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.

A New Era Of mRNA Potency And Safety

Selecting the right RNA polymerase and cap analog pairing significantly impacts mRNA yield, purity, and immunogenic safety, yet many production workflows still rely on a single industry-standard enzyme.

Top 5 Approaches To The Bioanalysis Of Oligonucleotides

Evaluate the strengths and limitations of each bioanalysis method carefully to choose the most effective strategy for your clinical study.

High-Throughput Characterization Of RNA

How a multi-capillary electrophoresis (CE) system is used to efficiently separate a wide range of RNA-based molecules by size and demonstrates the multiplex capability for RNA characterization.

Potent Immune Response With Vernal's LNP-mRNAs

As mRNA medicine continues to evolve, understand how trusted manufacturing partners will continue to play a pivotal role in translating innovative concepts into clinical reality.

Exploring Osmolality As A CQA Within RNA Therapeutics

Discover how osmolality can be applied throughout the bioprocessing workflow for RNA, and recognize the importance of this sensitive measurement, from formulation optimization to cryopreservation techniques.

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.

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.