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
Walk step-by-step instructions through how you can set up the Nova Benchtop System and make your first LNPs.
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.
In this segment, Replicate Biosciences’ Andy Geall, Qian Ruan, & Alex Aust identify the most promising technological and raw material innovations that are influencing the drug substance and drug product production processes at CDMOs.
The quest for the “extra-hepatic GalNAc” is on. In this Advancing RNA Live clip, NanoVation Therapeutics’ Dominik Witzigmann and Stealth Co’s John Zuris walk through some of the most popular targeting ligand choices being explored in the RNA delivery space today, sharing which they believe will be the most game-changing.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![PNI Feature 2]( "Strategies To Overcome Delays In The Development Of Genomic Medicines")
Learn how optimizing LNPs can help overcome drug-delivery challenges, unlocking potential new genomic medicines in a shorter time.
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![GettyImages-970394878-genetic-engineering-gene-editing-dna]( "Genome Editing Using mRNA-Lipid Nanoparticles For CAR T Cell Therapy")
Gene delivery platforms must support a diversity of genetic engineering strategies for genomic medicine development. Learn how lipid nanoparticles can accelerate development from discovery to scale-up.
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![mRNA GettyImages-1356994684]( "mRNA Manufacturing And Formulation")
From fermentation to formulation, mRNA manufacturing requires a toolbox of technologies, services, and expertise. Explore our portfolio of solutions for making, purifying, and formulating mRNA.
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![GettyImages-1219169373 lab, research, assay, RNA, DNA]( "Purification And QC Of An RNA 21‑Mer Oligonucleotide Via LC‑MS")
Learn how buffer choice, gradient strategy, and pretreatment steps influence recovery, purity, and resolution when purifying a native RNA oligo, with analytical confirmation and practical guidance.
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![iStock-1293772874-lab-plasmid-gene therapy-DNA]( "Novel Lipid Nanoparticle Delivery Reagent And Manufacturing Workflow")
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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![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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![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.
