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.

Selection And Evaluation Of Hybridization Capture Probes For LC/MS Analysis Of Oligonucleotides

To overcome challenges in oligonucleotide LC/MS bioanalysis, consider optimizing probe design by evaluating alternative chemistries like PNA for improved recovery and chromatographic performance.

mRNA Product Purification Process Strategies

Impurities from IVT can induce undesired immune responses and negatively impact translation efficiency. Explore the pros and cons of each option for mRNA purification.

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.

Lentiviral Vector Characterization For Cell And Gene Therapy Manufacturing

This study focuses on characterizing recombinant LVVs used in gene-modified cell therapies, utilizing two real-time PCR assays adapted to the QuantStudio™ Absolute Q™ Digital PCR System.

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.

Comparison Of Constant- And PulsedField Electrophoresis

Accurate sizing of large DNA fragments is crucial in long‑read sequencing. Examine how different electrophoresis methods perform with 10–20 kb smears and how to choose the most reliable approach.

Extending The Lower Limits Of Quantification Of A Therapeutic Oligonucleotide Through Microflow LC-MS/MS

Here a solution is presented to widely recognized challenges with ionpairing reversed phase liquid chromatography (IP-RP LC) through a novel microflow LC-MS strategy.