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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Manufacturing RNA-containing LNPs demands specialized expertise. Explore some of the challenges of RNA-LNP drug manufacturing and the advantages of outsourcing RNA-LNP formulations.
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Learn about one path forward to navigate the pitfalls with mRNA transfection and a medium to assist in lentivirus-mediated transduction of human mesenchymal stromal/stem cells (hMSCs).
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Explore process and analytical development capabilities that can help you advance your formulation to GMP Manufacturing.
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Early integration of standardized, GMP-aligned cryopreservation improves consistency, documentation, and scalability in advanced therapy programs as they progress toward regulatory submissions.
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Non-PEG stabilizer lipids may offer a new path for LNP design. Combining strong colloidal performance with greater chemical flexibility helps address growing concerns around PEG-related immunogenicity.
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Cryopreservation eliminates rigid timelines by decoupling collection from production, providing the flexibility needed to optimize facility use and stabilize advanced therapy supply chains.
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Lipid nanoparticles are redefining immune cell engineering. Delve into new data that demonstrates the performance across multiple cell types and bioreactor systems, supporting their potential workflows.