FORMULATION ARTICLES
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![neuroscience, neuromuscular disorders, pain signals, rehabilitation-GettyImages-2280116713]( "Why Delivery Is Defining The Next Era Of Neuromuscular Therapeutics")
Why Delivery Is Defining The Next Era Of Neuromuscular TherapeuticsEmerging 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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![intelligent technology interface, robot chat-GettyImages-2279572840]( "Analytical Platforms: The Foundation Of The Next Generation Of RNA Therapeutics")
Analytical Platforms: The Foundation Of The Next Generation Of RNA TherapeuticsRNA 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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![nucleic acid, DNA, RNA, purified protein-GettyImages-1350828873]( "A New Purification Strategy For The Removal Of dsRNA Impurities From IVT mRNA Products")
A New Purification Strategy For The Removal Of dsRNA Impurities From IVT mRNA ProductsNIST 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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![GettyImages-1316503044-dna-strand-double-helix-gene-editing]( "The Second Holy Grail Of Gene Editing: Large Knock-Ins")
The Second Holy Grail Of Gene Editing: Large Knock-InsExploring 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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![GettyImages-1358847958]( "From Baby KJ To Global Access: The 3 Pillars Of Next-Gen mRNA Expansion")
From Baby KJ To Global Access: The 3 Pillars Of Next-Gen mRNA ExpansionObviously, 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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![Leukodystrophy Awareness Month-GettyImages-2166836167]( "The Preclinical Gauntlet: Securing Capital And Designing Regulatory Pathways For Ultra-Rare ASO Drugs (Pt. 2)")
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.
FORMULATION VIDEOS
The clinical promise of mRNA therapeutics and vaccines is clear, but compared to monoclonal antibodies or viral vectors, their novelty also brings new challenges to the manufacturing process.
CRISPR gene editing and lipid nanoparticle delivery are revolutionizing precision medicine. Discover how optimized LNP systems and advanced analytics accelerate therapeutic development.
New technologies for the encapsulation of nucleic acids, such as FDmiX, can potentially overcome limitations of currently available standard mixing technologies.
Discover how polysarcosine-functionalized lipid nanoparticles are transforming mRNA delivery. Join Dr. Heinrich Haas of BioNTech as he unveils innovative research and its implications for therapeutic breakthroughs.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![Crispr gene editing-GettyImages-1139760176]( "CRISPR 101 - Your Guide To Understanding CRISPR")
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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![GettyImages-1067806062-scientist-lab-vaccine-vial]( "Linear Scalability In Turbulent‐Jet Mixing For Nanoparticle Processing")
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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![Scientists Discussing GettyImages-916075464]( "Is Standardization The Key To Scalable CGT?")
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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![iStock-1213713667-viral-vector-virus]( "Gene Therapy And Oligonucleotide Compendium")
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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![GettyImages-1334686671-cold-chain-cryopreservation-logistics]( "Validate Cryopreservation As A Defined Input, Not A Variable")
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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![LNP Formulations Feature]( "Fast Track RNA-LNP Drug Development By Lowering The Lipid Barrier")
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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![Cytiva-602047-2. Translation of RNA]( "Translation Of RNA Medicines From Design To Clinic")
Understand the advantages and challenges of lipid-based delivery systems for successful translation of RNA into clinical development.
