FORMULATION ARTICLES
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Beyond GalNAc: Reengineering siRNA For Extrahepatic Delivery
We discuss why effective extrahepatic delivery remains one of the field's biggest obstacles and how chemical engineering may provide a new path forward.
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Engineering CAR T Cells With mRNA-LNPs: Applying Oncology Learnings To Allergic Diseases
Targeted lipid nanoparticles (LNPs) could deliver mRNA encoding a CAR directly to T cells inside a patient with allergic disease. The potential implications extend beyond a single allergic condition.
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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.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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Messenger RNA-LNPs are a promising strategy for delivering gene-editing components. Learn how a continuous manufacturing platform can address the scalability and reproducibility challenges of this process.
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Repeat dosing may be essential for long-term mRNA applications. Explore safety, immune-response, and efficacy findings from multiple LNP administrations, including a nine-dose evaluation over six months.
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Polyethylene glycol and similar variants have driven innovative research for therapies. However, the continued use of PEG poses significant drawbacks.
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Advancements in purification, delivery mechanisms, and stability during storage and transport will enable the future of mRNA-based therapies.
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Batch processing platforms for LNPs lack efficiency, appropriate controls, and yield optimization. Continuous manufacturing platforms may address some of those issues.
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Lipid nanoparticles enable efficient gene editing in hard-to-transfect cells like T-cells and blood stem cells, which offers scalable, clinically relevant methods for developing advanced therapies for cancer and rare diseases.
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Explore key considerations for manufacturing RNA-LNP drug products surrounding process and analytical development, automation, environmental monitoring, and strategic collaborations.