FORMULATION ARTICLES
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The Delivery Challenge For In Vivo Cell Engineering
As RNA therapies expand beyond the liver, delivery—not payload design—has become the defining challenge for precise, scalable in vivo cell engineering.
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Building An ASO For TUBB4A-Related Leukodystrophy (Pt. 1)
SynaptixBio CEO Dan Williams explains how the company selected an ASO for TUBB4A leukodystrophy by balancing potency, safety, and translational rigor to advance a rare disease therapy.
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Location Matters: How The Nuclear Geography Of circRNAs Could Shape The Future Of RNA Therapeutics
CircRNA location may be as important as its sequence. Discover how nuclear organization could influence the next generation of RNA therapeutics.
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Building The RNA Knowledge Graph: How Better Annotation Is Accelerating Therapeutic Discovery
As RNA therapeutics grow more complex, better biological annotation is becoming the foundation for AI-driven discovery, target selection, and smarter drug development.
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Building A Self-Driving Lab For LNP Development
With commercially available components, MIT researchers built a platform that accelerates LNP process development, optimization, and autonomous manufacturing research.
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Beyond Vaccines: Can RNA Sense Gases?
Could RNA sense its environment? A new hypothesis explores how RNA may detect temperature and gases, inspiring smarter, adaptive RNA therapeutics.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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Understand how turbulent-jet mixing ensures predictable and reproducible nanoparticle size and uniformity across all scales, accelerating development and regulatory readiness.
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Risk analysis and lane qualification show supply chains perform consistently, meeting regulatory expectations by demonstrating control, reducing variability, and ensuring reliable delivery.
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Understand the advantages and challenges of lipid-based delivery systems for successful translation of RNA into clinical development.
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Gene therapies rely on efficient, precise methods of delivering genetic material. Examine how combining polymeric nanoparticles with CRISPR therapies is increasing the potential for improved treatment.
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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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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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Learn how optimizing LNPs can help overcome drug-delivery challenges, unlocking potential new genomic medicines in a shorter time.