FORMULATION ARTICLES
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Structure-Guided siRNA Design: How Chemistry, Context, And Constraint Are Shaping The Next Phase Of RNAi Therapeutics
As siRNA design matures, the next breakthroughs will depend on balancing chemical optimization, delivery challenges, and expanding beyond established design rules.
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Following The Data To The Heart: Atrium's Quest For Next-Gen Targeted RNA Delivery
In 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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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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From Exosomes To Nanodrugs: Advancing Therapeutic Delivery For CNS Repair
Exosomes 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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Why RNAi-Based Oncology Must Move Beyond Single-Target Thinking
RNAi therapeutics may help oncology move beyond single-target drugs by disrupting adaptive tumor survival networks driving resistance in solid tumors.
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What Makes An RNA Platform Investable? Lessons From The Last Wave Of Biotech Financing
RNA investing has shifted from hype to scalability, forcing companies to prove manufacturability, delivery strength, and platform-wide value.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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Explore ongoing work to harness different types of HDR templates in order to build cutting-edge systems with the potential to enable point-of-care cell and gene therapy manufacture.
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Compared to traditional protein-based therapies, mRNA drugs exhibit unique pharmacokinetic profiles and can be rapidly developed, making overcoming their challenges critical for the industry.
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We showcased the utility of microfluidics to enable low shear, rapid screening of preclinical candidates and the swift advancement to GMP-enabling studies.
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From fermentation to formulation, mRNA manufacturing requires a toolbox of technologies, services, and expertise. Explore our portfolio of solutions for making, purifying, and formulating mRNA.
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Polyethylene glycol and similar variants have driven innovative research for therapies. However, the continued use of PEG poses significant drawbacks.
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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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Review the fundamentals of CRISPR technology, development, and its use to manipulate genes in different ways, such as altering their nucleotide sequences or changing their expression.