FORMULATION ARTICLES
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Beyond Better Models: Why The Future Of AI-Designed mRNA Depends On More Relevant Biological Data
AI can optimize mRNA only if it's trained on biologically relevant data. Better therapeutic datasets — not just better algorithms — will drive the next generation of RNA medicines.
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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.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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Messenger RNA-LNPs are a promising strategy for delivering gene-editing components. Learn how a continuous manufacturing platform can address the scalability and reproducibility challenges of this process.
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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.
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Early integration of standardized, GMP-aligned cryopreservation improves consistency, documentation, and scalability in advanced therapy programs as they progress toward regulatory submissions.
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Discover how mRNA technologies open the doors to a whole range of new gene therapies and the role of lipid nanoparticles (LNPs) in current and future developments.
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Despite the momentum of the RNA-LNP therapeutic market, significant manufacturing challenges remain. However, the right mixing could solve three of these key challenges regarding instrumentation.
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The shift from "scale-up" to "scale-out" in genetic medicine enhances production speed, revolutionizing pharmaceutical manufacturing and accelerating life-saving therapies for global patient populations.
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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.