FORMULATION ARTICLES
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![Emergency medicine-GettyImages-2055655561]( "Targeting The Uncharted Heart: Why The Next Era Of siRNA Belongs In Cardiology")
Targeting The Uncharted Heart: Why The Next Era Of siRNA Belongs In CardiologyDuring our conversation, Gallagher walked me through what’s keeping her up at night as she leads the company and its first siRNA candidate toward an IND, elaborating on the role RNA can play in cardiomyopathies and identifying the unique challenges of developing RNA medicines today.
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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.
FORMULATION VIDEOS
In this Advancing RNA Live clip, Sanofi’s Sumit Luthra highlights the learnings we’ve made to-date about how our processes — both in terms of unit operations and process conditions — inform our LNP composition/heterogeneity.
Streamline CAR-T cell manufacturing with lipid nanoparticles delivering base editors to create fratricide-resistant, CD45-edited CAR45 T cells, which enables scalable, precise, and potent therapies for blood cancers.
In this Advancing RNA Live clip, NanoVation’s Dominik Witzigmann and Stealth Co’s John Zuris break down the scientific advancements we’re seeing for each of an LNP’s components (i.e., the ionizable lipid, helper lipid, and PEG), albeit with the caveat that, with these advancements, Freedom to Operate (FTO) continues to grow increasingly complicated.
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-970394878-genetic-engineering-gene-editing-dna]( "Genome Editing Using mRNA-Lipid Nanoparticles For CAR T Cell Therapy")
Gene delivery platforms must support a diversity of genetic engineering strategies for genomic medicine development. Learn how lipid nanoparticles can accelerate development from discovery to scale-up.
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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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![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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![Lipid Nanoparticle mRNA Vaccine GettyImages-1418152448]( "Fit-For-Purpose Lipids Can Accelerate, Scale, And Improve RNA-LNP Drugs")
Emerging biotech companies are investing in novel delivery capabilities to address different modalities' needs and extra-hepatic delivery. Explore steps to accelerate commercial nanomedicine delivery.
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![scientist laboratory microscope-GettyImages-1253518521]( "Cell And Virus Banking In OV Development And Manufacturing")
Delve into the exciting potential of OVs, including the critical role of cell and virus seed banks, and discover key considerations for establishing cell and virus banks for OV production.
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![Worker Holding Vaccine Vial GettyImages-1403976499]( "Potent Immune Response With Vernal's LNP-mRNAs")
As mRNA medicine continues to evolve, understand how trusted manufacturing partners will continue to play a pivotal role in translating innovative concepts into clinical reality.
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![KNAUER - Azura]( "The Hitchhiker´s Guide To Creating Oligonucleotides From Start To Finish")
Producing high-quality oligonucleotides demands precise execution across multiple stages. Gain a clearer understanding of the complete mid-scale production workflow and necessary quality control measures.
