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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Compared to traditional protein-based therapies, mRNA drugs exhibit unique pharmacokinetic profiles and can be rapidly developed, making overcoming their challenges critical for the industry.
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Technical summaries and detailed protocols for purity testing, sizing, sequencing and expression analysis of oligonucleotide-based active agents as well as protein and lipid-based delivery systems.
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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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Delve into the clinical landscape of hemophilia, a rare X-linked congenital bleeding disorder, across three key regions of the globe, namely Asia Pacific, Europe, and North America.
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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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In this paper, we present the foundation of an mRNA-LNP platform for encoding and expressing therapeutic antibodies in vivo, eliminating the need for costly and time-consuming manufacturing.
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Explore this proprietary ionizable lipid library which has shown promising potency towards various applications involving cell therapy, protein replacement therapy and RNA-based Vaccines.