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
Review strategies to assess the potential for human immune response following the administration of an mRNA therapeutic, the associated LNP, and the protein encoded for by the mRNA.
Nutcracker Therapeutics’ Oreanna Thomas shares her takeaways from a pre-IND meeting for one of the company’s mRNA therapeutic candidates.
NxGen™ microfluidic technology enables controlled and precise assembly of high-quality, uniform LNPs, ensuring reproducible scale-up of RNA-LNPs while maximizing drug loading and particle quality.
Overcome LNP upscaling challenges by leveraging fluid mechanical principles. Learn how Impingement Jets Mixing achieves reproducible turbulence and consistent LNP quality across all production scales.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![GettyImages-1405590436-lab-perfusion-cleanroom]( "Laminar And Turbulent Mixing Techniques For LNP Formulations Scale Up")
Explore the critical factors in scaling up lipid nanoparticle (LNP) formulation, comparing the advantages and limitations of both laminar (microfluidic) and turbulent (tee and jet) mixing technologies.
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![mRNA GettyImages-1356994723]( "The Future Of mRNA: Four Key Focuses For Industry")
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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![blood-cell_173710948-thinkstock_450x300]( "Hemophilia - Global Clinical Trial Landscape")
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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![GettyImages-2193144544-scientist-cell-culture-in-lab]( "Lipid Nanoparticles For Pulmonary Delivery Of mRNA")
Gain insight into how nebulized mRNA lipid nanoparticles show preclinical success in lung delivery, offering fresh momentum for inhaled gene therapies targeting respiratory diseases.
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![RNA abbreviation of ribonucleic acid-GettyImages-1453953422]( "Exploring Osmolality As A CQA Within RNA Therapeutics")
Discover how osmolality can be applied throughout the bioprocessing workflow for RNA, and recognize the importance of this sensitive measurement, from formulation optimization to cryopreservation techniques.
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![GettyImages-2160715091 lab analysis]( "Targeted LC-MS/MS Detection Of Lipid Impurities In Lipid Nanoparticles")
Learn about an HPLC-MS/MS method for detecting lipid impurities in lipid nanoparticles (LNPs) and why it is critical for quality control of LNP-based drug delivery systems.
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![Genomic sequencing, molecular biology, and genetic data analysis-GettyImages-2153790378]( "The Paradigm Shift: From Scale-Up To Scale-Out In Genetic Medicine")
The shift from "scale-up" to "scale-out" in genetic medicine enhances production speed, revolutionizing pharmaceutical manufacturing and accelerating life-saving therapies for global patient populations.
