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 how three companies collaborated to develop a new antibiotic to address the increasing development of antimicrobial resistance (AMR) that threatens the global health landscape.
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Explore the potential of polymeric nanoparticles (PNPs) as an alternative to viral vectors. PNPs offer design flexibility, enabling larger payloads, targeted release, and minimized side effects.
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Repeat dosing may be essential for long-term mRNA applications. Explore safety, immune-response, and efficacy findings from multiple LNP administrations, including a nine-dose evaluation over six months.
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Here, we highlight three important process considerations across the RNA-LNP manufacturing workflow which includes limit size behavior, in-line dilution and downstream tangential flow filtration (TFF).
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Learn how turbulent jet mixing enables linear scalability, guaranteeing consistent nanoparticle quality from research to commercial production and accelerating product timelines.
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We aim to highlight that commercially available LNP reagent mix, GenVoy-ILM, is an accessible and easy-to-use LNP formulation that allows for rapid preclinical development of RNA vaccines.
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Explore how lipid nanoparticles are revolutionizing cell therapy workflows to enable the precise delivery of mRNA and gene editing tools for next-generation treatments like CAR T and HSC therapies.