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
Russell Johnson, VP Formulation Research for RVAC Medicines, and Aalok Shah, Director of Formulation & Drug Delivery, Strand Therapeutics, share their expectations on which scientific advancements/characteristics will be most important for LNP-delivery of mRNA/RNA cargo in the future.
Camp4’s Satya Kuchimanchi highlights where he has seen the most growth in the oligo supply chain over the past few years, as well as which industry trends have been driving these shifts.
Together, Life Edit Therapeutics’ April Sena and Sanofi’s Troiano share their perspectives on raw material quality & IP-related considerations for managing the supply chain for next-generation RNA modalities.
Sanofi’s Sumit Luthra shares several of the analytical and manufacturing innovations/improvements he’s watching and waiting for that will improve our knowledge of LNP composition, structure, function, and quality.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![GettyImages-1194781834-cell]( "mRNA-Lipid Nanoparticles Circumvent Viral Vector Limitations")
Both physical and chemical nonviral gene delivery systems for ex vivo genetic modification offer advantages over viral vectors, including smaller scale production and the low risk of immunogenicity.
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![GettyImages-1409578453-mRNA-LNP-lipid-nanoparticle-RNA therapy]( "Formulation Process And Analytical Development Capabilities")
Explore process and analytical development capabilities that can help you advance your formulation to GMP Manufacturing.
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![Cytiva-602047-2. Translation of RNA]( "Translation Of RNA Medicines From Design To Clinic")
Understand the advantages and challenges of lipid-based delivery systems for successful translation of RNA into clinical development.
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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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![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-500323894 research tech]( "High-Throughput Lipid Separation For LNP Formulations")
Lipid nanoparticles (LNPs) carry immense potential as delivery systems. Learn about the development of isocratic and gradient methods for high-throughput separation of lipids used in LNP formulations.
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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.
