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.

Probing RNA Base Pairing And Ligand Interactions In Solution With Infrared-Based MMS

Discover why Microfluidic Modulation Spectroscopy (MMS) is a novel, label-free, solution-based infrared technique for probing RNA base pairing and ligand interactions.

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.

Metabolite ID And Relative Quantification Of Oligonucleotides In Plasma

This technical note describes the identification, relative quantification, and structural confirmation of the chain-shortened metabolites of a phosphorothioated oligonucleotide.

5 Key Questions On How To Accelerate mRNA Development

Stakeholders are working to solve key mRNA manufacturing challenges, including how raw materials impact product quality and how to address scale up. Though existing analytical and manufacturing technologies may fall short for mRNA, investment in innovative techniques will help meet increasing demand for personalized therapeutics.

Quality Control Of Oligonucleotides Using HPLC Coupled To UV And MS Detection

Efficient quality control of therapeutic oligonucleotides is crucial. Explore advanced methods combining HPLC-UV and mass spectrometry for precise impurity quantification and mass confirmation.

Optimizing mRNA For Success: A Guide To Enhanced Stability, Delivery, And Efficacy

RNA offers exciting potential for vaccines, gene therapies, and personalized medicine, but challenges related to stability, delivery, and manufacturing persist.

Limiting Off-Target Effects Of CRISPR-Based Products

Find out how to enhance CRISPR gene editing specificity by using Next Generation Sequencing to improve sgRNA quality control, addressing off-target effects and ensuring higher sequence fidelity.