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.

Establishing Analytical Methods For mRNA-Based Therapies

Here, we provide a detailed description of assays for sequence identification and LNP composition in mRNA-LNP products that support the development of safe and effective mRNA therapies.

Methods for mRNA Poly(A) Sizing

This study used Oxford Nanopore Technologies' Direct RNA Sequencing Kit and obtained successful sizing of poly(A) tail regions. The results are presented in a histogram and table format.

Accelerating IND-Enabling Studies With Automated Bioanalysis Workflows

Reimagined as automation-first, bioanalysis shifts from being an Investigational New Drug (IND) bottleneck to a catalyst for innovation—helping companies move therapies forward with greater confidence.

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.

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.

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.

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.