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.

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.

Optimizing qPCR For Broad-Range Transcript Detection In Anti-PD-1 Therapy

Optimize qPCR protocols for greater sensitivity and reproducibility to accurately measure immune-related gene expression and better identify patients who will benefit from anti-PD-1 immunotherapy.

RNA Biotherapeutics — A New Approach To An Old Problem

Check out a brief history of RNA-based therapy and how Droplet Digital™ PCR supports this field of work.

Expression Analysis Of CTLA-4, PD-1, And PD-L1

Advance personalized immunotherapy in NSCLC by leveraging qPCR-based analysis of CTLA-4, PD-1, and PD-L1 expression to identify predictive biomarkers and guide more effective treatment strategies.

dsRNA: Why Polymerase Choice Matters

Polymerase selection during IVT shapes dsRNA levels, processing costs, and product quality. See how enzyme engineering reduces abortive transcripts and improves purity profiles for complex mRNA manufacturing applications.

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.

Lessons From Benchmarking Of dsRNA Across Polymerase Systems

Not all low-dsRNA claims hold up under direct comparison. A 15-enzyme IVT study identifies the few systems that actually deliver across yield, integrity, and dsRNA simultaneously.