparticles (LNPs) is a process that demands robustness, scalability, and reproducibility. Examine the impact of key operational parameters on the efficacy of LNPs.
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Manual LNP formulation introduces process variability that threatens particle quality and yield. As programs scale toward clinical production, maintaining strict control over CQAs becomes essential.
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Explore the benefits of building a manufacturing process early, how research into LNPs in nucleic acid therapeutics will contribute to the development of future drugs, and more.
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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.