FORMULATION ARTICLES
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Why Delivery Is Defining The Next Era Of Neuromuscular Therapeutics
Emerging therapies are being designed to safely deliver oligonucleotides to disease-relevant tissues. Delivery is no longer a technical hurdle to overcome but a strategic lever.
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Analytical Platforms: The Foundation Of The Next Generation Of RNA Therapeutics
RNA therapeutics are entering a new era where analytical platforms drive scalability, regulatory confidence, manufacturing flexibility, and the development of increasingly complex products.
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A New Purification Strategy For The Removal Of dsRNA Impurities From IVT mRNA Products
NIST research introduces a gentle SEC method that separates dsRNA impurities from IVT mRNA by tweaking buffer pH, achieving 81.8% target recovery without transcript damage.
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The Second Holy Grail Of Gene Editing: Large Knock-Ins
Exploring the race to achieve large gene knock-ins, emerging editing platforms, and how overcoming this challenge could unlock therapies for many inherited diseases.
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From Baby KJ To Global Access: The 3 Pillars Of Next-Gen mRNA Expansion
Obviously, the transition from RNA vaccines to therapeutics is an “expansion” all on its own; but to do this successfully, we need to expand our science, our products’ deliverability, and our patient access. In the following article, I highlight a few of the best practices and/or innovations that came up in conversation during this event that will play a significant role in helping us expand mRNA’s reach, both scientifically and therapeutically.
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The Preclinical Gauntlet: Securing Capital And Designing Regulatory Pathways For Ultra-Rare ASO Drugs (Pt. 2)
SynaptixBio CEO Dan Williams explains why funding, regulatory strategy, toxicology, and disciplined execution — not just science — determine success in advancing ultra-rare ASO therapies.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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Building regulatory readiness early in cell and gene therapy development protects product integrity, prevents costly delays, and secures long-term program viability.
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Experts note CGT programs often struggle due to limited operational readiness; early standardization supports scalable, reproducible, regulatory‑ready development and helps prevent late‑stage bottlenecks.
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Realize optimal coupling efficiency and increase your productivity for oligonucleotide drugs by selecting the right synthesizer and prioritizing automation to define and tweak synthesis methods.
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Examine how a 21‑mer oligo was synthesized and purified through systematic resin screening, method optimization, and successful scale‑up to build reliable, high‑purity chromatography workflows.
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RNA therapeutics hold immense promise for precision treatment, but their development faces challenges. A CDMO with adaptable platforms and flexible timelines can help reduce time and costs.
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A comparative study examines how two mixing technologies, microfluidic and turbulent jet, influence the attributes of lipid nanoparticles and their suitability for different stages of drug development.
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Aseptic fill and finish operations gain efficiency and precision with modular isolator technology, inline weight checks, and lyophilization, which are ideal for mRNA-LNPs and other sensitive modalities.