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.

Best Practices For Analysis Of IVT mRNA

Discover how an automated capillary electrophoresis system provides robust and precise integrity, concentration, and size analysis for IVT mRNA.

Analytics And Bioassays Can Safely Fast-Track mRNA-LNP Drug Development

One main mRNA technology challenge is the formulation of delivery systems like lipid nanoparticles (LNPs). Learn about evading impurities in lipids, the future of mRNA-based genomic medicines, and more.

The Art Of Phosphitylation

Phosphorylation is key for bioactives, with phosphoramidites favored for high selectivity. Discover how choosing specific protecting groups and new technologies fine-tunes the synthesis process.

Streamlining The Importing & Exporting Of Biological Samples

Create a seamless and predictable import and export process by working with experienced bioanalytical laboratory partners, understanding what paperwork samples require, and proactive planning.

Understanding RNA Higher-Order Structure Dynamics For CRISPR Applications

Scientists are gaining valuable knowledge about the structural dynamics of guide RNAs used in CRISPR-Cas9. This deeper understanding could pave the way for improved gene editing tools.

Twist Of Two: 2D-LC For Oligos

Discover how coupling orthogonal mechanisms reveals hidden impurities and creates a more robust strategy for characterizing complex oligonucleotide samples.

The Next Generation Of mRNA Products: Implications For Quality Control

To fully realize the potential of mRNA as a therapeutic or vaccine modality, developers will need to employ cutting-edge analytical methods to ensure the safety and efficacy of these novel products.