FORMULATION ARTICLES
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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.
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![Cancer Detection-GettyImages-1477214805]( "Reprogramming The Tumor Microenvironment: Why RNA Immunotherapy Is Targeting Liver Metastases (Pt. 1)")
Reprogramming The Tumor Microenvironment: Why RNA Immunotherapy Is Targeting Liver Metastases (Pt. 1)RNA/DNA immunotherapy may overcome liver metastases by reprogramming innate immunity, restoring interferon signaling, and improving responses to checkpoint inhibitors.
FORMULATION VIDEOS
Unlock next-generation nanoparticle and LNP manufacturing. Achieve high throughput, minimal waste, and superior quality with a compact, automated, and contamination-controlled system.
It goes without saying that achieving cell-specific delivery is the most often discussed challenge facing the RNA-LNP field near- and long-term. However, as these three LNP experts share in this Advancing RNA Live clip, they have a whole list of additional R&D and CMC priorities we must address/accomplish to improve RNA-LNPs’ efficacy in personalized and gene therapy approaches.
Explore a structured approach to optimizing and scaling plasmid DNA production using E. coli, with insights into key growth parameters, benchtop feasibility, and pilot-scale performance.
This webinar is presented by Dr. John P. Cooke, Chair of the Department of Cardiovascular Sciences at the Houston Methodist Research Institute, Director of the Center for Cardiovascular Regeneration, and Medical Director of the RNA Therapeutics Program in the Houston Methodist DeBakey Heart and Vascular Center.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![Cleanroom Scientists At Computer GettyImages-1294339594]( "Drug Discovery And Screening, A Digital Era For RNA-LNP Therapeutics")
Learn about the key stages of drug discovery and screening as well as how to overcome bottlenecks in screening mRNA-LNPs to rapidly develop genomic medicines.
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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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![GettyImages-1182436133 scientist, lab, research, oncology]( "Precision In Nanoparticle Processing And Monodispersed Lipid Nanoparticles For Advanced Therapeutics")
Precision In Nanoparticle Processing And Monodispersed Lipid Nanoparticles For Advanced TherapeuticsPrecision in nanoparticle processing is key for drug delivery and vaccines. Learn how monodispersed formulations enhance reproducibility and stability for next-generation therapeutics, like RNA-LNPs.
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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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![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-1275317360-lab-cell-gene-research-scientist-development-discovery]( "Scale-Up And Manufacturing Of Self-Amplifying RNA-LNPs")
This poster shows the work to scale up COVID-19 self-amplifying RNA-Lipid nanoparticle using PNI developed ionizable lipid and formulation from 0.2, to 50 mg scale using PNI’s Manufacturing Platform.
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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.
