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.

The Art Of Phosphitylation

Phosphorylation is key for bioactives, with phosphoramidites favored for high selectivity. Discover how choosing specific protecting groups and new technologies fine-tunes the synthesis process.

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.

Gene Therapy And Oligonucleotide Compendium

Technical summaries and detailed protocols for purity testing, sizing, sequencing and expression analysis of oligonucleotide-based active agents as well as protein and lipid-based delivery systems.

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.

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.

Unlocking The IP Benefits Of Novel Nucleases In CRISPR Therapies

Incorporating novel nucleases into CRISPR therapies enhances intellectual property benefits through improved licensing, strategic protections, and access to new gene targets.

Ultra-Sensitive Assay For Anti-Sense Oligonucleotide Quantification

Learn about a new level in sensitivity for challenging analytes in matrix that opens doors for accurate bioanalysis and metabolism studies with specificity not achievable with orthogonal methods.