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 In Vitro Transcribed (IVT) mRNA

Learn best practices for high-resolution analysis of IVT RNA using automated capillary electrophoresis. Improve accuracy and precision in quantifying critical quality attributes.

Stick With Me: The Cool Chemistry Of HILIC Oligonucleotide Analysis

Discover a powerful alternative for analyzing polar sequences without ion-pairing reagents. By leveraging hydrophilic partitioning, you can achieve sharper separation and improved mass spectrometry compatibility.

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.

How To Navigate Through The Jungle Of Columns?

Whether prioritizing mass spectrometry compatibility or loading capacity, understanding the specific trade-offs between IP-RP, AEX, and SEC methodologies is essential for successful characterization.

Structural Characterization Of RNA And Detection Of RNA-Ligand Binding

Delve into the intricacies of riboswitch behavior and ligand interactions and gain valuable insights into gene regulation and potential therapeutic targets.

Oligonucleotides: Where We Are And Where We Would Like To Go 

This presentation provides an overview of oligonucleotide bioanalysis's current status and future directions. We will look into the advantages and disadvantages of various bioanalytical techniques.

CRISPR sgRNA For Successful Gene Editing: Discovery To Clinic

The single guide RNA (sgRNA) is one of the key components of successful CRISPR gene editing. Here we cover all the important aspects of sgRNA and Synthego’s sgRNA products.