Order, Chaos, And The mRNA Black Box: Takeaways From The USP/AMM Forum

To be clear, this year’s USP/AMM quality forum was just as focused on the intricacies of applying/using the methods. But what was equally if not more present in this year’s discussion was our increasing acknowledgement that our understanding of mRNA and its biological mechanisms remains quite messy.

From Supporting Component to Strategic Driver: The Evolving Role of gRNA in CRISPR Therapeutics

Guide RNA is evolving from a simple targeting reagent into a complex, engineered molecule shaped by expanding CRISPR modalities, increasing length and modification demands, and tighter integration with manufacturability, analytical control, and regulatory expectations.

The RNA Ties That Bind: 3 Takeaways From The OPT Congress

Here, I unpack three takeaways I had from the OPT Congress, each of which I’d argue are indicative of the interconnectedness across the genetic medicines field.

Rethinking ATMP Assurance When Sterility Timelines, Reality Misalign

The compendial 14-day sterility testing window exposes product and patient to numerous risks, but settling on a rapid alternative also presents its own set of challenges.

MicroRNA Therapeutics In Metabolic Liver Disease: Context-Driven Drug Discovery

Realizing the therapeutic potential of miRNAs in liver disease requires confronting a problem the field has not fully reckoned with.

Inside Alnylam's Playbook For High Volume siRNA Production

As one of the first companies to establish a chemoenzymatic ligation platform, Alnylam's Maines and Nechev are in the perfect position to espouse wisdom on the practical aspects and remaining challenges of implementing enzymatic ligation at a large scale. Here in this installment, Maines and Nechev outline what they’ve learned thus far about the science of enzymatic ligation, while also paying credence to the unknowns and existing barriers that, as a frontrunner, Alnylam will inevitably (but willingly) be tasked with facing in the future.

Demystifying CRISPR gRNA Chemical Modifications

Chemical modifications on CRISPR gRNAs enhance stability, editing efficiency, and reduce immune responses, enabling in vivo applications and clinical success.

Boost LNP Drug Potency With Optimized Delivery Formulations

Lipid nanoparticles (LNPs) function as a drug delivery system for nucleic acids. Learn about the biomedical applications of LNPs as well as the anatomy, formulation optimization, and safety of LNPs.

IR Spectral Signatures Of GC, AU, And GU Base Pairing In H2O-Based Buffer By MMS

Explore a label-free approach to characterize RNA structure with high sensitivity. See how specific base pair types are resolved, providing a clearer picture of RNA folding.

A Risk-Based Approach To Plasmid DNA And mRNA Process Development

Balancing robust analytics and clinical readiness is key for early-phase pDNA and mRNA therapeutics amid structural complexity and regulatory challenges.

Learn How Compliance-Ready LC-MS Workflows for Oligonucleotides Can Expand your Analytical Services Portfolio

CMO invests in solution to ensure data integrity and meet regulatory requirements as they expand their oligonucleotide manufacturing and analysis services.

Why Bioanalysis Needs To Break Free From Manual Bottlenecks

Bioanalysis remains one of the most persistent bottlenecks in drug development, with CROs displaying inefficiencies that are no longer operational nuisances but strategic liabilities.

Study Viral Infection In Resting Human CD4+ T Cells

Explore a study proving how synthetic single guide RNAs were used to optimize the editing conditions for resting human CD4+ T cells and maintain high cell viability for several weeks post Nucleofection.