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.

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.

Advancing RNA-LNP Therapeutics As An Alternative To Viral Vectors

RNA-LNP therapeutics offer a flexible, transient alternative to traditional viral vectors for genetic medicine. Learn about the advantages of LNPs in safety, scalability, and payload versatility.

Five Essentials For Accurate Oligonucleotide Chemistry

Explore five key areas in oligonucleotide chemistry where improvements will have a huge influence on your synthesis quality.

A Risk-Based Approach To Plasmid DNA And mRNA Process Development

Balancing robust analytics and clinical readiness is key for early-phase pDNA and mRNA therapeutics amid structural complexity and regulatory challenges.

Mastering mRNA Manufacturing: A Focus on Raw Materials & Supply Assurance

Rapid advancements in mRNA-based therapeutic development have underscored the role of raw material quality and supply chain management in ensuring their successful production.

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.

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.