FORMULATION ARTICLES
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![molecular scissors, manipulation of molecular biology, RNA molecule-GettyImages-2213997299]( "Structure-Guided siRNA Design: How Chemistry, Context, And Constraint Are Shaping The Next Phase Of RNAi Therapeutics")
Structure-Guided siRNA Design: How Chemistry, Context, And Constraint Are Shaping The Next Phase Of RNAi TherapeuticsAs siRNA design matures, the next breakthroughs will depend on balancing chemical optimization, delivery challenges, and expanding beyond established design rules.
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![GettyImages-1487902757]( "Following The Data To The Heart: Atrium's Quest For Next-Gen Targeted RNA Delivery")
Following The Data To The Heart: Atrium's Quest For Next-Gen Targeted RNA DeliveryIn this two-part series, Gallagher shares more about her goals for Atrium following its spin-out from Novartis, articulating what it looks like and means to “pave a path” to greater patient access in rare cardiomyopathies. Here in part one, we start with a look into the formation of Atrium, sharing the “why” behind the company’s formation and what’s next for the company’s targeted delivery platform.
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![RNA, siRNA, mRNA, CRISPR-GettyImages-1409578455]( "Scaling RNA Therapeutics: Purification, LNP Formulation, And GMP Manufacturing (Pt. 2)")
Scaling RNA Therapeutics: Purification, LNP Formulation, And GMP Manufacturing (Pt. 2)Part 2 explores the downstream technologies that turn RNA into a therapeutic product, from impurity removal and LNP formulation to GMP infrastructure and scalable manufacturing.
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![NeurExone_coverphoto]( "From Exosomes To Nanodrugs: Advancing Therapeutic Delivery For CNS Repair")
From Exosomes To Nanodrugs: Advancing Therapeutic Delivery For CNS RepairExosomes are biological vesicles used for cellular communication that can overcome CNS barriers, delivering therapeutic cargo for CNS and optic nerve injuries.
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![GettyImages-1447956737]( "Why RNAi-Based Oncology Must Move Beyond Single-Target Thinking")
Why RNAi-Based Oncology Must Move Beyond Single-Target ThinkingRNAi therapeutics may help oncology move beyond single-target drugs by disrupting adaptive tumor survival networks driving resistance in solid tumors.
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![Green Business-GettyImages-2179499504]( "What Makes An RNA Platform Investable? Lessons From The Last Wave Of Biotech Financing")
What Makes An RNA Platform Investable? Lessons From The Last Wave Of Biotech FinancingRNA investing has shifted from hype to scalability, forcing companies to prove manufacturability, delivery strength, and platform-wide value.
FORMULATION VIDEOS
Explore how lipid nanoparticles are revolutionizing non-viral gene editing with scalable, efficient delivery for both ex vivo and in vivo applications, which features new CRISPR-LNP data in T cells.
Learn how streamlined vector backbones can improve efficiency, consistency, and expression durability when reliability matters most.
How can PCR-based technology accelerate mRNA development and manufacturing and design principles/critical parameters to enable the rapid development of lipids for RNA formulation?
Dr. Gerrit Borchard, University of Geneva, presents a personal account of DNA vaccine development in a global pandemic.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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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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![LNP Formulations Feature]( "Fast Track RNA-LNP Drug Development By Lowering The Lipid Barrier")
Fast-track RNA-LNP development, streamline screening with off-the-shelf ionizable lipid mixes, and utilize validation strategies to generate reproducible and scalable LNPs for lead candidates.
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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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![Science techonlogy-research-development-discovery-GettyImages-1209662272]( "Turbulent Mixing: Scaling Up Nanoparticle Manufacturing For The Future")
Choosing between turbulent and microfluidic mixing methods in your lipid nanoparticle (LNP) production can have a significant impact when scaling up production.
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![GettyImages-2032712798-lab-vaccine-microscope-research-development]( "Transforming Nanoparticle Processing Through Innovation")
Learn about an innovative closed, single-use system and proprietary mixing technology aimed at improving reproducibility and reducing contamination risks in biopharma manufacturing.
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![GettyImages-1409578453-mRNA-LNP-lipid-nanoparticle-RNA therapy]( "An Introduction And Brief History Of mRNA-LNPs")
With adjustable components and increased stability during bloodstream circulation, LNPs are significantly advancing drug development and delivery, particularly for mRNA vaccines.
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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.
