FORMULATION ARTICLES
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![mAb, monoclonal antibody, antigen, vaccine-GettyImages-2245122166]( "Suppressor tRNA Therapeutics: A New Frontier In Treating Genetic Disease")
Suppressor tRNA Therapeutics: A New Frontier In Treating Genetic DiseaseSuppressor tRNAs could unlock a scalable new way to treat genetic diseases caused by nonsense mutations, restoring full-length proteins across multiple indications.
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![Virus, microscopy, vaccine, covid-GettyImages-1439691672]( "Engineering Transient In Vivo CAR-T: Extending Expression, Expanding Access, And Rethinking Immune Reset")
Engineering Transient In Vivo CAR-T: Extending Expression, Expanding Access, And Rethinking Immune ResetIn this Q&A, Michael Lam, Ph.D., explores how circular RNA and targeted delivery extend transient CAR expression to enable deep B-cell depletion and controllable immune reset.
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![body immune response viral infection, Cancer Immunotherapy-GettyImages-2172360428]( "Engineering Repeat-Dose RNA: Engineering Around Immune Recognition In Repeat-Dose RNA Therapeutics")
Engineering Repeat-Dose RNA: Engineering Around Immune Recognition In Repeat-Dose RNA TherapeuticsDesigning repeat-dose RNA means engineering around immune recognition — optimizing payloads, purity, and delivery early to sustain efficacy, tolerability, and durability across multiple administrations.
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![DNA Helix, Futuristic Digital Interface, genetics, molecular biology, gene editing-GettyImages-2221747428]( "A Novel Co-Tethered Transcription Platform For High-Yield, High-Purity mRNA Synthesis")
A Novel Co-Tethered Transcription Platform For High-Yield, High-Purity mRNA SynthesisCo-tethered transcription boosts mRNA yield and purity by organizing transcription machinery, reducing dsRNA impurities, and streamlining production for scalable RNA therapeutics.
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![Drone Point View of Overpass and City Traffic at Night-GettyImages-1279832748]( "A New Approach To RNA Synthesis And Purification: Rethinking A Persistent Bottleneck")
A New Approach To RNA Synthesis And Purification: Rethinking A Persistent BottleneckPhotocleavable supports enable light-triggered RNA release, reducing reliance on chromatography and streamlining synthesis, scalability, and purity for complex RNA therapeutics.
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![Genetic Engineering, biotechnology gene analysis-GettyImages-1485674887]( "Oligos, mRNA, Or Gene Editing: Where Should You Bet?")
Oligos, mRNA, Or Gene Editing: Where Should You Bet?Investing in RNA and gene editing requires balancing risk, timing, and scale — understanding how oligos, mRNA, and CRISPR each drive value across a converging biotech landscape.
FORMULATION VIDEOS
There are many different criteria for selecting a CDMO for your mRNA products. Arcturus’ Qian Ruan explains that a product’s stage of development and the specific needs inherent to that phase of development should be an important guide for companies today, especially given the expansion of the outsourcing sector and the wide range of capabilities.
Explore how lipid nanoparticles are driving breakthroughs in cell therapy, from mRNA-based immune cell engineering to genome editing for CAR T cells and HSPCs from leading experts.
Achieving cost-effective manufacturing while maintaining high quality standards today is one of the biggest challenges facing companies outsourcing mRNA/RNA development. In turn, panelists Qian Ruan, Andy Geall, and Alex Aust reflect on how and where they’ve seen the costs of mRNA-LNP manufacturing evolving/fluctuating in recent years.
Discover how continuous manufacturing of nanoparticles can improve process efficiency, reduce costs, and increase the quality of the final drug product.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![GettyImages-1317697233-cancer-t-cells]( "A Novel RNA Lipid Nanoparticle Platform: Gene-Edited CAR T Cells For Off-The-Shelf Cancer Therapy")
We report on the use of a novel lipid nanoparticle (LNP) reagent in a validated protocol to achieve successful complex gene editing in primary T cells with high efficiency while maintaining high cell viability.
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![GettyImages-1437856375-lnp-lipid-nanoparticle]( "Regulatory Considerations For Excipients Used In Lipid Nanoparticles")
Review the regulatory classification of LNPs, liposomes, and novel excipients as well as specific requirements outlined in regulatory guidance documents and strategies to navigate emerging challenges.
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![GettyImages-1307980860-dna-rna-vial-gene]( "Optimizing Lipid Formulations For Targeted RNA-LNP Applications")
Learn about the considerations and challenges of using ionizable lipids, including the 5 types of lipids used for RNA delivery.
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![mRNA GettyImages-1428646288]( "Establishing Analytical Methods For mRNA-Based Therapies")
Here, we provide a detailed description of assays for sequence identification and LNP composition in mRNA-LNP products that support the development of safe and effective mRNA therapies.
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![Pharmaceutical-Drug-Manufacturing-GettyImages-1405788565]( "Nanoparticle Engineering Addresses The Challenge Of Poor Aqueous Solubility")
As pharma companies aim to overcome development obstacles, it is important to incorporate GMP expertise alongside novel technologies that can enhance the properties of APIs and dosage forms.
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![Medical scientists analyzing research-GettyImages-1413600685]( "Developing A Comprehensive Regulatory Approach For LNP Drugs")
The path to regulatory approval for LNPs demands carefully considered strategies. By adopting proactive and strategic approaches, developers can overcome regulatory hurdles on the path to approval.
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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.
