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.

5 Key Questions On How To Accelerate mRNA Development

Stakeholders are working to solve key mRNA manufacturing challenges, including how raw materials impact product quality and how to address scale up. Though existing analytical and manufacturing technologies may fall short for mRNA, investment in innovative techniques will help meet increasing demand for personalized therapeutics.

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.

Lentiviral Vector Characterization For Cell And Gene Therapy Manufacturing

This study focuses on characterizing recombinant LVVs used in gene-modified cell therapies, utilizing two real-time PCR assays adapted to the QuantStudio™ Absolute Q™ Digital PCR System.

IR Spectral Signatures Of GC, AU, And GU Base Pairing In H2O-Based Buffer By MMS

Explore a label-free approach to characterize RNA structure with high sensitivity. See how specific base pair types are resolved, providing a clearer picture of RNA folding.

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.

Preventive Control Of Sequencing Through The Insert

Understanding library size distributions helps prevent sequencing through the insert, reducing adapter read‑through. Explore how smear analysis guides optimal read‑length selection to avoid noise.

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.

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.

Assessing Doggybone DNA For In Vitro Transcription

In this study, we evaluated the performance of two dbDNA IVT templates, each encoding the same mRNA (firefly luciferase or Fluc), by looking at the mRNA purity and in vivo potency of the mRNA IVT’d from each template.