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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Review the fundamentals of CRISPR technology, development, and its use to manipulate genes in different ways, such as altering their nucleotide sequences or changing their expression.
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Understand how turbulent-jet mixing ensures predictable and reproducible nanoparticle size and uniformity across all scales, accelerating development and regulatory readiness.
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Experts note CGT programs often struggle due to limited operational readiness; early standardization supports scalable, reproducible, regulatory‑ready development and helps prevent late‑stage bottlenecks.
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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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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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Fast-track RNA-LNP development, streamline screening with off-the-shelf ionizable lipid mixes, and utilize validation strategies to generate reproducible and scalable LNPs for lead candidates.
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Understand the advantages and challenges of lipid-based delivery systems for successful translation of RNA into clinical development.