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.

Quantification Of Large Oligonucleotides Using High Resolution MS/MS

Explore how to achieve sensitive, high-resolution analysis of large oligonucleotides, with the opportunity to perform both qualitative and quantitative analysis in a single run.

Suitability Testing For IVT mRNA Analysis

Automated capillary electrophoresis provides robust, precise analysis of IVT mRNA integrity, concentration, and size, demonstrating excellent method precision.

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.

CRISPR Off-Target Editing: Prediction, Analysis, And More

A crucial consideration for researchers is off-target editing – unintended DNA modifications at sites other than the intended target. Explore the complexities and strategies to mitigate these effects.

Assigning IR Absorption Bands For RNA Building Blocks Using Microfluidic Modulation Spectroscopy

Explore the power of MMS in identifying RNA base signatures. This study analyzes vibrational modes of A, G, C, and U, revealing insights into electronic environments and molecular interactions.

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.

Novel Plasmid DNA-Encoded Poly(A) Tails For mRNA Synthesis

Here, we evaluate the stability of these variant poly(A) regions and the biological activity of the mRNA containing the variant poly(A) tails made from these templates.