FORMULATION ARTICLES
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![Human Internal Organs Anatomy-GettyImages-1391174702]( "Beyond GalNAc: Reengineering siRNA For Extrahepatic Delivery")
Beyond GalNAc: Reengineering siRNA For Extrahepatic DeliveryWe 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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![Asthma inhaler-GettyImages-2240010472]( "Engineering CAR T Cells With mRNA-LNPs: Applying Oncology Learnings To Allergic Diseases")
Engineering CAR T Cells With mRNA-LNPs: Applying Oncology Learnings To Allergic DiseasesTargeted 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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![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.
FORMULATION VIDEOS
Here, Advancing RNA Live speakers Khaled Yamout, Sanofi’s Francoise Ribes, and Camp4’s Satya Kuchimanchi share which supply chain advancements they would like to see in the near future, as well as how they anticipate these changes & evolutions could impact each modality’s growth.
New technologies for the encapsulation of nucleic acids, such as FDmiX, can potentially overcome limitations of currently available standard mixing technologies.
Dr. Gerrit Borchard, University of Geneva, presents a personal account of DNA vaccine development in a global pandemic.
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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![GettyImages-1648451263 Scientist Holding Tray Of Medical Samples In Lab]( "Continuous Nanoparticle Manufacturing For Gene Editing Delivery")
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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![research-drugdevelopment-GettyImages-2254140660]( "mRNA LNPs Drive Safe, Effective Repeat Dosing In Disease Models")
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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![iStock-1213444268-vaccine-virus-syringe]( "PEG In LNP Formulations: Addressing Challenges And Seeking Solutions")
Polyethylene glycol and similar variants have driven innovative research for therapies. However, the continued use of PEG poses significant drawbacks.
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![GettyImages-1438509986-isolated-rna-lipid-nanoparticle-LNP]( "Reducing Risk In mRNA Therapeutic Development")
Advancements in purification, delivery mechanisms, and stability during storage and transport will enable the future of mRNA-based therapies.
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![GettyImages-1418152314]( "Unpacking The Importance Of Lipid Nanoparticle Production Platforms")
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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![GettyImages-1356994439-stem-cell-DNA-gene-editing-tweezers]( "Versatile Lipid Nanoparticle Platform For Efficient Gene Editing")
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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![GettyImages-1087219020 Scientists wearing protective coveralls working in manufacturing laboratory]( "Insights Into GMP Manufacturing Of RNA-LNP Drug Products")
Explore key considerations for manufacturing RNA-LNP drug products surrounding process and analytical development, automation, environmental monitoring, and strategic collaborations.
